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Trial Outcomes & Findings for Safety and Tolerability of HSC835 in Patients With Hematological Malignancies (NCT NCT01474681)

NCT ID: NCT01474681

Last Updated: 2020-12-30

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

27 participants

Primary outcome timeframe

32 days

Results posted on

2020-12-30

Participant Flow

Single arm study of HSC835 broken down into 4 subgroups for analyses and 6 subgroups for safety.

Participant milestones

Participant milestones
Measure
SUCBT < 18 Yrs
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Overall Study
STARTED
1
8
3
15
Overall Study
COMPLETED
1
5
1
9
Overall Study
NOT COMPLETED
0
3
2
6

Reasons for withdrawal

Reasons for withdrawal
Measure
SUCBT < 18 Yrs
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Overall Study
Death
0
3
2
6

Baseline Characteristics

Safety and Tolerability of HSC835 in Patients With Hematological Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
SUCBT
n=9 Participants
Single umbilical cord blood transplant (SUCBT) This is the combination of the SUCBT\<18 yrs and SUCBT \>= 18 yrs
DUCBT
n=18 Participants
Double umbilical cord blood transplant (DUCBT) This is the combination of the DUCBT\<18 yrs and DUCBT \>= 18 yrs
Total
n=27 Participants
Total of all reporting groups
Age, Continuous
33.3 years
STANDARD_DEVIATION 17.18 • n=5 Participants
31.3 years
STANDARD_DEVIATION 13.75 • n=7 Participants
32.0 years
STANDARD_DEVIATION 14.67 • n=5 Participants
Sex: Female, Male
Female
5 Participants
n=5 Participants
6 Participants
n=7 Participants
11 Participants
n=5 Participants
Sex: Female, Male
Male
4 Participants
n=5 Participants
12 Participants
n=7 Participants
16 Participants
n=5 Participants

PRIMARY outcome

Timeframe: 32 days

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

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.

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=1 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 Participants
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 Participants
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
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.
Infusional Toxicity 48 hrs after transplant
0 participants
0 participants
0 participants
1 participants
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.
Absence of graft failure after 32 days
0 participants
0 participants
0 participants
0 participants

SECONDARY outcome

Timeframe: 42 days

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

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.

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=1 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 Participants
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 Participants
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Incidence of Neutrophil Recovery Within 42 Days
1 participants
8 participants
3 participants
15 participants

SECONDARY outcome

Timeframe: 6 months

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

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.

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=1 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 Participants
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 Participants
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Incidence of Platelet Recovery Within Six Months
1 participants
6 participants
2 participants
11 participants

SECONDARY outcome

Timeframe: Day 100

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

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

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=3 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=15 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)
Predominance of HSC835
0 participants
6 participants
—
—
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)
Mixed dominance
3 participants
3 participants
—
—
Frequency of Expanded Unit Predominance at Day 100 (DUCBT Recipients Only)
Predominance of the unmaninpulated unit
0 participants
6 participants
—
—

SECONDARY outcome

Timeframe: Day 100 and Month 12

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

Number of participants with incidence of transplant related mortality (TRM) within 100 days and one year

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=1 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 Participants
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 Participants
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year
Day 100
0 participants
1 participants
1 participants
3 participants
Incidence of Transplant Related Mortality (TRM) Within 100 Days and One Year
Month 12
0 participants
1 participants
1 participants
6 participants

SECONDARY outcome

Timeframe: Day 100 and Monnth 12

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

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

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=1 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 Participants
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 Participants
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year
aGVHD-Day 100
0 participants
5 participants
3 participants
8 participants
Incidence of Acute Graft Versus Host Disease (aGVHD) Within 100 Days and Chronic Graft Versus Host Disease (cGVHD) Within 1 Year
cGVHD-Month12
0 participants
1 participants
0 participants
2 participants

