Track this trial free. DataLookout checks ClinicalTrials.gov daily and emails you which fields changed on NCT03282656 (status, enrollment, completion dates, endpoints, sites) when this record is updated. Free accounts track up to 5 trials.

Track this trial

Gene Transfer for Sickle Cell Disease

StatusActive, Not Recruiting
PhasePhase 1
Started
View on ClinicalTrials.gov ↗
Record status
This record was last updated January 9, 2026 (before its estimated May 13, 2026 completion). Its status may not reflect the trial's current state.

Record History

See every version on ClinicalTrials.gov ↗

  1. Primary completion date: Sep 13, 2024 to May 13, 2026
    • Study completion date: Dec 13, 2024 to Nov 13, 2026
    v18
  2. Primary completion date: Aug 13, 2024 to Sep 13, 2024
    • Study completion date: Nov 13, 2024 to Dec 13, 2024
    v17
  3. Primary completion date: May 13, 2024 to Aug 13, 2024v16
  4. Re-verified, no change to tracked fieldsv15
  5. Primary completion date: Oct 13, 2022 to May 13, 2024
    • Study completion date: Apr 13, 2024 to Nov 13, 2024
    v14
Show 12 earlier versionsHide 12 earlier versions
  1. Trial status: Recruiting to Active, Not Recruiting
    • Enrollment target: estimated 15 to confirmed 10
    • Primary completion date: Jun 13, 2022 to Oct 13, 2022
    • Site details revised at 2 of 2 sites
    v13
  2. Trial status: Suspended to Recruiting
    • Primary completion date: Jan 13, 2022 to Jun 13, 2022
    • Study completion date: Feb 13, 2024 to Apr 13, 2024
    • Site details revised at 2 of 2 sites
    v12
  3. Primary completion date: Jul 13, 2021 to Jan 13, 2022
    • Study completion date: Feb 13, 2023 to Feb 13, 2024
    v11
  4. Trial status: Recruiting to Suspended Sponsor's reason: Study paused per DSMB pending investigation of Adverse Event occurrence in an unrelated gene therapy study involving sickle cell patients.
    • Primary completion date: Feb 13, 2021 to Jul 13, 2021
    • Study completion date: Feb 13, 2024 to Feb 13, 2023
    • Site details revised at 2 of 2 sites
    v10
  5. Study sites: 1 to 2v9
  6. Enrollment target: 7 to 15v8
  7. Primary completion date: Feb 13, 2020 to Feb 13, 2021v7
  8. Study description revised
    • Eligibility criteria shortened, about 5 words
    v6
  9. Primary completion date: Dec 1, 2019 to Feb 13, 2020
    • Study completion date: Dec 1, 2024 to Feb 13, 2024
    • Start date changed from estimate of Jan 20, 2018 to Feb 13, 2018
    v5
  10. Start date: Nov 30, 2017 to Jan 20, 2018v4
  11. Trial status: Not Yet Recruiting to Recruitingv3 Show 4 more changesShow less
    • Start date: Oct 30, 2017 to Nov 30, 2017
    • Study sites: 0 to 1
    • Primary completion date: Oct 1, 2019 to Dec 1, 2019
    • Study completion date: Oct 1, 2024 to Dec 1, 2024
  12. Start date: Oct 1, 2017 to Oct 30, 2017v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of GVHD, reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to introduce the gene into the patient's own blood stem cells is to engineer and use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings. The investigators have recently discovered a gene that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition. In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid the strong medicines often required to prevent and treat GVHD and rejection. The goal is to test whether this approach is safe, and whether using gene therapy to change the expression of this particular gene will lead to increased fetal hemoglobin production in people with sickle cell disease.
Trial Details
NCT Number NCT03282656
Lead Sponsor David Williams
Conditions
(from sponsor)		 Sickle Cell Disease
Related terms Anemia, Sickle Cell, beta-Thalassemia
Enrollment 10 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov