3 recruiting, 2 completed, 1 not yet recruiting. 3 Phase 3, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 3 | Recruiting 2, Completed 1 |
| Not applicable | 1 | Not yet recruiting 1 |
Follow Genetic Disease, Inborn: new trials by email Literature Review of Genetic Disease, Inborn trials Research Report on Genetic Disease, Inborn
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 |
|---|---|---|---|---|---|---|
| Market Research of a Slow Release Protein Substitute for the Dietary Management of PKU Slow release, phenylalanine-free protein substitute tablets - Phenylketonuria (PKU) and Hyperphenylalaninemia, Brain Diseases, Metabolic, Inborn | Not applicable | Not yet recruiting | metaX Institut fuer Diatetik GmbH | 10 | 2026-10-15 | 2026-10-15 |
| Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) Zeleciment Rostudirsen (DYNE-251), Placebo - Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne | Phase 3 | Recruiting | Dyne Therapeutics | 90 | 2026-06-01 | 2026-05-27 |
| A Phase 3 Study of NTLA-2001 in ATTRv-PN nexiguran ziclumeran, Normal Saline as Placebo - Neuromuscular Disease, Neuromuscular Diseases (NMD) | Phase 3 | Recruiting | Intellia Therapeutics | 60 | 2024-11-22 | 2026-04-16 |
| Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies. Obicetrapib, Placebo - Dyslipidemias, High Cholesterol | Phase 3 | Completed | NewAmsterdam Pharma | 354 | 2022-07-25 | 2025-06-11 |
| A Study Comparing the Quality of Life of Patients in the Treatment of Eczema by Pediatric Generalists and Specialists Care provided by general pediatrician, Care provided by pediatric dermatologist - Eczema, Dermatitis | Completed | Yale University | 26 | 2016-09-01 | 2018-01-11 | |
| Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes Granuloma, Granulomatous Disease, Chronic | Recruiting | National Institute of Allergy and Infectious Diseases (NIAID) | 850 | 1994-02-27 | 2026-10-13 |
Genetic Diseases, Inborn (6) · Metabolic Diseases (3) · Genetic Diseases, X-Linked (2) · Metabolism, Inborn Errors (2) · Nervous System Diseases (2) · Neuromuscular Diseases (2) · Amino Acid Metabolism, Inborn Errors (1) · Amyloid Neuropathies (1) · Amyloid Neuropathies, Familial (1) · Amyloidosis (1) · Amyloidosis, Familial (1) · Amyloidosis, Hereditary, Transthyretin-Related (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Nutritional and Metabolic Diseases (2) · Chronic Disease (1) · Disease Attributes (1) · Hematologic Diseases (1) · Hemic and Lymphatic Diseases (1) · Heredodegenerative Disorders, Nervous System (1) · Hyperlipidemias (1) · Immune System Diseases (1) · Immunologic Deficiency Syndromes (1) · Leukocyte Disorders (1) · Lymphatic Diseases (1) · Lymphoproliferative Disorders (1) · Metabolism, Inborn Errors (1) · Muscular Diseases (1)
NTLA-2001 (1) · Saline Solution (1)
Dyne Therapeutics (1) · Intellia Therapeutics (1) · National Institute of Allergy and Infectious Diseases (NIAID) (1) · NewAmsterdam Pharma (1) · Yale University (1) · metaX Institut fuer Diatetik GmbH (1)
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Anita MacDonald, Professor (1) · Harry L Malech, M.D. (1) · Marc Ditmarsch (1) · Richard Antaya, MD (1)
2025 (1)