Who is sponsoring NCT04797260?

NCT04797260 is sponsored by Leiden University Medical Center.

What drugs are being tested in NCT04797260?

Interventions in NCT04797260: Gene therapy.

What condition does NCT04797260 study?

NCT04797260 studies Severe Combined Immunodeficiency Due to RAG1 Deficiency.

Is NCT04797260 still recruiting?

NCT04797260 is currently recruiting now. Last updated 18 April 2024.

Last reviewed 2024-04-18 · How we verify

NCT04797260: RAG1-SCID

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

Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID

Recruiting now NA Last updated 18 April 2024

What this trial tests

NA trial testing Gene therapy in Severe Combined Immunodeficiency Due to RAG1 Deficiency in 10 participants. Currently enrolling.

Timeline

23 July 2021

Primary endpoint
31 December 2029

31 December 2029

Quick facts

Lead sponsor	Leiden University Medical Center
Phase	NA
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	23 July 2021
Primary completion	31 December 2029
Estimated completion	31 December 2029
Sites	7 locations across Italy, Netherlands, United Kingdom, Poland, Australia, Turkey (Türkiye), Spain

Drugs / interventions tested

Gene therapy — full drug profile →

Conditions studied

Severe Combined Immunodeficiency Due to RAG1 Deficiency — all drugs for Severe Combined Immunodeficiency Due to RAG1 Deficiency →

Sponsor

Leiden University Medical Center

Who can join

Adults 8 Weeks to 24 Months, any sex, with Severe Combined Immunodeficiency Due to RAG1 Deficiency. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study is a prospective, non-randomized, open-label, two-centre phase I/II intervention study designed to treat children up to 24 months of age with RAG1-deficient SCID with an indication for allogeneic hematopoietic stem cell transplantation but lacking an HLA-matched donor. The study involves infusion of autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (hereafter called RAG1 LV CD34+ cells) in five patients with RAG1-deficient SCID.

Publications & conference data

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

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
Therapy Development by Genome Editing of Hematopoietic Stem Cells.
Koniali L, Lederer CW, Kleanthous M. · · 2021 · cited 25× · PMID 34198536 · DOI 10.3390/cells10061492
The recombinase activating genes: architects of immune diversity during lymphocyte development.
Braams M, Pike-Overzet K, Staal FJT. · · 2023 · cited 18× · PMID 37497222 · DOI 10.3389/fimmu.2023.1210818
Advances in gene therapy for inborn errors of immunity.
Ott de Bruin LM, Lankester AC, Staal FJT. · · 2023 · cited 17× · PMID 37846903 · DOI 10.1097/aci.0000000000000952
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Castiello MC, Brandas C, Ferrari S, Porcellini S, et al · · 2024 · cited 12× · PMID 38324638 · DOI 10.1126/scitranslmed.adh8162
Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.
Castiello MC, Ferrari S, Villa A. · · 2023 · cited 11× · PMID 36863140 · DOI 10.1016/j.smim.2023.101731
Precision medicine: The use of tailored therapy in primary immunodeficiencies.
Pinto MV, Neves JF. · · 2022 · cited 11× · PMID 36569887 · DOI 10.3389/fimmu.2022.1029560

Verify or expand the search:

Other trials of Gene therapy

Trials testing the same drug.

NCT05991336 — Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy · recruiting
NCT04883710 — The World Federation of Hemophilia Gene Therapy Registry · not yet recruiting

Other Leiden University Medical Center trials

Trials by the same sponsor.

NCT07312565 — Occurrence of Abdominal Aortic Aneurysms in Patients With Aneurysmal Subarachnoid Hemorrhage · not yet recruiting
NCT07090343 — Semaglutide in Patients Undergoing Transcatether Aortic Valve Replacement · Phase 3 · not yet recruiting
NCT07498738 — Making Diabetes Care Fit for Young Adults Living With Type 1 Diabetes: Observations, Reflections and Expert Opinions · NA · not yet recruiting
NCT06545292 — Microsampling for Therapeutic Drug Monitoring of Oral Oncolytics in Oncology Patients · NA · recruiting
NCT07021495 — SKIN Disease Profiling by an Exploratory, pRospective, Biomarker Study in dermatoloGY Practice (SKINERGY) · 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 NCT04797260 (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 Leiden University Medical Center
Last refreshed: 18 April 2024

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/NCT04797260.

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