Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
Public ClinicalTrials.gov record NCT04443907. 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.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease
Study identification
- NCT ID
- NCT04443907
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Enrollment
- 4 participants
Conditions and interventions
Conditions
Interventions
- OTQ923 Biological
Biological
Eligibility (public fields only)
- Age range
- 2 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
- Aug 24, 2020
- Primary completion
- Jan 5, 2025
- Completion
- Jan 5, 2025
- Last update posted
- Jan 7, 2026
2020 – 2025
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Chicago | Chicago | Illinois | 60637 | — |
| Memorial Sloan Kettering Cancer Ctr | New York | New York | 10065 | — |
| St Jude Children's Research Hospital | Memphis | Tennessee | 38105-3678 | — |
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 NCT04443907, 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 Jan 7, 2026 · Synced May 16, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04443907 live on ClinicalTrials.gov.