Heredodegenerative Disorders, Nervous System clinical trials
The registry lists 21 studies for Heredodegenerative Disorders, Nervous System across more than 10 countries. 5 are recruiting or about to start. 3 of them are mainly about Heredodegenerative Disorders, Nervous System (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-05. Counting its 29 narrower conditions too, such as Huntington Disease: 2,363 studies, 595 open.
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Status
Who runs them
PepGen Inc (2) · Vertex Pharmaceuticals Incorporated (2) · SwanBio Therapeutics, Inc. (2) · Siena Biotech S.p.A. (2) · State University of New York at Buffalo (2) · National Human Genome Research Institute (NHGRI) (1) · Prilenia (1) · Avidity Biosciences, Inc. (1)
What is being tested here
Drugs under test in Heredodegenerative Disorders, Nervous System studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2, phase 3 first
- Jul 2027A Phase 3 Study of NTLA-2001 in ATTRv-PN · Phase 3 · Intellia Therapeutics
- Feb 2027A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) · Vertex Pharmaceuticals Incorporated
Who these studies take
Of the 3 Heredodegenerative Disorders, Nervous System studies recruiting now, 2 (67%) set no upper age limit and 0 (0%) stop at 75 or younger. 1 (33%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (33%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 1 (33%) | 1 (33%) |
| 18 | 2 (67%) | 2 (67%) |
| 30 | 3 (100%) | 3 (100%) |
| 40 | 3 (100%) | 3 (100%) |
| 50 | 3 (100%) | 3 (100%) |
| 65 | 3 (100%) | 3 (100%) |
| 75 | 3 (100%) | 3 (100%) |
| 85 | 2 (67%) | 2 (67%) |
Across all 21 studies, recruiting or not: 38% stop at 75 or younger and 29% at 65; 29% take children; 0% are for women only and 14% for men only; 10% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 21 of 21. search them.
- A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type IEnrolling by invitationNCT06926621Phase 28 countries
- An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)RecruitingNCT07220603Phase 21 country
- Molecular and Functional Imaging in Monogenic PD.RecruitingNCT055186171 country
- MCLENA-2: A Phase II Clinical Trial for the Assessment of Lenalidomide in Patients With Mild Cognitive ImpairmentNot yet recruitingNCT06177028Phase 2
- The Parkinson's Genetic Research StudyRecruitingNCT015584791 country
- A Phase 3 Study of NTLA-2001 in ATTRv-PNActive, not recruitingNCT06672237Phase 36 countries
- A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type IIActive, not recruitingNCT05208281Phase 2/31 country
- Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) PatientsCompletedNCT05479981Phase 21 country
- A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)TerminatedNCT05394064Phase 1/22 countries
- A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)Active, not recruitingNCT06185764Phase 1/210 countries
- Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1CompletedNCT06204809Phase 13 countries
- Study of Disease Progression in Adults With Inherited Forms of Spastic ParaplegiaTerminatedNCT050088743 countries
- A Second Intermediate-Size Expanded Access Protocol (EAP) for Pridopidine in People With Amyotrophic Lateral Sclerosis (Pridopidine EAP 2)No longer availableNCT060699342 countries
- Natural History Study of FDXR Mutation-related MitochondriopathyCompletedNCT045809791 country
- Natural History Study of SLC25A46 Mutation-related MitochondriopathyCompletedNCT045945901 country
- A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A MutationCompletedNCT042014181 country
- Mitogen Activated Protein Kinase Kinase (MEK1/2) Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in People With Neurofibromatosis Type 1 (NF1) Mutated Gastrointestinal Stromal Tumors (GIST)WithdrawnNCT03109301Phase 2
- Clinical and Genetic Studies of Familial Presenile Dementia With Neuronal Inclusion BodiesCompletedNCT000061761 country
- An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) PatientsCompletedNCT024299471 country
- An Exploratory Clinical Trial in Early Stage Huntington's Disease Patients With SEN0014196CompletedNCT01485952Phase 11 country
- A Open-label Food Effect Study With SEN0014196 in Subjects With Huntington DiseaseCompletedNCT01485965Phase 11 country