Trial Outcomes & Findings for Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease (NCT NCT04207320)

NCT ID: NCT04207320

Last Updated: 2024-12-04

Results Overview

Graft Function: efficacy is defined as stable donor engraftment (\>5% total nucleated cell DNA) and donor erythropoiesis that corrects the SCD hematologic phenotype (\<50% HbS in the peripheral blood). Organ Toxicity: grade III/IV irreversible end organ toxicity based on NCI grading Graft Versus Host disease: grade III/IV aGvHD or death within 100 days post- Hap-HSCT

• Recruitment status: TERMINATED
• Study phase: NA
• Target enrollment: 3 participants
• Primary outcome timeframe: 100 days post-Hap-HSCT
• Results posted on: 2024-12-04

Participant Flow

Participant milestones

Measure	Stage I Stage I will include eligible subjects between the ages of 10-25 years. αβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.	Stage II Stage II will include eligible subjects between the ages of 2-25 years. αβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.
Overall Study STARTED	3	0
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	3	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease

Baseline characteristics by cohort

Measure	Stage I n=3 Participants Stage I will include eligible subjects between the ages of 10-25 years. αβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.	Stage II Stage II will include eligible subjects between the ages of 2-25 years. αβ+ T-cell depletion with Miltenyi CliniMACS system: Haploidentical CD34+ megadose hematopoietic stem cell transplant in which cells are purified using the Miltenyi CliniMACS system designed for αβ+ T-cell receptor selection using immunomagnetic beads.	Total n=3 Participants Total of all reporting groups
Age, Categorical <=18 years	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants	0 Participants n=7 Participants	2 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	20 years n=5 Participants	—	20 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	3 participants n=5 Participants	—	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days post-Hap-HSCT

Population: No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.

Graft Function: efficacy is defined as stable donor engraftment (>5% total nucleated cell DNA) and donor erythropoiesis that corrects the SCD hematologic phenotype (<50% HbS in the peripheral blood).

Organ Toxicity: grade III/IV irreversible end organ toxicity based on NCI grading

Graft Versus Host disease: grade III/IV aGvHD or death within 100 days post- Hap-HSCT

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year post transplant

Population: No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.

Proportion of patients at 1 year who have not died or had graft failure

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 100 days post transplant

Population: No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.

Proportion of subjects with grades I through IV acute GvHD

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 100 days post transplant

Population: No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.

Proportion of subjects with grades III through IV acute GvHD

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year post transplant

Population: No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.

Proportion of subjects with grades I through IV chronic GvHD

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 1 year post transplant

Population: No subjects underwent transplant while on study. All subjects withdrawn and study closed prior to any study procedures/outcome measures performed.

Proportion of subjects with grades III through IV chronic GvHD

Outcome measures

Outcome data not reported

Adverse Events

Stage I

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Stage II

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. John Cunningham

University of Chicago

Phone: 773-702-6808

Email: jcunning@bsd.uchicago.edu

Results disclosure agreements

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