Clinical trials: Neurodegenerative Disease, Hereditary
Familial Amyloid Polyneuropathy (FAP)
Study of SOM0226 in Familial Amyloid Polyneuropathy
Clinical proof of concept study to evaluate SOM0226 efficacy in TTR Amyloidosis.
- Trial name
- NCT02191826
- Testing
- SOM0226
- Where
- Spain
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- SOM Innovation Biotech SA
Steinert's Disease
Compare Train and 3D-4D Left Ventricular Systolic Function in Subjects Suffering From Dystrophy and Healthy Subjects
Steinert's disease is an orphan disease. The prognosis of patients with this disease is conditioned by cardiac involvement. Search an early stage alterations in contractile function in subjects suffering from dystrophy would…
- Trial name
- NCT02930408
- Testing
- electrocardiogram, Echocardiography
- Volunteers
- 70 people
- Sponsor
- Poitiers University Hospital
Charcot-Marie-Tooth Disease
Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with Charcot-Marie-Tooth Disease Type 1 and Type X (CMT1 and CMTX), to…
- Trial name
- NCT03124459
- Testing
- ACE-083
- Where
- 16 sites in United States
- Volunteers
- 63 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Burns
Determination of Nerve Growth Factor (NGF) Expression in Pediatric Patients With Severe Burns: a Pilot Study
The goal of this observational study is to determine the blood values of NGF in severely burned patients, to describe any changes over time and finally to correlate the expression of NGF to the severity of the burn.
- Trial name
- NCT05751174
- Testing
- pediatric patients affected by severe burns
- Where
- Italy
- Volunteers
- 12 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Recurrent Head and Neck Squamous Cell Carcinoma, Metastatic Head and Neck Squamous Cell Carcinoma
A Phase Ib/II Study of BYL719 and Cetuximab in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma
This was a multi-center, open-label, Phase Ib dose escalation /Phase II study in recurrent or metastatic head and neck squamous cell carcinoma (RM HNSCC) patients considered to be resistant, ineligible or intolerant to…
- Trial name
- NCT01602315
- Testing
- BYL719 as film-coated (FC) whole tablets, BYL719 as dispersible tablets (DT), cetuximab
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 179 people
- Sponsor
- Novartis Pharmaceuticals
Tourette Syndrome, Tic Disorders
Trajectories of Change in Tourette Syndrome
This K23 Career Development Award is designed to provide the training needed for the PI to achieve her long-term career goal of conducting independent, programmatic intervention research in developmental populations.
- Trial name
- NCT06679790
- Testing
- Comprehensive Behavioral Intervention for Tics
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Minnesota
Tourette Syndrome
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Guanfacine for Tourette Syndrome and Attention Deficit Hyperactivity Disorder
OBJECTIVES: I. Evaluate the safety and efficacy of the alpha-2 adrenergic agonist guanfacine in children and adolescents with Tourette syndrome or other chronic tic disorder, and attention deficit hyperactivity disorder.
- Trial name
- NCT00004376
- Testing
- guanfacine
- Volunteers
- 35 people
- Sponsor
- National Center for Research Resources (NCRR)
Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
Primary: The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus…
- Conditions
- Congenital Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome, Nystagmus, Acquired
- Trial name
- EAP-001 (NCT02189720)
- Testing
- Amifampridine Phosphate
- Where
- United States
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Tourette Syndrome, Cannabis
The Effect of Medical Cannabis on Tics, Premonitory Urge and Psychiatric Comorbidity in Adults With Tourette Syndrome
Medical cannabis (MC) is a standard treatment in Israel to adults with resistant Gilles de la Tourette syndrome (GTS). While small randomized control trials assessed THC efficacy on tics and premonitory urge, only small…
- Trial name
- NCT05115318
- Testing
- Medical Cannabis
- Where
- Israel
- Volunteers
- 18 people
- Sponsor
- Tel-Aviv Sourasky Medical Center
Leber's Hereditary Optic Neuropathy
Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy
The study is a dose-escalation study, phase 1. The objective of this proposed clinical trial is to evaluate the safety of mitochondrially targeted ND4 gene therapy with the adeno-associated viral vector in appropriate LHON…
- Trial name
- LHON (NCT02161380)
- Testing
- injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High)
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Byron Lam
Tourette Syndrome
Efficacy of a Therapeutic Combination of Dronabinol and PEA for Tourette Syndrome
This is an investigator-initiated proof of concept study with the purpose to examine the safety, tolerability and feasibility of Dronabinol (synthetic Δ9-THC) and PEA for the treatment of adults with Tourette syndrome.