SECONDARY outcome

Timeframe: Month 12

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

Number of participants with Incidence of relapse within one year

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=1 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 Participants
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 Participants
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Incidence of Relapse Within One Year
0 participants
2 participants
1 participants
0 participants

SECONDARY outcome

Timeframe: Month 12

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

Number of participants with Overall survival (OS) within one year

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=1 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 Participants
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 Participants
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Overall Survival (OS) Within One Year
1 participants
5 participants
1 participants
9 participants

SECONDARY outcome

Timeframe: Month 12

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

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)

Outcome measures

Outcome measures
Measure
SUCBT < 18 Yrs
n=1 Participants
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 Participants
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 Participants
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 Participants
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
Disease Free Survival (DFS) Within One Year
1 participants
5 participants
1 participants
9 participants

Adverse Events

SUCBT < 18 Yrs

Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths

SUCBT >= 18 Yrs

Serious events: 7 serious events
Other events: 8 other events
Deaths: 0 deaths

DUCBT < 18 Yrs

Serious events: 3 serious events
Other events: 3 other events
Deaths: 0 deaths

DUCBT >= 18 Yrs

Serious events: 14 serious events
Other events: 15 other events
Deaths: 0 deaths

SUCBT

Serious events: 8 serious events
Other events: 9 other events
Deaths: 0 deaths

DUCBT

Serious events: 17 serious events
Other events: 18 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
SUCBT < 18 Yrs
n=1 participants at risk
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 participants at risk
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 participants at risk
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 participants at risk
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
SUCBT
n=9 participants at risk
Single umbilical cord blood transplant (SUCBT) This is the combination of the SUCBT\<18 yrs and SUCBT \>= 18 yrs
DUCBT
n=18 participants at risk
Double umbilical cord blood transplant (DUCBT) This is the combination of the DUCBT\<18 yrs and DUCBT \>= 18 yrs
Blood and lymphatic system disorders
Febrile neutropenia
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Blood and lymphatic system disorders
Thrombotic thrombocytopenic purpura
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Gastrointestinal disorders
Gastrointestinal haemorrhage
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Gastrointestinal disorders
Impaired gastric emptying
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Gastrointestinal disorders
Nausea
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Hepatobiliary disorders
Cholecystitis
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Immune system disorders
Acute graft versus host disease
0.00%
0/1
62.5%
5/8
66.7%
2/3
53.3%
8/15
55.6%
5/9
55.6%
10/18
Immune system disorders
Acute graft versus host disease in skin
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Immune system disorders
Chronic graft versus host disease
0.00%
0/1
12.5%
1/8
0.00%
0/3
13.3%
2/15
11.1%
1/9
11.1%
2/18
Immune system disorders
Engraftment syndrome
0.00%
0/1
0.00%
0/8
0.00%
0/3
20.0%
3/15
0.00%
0/9
16.7%
3/18
Infections and infestations
