A Phase 1 Trial of a Single ProHema® CB Product for Pediatric Patients With Hematologic Malignancies
Study Details
Study Description
Brief Summary
This is an open-label, safety study of a single ProHema-CB product administered following myeloablative conditioning regimen in pediatric subjects with hematologic malignancies.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1 |
Detailed Description
A maximum of 18 eligible male and female subjects (1 to 18 years old, inclusive) will be enrolled and treated in the trial at approximately 3 to 5 centers within the U.S. These 18 subjects will consist of 3 cohorts of 6 subjects each. The cohorts will be defined by age: 1 to 4 years; > 4 to 12 years; and > 12 to 18 years. These cohorts will be enrolled simultaneously.
All subjects will be admitted to the hospital, per institutional practice, and will receive a myeloablative conditioning regimen, after which they will receive an HLA-matched or partially matched ProHema-CB unit on study Day 0.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: ProHema-CB All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour. |
Biological: Biological: ProHema-CB
Each subject will receive one administration of ProHema-CB unit transplant.
|
Outcome Measures
Primary Outcome Measures
- Safety Profile, Primarily Assessed by Neutrophil Engraftment [Neutrophil engraftment by Day 42]
To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Male and female subjects aged 1 to 18 years, inclusive.
-
Subjects with hematologic malignancies for whom allogeneic stem cell transplantation is deemed clinically appropriate.
-
Acute Myelogenous Leukemia (AML) in high risk 1st or subsequent CR
-
Acute Lymphoblastic Leukemia (ALL) in CR
-
NK cell lymphoblastic leukemia in any CR
-
Biphenotypic or undifferentiated leukemia in 1st or subsequent CR
-
Myelodysplastic Syndrome (MDS) at any stage.
-
Chronic Myelogenous Leukemia (CML) All subjects with evidence of CNS leukemia must be treated and be in CNS CR to be eligible for trial.
-
Lack of 5-6/6 HLA matched related or 8/8 HLA A, B, C, DRß1 matched unrelated donor; or unrelated donor not available within appropriate timeframe, as determined by the transplant physician.
-
Availability of suitable primary and secondary umbilical cord blood (UCB) units.
-
Adequate performance status, defined as:
-
Subjects ≥ 16 years: Karnofsky score ≥ 70%.
-
Subjects < 16 years: Lansky score ≥ 70%.
-
Cardiac: Left ventricular ejection fraction at rest must be > 40%, or shortening fraction > 26%.
-
Pulmonary:
-
Subjects > 10 years: DLCO (diffusion capacity) > 50% of predicted (corrected for hemoglobin)
-
FEV1, FVC > 50% of predicted; Note: If unable to perform pulmonary tests, then O2 saturation > 92% on room air.
-
Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) > 70mL/min/1.73m2.
-
Hepatic: Bilirubin ≤ 2.5 mg/dL (except in the case of Gilbert's syndrome or ongoing hemolytic anemia); and ALT, AST and Alkaline Phosphatase ≤ 5 × ULN.
-
Signed IRB approved Informed Consent Form (ICF).
Exclusion Criteria:
-
Female subjects that are pregnant or breastfeeding.
-
Evidence of HIV infection or HIV positive serology.
-
Current uncontrolled bacterial, viral or fungal infection.
-
Prior allogeneic hematopoietic stem cell transplant.
-
Autologous transplant < 12 months prior to enrollment.
-
Prior autologous transplant for the disease for which the UCB transplant is being performed.
-
Active malignancy other than the one for which the UCB transplant is being performed within 12 months of enrollment.
-
Inability to receive TBI.
-
Requirement of supplemental oxygen.
-
HLA-matched related donor able to donate.
-
Use of an investigational drug within 30 days prior to screening.
