Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT01474681

Collaborator

(none)

Enrollment

Location

Arm

56.8

Actual Duration (Months)

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study evaluated the safety and tolerability of using HSC835 in patients with hematological malignancies.

Condition or Disease	Intervention/Treatment	Phase
Acute Myelocytic Leukemia Acute Lymphocytic Leukemia Chronic Myelogenous Leukemia Myelodysplastic Syndrome Chronic Lymphocytic Leukemia Marginal Zone Lymphoma Follicular Lymphomas Large-cell Lymphoma Lymphoblastic Lymphoma Burkitt's Lymphoma High Grade Lymphomas Mantle-cell Lymphoma Lymphoplasmacytic Lymphoma	Biological: HSC835	Phase 1/Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

27 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A First-in-human, Single-arm, Single-center, Open-label, Proof-of-concept Study to Evaluate the Safety and Tolerability of Infusing HSC835 (LFU835-expanded Umbilical Cord Blood Hematopoietic Stem Cells) in Patients With Hematological Malignancies

Actual Study Start Date :

Jan 9, 2012

Actual Primary Completion Date :

Oct 3, 2016

Actual Study Completion Date :

Oct 3, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: HSC835 HSC835 infusion	Biological: HSC835

Arm

Intervention/Treatment

Experimental: HSC835

HSC835 infusion

Biological: HSC835

Outcome Measures

Primary Outcome Measures

Safety and Tolerability of HSC835 for Clinical Use Were Measured by Infusional Toxicity (Within First 48 Hours After Transplant) and Absence of Graft Failure After 32 Days in Excess of That Currently Observed With UCBT. [32 days]
The safety and tolerability of HSC835 for clinical use were measured by infusional toxicity and absence of graft failure in excess of that currently observed with UCBT. Infusional toxicity - AE from transplant until first 48 hours. Administration of the HSC835 expanded CD34-positive cell product, infused over a period of approximately 15 minutes may theoretically cause adverse reactions based on hemodynamic effects, the release of factors like cytokines through administration into the systemic circulation, or acute hypersensitivity, among others.

Secondary Outcome Measures

Incidence of Neutrophil Recovery Within 42 Days [42 days]
Neutrophil recovery (engraftment) is defined as the first of three consecutive days with ANC > 0.5 x 109/L which occurred for all patients before 42 days post transplant.
Incidence of Platelet Recovery Within Six Months [6 months]
Incidence of platelet recovery within six months. Number of participants recovering platelet to ≥50,000 × 109/L for at least one week without transfusion in the prior 7 days to the first measurement.
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only) [Day 100]
Frequency of expanded unit predominance at day 100 (DUCBT recipients only) unit predominance was assessed by differences in microsatellite patterns between the recipient, HSC835 and the unmanipulated cord blood unit. Evaluation of sorted CD15-positive/CD33-positive myeloid and CD3-positive T cells in the peripheral blood, revealed three patterns: predominance of HSC835, Mixed dominance an unique chimerism pattern was observed with the CD15/CD33 population predominantly derived from HSC835 and the CD3 population almost exclusively derived from the unmanipulated unit, and predominance of the unmanipulated unit
Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year [Day 100 and Month 12]
Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year
Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year [Day 100 and Monnth 12]
Number of participants with incidence of Acute Graft Versus Host Disease (aGVHD) within 100 days and Chronic Graft Versus Host Disease (cGVHD) within 1 year
Incidence of Relapse Within One Year [Month 12]
Number of participants with Incidence of relapse within one year
Overall Survival (OS) Within One Year [Month 12]
Number of participants with Overall survival (OS) within one year
Disease Free Survival (DFS) Within One Year [Month 12]
Number of participants with Disease Free Survival (DFS) within one year. Patients are considered to have achieved DFS or relapse-free survival if they had not experienced either relapse or death (of any cause)

Eligibility Criteria

Criteria

Ages Eligible for Study:

10 Years to 55 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patients with a diagnosis that qualifies them for a DUCBT
Absence of recent active mold infection
Adequate organ function
Availability of eligible donor material

Exclusion Criteria:

Pregnancy or breastfeeding women and women of child-bearing potential unless two acceptable forms of contraception are being used
Human immunodeficiency virus (HIV) infection
Active infection
Extensive prior chemotherapy
Prior myeloablative allotransplantation or autologous transplant.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigative Site	Minneapolis	Minnesota	United States	55455

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

A Plain Language Trial Summary is available on novartisclinicaltrials.com

Publications

None provided.

