HATCY: Safety and Efficacy of ATIR101 as Adjunctive Treatment to Blood Stem Cell Transplantation From a Haploidentical Family Donor Compared to Post-transplant Cyclophosphamide in Patients With Blood Cancer

• Sponsor: Kiadis Pharma (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT02999854
• Collaborator: (none)
• Enrollment: 63
• Locations: 42
• Arms: 2
• Actual Duration (Months): 48.6
• Patients Per Site: 1.5
• Patients Per Site Per Month: 0

Study Details

Study Description

Brief Summary

The primary objective of this study is to compare safety and efficacy of a haploidentical T-cell depleted HSCT and adjunctive treatment with ATIR101 versus a haploidentical T cell replete HSCT with post-transplant administration of high dose cyclophosphamide (PTCy) in patients with a hematologic malignancy. An additional objective of the study is to compare the effect of the two treatments on quality of life.

Condition or Disease	Intervention/Treatment	Phase
Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndrome	Biological: ATIR101; Drug: Cyclophosphamide; Procedure: T-cell depleted HSCT from a related, haploidentical donor; Procedure: T-cell replete HSCT from a related, haploidentical donor	Phase 3

Detailed Description

Study CR-AIR-009 is a Phase III randomized controlled multicenter open-label study comparing two parallel groups. After signing informed consent, a total of 250 patients will be randomized in a 1:1 fashion to receive either a T-cell depleted hematopoietic stem cell transplantation (HSCT; CD34 selection) from a related, haploidentical donor, followed by ATIR101 infusion, or a T-cell replete HSCT, followed by a high dose of post-transplant cyclophosphamide (PTCy).

Randomization will use minimization to balance treatment groups with respect to underlying disease (AML, ALL, or MDS), Disease Risk Index (DRI; intermediate risk, high risk, or very high risk) and center. A stochastic treatment allocation procedure will be used so that the treatment assignment is random for all patients entered in the study.

Patients randomized in the ATIR101 group will receive a single ATIR101 dose of 2×10E6 viable T-cells/kg between 28 and 32 days after the HSCT. Patients randomized in the PTCy group will receive cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT. All patients will be followed up for at least 24 months post HSCT.

Study Design

• Study Type: Interventional
• Actual Enrollment: 63 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Prevention
• Official Title: A Phase III, Multicenter, Randomized Controlled Study to Compare Safety and Efficacy of a Haploidentical HSCT and Adjunctive Treatment With ATIR101, a T-lymphocyte Enriched Leukocyte Preparation Depleted ex Vivo of Host Alloreactive T-cells, Versus a Haploidentical HSCT With Post-transplant Cyclophosphamide in Patients With a Hematologic Malignancy
• Actual Study Start Date: Nov 29, 2017
• Actual Primary Completion Date: Nov 9, 2021
• Actual Study Completion Date: Dec 17, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ATIR101 T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT	Biological: ATIR101; ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion); Procedure: T-cell depleted HSCT from a related, haploidentical donor; T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Active Comparator: PTCy T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT	Drug: Cyclophosphamide; High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion); Procedure: T-cell replete HSCT from a related, haploidentical donor; T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen

Outcome Measures

Primary Outcome Measures

1. Graft-versus-host Disease-free, Relapse-free Survival (GRFS) [24 months post-HSCT]

   Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.

Secondary Outcome Measures

1. Overall Survival (OS) [24 months post-HSCT]

   OS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.

2. Progression-free Survival (PFS) [24 months post-HSCT]

   Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.

3. Relapse-related Mortality (RRM) [Through study completion, at least two years post HSCT]

   Time from randomization to death due to disease relapse or disease progression

4. Transplant-related Mortality (TRM) [24 months post-HSCT]

   Defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.

