Last reviewed · How we verify
NCT04443907
Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
Phase 1 trial testing OTQ923 in Sickle Cell Disease in 4 participants. Terminated before completion.
6 January 2025
Quick facts
| Lead sponsor | Novartis Pharmaceuticals |
|---|---|
| Phase | Phase 1 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 4 |
| Start date | 25 August 2020 |
| Primary completion | 6 January 2025 |
| Estimated completion | 6 January 2025 |
| Sites | 3 locations across United States |
Drugs / interventions tested
Conditions studied
- Sickle Cell Disease — all drugs for Sickle Cell Disease →
Sponsor
Novartis Pharmaceuticals — full company profile →
Who can join
Adults 2 to 40, any sex, with Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This study evaluated a genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) product - OTQ923 to reduce the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
CRISPR-Cas9 Editing of the <i>HBG1</i> and <i>HBG2</i> Promoters to Treat Sickle Cell Disease.
Sharma A, Boelens JJ, Cancio M, Hankins JS, et al · · 2023 · cited 118× · PMID 37646679 · DOI 10.1056/nejmoa2215643 -
Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.
Piel FB, Rees DC, DeBaun MR, Nnodu O, et al · · 2023 · cited 100× · PMID 37451304 · DOI 10.1016/s2352-3026(23)00096-0 -
The gut-airway microbiome axis in health and respiratory diseases.
Özçam M, Lynch SV. · · 2024 · cited 71× · PMID 38778224 · DOI 10.1038/s41579-024-01048-8 -
Potent and uniform fetal hemoglobin induction via base editing.
Mayuranathan T, Newby GA, Feng R, Yao Y, et al · · 2023 · cited 61× · PMID 37400614 · DOI 10.1038/s41588-023-01434-7 -
Single-nucleotide-level mapping of DNA regulatory elements that control fetal hemoglobin expression.
Cheng L, Li Y, Qi Q, Xu P, et al · · 2021 · cited 58× · PMID 33958780 · DOI 10.1038/s41588-021-00861-8 -
CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments.
Laurent M, Geoffroy M, Pavani G, Guiraud S. · · 2024 · cited 45× · PMID 38786024 · DOI 10.3390/cells13100800 -
International Recommendations for Screening and Preventative Practices for Long-Term Survivors of Transplantation and Cellular Therapy: A 2023 Update.
Rotz SJ, Bhatt NS, Hamilton BK, Duncan C, et al · · 2024 · cited 43× · PMID 38413247 · DOI 10.1016/j.jtct.2023.12.001 -
Current trends of clinical trials involving CRISPR/Cas systems.
Zhang S, Wang Y, Mao D, Wang Y, et al · · 2023 · cited 35× · PMID 38020120 · DOI 10.3389/fmed.2023.1292452
Verify or expand the search:
- PubMed search for NCT04443907
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other trials of OTQ923
Trials testing the same drug.
- NCT06155500 — Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HS · Phase 1 · active not recruiting
Other recruiting trials for Sickle Cell Disease
Currently open trials in the same condition.
- NCT07369024 — Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults · Phase 2 · recruiting
- NCT06016634 — Alendronate for Osteonecrosis in Adults With Sickle Cell Disease · Phase 2 · recruiting
- NCT06260891 — Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial · Phase 2 · recruiting
- NCT07222475 — Writing Relaxing Beats in Adolescents Who Have Sickle Cell Disease · NA · recruiting
- NCT07224360 — Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD) · Phase 2 · recruiting
Other Novartis Pharmaceuticals trials
Trials by the same sponsor.
- NCT07498335 — Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Atrasentan in Pediatric Patients With Primary · Phase 3 · not yet recruiting
- NCT07489573 — Study of Efficacy and Safety of Secukinumab in Chinese Adult Patients With Moderate to Severe Hidradenitis Suppurativa · Phase 4 · not yet recruiting
- NCT07484269 — PULSE Registry: for Patients Receiving Lutetium (177Lu) Vipivotide Tetraxetan · not yet recruiting
- NCT07416162 — A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy · not yet recruiting
- NCT07387926 — Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+ · Phase 1, PHASE2 · not yet recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04443907 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Novartis Pharmaceuticals
- Last refreshed: 8 January 2026
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04443907.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing