Trial Outcomes & Findings for Reduced Intensity Preparative Regimen Followed by Stem Cell Transplant (FAB) (NCT NCT00579111)

NCT ID: NCT00579111

Last Updated: 2016-05-04

Results Overview

Each patient will be classified as a success or failure. A success will be defined as engraftment of at least 35% of cells 100 days after transplant.

• Recruitment status: TERMINATED
• Study phase: PHASE1/PHASE2
• Target enrollment: 4 participants
• Primary outcome timeframe: 100 days
• Results posted on: 2016-05-04

Participant Flow

Participant milestones

Measure	HLA-identical Sibling Transplant Recipients of HLA identical sibling stem cell transplants	Unrelated Matched or Single Antigen Mismatched Transplant Recipients of unrelated matched or single antigen mismatched donor stem cell transplant or single antigen mismatched family donor stem cell transplants
Overall Study STARTED	3	1
Overall Study COMPLETED	1	0
Overall Study NOT COMPLETED	2	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Reduced Intensity Preparative Regimen Followed by Stem Cell Transplant (FAB)

Baseline characteristics by cohort

Measure	HLA-identical Sibling Transplant n=3 Participants Recipients of HLA identical sibling stem cell transplants	Unrelated Matched or Single Antigen Mismatched Transplant n=1 Participants Recipients of unrelated matched or single antigen mismatched donor stem cell transplant or single antigen mismatched family donor stem cell transplants	Total n=4 Participants Total of all reporting groups
Age, Customized Between 50 and 65 years	3 participants n=5 Participants	0 participants n=7 Participants	3 participants n=5 Participants
Age, Customized >=65 years	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days

Each patient will be classified as a success or failure. A success will be defined as engraftment of at least 35% of cells 100 days after transplant.

Outcome measures

Measure	HLA-identical Sibling Transplant n=3 Participants Recipients of HLA identical sibling stem cell transplants	Unrelated Matched or Single Antigen Mismatched Transplant n=1 Participants Recipients of unrelated matched or single antigen mismatched donor stem cell transplant or single antigen mismatched family donor stem cell transplants
Number of Patients With Successful Donor Engraftment	2 participants	1 participants

SECONDARY outcome

Timeframe: 100 days

Outcome measures

Measure	HLA-identical Sibling Transplant n=3 Participants Recipients of HLA identical sibling stem cell transplants	Unrelated Matched or Single Antigen Mismatched Transplant n=1 Participants Recipients of unrelated matched or single antigen mismatched donor stem cell transplant or single antigen mismatched family donor stem cell transplants
Number of Patients With Treatment Related Grade III or IV Non-hematological Toxicity	1 participants	0 participants

Adverse Events

HLA-identical Sibling Transplant

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Unrelated Matched or Single Antigen Mismatched Transplant

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	HLA-identical Sibling Transplant n=3 participants at risk Recipients of HLA identical sibling stem cell transplants	Unrelated Matched or Single Antigen Mismatched Transplant n=1 participants at risk Recipients of unrelated matched or single antigen mismatched donor stem cell transplant or single antigen mismatched family donor stem cell transplants
Blood and lymphatic system disorders Blood/Bone Marrow - Other: Engraftment Failure	33.3% 1/3 • Number of events 1	0.00% 0/1
Infections and infestations Infection (documented clinically or microbiologically) with Grade 3 or 4 neutrophils (ANC <1.0 x 10e	33.3% 1/3 • Number of events 2	0.00% 0/1

Other adverse events

Adverse event data not reported

Additional Information

Rammurti Kamble, MD

Baylor

Phone: 713-394-6252

Email: rtkamble@tmhs.org

Results disclosure agreements

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