Other Outcome Measures

1. Immune Reconstitution [Through study completion, at least two years post HSCT]

   Time to CD3+ > 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)

2. Cumulative Incidence of Grade II-IV and Grade III-IV Acute Graft-versus-host-disease (GVHD) [Through study completion, at least two years post HSCT]

3. Cumulative Incidence of Moderate/Severe Chronic GVHD [Through study completion, at least two years post HSCT]

4. Cumulative Incidence of Chronic GVHD Requiring Systemic Immunosuppressive Treatment [Through study completion, at least two years post HSCT]

5. Duration of GVHD Episodes [Through study completion, at least two years post HSCT]

6. Cumulative Incidence of NCI CTCAE Grade 2-5 and Grade 3-5 Infections [Until 2 years after the HSCT]

   Viral, fungal, and bacterial infections

7. Cumulative Incidence of NCI CTCAE Grade 3-5 Adverse Events [Until 2 years after the HSCT]

   Viral, fungal, and bacterial infections

8. FACT-BMT Total Score (Change From Screening) [Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)]

   Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)

9. SF-36 Total Score (Change From Screening) [Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)]

   Quality of life: Short Form 36-item health survey (SF-36)

10. MDASI Total Score (Change From Screening) [Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)]

    Quality of life: MD Anderson Symptom Inventory (MDASI)

11. EQ-5D-5L (Change From Screening) [Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)]

    Quality of life: EQ-5D-5L

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 70 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Any of the following hematologic malignancies:

• Acute myeloid leukemia (AML) in first cytomorphological remission (with < 5% blasts in the bone marrow) with Disease Risk Index (DRI) intermediate or above, or in second or higher cytomorphological remission (with < 5% blasts in the bone marrow)

• Acute lymphoblastic leukemia (ALL) in first or higher remission (with < 5% blasts in the bone marrow)

• Myelodysplastic syndrome (MDS): transfusion-dependent (requiring at least one transfusion per month), or intermediate or higher Revised International Prognostic Scoring System (IPSS-R) risk group

• Clinical justification of allogeneic stem cell transplantation where a suitable HLA matched sibling or unrelated donor is unavailable in a timely manner

• Availability of a related haploidentical donor with one fully shared haplotype and 2 to 4 mismatches at the HLA-A, -B, -C, and -DRB1 loci of the unshared haplotype, as determined by high resolution human leukocyte antigen (HLA)-typing

• Karnofsky Performance Status (KPS) ≥ 70%

• Male or female, age ≥ 18 years and ≤ 70 years. Patients aged ≥ 65 years must have a Sorror score ≤ 3

• Patient weight ≥ 25 kg and ≤ 130 kg

• Availability of a donor aged ≥ 16 years and ≤ 75 years who is eligible according to local requirements and regulations. Donors aged < 16 years are allowed if they are the only option for an HSCT, if they are permitted by local regulations, and if the IRB/IEC approves participation in the study.

• For females of childbearing potential who are sexually active and males who have sexual contact with a female of childbearing potential: willingness to use of reliable methods of contraception (oral contraceptives, intrauterine device, hormone implants, contraceptive injection or abstinence) during study participation

• Given written informed consent (patient and donor)

Exclusion Criteria:

• Diagnosis of chronic myelomonocytic leukemia (CMML)

• Availability of a suitable HLA-matched sibling or unrelated donor in a donor search

• Prior allogeneic hematopoietic stem cell transplantation

• Diffusing capacity for carbon monoxide (hemoglobin corrected DLCO) < 50% predicted

• Left ventricular ejection fraction < 45% (evaluated by echocardiogram or MUGA scan)

• Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 2.5 × upper limit of normal (CTCAE grade 2)

• Creatinine clearance < 50 ml/min (calculated or measured)

• Positive pregnancy test or breastfeeding of patient or donor (women of childbearing age only)

• Estimated probability of surviving less than 3 months

• Known allergy to any of the components of ATIR101 (e.g., dimethyl sulfoxide)

• Known hypersensitivity to cyclophosphamide or any of its metabolites

• Any contraindication for GVHD prophylaxis with mycophenolate mofetil, cyclosporine A, or tacrolimus

• Known presence of HLA antibodies against the non-shared donor haplotype

• Positive viral test of the patient or donor for human immunodeficiency virus (HIV)-1, HIV-2, hepatitis B virus (HBV), hepatitis C virus (HCV), Treponema pallidum, human T-lymphotropic virus (HTLV)-1 (if tested), HTLV-2 (if tested), West Nile virus (WNV; if tested), or Zika virus (if tested)

• Any other condition that, in the opinion of the investigator, makes the patient or donor ineligible for the study

