Clinical trials: Brain Malformation
CCM, Cavernoma
CASH (Cavernous Angiomas With Symptomatic Hemorrhage) Trial Readiness
Brain Cavernous Angiomas with Symptomatic Hemorrhage (CASH) are rare, but they exact a heavy burden of neurologic disability from recurrent bleeding, for which there is no proven therapy.
- Conditions
- CCM, Cavernoma, Cerebral Cavernous Malformation, Cerebral Cavernous Malformations 1
- Trial name
- NCT03652181
- Where
- United States
- Volunteers
- 123 people
- Sponsor
- University of Chicago
Huntington Disease
Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease
The purpose of this research study is to study the safety and efficacy of fenofibrate, an FDA-approved drug for high cholesterol and/or elevated triglycerides (fats), as a treatment for Huntington's disease (HD).
- Trial name
- NCT03515213
- Testing
- Fenofibrate
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of California, Irvine
Fabry Disease
A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
People with Fabry Disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme.
- Trial name
- NCT00081497
- Testing
- agalsidase beta
- Where
- 25 sites in United States, Canada and 4 more
- Volunteers
- 67 people
- Sponsor
- Genzyme, a Sanofi Company
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) and how migalastat works in participants with Fabry disease.
- Trial name
- NCT00283959
- Testing
- migalastat HCl
- Where
- 2 sites in Australia, Brazil
- Volunteers
- 4 people
- Sponsor
- Amicus Therapeutics
Infantile Neronal Ceroid Lipofuscinosis
Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis
This study will examine the effectiveness of a drug called Cystagon in treating infantile neuronal ceroid lipofuscinosis (INCL), a progressive neurological disease affecting children.
- Trial name
- NCT00028262
- Testing
- Cystagon
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy
The objective of the study is to determine whether the combination of the bile acid TUDCA, and doxycycline will slow the progression of familial and senile amyloidosis.
- Trial name
- NCT01855360
- Testing
- Tauroursodeoxycholic Acid and Doxycycline
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Brigham and Women's Hospital
Phenylketonurias
Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU.
This is an open-label, randomized, 2-way crossover, monocentric controlled study in patients (≥ 16 years old) with phenylketonuria (PKU).
- Trial name
- NCT05827536
- Testing
- PKU GOLIKE, Standard of Care
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- APR Applied Pharma Research s.a.
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Opioid Titration Order Sheet or Standard Care in Treating Patients With Cancer Pain
RATIONALE: An Opioid Titration Order Sheet that allows healthcare providers to adjust the dose and schedule of pain medication may help improve pain treatment for patients with cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00666211
- Testing
- educational intervention, Titrated pain management, questionnaire administration
- Where
- 11 sites in United States
- Volunteers
- 98 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Huntington Disease, Chorea
Risperidone for the Treatment of Huntington's Disease Involuntary Movements
The purpose of this study is to assess the safety and benefit of risperidone for the treatment of chorea (involuntary movements) in Huntington's disease.
- Trial name
- NCT04201834
- Testing
- Risperidone, BioStamp nPoint device
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Rochester
Health Volunteers, Huntington Disease
A Study to Evaluate Sigma-1 and Dopamine-2 Receptor Occupancy by Pridopidine in the Human Brain of Healthy Volunteers and in Patients With Huntington's Disease
The purpose of this study is to demonstrate engagement of pridopidine with S1R and D2R (optional) in the living human brain. No formal statistical analysis will be conducted
- Trial name
- NCT03019289
- Testing
- pridopidine (90 mg)
- Where
- Germany
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Prilenia
Transthyretin (TTR) Amyloid Cardiomyopathy
Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy
Open label study to evaluate tafamidis for the treatment of transthyretin cardiomyopathy
- Trial name
- NCT02791230
- Testing
- Tafamidis
- Where
- 80 sites in United States, Argentina and 15 more
- Volunteers
- 1,733 people
- Sponsor
- Pfizer
Sickle Cell Disease
Measurement of Cerebral Blood Flow Using Transcranial Doppler Ultrasound in Children With Sickle Cell Disease
Sickle cell disease (SCD) affects haemoglobin - the molecule in blood cells which carries oxygen. It causes red blood cells to become abnormal crescent (or sickle)- shaped.
- Trial name
- NCT02090881
- Testing
- Ultrasound scan
- Where
- United Kingdom
- Volunteers
- 25 people
- Sponsor
- Imperial College Healthcare NHS Trust
Tourette Disorder, Tourette Syndrome
Guanfacine in Children With Tic Disorders
The goal of this pilot study is to obtain preliminary information on the tolerability and efficacy of extended release guanfacine (trade name Intuniv) in children with Tourette Disorder (TD, also called Tourette syndrome).
- Trial name
- NCT01547000
- Testing
- extended-release guanfacine (Intuniv)
- Where
- 3 sites in United States
- Volunteers
- 34 people
- Sponsor
- Yale University
Gaucher Disease, Type 3
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD.
- Trial name
- NCT04002830
- Testing
- Elelyso
- Where
- 3 sites in India, Israel and 1 more
- Volunteers
- 14 people
- Sponsor
- Ari Zimran
Pompe Disease (Late-onset)
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.
- Trial name
- NCT04138277
- Testing
- AT2221, ATB200
- Where
- 60 sites in United States, Argentina and 21 more
- Volunteers
- 119 people
- Sponsor
- Amicus Therapeutics
Phenylketonuria
The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study
Newborn screening and early treatment prevent the most severe manifestations of phenylketonuria (PKU). However, executive functioning deficits, attention deficit disorder, slow processing speed, and visual-motor problems commonly…
- Trial name
- NCT01917344
- Testing
- MRI
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Boston Children's Hospital
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
Phenylketonuria
Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period
The primary objective of the study is to evaluate the proportion of responders (that is, greater than or equal to [>=] 30 percent reduction from Baseline in blood phenylalanine [Phe] level) to treatment with Kuvan® (sapropterin…
- Trial name
- ENDURE (NCT01082328)
- Testing
- Kuvan®
- Where
- Norway
- Volunteers
- 59 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Arginase I Deficiency, Hyperargininemia
Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D.
- Trial name
- NCT03921541
- Testing
- Pegzilarginase
- Where
- 32 sites in United States, Austria and 5 more
- Volunteers
- 32 people
- Sponsor
- Immedica Pharma AB
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen