Acute Lymphocytic Leukemia, Chronic Lymphocytic Leukemia, Multiple Myeloma, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Non-myeloablative Conditioning, Single Umbilical Cord Blood Transplantation
Conditions
Keywords
HSC835, single umbilical cord blood transplantation, non-myeloablative conditioning regimen, Umbilical cord blood, UCB transplantation
Brief summary
This study was designed to evaluate the safety and tolerability of HSC835 for clinical use as measured by the absence of graft failure at day 42 in excess of that currently observed with double umbilical cord blood (UCB) transplantation (DUCBT) with non-myeloablative (NMA) conditioning.
Interventions
DRUGHSC835
HSC835 is a stem cell therapy product providing a source of Hematopoietic stem cell (HSC).
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients with a diagnosis that qualifies them for UCBT * 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Absence of Graft Failure at Day 42 | 42 days | This endpoint was to study safety and tolerability of HSC835 as measured by the absence of graft failure at day 42 in excess of that currently observed with double umbilical cord blood (UCB) transplantation (DUCBT) with non-myeloablative (NMA) conditioning. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Neutrophil Recovery Within 42 Days | 42 days | Engraftment is defined as the first of three consecutive days with ANC \> 0.5 x 109/L. |
| Incidence of Non-relapse Mortality (NRM) Within 100 Days and One Year | 1 year | NRM includes all patients who died from any other cause except relapse of the underlying disease during the study duration. |
| Incidence of Overall Survival Within One Year | 1 year | Overall survival is the proportion of patients who were alive at the end of the one year study period. |
| Incidence of Relapse-free Survival Within One Year | 1 year | Patients are considered to have achieved relapse-free survival if they had not experienced either relapse or death (of any cause) at the end of the study. |
Countries
United States
Participant flow
Recruitment details
Patients were enrolled into the study until approximately nine (9) patients were evaluable for safety as measured by the absence of infusional toxicity and sustained engraftment at Day 100.
Participants by arm
| Arm | Count |
|---|---|
| HSC835 Patients with hematologic malignancies requiring UCB transplant with a NMA conditioning regimen. | 9 |
| Total | 9 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Abnormal test procedure result(s) | 4 |
| Overall Study | Death | 1 |
Baseline characteristics
| Characteristic | HSC835 |
|---|---|
| Age, Continuous | 59.0 years STANDARD_DEVIATION 13.16 |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 8 / 9 | 6 / 9 |
| serious Total, serious adverse events | 0 / 9 | 9 / 9 |
Outcome results
Primary
Absence of Graft Failure at Day 42
This endpoint was to study safety and tolerability of HSC835 as measured by the absence of graft failure at day 42 in excess of that currently observed with double umbilical cord blood (UCB) transplantation (DUCBT) with non-myeloablative (NMA) conditioning.
Time frame: 42 days
Population: Safety Analysis Set: The Safety analysis included all enrolled patients who were transplanted with HSC835 (any patient with a date for HSC835 transplant).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HSC835 | Absence of Graft Failure at Day 42 | 0 Participants |
Secondary
Incidence of Neutrophil Recovery Within 42 Days
Engraftment is defined as the first of three consecutive days with ANC \> 0.5 x 109/L.
Time frame: 42 days
Population: Safety Analysis Set: The Safety analysis included all enrolled patients who were transplanted with HSC835 (any patient with a date for HSC835 transplant).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HSC835 | Incidence of Neutrophil Recovery Within 42 Days | 9 Participants |
Secondary
Incidence of Non-relapse Mortality (NRM) Within 100 Days and One Year
NRM includes all patients who died from any other cause except relapse of the underlying disease during the study duration.
Time frame: 1 year
Population: Safety Analysis Set: The Safety analysis included all enrolled patients who were transplanted with HSC835 (any patient with a date for HSC835 transplant).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HSC835 | Incidence of Non-relapse Mortality (NRM) Within 100 Days and One Year | 1 Participants |
Secondary
Incidence of Overall Survival Within One Year
Overall survival is the proportion of patients who were alive at the end of the one year study period.
Time frame: 1 year
Population: Safety Analysis Set: The Safety analysis included all enrolled patients who were transplanted with HSC835 (any patient with a date for HSC835 transplant).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HSC835 | Incidence of Overall Survival Within One Year | 5 Participants |
Secondary
Incidence of Relapse-free Survival Within One Year
Patients are considered to have achieved relapse-free survival if they had not experienced either relapse or death (of any cause) at the end of the study.
Time frame: 1 year
Population: Safety Analysis Set: The Safety analysis included all enrolled patients who were transplanted with HSC835 (any patient with a date for HSC835 transplant).
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| HSC835 | Incidence of Relapse-free Survival Within One Year | 4 Participants |