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	City of Hope National Medical Center	Duarte	California	United States	91010
2	Moores UC San Diego Cancer Center	La Jolla	California	United States	92037-0698
3	UCLA Center for Health Sciences	Los Angeles	California	United States	90095
4	Stanford University School of Medicine	Stanford	California	United States	94305
5	Emory University	Atlanta	Georgia	United States	30322
6	University of Kansas Cancer Center	Westwood	Kansas	United States	66205
7	Massachusetts General Hospital	Boston	Massachusetts	United States	02114
8	University of Michigan	Ann Arbor	Michigan	United States	48109-1274
9	Weill Cornell Medical College	New York	New York	United States	10021
10	Columbia University Medical Center	New York	New York	United States	10032
11	Stony Brook University Hospital	Stony Brook	New York	United States	11794
12	Oregon Health & Science University	Portland	Oregon	United States	97239
13	Universitair Ziekenhuis Antwerpen	Antwerp		Belgium	2650
14	Algemeen Ziekenhuis Sint-Jan	Brugge		Belgium	8000
15	Institut Jules Bordet	Brussels		Belgium	1000
16	Universitair Ziekenhuis Gasthuisberg	Leuven		Belgium	3000
17	Centre Hospitalier Universitaire de Liège	Liège		Belgium	4000
18	Maisonneuve-Rosemont Hospital	Montreal	Quebec	Canada	H1T 2M4
19	University Hospital Centre Zagreb	Zagreb		Croatia	10000
20	APHP Hospital Saint Louis	Paris		France	75475
21	University Hospital Frankfurt, Goethe University	Frankfurt		Germany
22	University Medical Center Mainz	Mainz		Germany	55131
23	Ludwig-Maximilians-University Hospital of Munich-Grosshadern	Munich		Germany	81377
24	Universitätsklinikum Würzburg	Würzburg		Germany	97080
25	Rambam Medical Center	Haifa		Israel	3109601
26	Hadassah Medical Center & Hadassah Hospital Ein Karem	Jerusalem		Israel	91120
27	Sourasky Medical Center & Tel Aviv University	Tel Aviv		Israel	6423906
28	Chaim Sheba Medical Center	Tel-Hashomer		Israel	5265601
29	Milano Hospital, Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milano		Italy	20122
30	Fondazione IRCCS Policlinico San Matteo	Pavia		Italy	27100
31	Academisch Ziekenhuis Maastricht	Maastricht		Netherlands	6229 HX
32	Faculdade de Medicina da Universidade de Lisboa	Lisboa		Portugal	1649-028
33	University Hospital Barcelona Vall d' Hebron	Barcelona		Spain	08035
34	Hospital Puerta de Hierro Majadahonda	Madrid		Spain	28220
35	UGC Hematología y Hemoterapia	Sevilla		Spain	41013
36	Servicio de Hematología Hospital, Universitari I politècnic La Fe	Valencia		Spain	46026
37	Karolinska University Hospital	Stockholm		Sweden	SE-141 86
38	Heartlands Hospital	Birmingham		United Kingdom	B9 5SS
39	St James University Hospital	Leeds		United Kingdom	LS9 7TF
40	Royal Liverpool University Hospital	Liverpool		United Kingdom	L7 8XP
41	Hammersmith Hospital	London		United Kingdom	W12 0HS
42	Manchester Royal Infirmary	Manchester		United Kingdom	M13 9WL

Sponsors and Collaborators

• Kiadis Pharma

Investigators

• Principal Investigator: Denis Claude Roy, Prof MD, Research Center and Cellular Therapy Laboratory, Maisonneuve-Rosemont Hospital (Montreal, Canada)
• Principal Investigator: Stephan Mielke, Prof MD, Centre for Allogeneic Stem Cell Transplantation, Karolinska University Hospital (Stockholm, Sweden)

Study Documents (Full-Text)

• Study Protocol - Aug 23, 2018
• Statistical Analysis Plan - Dec 13, 2021

More Information

Publications

None provided.

• Responsible Party: Kiadis Pharma
• ClinicalTrials.gov Identifier: NCT02999854
• Other Study ID Numbers: CR-AIR-009; 2016-004672-21
• First Posted: Dec 21, 2016
• Last Update Posted: May 24, 2022
• Last Verified: Apr 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Kiadis Pharma: Haploidentical stem cell transplantation; Graft-versus-host disease; Immune reconstitution; Alloreactive T-cells; Photodynamic treatment; Hematologic malignancy; Transplant-related mortality; Overall survival; GRFS; GVHD
• Additional relevant MeSH terms: Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Myelodysplastic Syndromes; Cyclophosphamide

