NCT04819841 (Restore) is a Phase 1, PHASE2 clinical trial of nula-cel Drug Product in Sickle Cell Disease, sponsored by Kamau Therapeutics.

Who is sponsoring NCT04819841?

NCT04819841 is sponsored by Kamau Therapeutics.

What drugs are being tested in NCT04819841?

Interventions in NCT04819841: nula-cel Drug Product.

What condition does NCT04819841 study?

NCT04819841 studies Sickle Cell Disease.

Is NCT04819841 still recruiting?

NCT04819841 is currently recruiting now. Last updated 11 March 2026.

Last reviewed 2026-03-11 · How we verify

NCT04819841: Restore

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

Recruiting now Phase 1, PHASE2 Last updated 11 March 2026

What this trial tests

Phase 1, PHASE2 trial testing nula-cel Drug Product in Sickle Cell Disease in 15 participants. Currently enrolling.

Timeline

15 November 2021

Primary endpoint
31 December 2026

31 December 2028

Quick facts

Lead sponsor	Kamau Therapeutics
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	15
Start date	15 November 2021
Primary completion	31 December 2026
Estimated completion	31 December 2028
Sites	4 locations across United States

Drugs / interventions tested

nula-cel Drug Product

Conditions studied

Sickle Cell Disease — all drugs for Sickle Cell Disease →

Sponsor

Kamau Therapeutics — full company profile →

Who can join

Adults 12 to 40, any sex, with Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

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
Recent advances in the delivery and applications of nonviral CRISPR/Cas9 gene editing.
Sinclair F, Begum AA, Dai CC, Toth I, et al · · 2023 · cited 61× · PMID 36988873 · DOI 10.1007/s13346-023-01320-z
CRISPR/Cas9 Delivery System Engineering for Genome Editing in Therapeutic Applications.
Cheng H, Zhang F, Ding Y. · · 2021 · cited 59× · PMID 34683943 · DOI 10.3390/pharmaceutics13101649
Immunological barriers to haematopoietic stem cell gene therapy.
Charlesworth CT, Hsu I, Wilkinson AC, Nakauchi H. · · 2022 · cited 47× · PMID 35301483 · DOI 10.1038/s41577-022-00698-0
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
Gene editing and its applications in biomedicine.
Li G, Li X, Zhuang S, Wang L, et al · · 2022 · cited 44× · PMID 35235150 · DOI 10.1007/s11427-021-2057-0
An Update on the Application of CRISPR Technology in Clinical Practice.
Morshedzadeh F, Ghanei M, Lotfi M, Ghasemi M, et al · · 2024 · cited 33× · PMID 37269466 · DOI 10.1007/s12033-023-00724-z
Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease.
Germino-Watnick P, Hinds M, Le A, Chu R, et al · · 2022 · cited 33× · PMID 35681538 · DOI 10.3390/cells11111843

Verify or expand the search:

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

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04819841 (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 Kamau Therapeutics
Last refreshed: 11 March 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/NCT04819841.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing