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Hematopoietic Cell Transplantation

Stem Cell Transplant for Sickle Cell Disease

Phase 1 & 2
Recruiting
Led By Courtney F Joseph, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Recipient: Participants must fulfill one disease category, either at least one organ damage or no other access to curative therapy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year (+/- 3 months)
Awards & highlights

Study Summary

This trial will evaluate a transplant regimen for reversing sickle cell disease, measuring success rate, survival, GVHD, viral reactivation, organ function, and gene therapy research.

Who is the study for?
This trial is for people aged 16+ with Sickle Cell Disease (SCD) at high risk of health issues due to the disease. They need a family donor match who's willing and suitable to donate stem cells. Participants must agree to use birth control post-treatment and be able to follow the study procedures for its duration.Check my eligibility
What is being tested?
The trial tests a new drug combo (briquilimab, abatacept, alemtuzumab) plus radiation in HCT for SCD. It aims to make blood stem cell transplants from family donors safer, even when patients have organ damage from SCD.See study design
What are the potential side effects?
Potential side effects may include reactions related to immune suppression by briquilimab and abatacept, infusion-related complications, and typical risks associated with radiation therapy such as fatigue or skin irritation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have organ damage from my condition or no access to curative treatments.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year (+/- 3 months)
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year (+/- 3 months) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Evaluate the regimen success rate where success defined as successful engraftment and absence of acute grade 3 or higher GVHD or moderate to severe chronic GVHD evaluated at 1 yr post-transplant
Secondary outcome measures
Autoimmune and hyperinflammatory complications
Event-free survival and overall survival
Viral reactivation and disease

Side effects data

From 2016 Phase 4 trial • 3 Patients • NCT02078193
67%
COPD exacerbation
100%
80%
60%
40%
20%
0%
Study treatment Arm
Belatacept

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort 2Experimental Treatment1 Intervention
All participants will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. Cohort 2 will receive a total of two doses of PT-Cy; 50mg/kg, on days +3 and +4 (total 100 mg/kg) post-HCT
Group II: Cohort 1Experimental Treatment1 Intervention
All participants will receive the same dose of the investigational briquilimab antibody and abatacept added to the NIH-established regimen of alemtuzumab-TBI-sirolimus and infusion of filgrastim-mobilized peripheral blood hematopoietic cells from haploidentical related donors. Cohort 1 will receive a single dose of PT-Cy; 50 mg/kg, on day +3 post-HCT

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,840 Previous Clinical Trials
47,852,052 Total Patients Enrolled
Courtney F Joseph, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
3 Previous Clinical Trials
287 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Am I a viable participant in this medical research?

"For consideration in this clinical trial, patients must possess the sickle cell disease condition and be between 4-100 years old. This study is currently looking for 72 volunteers to participate."

Answered by AI

Are additional participants being enrolled in this investigation?

"Accurate. It appears that clinicaltrials.gov is reporting this trial as no longer enrolling patients, having been last updated on November 22nd 2023 after initially being posted in late November of the same year. Although not active at present, there are 185 other trials actively recruiting participants currently."

Answered by AI

Does this research project accept individuals in their seventh decade or older?

"This clinical trial seeks out individuals aged 4 to 100. Altogether, there are 198 trials for people below the age of 18 and over 65 years old."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function
2023. "Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06145282.
~48 spots leftby Sep 2032
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