Responsible Party:

Novartis Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT01474681

Other Study ID Numbers:

CHSC835X2201

First Posted:

Nov 18, 2011

Last Update Posted:

Dec 30, 2020

Last Verified:

Mar 1, 2019

Keywords provided by Novartis Pharmaceuticals

hematologic malignancies

leukemia

lymphoma

Additional relevant MeSH terms:

Leukemia, Lymphocytic, Chronic, B-Cell

Leukemia, Myeloid

Lymphoma, Mantle-Cell

Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Hematologic Neoplasms

Waldenstrom Macroglobulinemia

Leukemia, Myeloid, Acute

Lymphoma, Non-Hodgkin

Myelodysplastic Syndromes

Study Results

Participant Flow

Recruitment Details	Single arm study of HSC835 broken down into 4 subgroups for analyses and 6 subgroups for safety.
Pre-assignment Detail

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Period Title: Overall Study
STARTED	1	8	3	15
COMPLETED	1	5	1	9
NOT COMPLETED	0	3	2	6

Baseline Characteristics

Arm/Group Title	SUCBT	DUCBT	Total
Arm/Group Description	Single umbilical cord blood transplant (SUCBT) This is the combination of the SUCBT<18 yrs and SUCBT >= 18 yrs	Double umbilical cord blood transplant (DUCBT) This is the combination of the DUCBT<18 yrs and DUCBT >= 18 yrs	Total of all reporting groups
Overall Participants	9	18	27
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	33.3 (17.18)	31.3 (13.75)	32.0 (14.67)
Sex: Female, Male (Count of Participants)
Female	5 55.6%	6 33.3%	11 40.7%
Male	4 44.4%	12 66.7%	16 59.3%

Outcome Measures

1. Primary Outcome

Title	Safety and Tolerability of HSC835 for Clinical Use Were Measured by Infusional Toxicity (Within First 48 Hours After Transplant) and Absence of Graft Failure After 32 Days in Excess of That Currently Observed With UCBT.
Description	The safety and tolerability of HSC835 for clinical use were measured by infusional toxicity and absence of graft failure in excess of that currently observed with UCBT. Infusional toxicity - AE from transplant until first 48 hours. Administration of the HSC835 expanded CD34-positive cell product, infused over a period of approximately 15 minutes may theoretically cause adverse reactions based on hemodynamic effects, the release of factors like cytokines through administration into the systemic circulation, or acute hypersensitivity, among others.
Time Frame	32 days

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	1	8	3	15
Infusional Toxicity 48 hrs after transplant	0 0%	0 0%	0 0%	1 NaN
Absence of graft failure after 32 days	0 0%	0 0%	0 0%	0 NaN

2. Secondary Outcome

Title	Incidence of Neutrophil Recovery Within 42 Days
Description	Neutrophil recovery (engraftment) is defined as the first of three consecutive days with ANC > 0.5 x 109/L which occurred for all patients before 42 days post transplant.
Time Frame	42 days

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	1	8	3	15
Number [participants]	1 11.1%	8 44.4%	3 11.1%	15 NaN

3. Secondary Outcome

Title	Incidence of Platelet Recovery Within Six Months
Description	Incidence of platelet recovery within six months. Number of participants recovering platelet to ≥50,000 × 109/L for at least one week without transfusion in the prior 7 days to the first measurement.
Time Frame	6 months

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	1	8	3	15
Number [participants]	1 11.1%	6 33.3%	2 7.4%	11 NaN

