1 active not recruiting, 1 recruiting. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Severe Combined Immunodeficiency, X Linked: new trials by email Literature Review of Severe Combined Immunodeficiency, X Linked trials Research Report on Severe Combined Immunodeficiency, X Linked
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 |
|---|---|---|---|---|---|---|
| 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 | |
| Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning autologous CD34+ cell transduced with G2SCID vector - Severe Combined Immunodeficiency, X Linked, Gene Therapy | Phase 1 / Phase 2 | Recruiting | David Williams | 12 | 2018-02-26 | 2025-12-11 |
X-Linked Combined Immunodeficiency Diseases (2) · 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)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Genetic Diseases, X-Linked (2) · Immune System Diseases (2) · Immunologic Deficiency Syndromes (2) · Infant, Newborn, Diseases (2) · Primary Immunodeficiency Diseases (2) · Severe Combined Immunodeficiency (2) · Abnormalities, Multiple (1) · Albinism (1) · Albinism, Oculocutaneous (1) · Amino Acid Metabolism, Inborn Errors (1) · Anemia (1) · Anemia, Hemolytic (1) · Anemia, Hemolytic, Congenital (1)
David Williams (1) · RTI International (1)
United States (2)
Curt Scharfe, MD, PhD (1) · Sung-Yun Pai, MD (1)