Trial Outcomes & Findings for CHP 834 Unrelated and Partially Matched Related Donor Peripheral Stem Cell Transportation for T and B Cell Depletion (NCT NCT00579124)

NCT ID: NCT00579124

Last Updated: 2025-02-03

Results Overview

Successful engraftment will be whether subjects have achieved an absolute neutrophil count (ANC) \>500 and platelet count \>20 x 109/l by 100 days after transplant

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 93 participants
• Primary outcome timeframe: 100 days Post Transplant
• Results posted on: 2025-02-03

Participant Flow

Participant milestones

Measure	1. CliniMACS CD3+/CD19+ Depletion * 6/6 or 8/8 matched (fully matched) * 1 antigen or allele mismatched (mismatch at A or B or DRB1) * 2 antigen or allele mismatched (mismatch ONLY at A and B but NOT at DRB1 plus either A or B). Patients will receive grafts that have undergone CD3+ and CD19+ depletion. The CD3(-) fraction will be infused. CliniMACs: T and B Cell depletion	2. CliniMACS CD3+/CD19+ Depletion Stratum 2. CliniMACS CD3+/CD19+ depletion: * Haploidentical match * 2 antigen and/or allele mismatched where one of the mismatches includes DRB1 For patients in Stratum 2 we will perform CD3+ (T cell) and CD19+ (B cell) depletion. There will be no T cell add back in this stratum. CliniMACs: T and B Cell depletion
Overall Study STARTED	87	6
Overall Study COMPLETED	82	6
Overall Study NOT COMPLETED	5	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

CHP 834 Unrelated and Partially Matched Related Donor Peripheral Stem Cell Transportation for T and B Cell Depletion

Baseline characteristics by cohort

Measure	Stratum 1 n=87 Participants Stratum 1. CliniMACS CD3+/CD19+ depletion: Patients with donors with the following levels of matching: * 6/6 or 8/8 matched (fully matched) * 1 antigen or allele mismatched (mismatch at A or B or DRB1) * 2 antigen or allele mismatched (mismatch ONLY at A and B but NOT at DRB1 plus either A or B).	Stratum 2 n=6 Participants * Haploidentical match * 2 antigen and/or allele mismatched where one of the mismatches includes DRB1	Total n=93 Participants Total of all reporting groups
Age, Categorical <=18 years	80 Participants n=5 Participants	6 Participants n=7 Participants	86 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants	0 Participants n=7 Participants	7 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	12 years n=5 Participants	0.5 years n=7 Participants	12 years n=5 Participants
Sex: Female, Male Female	44 Participants n=5 Participants	0 Participants n=7 Participants	44 Participants n=5 Participants
Sex: Female, Male Male	43 Participants n=5 Participants	6 Participants n=7 Participants	49 Participants n=5 Participants
Region of Enrollment United States	87 Participants n=5 Participants	6 Participants n=7 Participants	93 Participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days Post Transplant

Successful engraftment will be whether subjects have achieved an absolute neutrophil count (ANC) >500 and platelet count >20 x 109/l by 100 days after transplant

Outcome measures

Measure	Stratum 1 n=87 Participants Stratum 1. CliniMACS CD3+/CD19+ depletion: Patients with donors with the following levels of matching: * 6/6 or 8/8 matched (fully matched) * 1 antigen or allele mismatched (mismatch at A or B or DRB1) * 2 antigen or allele mismatched (mismatch ONLY at A and B but NOT at DRB1 plus either A or B).	Stratum 2 n=5 Participants Patients with donors with the following levels of matching: * Haploidentical match * 2 antigen and/or allele mismatched where one of the mismatches includes DRB1
Number of Participants With Successful Engraftment	87 Participants	5 Participants

Adverse Events

AEs for Total Number of Participants: Stratum 1

Serious events: 6 serious events

Other events: 0 other events

Deaths: 0 deaths

AEs for Total Number of Participants: Stratum 2

Serious events: 2 serious events

Other events: 0 other events

Deaths: 2 deaths

Serious adverse events

Measure	AEs for Total Number of Participants: Stratum 1 n=87 participants at risk this group is total subject count evaluated for AE results in Stratum 1	AEs for Total Number of Participants: Stratum 2 n=6 participants at risk this group is total subject count evaluated for AE results in Stratum 2
General disorders Non-relapsed mortality	6.9% 6/87 • Number of events 6	33.3% 2/6 • Number of events 2

Other adverse events

Adverse event data not reported

Additional Information

Patricia Hankins

Children's Hospital of Philadelphia

Phone: 215 590 5168

Email: hankinsp@chop.edu

Results disclosure agreements

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