Severe Combined Immunodeficiency Due to RAG1 Deficiency: Clinical Trials

1 active not recruiting, 1 suspended. 1 Phase 1, 1 Phase 2.

By phase and status

Phase	Trials	Status
Phase 1	1	Suspended 1
Phase 2	1	Suspended 1

Follow Severe Combined Immunodeficiency Due to RAG1 Deficiency: new trials by email Literature Review of Severe Combined Immunodeficiency Due to RAG1 Deficiency trials Research Report on Severe Combined Immunodeficiency Due to RAG1 Deficiency

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

Trial	Phase	Status	Sponsor	Enrollment	Start	Updated
Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID Gene therapy - Severe Combined Immunodeficiency Due to RAG1 Deficiency	Phase 1 / Phase 2	Suspended	Videja B.V.	10	2021-07-23	2026-07-16
Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome		Active not recruiting	RTI International	30000	2018-10-15	2026-07-01

Narrower conditions

3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1)  ·  3-Hydroxyacyl-CoA Dehydrogenase Deficiency (1)  ·  3-methylcrotonyl CoA carboxylase 1 deficiency (1)  ·  3-methylcrotonyl CoA carboxylase 2 deficiency (1)  ·  Acidemia, isovaleric (1)  ·  Acrodermatitis enteropathica (1)  ·  Adenine phosphoribosyltransferase deficiency (1)  ·  Adrenal hyperplasia, congenital, type 5 (1)  ·  Adrenocorticotropic hormone deficiency (1)  ·  Amaurosis congenita of Leber, type 2 (1)  ·  Anemia, Sickle Cell (1)  ·  Angelman Syndrome (1)  ·  Aortic Stenosis, Supravalvular (1)  ·  Arginine-Glycine Amidinotransferase Deficiency (1)  ·  Argininosuccinic Aciduria (1)

Broader conditions

Abnormalities, Multiple (1)  ·  Albinism (1)  ·  Albinism, Oculocutaneous (1)  ·  Amino Acid Metabolism, Inborn Errors (1)  ·  Anemia (1)  ·  Anemia, Hemolytic (1)  ·  Anemia, Hemolytic, Congenital (1)  ·  Aortic Valve Disease (1)  ·  Aortic Valve Stenosis (1)  ·  Arrhythmias, Cardiac (1)  ·  Avitaminosis (1)  ·  Basal Ganglia Diseases (1)  ·  Blood Coagulation Disorders (1)  ·  Blood Coagulation Disorders, Inherited (1)  ·  Blood Platelet Disorders (1)

Interventions

Genetic Therapy (1)

Sponsors

RTI International (1)  ·  Videja B.V. (1)

Countries

Netherlands (1)  ·  Poland (1)  ·  Spain (1)  ·  Turkey (Türkiye) (1)  ·  United States (1)

Top investigators

Arjan C Lankester, Prof.dr. (1)  ·  Curt Scharfe, MD, PhD (1)

Results publications

PMID 31315600, PMID 32322605, PMID 35142618

NIH funding

2019 (1), 2023 (1)