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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

No Placebo-Only Group

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.

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.

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year (+/- 3 months)

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Side effects data

From 2016 Phase 4 trial • 3 Patients • NCT02078193

67%

COPD exacerbation

100%

80%

60%

40%

20%

Study treatment Arm

Belatacept

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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

0 / 1Eligibility criteria met