Study Results

Participant Flow

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Period Title: Active Study Phase (up to 18 Months)
STARTED	32	31
COMPLETED	8	22
NOT COMPLETED	24	9
Period Title: Active Study Phase (up to 18 Months)
STARTED	8	22
COMPLETED	5	19
NOT COMPLETED	3	3

Baseline Characteristics

Arm/Group Title	ATIR101	PTCy	Total
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	Total of all reporting groups
Overall Participants	16	27	43
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	36.2 (9.63)	32.8 (13.20)	34.5 (11.42)
Sex: Female, Male (Count of Participants)
Female	10 62.5%	20 74.1%	30 69.8%
Male	6 37.5%	7 25.9%	13 30.2%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	3 18.8%	1 3.7%	4 9.3%
Not Hispanic or Latino	12 75%	22 81.5%	34 79.1%
Unknown or Not Reported	1 6.3%	4 14.8%	5 11.6%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%
Asian	0 0%	1 3.7%	1 2.3%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%
Black or African American	0 0%	1 3.7%	1 2.3%
White	14 87.5%	23 85.2%	37 86%
More than one race	0 0%	0 0%	0 0%
Unknown or Not Reported	2 12.5%	2 7.4%	4 9.3%
Hematologic malignancy (Count of Participants)
Acute lymphatic leukemia (ALL)	4 25%	2 7.4%	6 14%
Acute myeloid leukemia (AML)	9 56.3%	18 66.7%	27 62.8%
Myelodysplastic syndrome (MDS)	3 18.8%	7 25.9%	10 23.3%

Outcome Measures

1. Primary Outcome

Title	Graft-versus-host Disease-free, Relapse-free Survival (GRFS)
Description	Defined as the time until acute GVHD grade III/IV, chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of GRFS were calculated at 24 months post HSCT.
Time Frame	24 months post-HSCT

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	16	27
Number (95% Confidence Interval) [Percentage of participants]	25 156.3%	62 229.6%

2. Secondary Outcome

Title	Overall Survival (OS)
Description	OS is defined as the time from HSCT until death from any cause. Kaplan-Meier estimates (percentage of participants) of OS were calculated at 24 months post HSCT.
Time Frame	24 months post-HSCT

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	16	27
Number (95% Confidence Interval) [percentage of participants]	49 306.3%	77 285.2%

3. Secondary Outcome

Title	Progression-free Survival (PFS)
Description	Defined as the time from HSCT until relapse, disease progression, or death, whichever occurs first. Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
Time Frame	24 months post-HSCT

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	16	27
Number (95% Confidence Interval) [percentage of participants]	44 275%	73 270.4%

4. Secondary Outcome

Title	Relapse-related Mortality (RRM)
Description	Time from randomization to death due to disease relapse or disease progression
Time Frame	Through study completion, at least two years post HSCT

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

5. Secondary Outcome

Title	Transplant-related Mortality (TRM)
Description	Defined as death due to causes other than disease relapse or progression, or other causes which are unrelated to the transplantation procedure (e.g. accident, suicide). Kaplan-Meier estimates (percentage of participants) of PFS were calculated at 24 months post HSCT.
Time Frame	24 months post-HSCT

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	16	27
Number (95% Confidence Interval) [percentage of participants]	44 275%	15 55.6%

6. Other Pre-specified Outcome

Title	Immune Reconstitution
Description	Time to CD3+ > 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)
Time Frame	Through study completion, at least two years post HSCT

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

7. Other Pre-specified Outcome

Title	Cumulative Incidence of Grade II-IV and Grade III-IV Acute Graft-versus-host-disease (GVHD)
Time Frame	Through study completion, at least two years post HSCT

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

8. Other Pre-specified Outcome

Title	Cumulative Incidence of Moderate/Severe Chronic GVHD
Time Frame	Through study completion, at least two years post HSCT

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

9. Other Pre-specified Outcome

Title	Cumulative Incidence of Chronic GVHD Requiring Systemic Immunosuppressive Treatment
Time Frame	Through study completion, at least two years post HSCT

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

10. Other Pre-specified Outcome

Title	Duration of GVHD Episodes
Time Frame	Through study completion, at least two years post HSCT

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

11. Other Pre-specified Outcome

Title	Cumulative Incidence of NCI CTCAE Grade 2-5 and Grade 3-5 Infections
Description	Viral, fungal, and bacterial infections
Time Frame	Until 2 years after the HSCT