4. Secondary Outcome

Title	Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)
Description	Frequency of expanded unit predominance at day 100 (DUCBT recipients only) unit predominance was assessed by differences in microsatellite patterns between the recipient, HSC835 and the unmanipulated cord blood unit. Evaluation of sorted CD15-positive/CD33-positive myeloid and CD3-positive T cells in the peripheral blood, revealed three patterns: predominance of HSC835, Mixed dominance an unique chimerism pattern was observed with the CD15/CD33 population predominantly derived from HSC835 and the CD3 population almost exclusively derived from the unmanipulated unit, and predominance of the unmanipulated unit
Time Frame	Day 100

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	3	15
Predominance of HSC835	0 0%	6 33.3%
Mixed dominance	3 33.3%	3 16.7%
Predominance of the unmaninpulated unit	0 0%	6 33.3%

5. Secondary Outcome

Title	Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year
Description	Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year
Time Frame	Day 100 and Month 12

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	1	8	3	15
Day 100	0 0%	1 5.6%	1 3.7%	3 NaN
Month 12	0 0%	1 5.6%	1 3.7%	6 NaN

6. Secondary Outcome

Title	Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year
Description	Number of participants with incidence of Acute Graft Versus Host Disease (aGVHD) within 100 days and Chronic Graft Versus Host Disease (cGVHD) within 1 year
Time Frame	Day 100 and Monnth 12

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	1	8	3	15
aGVHD-Day 100	0 0%	5 27.8%	3 11.1%	8 NaN
cGVHD-Month12	0 0%	1 5.6%	0 0%	2 NaN

7. Secondary Outcome

Title	Incidence of Relapse Within One Year
Description	Number of participants with Incidence of relapse within one year
Time Frame	Month 12

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	1	8	3	15
Number [participants]	0 0%	2 11.1%	1 3.7%	0 NaN

8. Secondary Outcome

Title	Overall Survival (OS) Within One Year
Description	Number of participants with Overall survival (OS) within one year
Time Frame	Month 12

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	1	8	3	15
Number [participants]	1 11.1%	5 27.8%	1 3.7%	9 NaN

9. Secondary Outcome

Title	Disease Free Survival (DFS) Within One Year
Description	Number of participants with Disease Free Survival (DFS) within one year. Patients are considered to have achieved DFS or relapse-free survival if they had not experienced either relapse or death (of any cause)
Time Frame	Month 12

Outcome Measure Data

Analysis Population Description
Safety Analysis Set (SAS) comprising all patients who were transplanted is used for analysis of all demographic, safety and engraftment outputs.

Arm/Group Title	SUCBT < 18 Yrs	SUCBT >= 18 Yrs	DUCBT < 18 Yrs	DUCBT >= 18 Yrs
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs	Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs	Double umbilical cord blood transplant (DUCBT) less than 18 yrs	Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Measure Participants	1	8	3	15
Number [participants]	1 11.1%	5 27.8%	1 3.7%	9 NaN

Adverse Events

	SUCBT < 18 Yrs		SUCBT >= 18 Yrs		DUCBT < 18 Yrs		DUCBT >= 18 Yrs		SUCBT		DUCBT
Time Frame
Adverse Event Reporting Description

