Clinical trials: Neural Degeneration
Pantothenate Kinase-Associated Neurodegeneration
Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)
A multi-center, placebo controlled, double-blind trial comparing the efficacy and safety of 18 months of treatment with deferiprone versus placebo in patients with PKAN.
- Trial name
- NCT01741532
- Testing
- Deferiprone oral solution
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 89 people
- Sponsor
- ApoPharma
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Amyotrophic Lateral Sclerosis
Multiple Doses of AT-1501-A201 in Adults With ALS
This is a Phase 2a, multi-center, open label, multiple dose study of AT-1501, a humanized monoclonal antibody antagonist to CD40 ligand (CD40L).
- Trial name
- NCT04322149
- Testing
- AT-1501
- Where
- 13 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Anelixis Therapeutics, LLC
Pantothenate Kinase-Associated Neurodegeneration
Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants
This study investigated whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, was safe and effective in treating participants with PKAN.
- Trial name
- PKAN (NCT03041116)
- Testing
- Fosmetpantotenate
- Where
- 20 sites in United States, Canada and 7 more
- Volunteers
- 84 people
- Sponsor
- Travere Therapeutics, Inc.
Parkinson's Disease
Early Parkinson's Disease Monotherapy With CVN424
This is a multicenter, 12-week, placebo-controlled clinical trial of CVN424 150 milligrams (mg) tablets in early, untreated Parkinson's Disease (PD).
- Trial name
- NCT06006247
- Testing
- CVN424 150 mg
- Where
- 42 sites in United States
- Volunteers
- 64 people
- Sponsor
- Cerevance Beta, Inc.
Parkinson's Disease
31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Parkinson's Disease
REPAIR-PD is a single-center open label pilot, sequential group, investigator and patient blinded study to assess the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 in patients who have been…
- Trial name
- REPAIR-PD (NCT03815916)
- Testing
- Gold Nanocrystals
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Clene Nanomedicine
Pantothenate Kinase-Associated Neurodegeneration
Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration
Patients with PKAN will be treated with the iron chelator deferiprone for 18 months. Only patients who have completed the earlier study TIRCON2012V1 (NCT01741532), a double-blind placebo-controlled trial in which participants…
- Trial name
- TIRCON-EXT (NCT02174848)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 68 people
- Sponsor
- ApoPharma
Cognition, Brain Volume Loss
Effect of Fingolimod on Neurodegeneration
This was a 24-month, open-label, multicenter study with a single treatment arm design. Primary objective of this study was: -To investigate the effects of Fingolimod on cognitive performance in highly active relapsing remitting…
- Trial name
- NCT02575365
- Testing
- 0,5 mg Fingolimod
- Where
- 3 sites in Turkey (Türkiye)
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals