Trial Outcomes & Findings for Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders (NCT NCT01050855)

Results Overview

engraftment of patients with non-malignant disorders will be evaluated using a reduced-intensity conditioning regimen

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

56 participants

Primary outcome timeframe

Post Transplant -100 days

Results posted on

2026-07-21

Participant milestones
Measure	Reduced-Intensity Conditioning (RIC) HSCT Participants receive reduced-intensity conditioning with alemtuzumab-, fludarabine-, and melphalan-based regimens, varying by timing of alemtuzumab (distal or intermediate). Regimens are based on established protocols used in non-malignant disorders. Alemtuzumab is administered prior to other agents to reduce risk of declining donor chimerism. Participants then undergo allogeneic stem cell transplantation with standard post-transplant care.
Overall Study STARTED	56
Overall Study COMPLETED	54
Overall Study NOT COMPLETED	2

Reasons for withdrawal
Measure	Reduced-Intensity Conditioning (RIC) HSCT Participants receive reduced-intensity conditioning with alemtuzumab-, fludarabine-, and melphalan-based regimens, varying by timing of alemtuzumab (distal or intermediate). Regimens are based on established protocols used in non-malignant disorders. Alemtuzumab is administered prior to other agents to reduce risk of declining donor chimerism. Participants then undergo allogeneic stem cell transplantation with standard post-transplant care.
Overall Study Physician Decision	2

Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

Baseline characteristics by cohort
Measure	Reduced-Intensity Conditioning (RIC) HSCT n=54 Participants Participants receive reduced-intensity conditioning with alemtuzumab-, fludarabine-, and melphalan-based regimens, varying by timing of alemtuzumab (distal or intermediate). Regimens are based on established protocols used in non-malignant disorders. Alemtuzumab is administered prior to other agents to reduce risk of declining donor chimerism. Participants then undergo allogeneic stem cell transplantation with standard post-transplant care.
Age, Continuous	7.87 years n=9 Participants
Sex: Female, Male Female	25 Participants n=9 Participants
Sex: Female, Male Male	29 Participants n=9 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=9 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	49 Participants n=9 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	4 Participants n=9 Participants
HLH vs Non-HLH HLH	6 Participants n=9 Participants
HLH vs Non-HLH Non-HLH	48 Participants n=9 Participants

Timeframe: Post Transplant -100 days

engraftment of patients with non-malignant disorders will be evaluated using a reduced-intensity conditioning regimen

Outcome measures
Measure	Reduced-Intensity Conditioning (RIC) HSCT n=54 Participants Participants receive reduced-intensity conditioning with alemtuzumab-, fludarabine-, and melphalan-based regimens, varying by timing of alemtuzumab (distal or intermediate). Regimens are based on established protocols used in non-malignant disorders. Alemtuzumab is administered prior to other agents to reduce risk of declining donor chimerism. Participants then undergo allogeneic stem cell transplantation with standard post-transplant care.
Engraftment	53 Participants

Timeframe: 1 year post transplant

Event-free survival is defined as the time interval to either primary or late graft failure, disease recurrence, or death.

Outcome measures
Measure	Reduced-Intensity Conditioning (RIC) HSCT n=54 Participants Participants receive reduced-intensity conditioning with alemtuzumab-, fludarabine-, and melphalan-based regimens, varying by timing of alemtuzumab (distal or intermediate). Regimens are based on established protocols used in non-malignant disorders. Alemtuzumab is administered prior to other agents to reduce risk of declining donor chimerism. Participants then undergo allogeneic stem cell transplantation with standard post-transplant care.
Event-free Survival	51 Participants

Serious events: 4 serious events

Other events: 0 other events

Deaths: 4 deaths

Serious adverse events
Measure	Reduced-Intensity Conditioning (RIC) HSCT n=54 participants at risk Participants receive reduced-intensity conditioning with alemtuzumab-, fludarabine-, and melphalan-based regimens, varying by timing of alemtuzumab (distal or intermediate). Regimens are based on established protocols used in non-malignant disorders. Alemtuzumab is administered prior to other agents to reduce risk of declining donor chimerism. Participants then undergo allogeneic stem cell transplantation with standard post-transplant care.
Infections and infestations CMV Pneumonitis	1.9% 1/54 • Number of events 1 • Adverse event data were collected from the time of transplant through 2 year (24 months) post-transplant.
Cardiac disorders Pulmonary Hypertension	1.9% 1/54 • Number of events 1 • Adverse event data were collected from the time of transplant through 2 year (24 months) post-transplant.
Respiratory, thoracic and mediastinal disorders Bronchiolitis Obliterans	1.9% 1/54 • Number of events 1 • Adverse event data were collected from the time of transplant through 2 year (24 months) post-transplant.
Vascular disorders Stroke	1.9% 1/54 • Number of events 1 • Adverse event data were collected from the time of transplant through 2 year (24 months) post-transplant.
Nervous system disorders Posterior Reversible Encephalopathy Syndrome	1.9% 1/54 • Number of events 1 • Adverse event data were collected from the time of transplant through 2 year (24 months) post-transplant.

Adverse event data not reported

The Children's Hospital of Philadelphia

Phone: 215-590-2820