- Trial name
- NCT03066193
- Testing
- Dronabinol and Palmitoylethanolamide
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Yale University
Optic, Atrophy, Hereditary, Leber
Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)
The goal of this clinical trial is to assess the effectiveness of GS010, a gene therapy, in improving the visual outcome in participants with Leber Hereditary Optic Neuropathy (LHON) due to the G11778A ND4 mitochondrial mutation…
- Trial name
- REVERSE (NCT02652780)
- Testing
- GS010
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 37 people
- Sponsor
- GenSight Biologics
Huntington Disease
Extracellular Vesicles for HD
The primary objective of this study is to discover blood-based biomarker of brain Huntingtin (HTT) protein using extracellular vesicles to be used in evaluating target engagement in HTT lowering clinical trials.
- Trial name
- NCT06082713
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University of Central Florida
Myotonic Dystrophy 1
Effects of a 12-week Strength Training Program in Men With Myotonic Dystrophy Type 1
Eleven men with myotonic dystrophy type 1 (DM1) underwent a 12-week lower-limb strength training program. The training program consisted of 3 series of 6 to 8 maximal repetitions of 5 different exercises: Leg extension, leg…
- Trial name
- NCT04018820
- Testing
- Training program
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- Élise Duchesne
Myotonic Dystrophy 1, Muscular Dystrophies
Myotonic Dystrophy Type 1 Aerobic Exercise Study
Myotonic dystrophy type 1 (DM1) is a genetic disease that primarily targets skeletal muscle resulting in severe weakness and muscle loss.
- Trial name
- DM1ex (NCT04187482)
- Testing
- Exercise training
- Where
- Canada
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- McMaster University
Advanced Cancer
A Study of WXFL10030390 in Patients With Advanced Solid Tumors or Lymphoma
WXFL10030390 (WX390) is a novel oral small molecular that inhibits phosphoinositide-3 kinase (PI3K) and mammalian target of rapamycin (mTOR) and has demonstrated potent inhibitory effects on multiple human tumor xenografts.
- Trial name
- NCT03730142
- Testing
- WXFL10030390
- Where
- China
- Volunteers
- 82 people
- Sponsor
- Shanghai Jiatan Pharmatech Co., Ltd
DM1, Myotonic Dystrophy 1
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to…
- Conditions
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- MARINA (NCT05027269)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 39 people
- Sponsor
- Avidity Biosciences, Inc.
Myotonic Dystrophy, Sudden Cardiac Death
RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy
This is a prospective multicentric Italian study to evaluate the arrhythmic risk in myotonic dystrophy type 1.
- Trial name
- NCT00127582
- Testing
- Electrophysiological study, pacemaker , implantable cardiac defibrillator or loop-recorder implant
- Where
- Italy
- Volunteers
- 537 people
- Sponsor
- Catholic University of the Sacred Heart
Amyloidosis, Amyloid
Monitoring of Early Disease Progression in Hereditary Transthyretin Amyloidosis
This study measures circulating, misfolded ATTR oligomers in asymptomatic ATTRm amyloidosis genetic carriers longitudinally over five years.
- Conditions
- Amyloidosis, Amyloid, Amyloid Neuropathies, Familial, Amyloid Cardiomyopathy
- Trial name
- MED-hATTR (NCT03431896)
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- The Cleveland Clinic
Breast Cancer
A Study of the Experimental Drug BKM120 With Paclitaxel in Patients With HER2 Negative, Locally Advanced or Metastatic Breast Cancer, With or Without PI3K Activation
This study evaluated whether the addition of daily BKM120 to weekly paclitaxel was effective and safe in treating patients with HER2- locally advanced or metastatic breast cancer.
- Trial name
- BELLE-4 (NCT01572727)
- Testing
- Paclitaxel, BKM120
- Where
- 112 sites in United States, Australia and 20 more
- Volunteers
- 416 people
- Sponsor
- Novartis Pharmaceuticals
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