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NCT03282656 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer for Sickle Cell Disease
A Phase 1 study of Sickle Cell Disease, sponsored by David Williams.
- Active
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 2
- Study locations
NCT03282656: Active Phase 1 study of Sickle Cell Disease, sponsored by David Williams.
NCT03282656 is a Phase 1 study of Sickle Cell Disease that is active but no longer recruiting, run by David Williams. The registered enrollment target is 10 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (97% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03282656, a Phase 1 study of Sickle Cell Disease, is active but no longer recruiting, sponsored by David Williams.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 2
- Study locations
Study Summary
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 reje
Primary Outcome
Rescue of hematopoiesis after conditioning (defined by absolute neutrophil count (ANC) greater than or equal to 0.5 x 109 /L for three consecutive days), achieved within 7 weeks following infusion (i.e., "primary engraftment").
Conditions Studied
Interventions
- BIOLOGICAL single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
Study Locations (2)
California
- UCLA - Mattel Children's Hospital - Los Angeles
Massachusetts
- Boston Children's Hospital - Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2018-02-13 |
| Est. Completion | 2026-11-13 |
| Phase | Phase 1 |
What the registry record for NCT03282656 still lists
NCT03282656 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a is the first listed.
NCT03282656 reports a single indexed study location in California, Massachusetts.
Frequently Asked Questions
What is clinical trial NCT03282656 about?
NCT03282656 is a clinical study titled "Gene Transfer for Sickle Cell Disease". 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 t...
What is the current status of trial NCT03282656?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2018-02-13. Estimated completion is 2026-11-13.
What conditions does trial NCT03282656 study?
This clinical trial studies the following conditions: Sickle Cell Disease.
What interventions are being tested in trial NCT03282656?
The interventions under investigation include: single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a (BIOLOGICAL).
Who is sponsoring clinical trial NCT03282656?
This trial is sponsored by David Williams, which has 5 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03282656 being conducted?
This trial has 2 study locations across California, Massachusetts. 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 NCT03282656's enrollment target sits among peer trials
10 196th of 213 higher than 16 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.
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