Clinical trials: Brain Malformation
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 a-galactosidase A enzyme.
- Trial name
- NCT00074971
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 20 sites in United States, France and 3 more
- Volunteers
- 58 people
- Sponsor
- Genzyme, a Sanofi Company
Hypoxic-Ischemic Encephalopathy, Bronchopulmonary Dysplasia
Efficacy Evaluation of UCB-MNCs in the Treatment of Refractory Neonatal Diseases
Hypoxic-ischemic encephalopathy (HIE), bronchopulmonary dysplasia (BPD), short bowel syndrome (SBS) are refractory in clinical treatment.
- Conditions
- Hypoxic-Ischemic Encephalopathy, Bronchopulmonary Dysplasia, Short Bowel Syndrome
- Trial name
- NCT06427642
- Testing
- Mononuclear cells, Mild hypothermia therapy, Breathing support technique
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Shandong Qilu Stem Cells Engineering Co., Ltd.
Friedreich's Ataxia
A First in Human Study of RT001 in Patients With Friedreich's Ataxia
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of RT001 in patients with Friedreich's ataxia.
- Trial name
- NCT02445794
- Testing
- Low dose cohort, High dose cohort
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Biojiva LLC
Phenylketonuria
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
The purpose of this study is to evaluate whether weekly injections of phenylalanine ammonia lyase (rAvPAL-PEG) can reduce blood phenylalanine concentrations in PKU subjects and whether repeated administration is safe.
- Trial name
- NCT00925054
- Testing
- rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg
- Where
- 11 sites in United States
- Volunteers
- 40 people
- Sponsor
- BioMarin Pharmaceutical
Ornithine Transcarbamylase Deficiency, OTC Deficiency
Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810
The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.
- Conditions
- Ornithine Transcarbamylase Deficiency, OTC Deficiency, OTCD
- Trial name
- NCT05526066
- Testing
- ARCT-810
- Where
- 14 sites in Belgium, France and 4 more
- Volunteers
- 8 people
- Sponsor
- Arcturus Therapeutics, Inc.
Huntington Disease
Open-label Extension Study of Pridopidine (ACR16) in the Symptomatic Treatment of Huntington Disease
Huntington disease (HD) is a hereditary neurodegenerative disorder causing impairment in movement, behavioral dysfunction and dementia.
- Trial name
- OPEN-HART (NCT01306929)
- Testing
- pridopidine
- Volunteers
- 134 people
- Sponsor
- Prilenia
Fabry Disease
Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry)
Primary objective: To estimate the prevalence of patients who are at high-risk for Fabry Disease (FD) in the Cleveland Clinic, Abu Dhabi (CCAD) United Arab Emirates (EMR) database from May 2016 to May 2022, according to the…
- Trial name
- NCT05671770
- Where
- United Arab Emirates
- Volunteers
- 15 people
- Sponsor
- Sanofi
Wilson Disease
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation…
- Trial name
- NCT03403205
- Testing
- ALXN1840, SoC Therapy
- Where
- 56 sites in United States, Australia and 20 more
- Volunteers
- 214 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Phenylketonurias
Evaluation of PKU Explore France
PKU explore France is an exploratory study to evaluate the acceptability of PKU explore, a food for special medical purposes, for use in the dietary management of phenylketonuria in infants from 6 months to 3 years of age…
- Trial name
- NCT04898829
- Testing
- PKU explore
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Vitaflo International, Ltd
Hereditary Tyrosinemia, Type I
Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1
The purpose of this study is to look at the steady-state serum concentrations of nitisinone when switching from twice daily and once daily dosing.
- Trial name
- HT-1 (NCT02323529)
- Testing
- Nitisinone
- Where
- 6 sites in Belgium, Denmark and 3 more
- Volunteers
- 18 people
- Sponsor
- Swedish Orphan Biovitrum
Gaucher Disease Type I, Cerebroside Lipidosis Syndrome
A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating patients with skeletal manifestations secondary to Type I Gaucher disease.
- Conditions
- Gaucher Disease Type I, Cerebroside Lipidosis Syndrome, Clucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease
- Trial name
- NCT00365131
- Testing
- Cerezyme (imiglucerase for injection)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Genzyme, a Sanofi Company
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia.
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Trial name
- NCT06255782
- Testing
- ECUR-506
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 20 people
- Sponsor
- iECURE, Inc.
Patent Foramen Ovale, Cryptogenic Stroke
Safety and Efficacy of IrisFIT™ Patent Foramen Ovale Occluders
To evaluate the performance of patent foramen ovale (PFO) device developed by Lifetech Technology (Shenzhen) Co., LTD
- Trial name
- NCT05312554
- Testing
- Iris FIT™ PFO closure system
- Where
- China
- Sponsor
- Lifetech Scientific (Shenzhen) Co., Ltd.
Tourette Syndrome
A Study to Test if TEV-50717 is Effective in Relieving Tics Associated With Tourette Syndrome (TS)
Standard placebo-controlled, double-blind study design (TEV-50717 [low dose and high dose] vs. placebo in a 1:1:1 ratio) was chosen to determine whether study drug treatment results in a statistically significant effect on the…
- Trial name
- ARTISTS2 (NCT03571256)
- Testing
- TEV-50717
- Where
- 56 sites in United States, Argentina and 8 more
- Volunteers
- 158 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Coronary Artery Disease
Observational Study of OCT in a Patients Undergoing FFR
The purpose of this study is to define and evaluate optical coherence tomography (OCT) stent guidance parameters through prospective data collection in percutaneous coronary intervention (PCI) procedures of de novo lesions.
- Trial name
- ILUMIEN I (NCT01663896)
- Testing
- OCT stent guidance
- Where
- 36 sites in United States, Australia and 12 more
- Volunteers
- 418 people
- Sponsor
- Abbott Medical Devices
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Gaucher Disease
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease
The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants up to 5 years of age with Gaucher disease. Symptoms will be checked with blood tests.
- Trial name
- PEDS (NCT04721366)
- Testing
- Standard of Care
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Takeda
Lennox Gastaut Syndrome (LGS)
A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome
The aims of the study are: to learn if soticlestat, when given as add-on therapy, reduces the number of major motor drop seizures in children, teenagers, and adults with Lennox-Gastaut Syndrome.
- Trial name
- NCT04938427
- Testing
- Soticlestat
- Where
- 98 sites in United States, Australia and 16 more
- Volunteers
- 270 people
- Sponsor
- Takeda
Phenylketonuria
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
The primary objective of the study is to evaluate the effect of dosing regimens of multiple subcutaneous (SC) doses of rAvPAL-PEG to induce an early and sustained Phe reduction while decreasing the frequency and severity of…
- Trial name
- NCT01560286
- Testing
- BMN 165 (rAvPAL-PEG)
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- BioMarin Pharmaceutical
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