Duplication Clinical Trials in San Diego, California
3 recruitingSan Diego, California
Showing 1–3 of 3 trials
Recruiting
Phase 1Phase 2
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Ionis Pharmaceuticals, Inc.48 enrolled11 locationsNCT06430385
Recruiting
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Mitochondrial DiseasesMitochondrial DisordersMitochondrial Genetic Disorders+2 more
Columbia University1,000 enrolled17 locationsNCT01694940
Recruiting
The Myelin Disorders Biorepository Project
AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more
Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369