Clinical trials: Brain Malformation
Late Infantile Metachromatic Leukodystrophy
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
This is a single center, open-label study of patients with late infantile MLD. All patients were previous treated 26 weeks in the phase I trial (EudraCT number: 2006-005341-11, NCT00418561).
- Trial name
- NCT00633139
- Testing
- Recombinant human Arylsulfatase A (rhASA)
- Where
- Denmark
- Volunteers
- 13 people
- Sponsor
- Shire
Phenylketonurias
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
The primary objective of this trial is to evaluate the ability of Phenoptin to increase phenylalanine (phe) tolerance in children with phenylketonuria who are following a phe-restricted diet.
- Trial name
- NCT00272792
- Testing
- Sapropterin Dihydrochloride
- Where
- 9 sites in United States
- Volunteers
- 45 people
- Sponsor
- BioMarin Pharmaceutical
Epilepsy, Dravet Syndrome (DS)
A Study of Soticlestat in Adults and Children With Rare Epilepsies
The main aim is to assess the long-term safety and tolerability of soticlestat when used along with other anti-seizure treatment. Participants will receive soticlestat twice a day.
- Conditions
- Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
- Trial name
- Endymion 1 (NCT03635073)
- Testing
- Soticlestat
- Where
- 59 sites in United States, Australia and 6 more
- Volunteers
- 156 people
- Sponsor
- Takeda
Wilson Disease
Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
Hypothesis: The investigators postulate that patients with Wilson disease who are asymptomatic or who have been effectively treated for their symptoms and are in a maintenance phase therapy can be safely and effectively treated…
- Trial name
- NCT01472874
- Testing
- Once a day Trientine
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Yale University
Familial Amyloid Polyneuropathy
Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis
This study will examine whether Fx-1006A is effective in halting the progression of Familial Amyloid Polyneuropathy (FAP). Deposition of TTR amyloid is associated with a variety of human diseases.
- Trial name
- NCT00409175
- Testing
- Fx-1006A
- Where
- 10 sites in United States, Argentina and 7 more
- Volunteers
- 128 people
- Sponsor
- Pfizer
Tourette's Disorder, Tic Disorder
Study Evaluating the Safety and Efficacy of Fixed-dose Once-daily Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current trial is to determine efficacy and safety of Once-daily aripiprazole in reducing Total Tic Severity in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01727700
- Testing
- Aripiprazole
- Where
- 75 sites in United States, Canada and 7 more
- Volunteers
- 133 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Gaucher Disease Type 1
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
Although miglustat has been approved as a treatment for mild to moderate type 1 Gaucher disease in patients who are unsuitable for enzyme replacement therapy (ERT), more data are required to establish the long term efficacy…
- Trial name
- NCT00319046
- Testing
- Miglustat
- Volunteers
- 42 people
- Sponsor
- Actelion
Sickle Cell Disease, Stroke
Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease
This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease.
- Trial name
- HUPrevent (NCT01389024)
- Testing
- Hydroxyurea
- Where
- 5 sites in United States
- Volunteers
- 28 people
- Sponsor
- Johns Hopkins University
Migraine With Aura, Patent Foramen Ovale
Comorbidities Associated With Migraine and Patent Foramen Ovale (CAMP)
The purpose of the study is to compare the rate of comorbidities associated with migraine aura (MA) between persons who have a large circulatory right-to-left shunt (RLS) and those who do not have RLS.
- Trial name
- CAMP (NCT01257880)
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Swedish Medical Center
Tourette's Disorder (TD)
Trial to Evaluate the Long-term Efficacy of Oral Aripiprazole in the Treatment of Pediatric Participants With Tourette's Disorder
To evaluate the long-term efficacy of oral aripiprazole in pediatric participants for the treatment of Tourette's Disorder (TD).