Arm/Group Title	SUCBT < 18 Yrs		SUCBT >= 18 Yrs		DUCBT < 18 Yrs		DUCBT >= 18 Yrs		SUCBT		DUCBT
Arm/Group Description	Single umbilical cord blood transplant(SUCBT) less than 18 yrs		Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs		Double umbilical cord blood transplant (DUCBT) less than 18 yrs		Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs		Single umbilical cord blood transplant (SUCBT) This is the combination of the SUCBT<18 yrs and SUCBT >= 18 yrs		Double umbilical cord blood transplant (DUCBT) This is the combination of the DUCBT<18 yrs and DUCBT >= 18 yrs
All Cause Mortality
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)		/ (NaN)		/ (NaN)		/ (NaN)		/ (NaN)
Serious Adverse Events
	SUCBT < 18 Yrs		SUCBT >= 18 Yrs		DUCBT < 18 Yrs		DUCBT >= 18 Yrs		SUCBT		DUCBT
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/1 (100%)		7/8 (87.5%)		3/3 (100%)		14/15 (93.3%)		8/9 (88.9%)		17/18 (94.4%)
Blood and lymphatic system disorders
Febrile neutropenia	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Thrombotic thrombocytopenic purpura	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Gastrointestinal disorders
Gastrointestinal haemorrhage	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Impaired gastric emptying	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Nausea	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Hepatobiliary disorders
Cholecystitis	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Immune system disorders
Acute graft versus host disease	0/1 (0%)		5/8 (62.5%)		2/3 (66.7%)		8/15 (53.3%)		5/9 (55.6%)		10/18 (55.6%)
Acute graft versus host disease in skin	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Chronic graft versus host disease	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		2/15 (13.3%)		1/9 (11.1%)		2/18 (11.1%)
Engraftment syndrome	0/1 (0%)		0/8 (0%)		0/3 (0%)		3/15 (20%)		0/9 (0%)		3/18 (16.7%)
Infections and infestations
Adenovirus infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Aspergillus infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
BK virus infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Candida pneumonia	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Cerebral aspergillosis	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Cytomegalovirus viraemia	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Encephalitis	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Enterobacter infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Enterococcal bacteraemia	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Enterococcal infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Human herpesvirus 6 infection	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		2/15 (13.3%)		0/9 (0%)		3/18 (16.7%)
Influenza	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Meningitis enterococcal	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Meningitis staphylococcal	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Oral herpes	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Parainfluenzae virus infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Pneumonia	0/1 (0%)		1/8 (12.5%)		1/3 (33.3%)		2/15 (13.3%)		1/9 (11.1%)		3/18 (16.7%)
Pneumonia cytomegaloviral	0/1 (0%)		0/8 (0%)		0/3 (0%)		3/15 (20%)		0/9 (0%)		3/18 (16.7%)
Pneumonia fungal	0/1 (0%)		2/8 (25%)		0/3 (0%)		2/15 (13.3%)		2/9 (22.2%)		2/18 (11.1%)
Pneumonia streptococcal	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Rhinovirus infection	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Sepsis	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Sinusitis	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Staphylococcal bacteraemia	0/1 (0%)		0/8 (0%)		2/3 (66.7%)		0/15 (0%)		0/9 (0%)		2/18 (11.1%)
Staphylococcal sepsis	0/1 (0%)		2/8 (25%)		0/3 (0%)		0/15 (0%)		2/9 (22.2%)		0/18 (0%)
Streptococcal bacteraemia	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Tooth abscess	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Viral myocarditis	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Injury, poisoning and procedural complications
Overdose	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Musculoskeletal and connective tissue disorders
Back pain	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute myeloid leukaemia recurrent	0/1 (0%)		2/8 (25%)		0/3 (0%)		0/15 (0%)		2/9 (22.2%)		0/18 (0%)
Leukaemia recurrent	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Nervous system disorders