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

12. Other Pre-specified Outcome

Title	Cumulative Incidence of NCI CTCAE Grade 3-5 Adverse Events
Description	Viral, fungal, and bacterial infections
Time Frame	Until 2 years after the HSCT

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

13. Other Pre-specified Outcome

Title	FACT-BMT Total Score (Change From Screening)
Description	Quality of life: Foundation for the Accreditation of Cellular Therapy - Bone Marrow Transplantation questionnaire (FACT-BMT)
Time Frame	Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

14. Other Pre-specified Outcome

Title	SF-36 Total Score (Change From Screening)
Description	Quality of life: Short Form 36-item health survey (SF-36)
Time Frame	Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

15. Other Pre-specified Outcome

Title	MDASI Total Score (Change From Screening)
Description	Quality of life: MD Anderson Symptom Inventory (MDASI)
Time Frame	Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

16. Other Pre-specified Outcome

Title	EQ-5D-5L (Change From Screening)
Description	Quality of life: EQ-5D-5L
Time Frame	Through study completion (Month 3, 6, 12, 24, 36, 48 post HSCT)

Outcome Measure Data

Analysis Population Description

Study was pre-maturely terminated. Data not collected.

Arm/Group Title	ATIR101	PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen	T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
Measure Participants	0	0

Adverse Events

Time Frame	Long term safety follow-up, 24 months.
Adverse Event Reporting Description	Study was pre-maturely terminated. For the long term safety follow-up (LTFU), up to 24 months post-HSCT, only cumulative SAEs were recorded. (63 patients had been enrolled (randomized); 32 to ATIR101 group and 31 to PTCy group (intention-to-treat [ITT] population). Only patients who received an HSCT and ATIR101 (n=16) or at least one dose of PTCy (n=27) were included in the modified ITT population (MITT). Eight ATIR101, and 22 PTCy treated patients entered 2 year LTFU).
Arm/Group Title	ATIR101		PTCy
Arm/Group Description	T-cell depleted HSCT from a related, haploidentical donor, followed by IV infusion with ATIR101 at a single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT ATIR101: ATIR101 is a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells using photodynamic treatment; single dose of 2×10E6 viable T-cells/kg body weight between 28 and 32 days after the HSCT (intravenous infusion) T-cell depleted HSCT from a related, haploidentical donor: T-cell depleted graft prepared from peripheral blood stem cells using the CD34+ cell selection method; infused after a total body irradiation (TBI) or non-TBI conditioning regimen		T-cell replete HSCT from a related, haploidentical donor, followed by IV infusion of post-transplant cyclophosphamide (PTCy) 50 mg/kg/day at 3 and 4/5 days after the HSCT Cyclophosphamide: High dose post-transplant cyclophosphamide 50 mg/kg/day at 3 and 4/5 days after the HSCT (powder for intravenous infusion) T-cell replete HSCT from a related, haploidentical donor: T-cell replete (full, non-manipulated) graft prepared from either bone marrow or peripheral blood stem cells; infused after a total body irradiation (TBI) or non-TBI conditioning regimen
All Cause Mortality
	ATIR101		PTCy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	8/16 (50%)		6/27 (22.2%)
Serious Adverse Events
	ATIR101		PTCy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	13/16 (81.3%)		19/27 (70.4%)
Blood and lymphatic system disorders
Blood and lymphatic system disorders	5/16 (31.3%)		5/27 (18.5%)
Cardiac disorders
Cardiac disorders	2/16 (12.5%)		5/27 (18.5%)
Gastrointestinal disorders
Gastrointestinal disorders	0/16 (0%)		1/27 (3.7%)
General disorders
General disorders and administration site conditions	4/16 (25%)		6/27 (22.2%)
Infections and infestations
Infections and infestations	10/16 (62.5%)		12/27 (44.4%)
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications	2/16 (12.5%)		0/27 (0%)
Investigations
Investigations	0/16 (0%)		2/27 (7.4%)
Metabolism and nutrition disorders
Metabolism and nutrition disorders	0/16 (0%)		3/27 (11.1%)
Nervous system disorders
Nervous system disorders	3/16 (18.8%)		0/27 (0%)
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders	2/16 (12.5%)		1/27 (3.7%)
Other (Not Including Serious) Adverse Events
	ATIR101		PTCy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/16 (0%)		0/27 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.