Adenovirus infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Aspergillus infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
BK virus infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Candida pneumonia
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Infections and infestations
Cerebral aspergillosis
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Cytomegalovirus viraemia
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Encephalitis
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Enterobacter infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Enterococcal bacteraemia
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Enterococcal infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Human herpesvirus 6 infection
0.00%
0/1
0.00%
0/8
33.3%
1/3
13.3%
2/15
0.00%
0/9
16.7%
3/18
Infections and infestations
Influenza
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Meningitis enterococcal
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Meningitis staphylococcal
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Oral herpes
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Parainfluenzae virus infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Pneumonia
0.00%
0/1
12.5%
1/8
33.3%
1/3
13.3%
2/15
11.1%
1/9
16.7%
3/18
Infections and infestations
Pneumonia cytomegaloviral
0.00%
0/1
0.00%
0/8
0.00%
0/3
20.0%
3/15
0.00%
0/9
16.7%
3/18
Infections and infestations
Pneumonia fungal
0.00%
0/1
25.0%
2/8
0.00%
0/3
13.3%
2/15
22.2%
2/9
11.1%
2/18
Infections and infestations
Pneumonia streptococcal
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Infections and infestations
Rhinovirus infection
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Sepsis
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Infections and infestations
Sinusitis
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Staphylococcal bacteraemia
0.00%
0/1
0.00%
0/8
66.7%
2/3
0.00%
0/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Staphylococcal sepsis
0.00%
0/1
25.0%
2/8
0.00%
0/3
0.00%
0/15
22.2%
2/9
0.00%
0/18
Infections and infestations
Streptococcal bacteraemia
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Tooth abscess
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Infections and infestations
Viral myocarditis
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Injury, poisoning and procedural complications
Overdose
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Musculoskeletal and connective tissue disorders
Back pain
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute myeloid leukaemia recurrent
0.00%
0/1
25.0%
2/8
0.00%
0/3
0.00%
0/15
22.2%
2/9
0.00%
0/18
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leukaemia recurrent
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Nervous system disorders
Cognitive disorder
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Nervous system disorders
Encephalopathy
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Nervous system disorders
Leukoencephalopathy
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Renal and urinary disorders
Acute kidney injury
0.00%
0/1
0.00%
0/8
0.00%
0/3
33.3%
5/15
0.00%
0/9
27.8%
5/18
Renal and urinary disorders
Cystitis haemorrhagic
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Renal and urinary disorders
Urinary bladder haemorrhage
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Respiratory, thoracic and mediastinal disorders
Acute respiratory distress syndrome
0.00%
0/1
0.00%
0/8
0.00%
0/3
20.0%
3/15
0.00%
0/9
16.7%
3/18
Respiratory, thoracic and mediastinal disorders
Epistaxis
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Respiratory, thoracic and mediastinal disorders
Pulmonary alveolar haemorrhage
0.00%
0/1
25.0%
2/8
0.00%
0/3
26.7%
4/15
22.2%
2/9
22.2%
4/18
Vascular disorders
Hypotension
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Vascular disorders
Venoocclusive disease
100.0%
1/1
0.00%
0/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18