-
Subject is unlikely to comply with the protocol requirements, instructions and study-related restrictions
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | City of Hope | Duarte | California | United States | 91010 |
| 2 | Boston Children's Hospital | Boston | Massachusetts | United States | 02115-5450 |
Sponsors and Collaborators
- Fate Therapeutics
Investigators
- Study Director: Chris Storgard, MD, Fate Therapeutics
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- FT1050-04
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | ProHema-CB |
|---|---|
| Arm/Group Description | All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour. Biological: ProHema-CB: Each subject will receive o |
| Period Title: Overall Study | |
| STARTED | 3 |
| COMPLETED | 2 |
| NOT COMPLETED | 1 |
Baseline Characteristics
| Arm/Group Title | ProHema-CB |
|---|---|
| Arm/Group Description | All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour. Biological: ProHema-CB: Each subject will receive one administration of ProHema-CB unit transplant. |
| Overall Participants | 3 |
| Age (Years) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [Years] |
15.7
(3.2)
|
| Sex: Female, Male (Count of Participants) | |
| Female |
0
0%
|
| Male |
3
100%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |
| Hispanic or Latino |
2
66.7%
|
| Not Hispanic or Latino |
1
33.3%
|
| Unknown or Not Reported |
0
0%
|
| Race (NIH/OMB) (Count of Participants) | |
| American Indian or Alaska Native |
0
0%
|
| Asian |
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
| Black or African American |
1
33.3%
|
| White |
1
33.3%
|
| More than one race |
0
0%
|
| Unknown or Not Reported |
1
33.3%
|
| Region of Enrollment (participants) [Number] | |
| United States |
3
100%
|
| Weight (kg) (kg) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [kg] |
73.4
(28.8)
|
Outcome Measures
| Title | Safety Profile, Primarily Assessed by Neutrophil Engraftment |
|---|---|
| Description | To describe the safety profile of ProHema-CB after myeloablative conditioning in pediatric patients with hematologic malignancies. The safety profile will primarily be assessed by neutrophil engraftment. |
| Time Frame | Neutrophil engraftment by Day 42 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | ProHema-CB |
|---|---|
| Arm/Group Description | All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour. Biological: ProHema-CB: Each subject will receive one administration of ProHema-CB unit transplant. |
| Measure Participants | 3 |
| Count of Participants [Participants] |
3
100%
|
Adverse Events
| Time Frame | During the First Year of Transplant | |
|---|---|---|
| Adverse Event Reporting Description | Serious Adverse Events and Adverse Events assessed as possibly or probably related to ProHema are included below. | |
| Arm/Group Title | ProHema-CB | |
| Arm/Group Description | All subjects will receive treatment with ProHema-CB (ex-vivo modulated human cord blood cells) transplant. ProHema-CB (the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 also referred to as FT1050) will be prepared and administered in one of two formulations, based upon subject weight: For subjects > 35 kg, ProHema-CB will be administered as 150 mL product in a blood bag via gravity infusion. It will be infused at 10 mL to 15 mL per minute, for a total infusion time of 10 to 15 min. For subject's ≤ 35 kg, ProHema-CB will be administered as a 50 mL product in a syringe via syringe pump.o It will be infused at 5 mL/kg per hour for a total infusion time of up to ~1 hour. Biological: ProHema-CB: Each subject will receive o | |
All Cause Mortality |
||
| ProHema-CB | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/3 (0%) | |
Serious Adverse Events |
||
| ProHema-CB | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/3 (0%) | |
Other (Not Including Serious) Adverse Events |
||
| ProHema-CB | ||
| Affected / at Risk (%) | # Events | |
| Total | 2/3 (66.7%) | |
| General disorders | ||
| Mucosal inflammation | 1/3 (33.3%) | |
| Vascular disorders | ||
| Hypertension | 1/3 (33.3%) | |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
Results Point of Contact
| Name/Title | Chris Storgard, Chief Medical Officer |
|---|---|
| Organization | Fate Therapeutics, Inc. |
| Phone | 866-875-1833 |
| chris.storgard@fatetherapeutics.com |
- FT1050-04