- Trial name
- NCT03661983
- Testing
- Aripiprazole
- Where
- 42 sites in United States, Canada and 1 more
- Volunteers
- 36 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Hemianopia, Hemianopsia
Collision Warning Device for Blind and Visually Impaired
This study evaluates a novel collision warning device to help people with severe vision impairment or blindness avoid collisions with obstacles.
- Conditions
- Hemianopia, Hemianopsia, Peripheral Visual Field Defect, Blindness
- Trial name
- NCT03057496
- Testing
- Collision warning device
- Where
- United States
- Volunteers
- 49 people, healthy volunteers welcome
- Sponsor
- Massachusetts Eye and Ear Infirmary
Tourette Syndrome
Tourette Deep Brain Stimulation (DBS) Target Detection & Suppression
The investigators will implant and monitor 8 research subjects with a subcortical closed-loop system for detection and suppression of tics in subjects with medically refractory debilitating Tourette Syndrome.
- Trial name
- NCT05371041
- Testing
- Medtronic Percept Neurostimulator
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Florida
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617860
- Testing
- WVE-120102
- Where
- 20 sites in Australia, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Obsessive-Compulsive Disorder, Tic Disorders
Effects of Ondansetron in Obsessive-compulsive and Tic Disorders
This project investigates the use of 4 weeks of 24 mg/day ondansetron as compared to placebo on symptoms and brain functioning in patients with obsessive-compulsive disorder (OCD) and tic disorders (TD).
- Conditions
- Obsessive-Compulsive Disorder, Tic Disorders, Tourette Syndrome
- Trial name
- NCT03239210
- Testing
- Ondansetron
- Where
- 2 sites in United States
- Volunteers
- 110 people
- Sponsor
- NYU Langone Health
Wilson Disease
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
- Trial name
- NCT04573309
- Testing
- ALXN1840
- Where
- 3 sites in United States, New Zealand and 1 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Migraine, Patent Foramen Ovale
GORE® CARDIOFORM Septal Occluder Migraine Clinical Study
Multi-center, prospective, randomized, placebo- and sham-controlled study to evaluate the GORE® CARDIOFORM Septal Occluder for migraine headache relief
- Conditions
- Migraine, Patent Foramen Ovale, PFO - Patent Foramen Ovale
- Trial name
- RELIEF (NCT04100135)
- Testing
- Actual device PFO closure, Thienopyridine (clopidogrel or prasugrel)
- Where
- 15 sites in United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- W.L.Gore & Associates
Huntington Disease
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.
- Trial name
- NCT05358717
- Testing
- PTC518
- Where
- 28 sites in United States, Australia and 9 more
- Volunteers
- 159 people
- Sponsor
- Novartis Pharmaceuticals
Cerebral Palsy, Muscle Spasticity
Dysport® Pediatric Lower Limb Spasticity Follow-on Study
The purpose of this research study was to determine the long term safety and efficacy of repeated treatments with Dysport® used in the treatment of lower limb spasticity in children with dynamic equinus foot deformity due to…
- Conditions
- Cerebral Palsy, Muscle Spasticity, Children
- Trial name
- NCT01251380
- Testing
- Botulinum toxin type A
- Where
- 27 sites in United States, Chile and 4 more
- Volunteers
- 216 people
- Sponsor
- Ipsen
Adrenoleukodystrophy, Metachromatic Leukodystrophy
Stem Cell Transplant for Inborn Errors of Metabolism
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic…
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease
- Trial name
- NCT00176904
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 135 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Tourette Syndrome, Tic Disorders
Median Nerve Stimulation Pilot
Results from the University of Nottingham suggested that rhythmic median nerve stimulation (MNS) improves tic symptoms in Tourette syndrome (TS).
- Conditions
- Tourette Syndrome, Tic Disorders, Tic Disorder, Chronic Motor or Vocal
- Trial name
- MNS (NCT04731714)
- Testing
- Rhythmic median nerve stimulation, Arrhythmic median nerve stimulation
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Washington University School of Medicine