Other adverse events

Other adverse events
Measure
SUCBT < 18 Yrs
n=1 participants at risk
Single umbilical cord blood transplant(SUCBT) less than 18 yrs
SUCBT >= 18 Yrs
n=8 participants at risk
Single umbilical cord blood transplant (SUCBT) greater than or equal to 18 yrs
DUCBT < 18 Yrs
n=3 participants at risk
Double umbilical cord blood transplant (DUCBT) less than 18 yrs
DUCBT >= 18 Yrs
n=15 participants at risk
Double umbilical cord blood transplant (DUCBT) greater than or equal to 18 yrs
SUCBT
n=9 participants at risk
Single umbilical cord blood transplant (SUCBT) This is the combination of the SUCBT\<18 yrs and SUCBT \>= 18 yrs
DUCBT
n=18 participants at risk
Double umbilical cord blood transplant (DUCBT) This is the combination of the DUCBT\<18 yrs and DUCBT \>= 18 yrs
Blood and lymphatic system disorders
Febrile neutropenia
100.0%
1/1
50.0%
4/8
66.7%
2/3
80.0%
12/15
55.6%
5/9
77.8%
14/18
Cardiac disorders
Atrial fibrillation
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Cardiac disorders
Sinus tachycardia
0.00%
0/1
12.5%
1/8
33.3%
1/3
6.7%
1/15
11.1%
1/9
11.1%
2/18
Cardiac disorders
Tachycardia
0.00%
0/1
12.5%
1/8
0.00%
0/3
13.3%
2/15
11.1%
1/9
11.1%
2/18
Eye disorders
Eye pain
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Gastrointestinal disorders
Abdominal pain
0.00%
0/1
25.0%
2/8
0.00%
0/3
20.0%
3/15
22.2%
2/9
16.7%
3/18
Gastrointestinal disorders
Constipation
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Gastrointestinal disorders
Diarrhoea
0.00%
0/1
25.0%
2/8
0.00%
0/3
33.3%
5/15
22.2%
2/9
27.8%
5/18
Gastrointestinal disorders
Dyspepsia
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Gastrointestinal disorders
Gastritis
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Gastrointestinal disorders
Nausea
100.0%
1/1
25.0%
2/8
33.3%
1/3
13.3%
2/15
33.3%
3/9
16.7%
3/18
Gastrointestinal disorders
Stomatitis
100.0%
1/1
87.5%
7/8
33.3%
1/3
66.7%
10/15
88.9%
8/9
61.1%
11/18
Gastrointestinal disorders
Vomiting
0.00%
0/1
0.00%
0/8
66.7%
2/3
20.0%
3/15
0.00%
0/9
27.8%
5/18
General disorders
Chills
0.00%
0/1
12.5%
1/8
0.00%
0/3
40.0%
6/15
11.1%
1/9
33.3%
6/18
General disorders
Face oedema
0.00%
0/1
12.5%
1/8
33.3%
1/3
6.7%
1/15
11.1%
1/9
11.1%
2/18
General disorders
Oedema peripheral
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
General disorders
Pain
0.00%
0/1
0.00%
0/8
66.7%
2/3
0.00%
0/15
0.00%
0/9
11.1%
2/18
Hepatobiliary disorders
Hyperbilirubinaemia
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Adenovirus infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Infections and infestations
BK virus infection
0.00%
0/1
37.5%
3/8
33.3%
1/3
26.7%
4/15
33.3%
3/9
27.8%
5/18
Infections and infestations
Bacillus bacteraemia
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Infections and infestations
Bacteraemia
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Clostridium difficile colitis
100.0%
1/1
0.00%
0/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Infections and infestations
Clostridium difficile infection
0.00%
0/1
12.5%
1/8
33.3%
1/3
13.3%
2/15
11.1%
1/9
16.7%
3/18
Infections and infestations
Corynebacterium bacteraemia
0.00%
0/1
12.5%
1/8
0.00%
0/3
6.7%
1/15
11.1%
1/9
5.6%
1/18
Infections and infestations
Cytomegalovirus viraemia
0.00%
0/1
0.00%
0/8
33.3%
1/3
26.7%
4/15
0.00%
0/9
27.8%
5/18
Infections and infestations
Enterococcal bacteraemia
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Enterococcal infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Folliculitis
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Granulicatella infection
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Human herpesvirus 6 infection
0.00%
0/1
0.00%
0/8
33.3%
1/3
13.3%
2/15
0.00%
0/9
16.7%
3/18
Infections and infestations
Influenza
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Klebsiella bacteraemia
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Infections and infestations
Lung infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Moraxella infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Mycobacterium avium complex infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Osteomyelitis viral
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Infections and infestations
Pneumonia fungal
0.00%
0/1
0.00%
0/8
33.3%
1/3
6.7%
1/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Sinusitis
0.00%
0/1
12.5%
1/8
0.00%
0/3
6.7%
1/15
11.1%
1/9
5.6%
1/18
Infections and infestations
