Trial Outcomes & Findings for Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism (NCT NCT00668564)

NCT ID: NCT00668564

Last Updated: 2017-12-28

Results Overview

Rate of successful engraftment - patients who achieved and sustained donor engraftment; donor chimerism by day 100 of at least 90% after undergoing hematopoietic stem cell transplantation.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 18 participants
• Primary outcome timeframe: Day 100
• Results posted on: 2017-12-28

Participant Flow

Participant milestones

Measure	Intent-to-Treat All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous \[IV\] days 1-4 prior to transplant); Busulfan (if \< or = 12 kg: 1.1 mg/kg or if \> 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
Overall Study STARTED	18
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Baseline characteristics by cohort

Measure	Intent-to-Treat n=18 Participants All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous \[IV\] days 1-4 prior to transplant); Busulfan (if \< or = 12 kg: 1.1 mg/kg or if \> 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
Age, Categorical <=18 years	18 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	4.7 years STANDARD_DEVIATION 4.3 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	11 Participants n=5 Participants
Region of Enrollment United States	18 participants n=5 Participants

PRIMARY outcome

Timeframe: Day 100

Rate of successful engraftment - patients who achieved and sustained donor engraftment; donor chimerism by day 100 of at least 90% after undergoing hematopoietic stem cell transplantation.

Outcome measures

Measure	Intent-to-Treat n=18 Participants All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous \[IV\] days 1-4 prior to transplant); Busulfan (if \< or = 12 kg: 1.1 mg/kg or if \> 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
Number of Patients Achieving Engraftment	14 Participants

SECONDARY outcome

Timeframe: Day 100, 1 Year, 3 Years

Population: Year 3 survival endpoint was not done due to study being terminated prematurely.

Number of patients alive at timepoints.

Outcome measures

Measure	Intent-to-Treat n=18 Participants All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous \[IV\] days 1-4 prior to transplant); Busulfan (if \< or = 12 kg: 1.1 mg/kg or if \> 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
Overall Survival Day 100	14 Participants
Overall Survival 1 Year	12 Participants

Adverse Events

Intent-to-Treat

Serious events: 8 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Intent-to-Treat n=18 participants at risk All patients treated with study regimen; Bone Marrow Transplant - cord blood transplant; Cyclophosphamide (50 mg/kg intravenous \[IV\] days 1-4 prior to transplant); Busulfan (if \< or = 12 kg: 1.1 mg/kg or if \> 12 kg: 0.8 mg/kg IV every 6 hours on days 6-9 before transplant) and Campath-1H (once per day 0.3 mg/kg IV on days 10-12 before transplant.
General disorders Death	33.3% 6/18 • Number of events 6 • Day 1 through Day 100 post-transplant. Only Serious Adverse Events were collected for this study.
Blood and lymphatic system disorders Graft failure	11.1% 2/18 • Number of events 2 • Day 1 through Day 100 post-transplant. Only Serious Adverse Events were collected for this study.
General disorders Auto recovery	11.1% 2/18 • Number of events 2 • Day 1 through Day 100 post-transplant. Only Serious Adverse Events were collected for this study.

Other adverse events

Adverse event data not reported

Additional Information

Paul Orchard, M.D.

Masonic Cancer Center, University of Minnesota

Phone: 612-626-2313

Email: orcha001@umn.edu

Results disclosure agreements

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