Cognitive disorder	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Encephalopathy	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Leukoencephalopathy	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Renal and urinary disorders
Acute kidney injury	0/1 (0%)		0/8 (0%)		0/3 (0%)		5/15 (33.3%)		0/9 (0%)		5/18 (27.8%)
Cystitis haemorrhagic	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Urinary bladder haemorrhage	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome	0/1 (0%)		0/8 (0%)		0/3 (0%)		3/15 (20%)		0/9 (0%)		3/18 (16.7%)
Epistaxis	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Pulmonary alveolar haemorrhage	0/1 (0%)		2/8 (25%)		0/3 (0%)		4/15 (26.7%)		2/9 (22.2%)		4/18 (22.2%)
Vascular disorders
Hypotension	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Venoocclusive disease	1/1 (100%)		0/8 (0%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Other (Not Including Serious) Adverse Events
	SUCBT < 18 Yrs		SUCBT >= 18 Yrs		DUCBT < 18 Yrs		DUCBT >= 18 Yrs		SUCBT		DUCBT
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/1 (100%)		8/8 (100%)		3/3 (100%)		15/15 (100%)		9/9 (100%)		18/18 (100%)
Blood and lymphatic system disorders
Febrile neutropenia	1/1 (100%)		4/8 (50%)		2/3 (66.7%)		12/15 (80%)		5/9 (55.6%)		14/18 (77.8%)
Cardiac disorders
Atrial fibrillation	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Sinus tachycardia	0/1 (0%)		1/8 (12.5%)		1/3 (33.3%)		1/15 (6.7%)		1/9 (11.1%)		2/18 (11.1%)
Tachycardia	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		2/15 (13.3%)		1/9 (11.1%)		2/18 (11.1%)
Eye disorders
Eye pain	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Gastrointestinal disorders
Abdominal pain	0/1 (0%)		2/8 (25%)		0/3 (0%)		3/15 (20%)		2/9 (22.2%)		3/18 (16.7%)
Constipation	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Diarrhoea	0/1 (0%)		2/8 (25%)		0/3 (0%)		5/15 (33.3%)		2/9 (22.2%)		5/18 (27.8%)
Dyspepsia	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Gastritis	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Nausea	1/1 (100%)		2/8 (25%)		1/3 (33.3%)		2/15 (13.3%)		3/9 (33.3%)		3/18 (16.7%)
Stomatitis	1/1 (100%)		7/8 (87.5%)		1/3 (33.3%)		10/15 (66.7%)		8/9 (88.9%)		11/18 (61.1%)
Vomiting	0/1 (0%)		0/8 (0%)		2/3 (66.7%)		3/15 (20%)		0/9 (0%)		5/18 (27.8%)
General disorders
Chills	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		6/15 (40%)		1/9 (11.1%)		6/18 (33.3%)
Face oedema	0/1 (0%)		1/8 (12.5%)		1/3 (33.3%)		1/15 (6.7%)		1/9 (11.1%)		2/18 (11.1%)
Oedema peripheral	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Pain	0/1 (0%)		0/8 (0%)		2/3 (66.7%)		0/15 (0%)		0/9 (0%)		2/18 (11.1%)
Hepatobiliary disorders
Hyperbilirubinaemia	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Infections and infestations
Adenovirus infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
BK virus infection	0/1 (0%)		3/8 (37.5%)		1/3 (33.3%)		4/15 (26.7%)		3/9 (33.3%)		5/18 (27.8%)
Bacillus bacteraemia	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Bacteraemia	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Clostridium difficile colitis	1/1 (100%)		0/8 (0%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Clostridium difficile infection	0/1 (0%)		1/8 (12.5%)		1/3 (33.3%)		2/15 (13.3%)		1/9 (11.1%)		3/18 (16.7%)
Corynebacterium bacteraemia	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		1/15 (6.7%)		1/9 (11.1%)		1/18 (5.6%)
Cytomegalovirus viraemia	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		4/15 (26.7%)		0/9 (0%)		5/18 (27.8%)
Enterococcal bacteraemia	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Enterococcal infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Folliculitis	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Granulicatella infection	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Human herpesvirus 6 infection	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		2/15 (13.3%)		0/9 (0%)		3/18 (16.7%)
Influenza	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Klebsiella bacteraemia	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Lung infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Moraxella infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Mycobacterium avium complex infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Osteomyelitis viral	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Pneumonia fungal	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		1/15 (6.7%)		0/9 (0%)		2/18 (11.1%)
