ClinicalTrials.gov record
Active, not recruiting Phase 2 Interventional

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

ClinicalTrials.gov ID: NCT05353647

Public ClinicalTrials.gov record NCT05353647. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced May 19, 2026, 9:07 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Multi-Center, Phase 2 Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell (GRASP, BMT CTN 2001)

Study identification

NCT ID
NCT05353647
Recruitment status
Active, not recruiting
Study type
Interventional
Phase
Phase 2
Lead sponsor
David Williams
Other
Enrollment
25 participants

Conditions and interventions

Interventions

  • Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a Biological

Biological

Eligibility (public fields only)

Age range
13 Years to 40 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Jul 11, 2022
Primary completion
Sep 30, 2027
Completion
Sep 30, 2027
Last update posted
May 11, 2026

2022 – 2027

United States locations

U.S. sites
9
U.S. states
5
U.S. cities
7
Facility City State ZIP Site status
Children's Hospital of Los Angeles Los Angeles California 90027
UCLA Medical Center Los Angeles California 90095
UCSF Benioff Children's Hospital Oakland Oakland California 94609
UC Davis Medical Center Sacramento California 95817
Children's Healthcare of Atlanta/Emory University Atlanta Georgia 30322
Lurie Children's Hospital of Chicago Chicago Illinois 60611
Boston Children's Hospital Boston Massachusetts 02115
Dana-Farber Cancer Institute/Brigham and Women's Hospital Boston Massachusetts 02115
Medical College of Wisconsin Milwaukee Wisconsin 53226

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT05353647, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted May 11, 2026 · Synced May 19, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT05353647 live on ClinicalTrials.gov.

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