Trial Outcomes & Findings for Stem Cell Transplant for Inborn Errors of Metabolism (NCT NCT00176904)

Results Overview

Number of patients alive at designated timepoints after transplant.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

135 participants

Primary outcome timeframe

100 Days, 1 Year and 3 Years

Results posted on

2017-12-28

Participant Flow

Participant milestones

Participant milestones
Measure	Patients Treated With Stem Cell Transplant All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Study STARTED	135
Overall Study COMPLETED	135
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplant for Inborn Errors of Metabolism

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patients Treated With Stem Cell Transplant n=135 Participants All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Age, Categorical <=18 years	117 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	18 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	8.7 years STANDARD_DEVIATION 8.9 • n=5 Participants
Sex: Female, Male Female	44 Participants n=5 Participants
Sex: Female, Male Male	91 Participants n=5 Participants
Region of Enrollment United States	135 participants n=5 Participants

PRIMARY outcome

Timeframe: 100 Days, 1 Year and 3 Years

Number of patients alive at designated timepoints after transplant.

Outcome measures

Outcome measures
Measure	Patients Treated With Stem Cell Transplant n=135 Participants All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Survival Day 100	120 Participants
Overall Survival 1 Year	92 Participants
Overall Survival 3 Years	81 Participants

SECONDARY outcome

Timeframe: Day 100

Population: 1 Patient not included due to early death (before day 40).

Number of patients with full donor chimerism (state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease) by Day 100 post-transplant of at least 90%.

Outcome measures

Outcome measures
Measure	Patients Treated With Stem Cell Transplant n=134 Participants All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Overall Donor Engraftment	123 Participants

SECONDARY outcome

Timeframe: Day 100

Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening.

Outcome measures

Outcome measures
Measure	Patients Treated With Stem Cell Transplant n=135 Participants All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease	34 Participants

SECONDARY outcome

Timeframe: Day 100

Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening.

Outcome measures

Outcome measures
Measure	Patients Treated With Stem Cell Transplant n=135 Participants All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease	13 Participants

SECONDARY outcome

Timeframe: 1 Year Post Transplant

Number of patients who exhibited chronic graft-versus-host disease by 1 Year post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Chronic GVHD is an extension of this syndrome.

Outcome measures

Outcome measures
Measure	Patients Treated With Stem Cell Transplant n=135 Participants All patients treated with protocol regimen (chemotherapy and stem cell transplant).
Number of Patients With Chronic Graft-Versus-Host Disease	13 Participants

Adverse Events

Patients Treated With Stem Cell Transplant

Serious events: 62 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Patients Treated With Stem Cell Transplant n=135 participants at risk All patients treated with protocol regimen (chemotherapy and stem cell transplant).
General disorders Auto recovery	6.7% 9/135 • Number of events 9 • Serious adverse experiences were collected during the first 100 days after transplant then 6 months and annually for 3 years. Selected serious adverse experiences (graft failure/autologous recovery, severe acute GVHD (grades III and IV) and death were collected during the first 100 days after transplant then 6 months and annually for 3 years. After day 100, only death or an unexpected adverse event will be reported. Adverse events were not collected in this study.
General disorders Death	40.0% 54/135 • Number of events 54 • Serious adverse experiences were collected during the first 100 days after transplant then 6 months and annually for 3 years. Selected serious adverse experiences (graft failure/autologous recovery, severe acute GVHD (grades III and IV) and death were collected during the first 100 days after transplant then 6 months and annually for 3 years. After day 100, only death or an unexpected adverse event will be reported. Adverse events were not collected in this study.
Blood and lymphatic system disorders Primary graft failure	1.5% 2/135 • Number of events 2 • Serious adverse experiences were collected during the first 100 days after transplant then 6 months and annually for 3 years. Selected serious adverse experiences (graft failure/autologous recovery, severe acute GVHD (grades III and IV) and death were collected during the first 100 days after transplant then 6 months and annually for 3 years. After day 100, only death or an unexpected adverse event will be reported. Adverse events were not collected in 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: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place