3 completed, 2 active not recruiting, 1 approved for marketing, 1 recruiting. 2 Phase 1, 2 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Completed 1, Recruiting 1 |
| Phase 2 | 2 | Completed 1, Recruiting 1 |
| Phase 3 | 1 | Completed 1 |
Follow Menkes Disease: new trials by email Literature Review of Menkes Disease trials Research Report on Menkes Disease
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 |
|---|---|---|---|---|---|---|
| NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease Droxidopa Oral Product, Placebo Control - Menkes Disease | Phase 1 / Phase 2 | Recruiting | Stephen G. Kaler | 6 | 2026-08-10 | 2026-10-05 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome Droxidopa, Placebo - Menkes Disease, Occipital Horn Syndrome | Phase 1 / Phase 2 | Completed | Stephen G. Kaler, MD | 3 | 2021-07-12 | 2026-04-13 |
| Long Term Follow-up on Menkes Disease Patients Long Term Follow-Up - Menkes Disease | Active not recruiting | Sentynl Therapeutics, Inc. | 50 | 2019-12-01 | 2026-03-02 | |
| 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 | |
| Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency Copper Histidine - Menkes Disease, Occipital Horn Syndrome | Phase 3 | Completed | Cyprium Therapeutics, Inc. | 93 | 2009-02-27 | 2020-11-19 |
| Copper Histidinate Treatment for Menkes Disease copper histidinate - Menkes Disease | Approved for marketing | Sentynl Therapeutics, Inc. | 2026-03-02 |
Menkes Kinky Hair Syndrome (7) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (2) · Acidemia, isovaleric (2) · Anemia, Sickle Cell (2) · Argininosuccinic Aciduria (2) · Ataxia with vitamin E deficiency (2) · Autonomic Nervous System Diseases (2) · Basal ganglia disease, biotin-responsive (2) · Beta ketothiolase deficiency (2) · Biotinidase Deficiency (2) · Brown-Vialetto-Van Laere syndrome (2) · Carbamoyl-Phosphate Synthase I Deficiency Disease (2) · Carnitine palmitoyl transferase 1A deficiency (2) · Carnitine palmitoyl transferase 2 deficiency (2) · Carnitine-Acylcarnitine Translocase Deficiency (2)
Brain Diseases (7) · Brain Diseases, Metabolic (7) · Brain Diseases, Metabolic, Inborn (7) · Central Nervous System Diseases (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Genetic Diseases, X-Linked (7) · Hair Diseases (7) · Heredodegenerative Disorders, Nervous System (7) · Intellectual Disability (7) · Metabolic Diseases (7) · Metabolism, Inborn Errors (7) · Metal Metabolism, Inborn Errors (7) · Nervous System Diseases (7) · Neurobehavioral Manifestations (7)
Droxidopa (2) · copper bis(histidinate) (1) · copper histidine (1)
Sentynl Therapeutics, Inc. (2) · Centre Hospitalier Universitaire de Liege (1) · Cyprium Therapeutics, Inc. (1) · RTI International (1) · Stephen G. Kaler (1) · Stephen G. Kaler, MD (1)
United States (6) · Belgium (1)
Curt Scharfe, MD, PhD (1) · Laurent Servais (1) · Stephen G Kaler, MD (1) · Stephen G Kaler, MD, MPH (1)