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A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease

NCT03653247 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 7

Last updated 2025-09-12

No results posted yet for this study

Summary

This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using BIVV003.

Conditions

Interventions

BIOLOGICAL

Plerixafor

Plerixafor subcutaneous injection will be administered prior to apheresis.

DRUG

Busulfan

Busulfan IV infusion will be administered as myeloablative conditioning therapy.

GENETIC

BIVV003

BIVV003 will be administered as an IV infusion following myeloablative conditioning with busulfan.

Sponsors & Collaborators

  • Sangamo Therapeutics

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Sangamo Therapeutics

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
40 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-03-06
Primary Completion
2025-07-17
Completion
2025-07-17
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03653247 on ClinicalTrials.gov