Sinusitis	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		1/15 (6.7%)		1/9 (11.1%)		1/18 (5.6%)
Staphylococcal bacteraemia	0/1 (0%)		4/8 (50%)		2/3 (66.7%)		6/15 (40%)		4/9 (44.4%)		8/18 (44.4%)
Staphylococcal infection	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Urinary tract infection enterococcal	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Investigations
Alanine aminotransferase increased	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Electrocardiogram QT prolonged	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
International normalised ratio increased	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Metabolism and nutrition disorders
Decreased appetite	1/1 (100%)		4/8 (50%)		3/3 (100%)		10/15 (66.7%)		5/9 (55.6%)		13/18 (72.2%)
Fluid overload	1/1 (100%)		4/8 (50%)		1/3 (33.3%)		6/15 (40%)		5/9 (55.6%)		7/18 (38.9%)
Hyperglycaemia	0/1 (0%)		0/8 (0%)		0/3 (0%)		3/15 (20%)		0/9 (0%)		3/18 (16.7%)
Hyponatraemia	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Musculoskeletal and connective tissue disorders
Arthralgia	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Musculoskeletal pain	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Pain in extremity	1/1 (100%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		1/9 (11.1%)		2/18 (11.1%)
Nervous system disorders
Dizziness	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Headache	0/1 (0%)		4/8 (50%)		1/3 (33.3%)		7/15 (46.7%)		4/9 (44.4%)		8/18 (44.4%)
Neuropathy peripheral	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Restless legs syndrome	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Psychiatric disorders
Anxiety	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		2/15 (13.3%)		1/9 (11.1%)		2/18 (11.1%)
Depression	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Insomnia	0/1 (0%)		2/8 (25%)		0/3 (0%)		6/15 (40%)		2/9 (22.2%)		6/18 (33.3%)
Restlessness	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Substance-induced psychotic disorder	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Renal and urinary disorders
Haematuria	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Urinary incontinence	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Urinary retention	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Reproductive system and breast disorders
Menstruation irregular	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Penile pain	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Scrotal oedema	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Respiratory, thoracic and mediastinal disorders
Dyspnoea	0/1 (0%)		0/8 (0%)		0/3 (0%)		2/15 (13.3%)		0/9 (0%)		2/18 (11.1%)
Epistaxis	0/1 (0%)		2/8 (25%)		0/3 (0%)		2/15 (13.3%)		2/9 (22.2%)		2/18 (11.1%)
Hiccups	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		2/15 (13.3%)		1/9 (11.1%)		2/18 (11.1%)
Hypoxia	0/1 (0%)		1/8 (12.5%)		1/3 (33.3%)		2/15 (13.3%)		1/9 (11.1%)		3/18 (16.7%)
Nasal congestion	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Oropharyngeal pain	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Pleural effusion	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Pulmonary oedema	0/1 (0%)		0/8 (0%)		0/3 (0%)		1/15 (6.7%)		0/9 (0%)		1/18 (5.6%)
Tachypnoea	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Skin and subcutaneous tissue disorders
Angioedema	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		0/15 (0%)		1/9 (11.1%)		0/18 (0%)
Rash	0/1 (0%)		1/8 (12.5%)		0/3 (0%)		5/15 (33.3%)		1/9 (11.1%)		5/18 (27.8%)
Vascular disorders
Haematoma	0/1 (0%)		0/8 (0%)		1/3 (33.3%)		0/15 (0%)		0/9 (0%)		1/18 (5.6%)
Hypertension	0/1 (0%)		6/8 (75%)		1/3 (33.3%)		10/15 (66.7%)		6/9 (66.7%)		11/18 (61.1%)
Hypotension	0/1 (0%)		2/8 (25%)		2/3 (66.7%)		0/15 (0%)		2/9 (22.2%)		2/18 (11.1%)

Limitations/Caveats

[Not Specified]

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Certain Agreements

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

The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.

Results Point of Contact

Name/Title	Study Director
Organization	Novartis Pharmaceuticals
Phone	862-778-8300
Email

Responsible Party:

Novartis Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT01474681

Other Study ID Numbers:

CHSC835X2201

First Posted:

Nov 18, 2011

Last Update Posted:

Dec 30, 2020

Last Verified:

Mar 1, 2019

Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

Study Details

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Results Point of Contact