Staphylococcal bacteraemia
0.00%
0/1
50.0%
4/8
66.7%
2/3
40.0%
6/15
44.4%
4/9
44.4%
8/18
Infections and infestations
Staphylococcal infection
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Infections and infestations
Urinary tract infection enterococcal
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Investigations
Alanine aminotransferase increased
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Investigations
Electrocardiogram QT prolonged
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Investigations
International normalised ratio increased
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Metabolism and nutrition disorders
Decreased appetite
100.0%
1/1
50.0%
4/8
100.0%
3/3
66.7%
10/15
55.6%
5/9
72.2%
13/18
Metabolism and nutrition disorders
Fluid overload
100.0%
1/1
50.0%
4/8
33.3%
1/3
40.0%
6/15
55.6%
5/9
38.9%
7/18
Metabolism and nutrition disorders
Hyperglycaemia
0.00%
0/1
0.00%
0/8
0.00%
0/3
20.0%
3/15
0.00%
0/9
16.7%
3/18
Metabolism and nutrition disorders
Hyponatraemia
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Musculoskeletal and connective tissue disorders
Arthralgia
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Musculoskeletal and connective tissue disorders
Pain in extremity
100.0%
1/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
11.1%
1/9
11.1%
2/18
Nervous system disorders
Dizziness
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Nervous system disorders
Headache
0.00%
0/1
50.0%
4/8
33.3%
1/3
46.7%
7/15
44.4%
4/9
44.4%
8/18
Nervous system disorders
Neuropathy peripheral
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Nervous system disorders
Restless legs syndrome
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Psychiatric disorders
Anxiety
0.00%
0/1
12.5%
1/8
0.00%
0/3
13.3%
2/15
11.1%
1/9
11.1%
2/18
Psychiatric disorders
Depression
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Psychiatric disorders
Insomnia
0.00%
0/1
25.0%
2/8
0.00%
0/3
40.0%
6/15
22.2%
2/9
33.3%
6/18
Psychiatric disorders
Restlessness
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Psychiatric disorders
Substance-induced psychotic disorder
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Renal and urinary disorders
Haematuria
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Renal and urinary disorders
Urinary incontinence
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Renal and urinary disorders
Urinary retention
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Reproductive system and breast disorders
Menstruation irregular
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Reproductive system and breast disorders
Penile pain
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Reproductive system and breast disorders
Scrotal oedema
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Respiratory, thoracic and mediastinal disorders
Dyspnoea
0.00%
0/1
0.00%
0/8
0.00%
0/3
13.3%
2/15
0.00%
0/9
11.1%
2/18
Respiratory, thoracic and mediastinal disorders
Epistaxis
0.00%
0/1
25.0%
2/8
0.00%
0/3
13.3%
2/15
22.2%
2/9
11.1%
2/18
Respiratory, thoracic and mediastinal disorders
Hiccups
0.00%
0/1
12.5%
1/8
0.00%
0/3
13.3%
2/15
11.1%
1/9
11.1%
2/18
Respiratory, thoracic and mediastinal disorders
Hypoxia
0.00%
0/1
12.5%
1/8
33.3%
1/3
13.3%
2/15
11.1%
1/9
16.7%
3/18
Respiratory, thoracic and mediastinal disorders
Nasal congestion
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Respiratory, thoracic and mediastinal disorders
Pleural effusion
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
0.00%
0/1
0.00%
0/8
0.00%
0/3
6.7%
1/15
0.00%
0/9
5.6%
1/18
Respiratory, thoracic and mediastinal disorders
Tachypnoea
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Skin and subcutaneous tissue disorders
Angioedema
0.00%
0/1
12.5%
1/8
0.00%
0/3
0.00%
0/15
11.1%
1/9
0.00%
0/18
Skin and subcutaneous tissue disorders
Rash
0.00%
0/1
12.5%
1/8
0.00%
0/3
33.3%
5/15
11.1%
1/9
27.8%
5/18
Vascular disorders
Haematoma
0.00%
0/1
0.00%
0/8
33.3%
1/3
0.00%
0/15
0.00%
0/9
5.6%
1/18
Vascular disorders
Hypertension
0.00%
0/1
75.0%
6/8
33.3%
1/3
66.7%
10/15
66.7%
6/9
61.1%
11/18
Vascular disorders
Hypotension
0.00%
0/1
25.0%
2/8
66.7%
2/3
0.00%
0/15
22.2%
2/9
11.1%
2/18

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Results disclosure agreements

  • Principal investigator is a sponsor employee 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.
  • Publication restrictions are in place

Restriction type: OTHER