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NCT03653247 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of Sickle Cell Disease, sponsored by Sangamo Therapeutics.

Completed
Registry status
Phase 1
Development phase
7
Enrollment target
5
Study locations

NCT03653247: Completed Phase 1 study of Sickle Cell Disease, sponsored by Sangamo Therapeutics.

NCT03653247 is a Phase 1 study of Sickle Cell Disease that has completed, run by Sangamo Therapeutics. The registered enrollment target is 7 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (98% lower). The trial reports 5 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT03653247, a Phase 1 study of Sickle Cell Disease, has completed, sponsored by Sangamo Therapeutics.

COMPLETED
Registry status
Phase 1
Development phase
7 participants
Enrollment target
5
Study locations

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.

Primary Outcome

The percentage of participants who are alive at post-transplantation Day 100 will be calculated using the Kaplan-Meier estimate.

Conditions Studied

Sickle Cell Disease

Interventions

  • DRUG Busulfan
  • BIOLOGICAL Plerixafor
  • GENETIC BIVV003

Study Locations (5)

California

  • UCSF Benioff Children's Hospital - Oakland
  • University of California Davis Comprehensive Cancer Center - Sacramento

Georgia

  • Children's Healthcare of Atlanta - Atlanta

Maryland

  • Investigational Site Number 101 - Bethesda

Michigan

  • Karmanos Cancer Institute - Detroit

Trial Details

FieldValue
Enrollment Target 7 participants
Start Date 2019-03-06
Est. Completion 2025-07-17
Phase Phase 1
Sangamo Therapeutics

25 total trials

What the finished NCT03653247 record still lists

NCT03653247 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 7 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (98% lower).

The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 3 interventions - of which Busulfan is the first listed.

NCT03653247 lists 5 locations in 4 states (California, Georgia, Maryland).

Frequently Asked Questions

What is clinical trial NCT03653247 about?

NCT03653247 is a clinical study titled "A Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease". 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.

What is the current status of trial NCT03653247?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 7 participants. The study started on 2019-03-06. Estimated completion is 2025-07-17.

What conditions does trial NCT03653247 study?

This clinical trial studies the following conditions: Sickle Cell Disease.

What interventions are being tested in trial NCT03653247?

The interventions under investigation include: Busulfan (DRUG), Plerixafor (BIOLOGICAL), BIVV003 (GENETIC).

Who is sponsoring clinical trial NCT03653247?

This trial is sponsored by Sangamo Therapeutics, which has 25 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT03653247 being conducted?

This trial has 5 study locations across California, Georgia, Maryland, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for Sickle Cell Disease

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT03653247's enrollment target sits among peer trials

7 202nd of 213 higher than 10 of 213 other Sickle Cell Disease trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Sickle Cell Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Related registry views

Source: ClinicalTrials.gov NCT03653247, the US trial registry maintained by the National Library of Medicine. NCT03653247 (small enrollment · multi site footprint · completed) retrieved and formatted by PlainTrial, see methodology.