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

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

40

Participants

Timeline

Start Date

May 12, 2022

Primary Completion Date

January 31, 2026

Study Completion Date

January 31, 2027

Conditions
Sickle Cell AnemiaBeta Thalassemia
Interventions
RADIATION

TBI

300 cGy total body irradiation (TBI, day -2)

DRUG

Hydroxyurea

Optimized 4-12 weeks prior to planned transplant date

BIOLOGICAL

briquilimab

briquilimab, anti-CD117 monoclonal antibody at 0.6mg/kg at day -11 prior to infusion of allogeneic HSCs

DRUG

Filgrastim (G-CSF)

May be used to minimize the days of neutropenia, and may be administered beginning near day 10 post stem cell infusion at 5 mcg/kg (rounding to the nearest vial) at the discretion of investigator.

DRUG

Sirolimus

For immune suppression (day -1)

BIOLOGICAL

Alemtuzumab

Alemtuzumab 1 mg/kg of alemtuzumab divided over 5 days (-7 through -3),

DRUG

Plerixafor

For autologous HSC collection; dose of plerixafor at 240 mcg/kg (capped at 20mg)

Trial Locations (1)

20892

National Institutes of Health Clinical Center, Bethesda

All Listed Sponsors
lead

National Heart, Lung, and Blood Institute (NHLBI)

NIH