2 recruiting. 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Recruiting 2 |
Follow Death: new trials by email Literature Review of Death trials Research Report on Death
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 |
|---|---|---|---|---|---|---|
| A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) RP-A601 - PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | Phase 1 | Recruiting | Rocket Pharmaceuticals Inc. | 9 | 2023-08-29 | 2026-04-16 |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
Arrhythmogenic Right Ventricular Dysplasia (1) · Death, Sudden, Cardiac (1) · Gaucher Disease (1) · Heart Arrest (1) · Mucopolysaccharidosis IV (1) · Mucopolysaccharidosis VI (1) · Mucopolysaccharidosis VII (1) · Sudden Infant Death (1) · Wolman Disease (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Death, Sudden (2) · Pathologic Processes (2) · Pathological Conditions, Signs and Symptoms (2) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Carbohydrate Metabolism, Inborn Errors (1) · Cardiomyopathies (1) · Cardiovascular Abnormalities (1) · Cardiovascular Diseases (1) · Central Nervous System Diseases (1) · Cholesterol Ester Storage Disease (1) · Congenital Abnormalities (1)
GAA protein, human (1) · GALNS protein, human (1) · Iduronidase (1) · Sebelipase alfa (1) · galsulfase (1) · idursulfase (1) · vestronidase alfa (1)
Rocket Pharmaceuticals Inc. (1) · University of California, San Francisco (1)
United States (2)
Barry Greenberg, MD (1) · Tippi MacKenzie, MD (1)
PMID 15324318, PMID 19948615, PMID 21637107, PMID 23510062, PMID 28374935, PMID 28657663, PMID 30337566, PMID 32102934, PMID 37955580, PMID 37979797, PMID 39891377, PMID 9918480