Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

Phase 1

Recruiting

• Conditions: Sickle Cell Anemia; Beta Thalassemia
• Interventions: Radiation: TBI; Drug: Hydroxyurea; Biological: briquilimab; Drug: Filgrastim (G-CSF); Drug: Sirolimus; Biological: Alemtuzumab; Drug: Plerixafor

• Registration Number: NCT05357482
• Lead Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Brief Summary

Background:

Sickle cell disease (SCD) is an inherited disorder of the blood. It can damage a person s organs and cause serious illness and death. A blood stem cell transplant is the only potential cure for SCD. Treatments that improve survival rates are needed.

Objective:
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Detailed Description

Study Description:
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Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2022-04-30
• Study First Submitted QC: 2022-05-02
• Study First Posted: 2022-05-03
• Study Start: 2022-05-12
• Status Verified: 2024-11-26
• Last Update Submitted: 2024-11-28
• Last Update Posted: 2024-12-02
• Primary Completion: 2032-11-01
• Study Completion: 2033-11-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
briquilimab in stem cell transplant recipients for SCD	TBI	Affected SCD and beta-thal subjects will receive briquilimab
briquilimab in stem cell transplant recipients for SCD	Filgrastim (G-CSF)	Affected SCD and beta-thal subjects will receive briquilimab
briquilimab in stem cell transplant recipients for SCD	briquilimab	Affected SCD and beta-thal subjects will receive briquilimab
briquilimab in stem cell transplant recipients for SCD	Hydroxyurea	Affected SCD and beta-thal subjects will receive briquilimab
briquilimab in stem cell transplant recipients for SCD	Sirolimus	Affected SCD and beta-thal subjects will receive briquilimab
briquilimab in stem cell transplant recipients for SCD	Alemtuzumab	Affected SCD and beta-thal subjects will receive briquilimab
briquilimab in stem cell transplant recipients for SCD	Plerixafor	Affected SCD and beta-thal subjects will receive briquilimab

Primary Outcome Measures

Name	Time	Method
percent myeloid (CD14/15) chimerism	1 year post transplant	The primary objective is to determine if addition of CD117 antibody (JSP191) would increase proportion of patients with donor myeloid chimerism =98% at 1 year post transplant.

Secondary Outcome Measures

Name	Time	Method
JSP antibody PK levels	day 100	To measure JSP191 and alemtuzumab clearance at 1 and 2 years post transplant
Rates of graft failure	1 and 2 years post transplant	Rates of graft failure
Rate of bacterial infection	1 and 2 years post transplant	Rate of bacterial infection
Transplant related mortality	1 and 2 years post transplant	Transplant related mortality
Alemtuzumab levels	day 100	To measure alemtuzumab clearance at 1 and 2 years post transplant
Proportion of patients with donor myeloid chimerism at or above 75%	1 and 2 years post transplant	Estimate the proportion of patients with donor myeloid chimerism at or above 75%
Rate of viral infection and/or reactivation	day 100	Rate of viral infection and/or reactivation
Rate of chronic GVHD	1 and 2 years post transplant	Rate of chronic graft vs host disease (GvHD)
Days to neutrophil engraftment	day 100	Day of neutrophil engraftment
Non-transplant related mortality	1 and 2 years post transplant	Non-transplant related mortality
Rates of acute GVHD	1 and 2 years post transplant	Rate of acute graft vs host disease (GvHD)

Trial Locations

Locations (1)

National Institutes of Health Clinical Center; 🇺🇸 Bethesda, Maryland, United States