Clinical trials: Brain Malformation
Cerebral Cavernous Malformation
Atorvastatin Treatment of Cavernous Angiomas With Symptomatic Hemorrhage Exploratory Proof of Concept (AT CASH EPOC) Trial
This phase I/II randomized, placebo-controlled, double-blinded, single-site clinical trial is designed to investigate the effect of a prolonged course of atorvastatin versus placebo on CCM lesional iron deposition assessed by…
- Trial name
- AT CASH EPOC (NCT02603328)
- Testing
- Atorvastatin
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- University of Chicago
Niemann-Pick Disease, Type C
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B…
- Trial name
- NCT03879655
- Testing
- VTS-270
- Where
- Costa Rica
- Volunteers
- 2 people
- Sponsor
- Mandos LLC
Phenylketonuria (PKU)
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
This is a Phase 3 open-label randomized controlled study enrolling approximately 54 adolescents with PKU. The study is designed to assess the safety and efficacy of pegvaliase injections.
- Trial name
- PEGASUS (NCT05270837)
- Testing
- Pegvaliase, Diet Only
- Where
- 16 sites in United States, Germany
- Volunteers
- 55 people
- Sponsor
- BioMarin Pharmaceutical
Intraventricular Hemorrhage, Periventricular Leukomalacia
Effects of Delayed Cord Clamp and/or Indomethacin on Preterm Infant Brain Injury
Intraventricular hemorrhage (IVH) and periventricular leukomalacia (PVL) are brain lesions that commonly occur in preterm infants and are well-recognized major contributors to long-term brain injury and related disabilities later…
- Conditions
- Intraventricular Hemorrhage, Periventricular Leukomalacia, Brain Injury, Renal Injury
- Trial name
- NCT02221219
- Testing
- Indomethacin, delay in umbilical cord clamp at birth, immediate cord clamp at birth
- Where
- United States
- Volunteers
- 256 people
- Sponsor
- Hong Huang
Tourette Syndrome
Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome
This is an open-label, rollover study to collect long-term safety, tolerability, and investigator- and participant-reported pharmacodynamic (PD) data after chronic administration of NBI-98854 in pediatric participants with…
- Trial name
- NCT03732534
- Testing
- NBI-98854
- Where
- 10 sites in United States, Puerto Rico
- Volunteers
- 6 people
- Sponsor
- Neurocrine Biosciences
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
Huntington's Disease
Examination of Quantitative Electroencephalographic (QEEG) Biomarkers in Huntington's Disease
The pace of basic science research defining the mechanisms of selective neuronal degeneration in Huntington disease (HD) has far exceeded the pace of translation of this information into clinically effective treatments for the…
- Trial name
- HD (NCT00670709)
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
Neurodegenerative Disease, Weight Loss
Electronic-health Application To Measure Outcomes REmotely Clinical Trial
This is a phase II feasibility, safety, tolerability and preliminary efficacy study of an e-Health application versus in-person nutritional counseling to maintain or increase weight in patients with neurodegenerative diseases…
- Conditions
- Neurodegenerative Disease, Weight Loss, Amyotrophic Lateral Sclerosis, Parkinson's Disease
- Trial name
- EAT MORE (NCT02418546)
- Testing
- In-Person Nutritional Counseling by a Registered Dietitian, Nutritional counseling using an e-Health Application
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Massachusetts General Hospital
Fabry Disease
Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
This is the first in human treatment with ST-920, an adeno-associated virus (AAV2/6) vector encoding the complementary deoxyribonucleic acid (cDNA) for human a-Gal A.
- Trial name
- NCT04046224
- Testing
- ST-920
- Where
- 18 sites in United States, Australia and 5 more
- Volunteers
- 36 people
- Sponsor
- Sangamo Therapeutics
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
Late Infantile Metachromatic Leukodystrophy
Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
This is a multi-center, open-label, extension study of patients with late infantile MLD who have previously completed clinical study HGT-MLD-048 (NCT00633139), defined as the completion of all Week 52 procedures.
- Trial name
- NCT00681811
- Testing
- HGT-1111
- Where
- Denmark
- Volunteers
- 11 people
- Sponsor
- Shire
Huntington Disease, Irritability
Evaluating the Efficacy of Dextromethorphan/Quinidine in Treating Irritability in Huntington's Disease
The purpose of this study is to assess efficacy and safety of dextromethorphan/quinidine 20mg/10mg (DM/Q 20mg/10mg) in patients with irritability due to Huntington's disease.
- Trial name
- NCT03854019
- Testing
- Dextromethorphan/quinidine 20mg/10mg (DM/Q 20mg/10mg)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Cancer
Cyproheptadine in Preventing Weight Loss in Children Receiving Chemotherapy for Cancer
RATIONALE: Cyproheptadine hydrochloride may prevent weight loss caused by cancer or cancer treatment. It is not yet known whether cyproheptadine is more effective than a placebo in preventing weight loss in young patients…
- Trial name
- NCT01132547
- Testing
- cyproheptadine hydrochloride
- Where
- 14 sites in United States
- Volunteers
- 22 people
- Sponsor
- University of South Florida
Small Fiber Neuropathy, Fabry Disease
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Sudoscan™ (Impeto Medical, Paris France) uses electrochemical skin conductance as a novel noninvasive method to detect sudomotor dysfunction.
- Conditions
- Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
- Trial name
- NCT02985710
- Testing
- Sudoscan, Skin biopsy, QSART
- Where
- United States
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Epilepsy, Lennox-Gastaut Syndrome
A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
- Trial name
- GWPCARE4 (NCT02224690)
- Testing
- GWP42003-P 20 mg/kg/day Dose
- Where
- 24 sites in United States, Netherlands and 1 more
- Volunteers
- 171 people
- Sponsor
- Jazz Pharmaceuticals
Huntington Disease
Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease
Huntington disease (HD) is an inherited neurodegenerative disease which affects over 30,000 people in both the United States and Australia.
- Trial name
- Reach2HD (NCT01590888)
- Testing
- PBT2
- Where
- 19 sites in United States, Australia
- Volunteers
- 109 people
- Sponsor
- Prana Biotechnology Limited
Gaucher Disease
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00376168
- Testing
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Where
- 11 sites in United States, Canada and 6 more
- Volunteers
- 32 people
- Sponsor
- Pfizer
Fabry Disease
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study.
- Trial name
- NCT02194985
- Testing
- migalastat HCl 150 mg
- Where
- 28 sites in United States, Argentina and 13 more
- Volunteers
- 84 people
- Sponsor
- Amicus Therapeutics
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme.
- Trial name
- NCT00074984
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 26 sites in United States, Canada and 4 more
- Volunteers
- 82 people
- Sponsor
- Genzyme, a Sanofi Company
Friedreich Ataxia
Neurology Measures in FA Children
The purpose of this study is to identify ways to follow progression of Friedreich's Ataxia (FA) and be able to measure changes over time in children with FA.
- Trial name
- NCT03418740
- Where
- 3 sites in United States
- Volunteers
- 108 people
- Sponsor
- Children's Hospital of Philadelphia