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Conditions: Phenylketonurias
Interventions: nointervention; Other
Lead sponsor: University of Missouri-Columbia; Other
Eligibility: 18 Years to 40 Years
Enrollment: 84 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2021
U.S. locations: 2
States / cities: Atlanta, Georgia • Columbia, Missouri

Conditions: Endothelial Dysfunction
Interventions: Sapropterin Dihydrochloride, Sapropterin Dihydrochloride and Vitamin C; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2009
U.S. locations: 1
States / cities: Hackensack, New Jersey

Conditions: Fabry Disease
Interventions: migalastat HCl 150 mg; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 12 Years to 17 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2024
U.S. locations: 5
States / cities: Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 2 more

Conditions: Fabry Disease
Interventions: migalastat hydrochloride, agalsidase; Drug · Biological
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 16 Years to 74 Years
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2015
U.S. locations: 8
States / cities: Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more

Conditions: Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions: Family Centered pediatric palliative care for family caregivers of children with rare diseases.; Behavioral
Lead sponsor: Children's National Research Institute; Other
Eligibility: 12 Months to 99 Years
Enrollment: 480 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2029
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Phenylketonuria
Interventions: rAvPAL-PEG; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 16 Years to 55 Years
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2019
U.S. locations: 14
States / cities: Aurora, Colorado • Gainesville, Florida • Chicago, Illinois + 11 more

Conditions: Hypopituitarism
Interventions: Arimidex (Anastrozole), Placebo, Growth Hormone; Drug
Lead sponsor: Nemours Children's Clinic; Other
Eligibility: 11 Years to 18 Years · Male only
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2010
U.S. locations: 1
States / cities: Jacksonville, Florida

Conditions: Arnold-Chiari Malformation, Posterior Fossa Tumors
Interventions: IV acetaminophen, normal saline; Drug
Lead sponsor: Children's National Research Institute; Other
Eligibility: 2 Years to 12 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Fabry Disease
Interventions: migalastat; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: Female only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Gaucher Disease
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 200 participants
Timeline: 2017 – 2027
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Behavioral Manifestations of Lesch-Nyhan Disease
Interventions: sapropterin; Drug
Lead sponsor: University of California, San Diego; Other
Eligibility: 4 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2010
U.S. locations: 1
States / cities: San Diego, California

Conditions: Lysosomal Diseases, Gangliosidosis, GM1
Interventions: AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone, Prednisone, Audiology assessment with ABR, Bone density scan (DEXA), Electrocardiogram (EKG), Echocardiogram, Electroencephalogram (EEG) awake and extended overnight, Laboratory tests, Lumbar puncture, Brain MRI/MRS/fMRI, Neurocognitive testing, Neurology exam, PICC or other Central line placement, Skeletal survey, Skin biopsy, Speech and modified barium swallow study, Ophthalmology exam; Biological · Procedure · Drug + 3 more
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 6 Months to 12 Years
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2028
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Tics, Tourette Syndrome
Interventions: CBIT +cTBS, CBIT +sham cTBS; Other
Lead sponsor: University of Minnesota; Other
Eligibility: 12 Years to 21 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Metastatic Colorectal Cancer
Interventions: Pembrolizumab, Binimetinib, Oxaliplatin, Leucovorin, 5-Fluorouracil [5-FU], Irinotecan; Biological · Drug
Lead sponsor: Merck Sharp & Dohme LLC; Industry
Eligibility: 18 Years and older
Enrollment: 116 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 10
States / cities: Duarte, California • Aurora, Colorado • New Haven, Connecticut + 7 more

Conditions: Solid Tumors, CNS Tumors
Interventions: Entrectinib; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 0 Years to 18 Years
Enrollment: 69 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2026
U.S. locations: 15
States / cities: La Jolla, California • San Francisco, California • Aurora, Colorado + 12 more

Conditions: Sjogren-Larsson Syndrome (SLS)
Interventions: Not listed
Lead sponsor: University of Nebraska; Other
Eligibility: Not listed
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2023
U.S. locations: 2
States / cities: Omaha, Nebraska • Pittsburgh, Pennsylvania

Conditions: Huntington Disease
Interventions: active tDCS; Device
Lead sponsor: The University of Texas Health Science Center, Houston; Other
Eligibility: 18 Years to 80 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Zellweger Spectrum
Interventions: Not listed
Lead sponsor: University of South Florida; Other
Eligibility: 18 Years and older
Enrollment: 92 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018
U.S. locations: 1
States / cities: Tampa, Florida

Conditions: Leukodystrophy, Globoid Cell
Interventions: PBKR03; Biological
Lead sponsor: Gemma Biotherapeutics; Industry
Eligibility: 1 Month to 9 Months
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2030
U.S. locations: 4
States / cities: Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more

Conditions: Tourette's Disorder
Interventions: vigabatrin; Drug
Lead sponsor: Barbara J. Coffey; Other
Eligibility: 18 Years to 35 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2015
U.S. locations: 1
States / cities: New York, New York

Conditions: Brain Malformation, Neuronal Migration Disorder, Cognition Disorder, Epilepsy
Interventions: Not listed
Lead sponsor: Harvard University Faculty of Medicine; Other
Eligibility: Not listed
Enrollment: 3,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1996 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Friedreich's Ataxia
Interventions: Placebo, EPI-743 400 mg, EPI-743 200 mg; Drug
Lead sponsor: PTC Therapeutics; Industry
Eligibility: 18 Years to 45 Years
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 3
States / cities: Los Angeles, California • Tampa, Florida • Philadelphia, Pennsylvania

Conditions: Huntington's Disease
Interventions: SEN0014196; Drug
Lead sponsor: Siena Biotech S.p.A.; Industry
Eligibility: 25 Years to 65 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 7
States / cities: La Jolla, California • Sacramento, California • St Louis, Missouri + 4 more

Conditions: Cranial Waveform in Normal Patients as a Result of Cardiac Cycle, Healthy
Interventions: Harmony Headset; Device
Lead sponsor: MindRhythm, Inc.; Industry
Eligibility: 18 Years to 100 Years
Enrollment: 500 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023
U.S. locations: 1
States / cities: Cupertino, California

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Remote Neurocognitive and Psychological Assessment in PKU

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

Palliative Care Needs of Children With Rare Diseases and Their Families

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

Arimidex Multicenter Trial in Growth Hormone (GH) Deficient Boys

Efficacy of Acetaminophen in Posterior Fossa Surgery

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

A Long-term Follow-up Study of Gaucher Disease

Trial of Kuvan in Lesch-Nyhan Disease

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

CBIT+TMS R33 Phase

Safety and Efficacy of Pembrolizumab (MK-3475) Plus Binimetinib Alone or Pembrolizumab Plus Chemotherapy With or Without Binimetinib in Metastatic Colorectal Cancer (mCRC) Participants (MK-3475-651/KEYNOTE-651)

Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

Home-based Transcranial Direct Current Stimulation Open Trial for Behavioral and Cognitive Symptoms in Huntington's Disease

Proxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum Disorders

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

Proof-of-Concept Safety Study of CPP-109 (Vigabatrin) for Treatment Refractory Tourette's Disorder

Human Epilepsy Genetics--Neuronal Migration Disorders Study

Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

A Open-label Food Effect Study With SEN0014196 in Subjects With Huntington Disease

500 "Normal" Adult Subjects Who Have Experienced no Known Brain Injury Will Participate in Collecting Data That Will Assist With the Optimization of the Diagnostic Capabilities of the Harmony in Detecting Neurologic Abnormalities.