Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
Public ClinicalTrials.gov record NCT04819841. 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 Phase I/II Study of Nula-cel in Autologous CD34+ Hematopoietic Stem Cells to Convert HbS to HbA for Treating Severe Sickle Cell Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
Study identification
- NCT ID
- NCT04819841
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 15 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 12 Years to 50 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
- Nov 14, 2021
- Primary completion
- Dec 30, 2026
- Completion
- Dec 30, 2028
- Last update posted
- Jul 1, 2026
2021 – 2028
United States locations
- U.S. sites
- 6
- U.S. states
- 4
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | Recruiting |
| Lucile Packard Children's Hospital | Palo Alto | California | 94304 | Recruiting |
| Washington University | St Louis | Missouri | 63110 | Recruiting |
| Columbia University Irving Medical Center | New York | New York | 10032 | Recruiting |
| Memorial Sloan Kettering | New York | New York | 10065 | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | Recruiting |
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 NCT04819841, 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 Jul 1, 2026 · Synced Sep 12, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04819841 live on ClinicalTrials.gov.