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Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
Interventions: Adalimumab Injection [Humira], Saline Solution for Injection; Drug
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center; Other
Eligibility: 5 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2027
U.S. locations: 1
States / cities: Torrance, California

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions: There is no intervention; Other
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 64 Years
Enrollment: 250 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2050
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Lysosomal Storage Disease, Peroxisomal Disorder
Interventions: Campath-1H, Clofarabine, Melphalan, Total Body Irradiation with Marrow Boosting, Hematopoietic stem cell transplantation, Cyclosporine A, Mycophenolate mofetil; Drug · Radiation · Biological
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 55 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Rett Syndrome
Interventions: Trofinetide; Drug
Lead sponsor: ACADIA Pharmaceuticals Inc.; Industry
Eligibility: 2 Years to 5 Years · Female only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 7
States / cities: Birmingham, Alabama • Aurora, Colorado • Chicago, Illinois + 4 more

Conditions: Rett Syndrome
Interventions: Not listed
Lead sponsor: Edison Pharmaceuticals Inc; Industry
Eligibility: 2 Years to 10 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015
U.S. locations: 1
States / cities: Birmingham, Alabama

Conditions: Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions: CliniMACS CD34 Reagent System; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: Up to 65 Years
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Rett Syndrome, RTT
Interventions: GWP42003-P; Drug
Lead sponsor: Jazz Pharmaceuticals; Industry
Eligibility: 2 Years to 18 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 14
States / cities: Birmingham, Alabama • La Jolla, California • Aurora, Colorado + 11 more

Conditions: Angelman Syndrome, Fragile X Syndrome
Interventions: OV101; Drug
Lead sponsor: Ovid Therapeutics Inc.; Industry
Eligibility: 13 Years to 17 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017
U.S. locations: 2
States / cities: Chicago, Illinois • Nashville, Tennessee

Conditions: Autism Spectrum Disorder, Fragile X Syndrome
Interventions: Mismatch negativity; Behavioral
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 15 Years to 35 Years
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Hunter Syndrome
Interventions: Neurobehavioral testing, Visual and auditory assessments; Behavioral · Other
Lead sponsor: Shire; Industry
Eligibility: 32 Months to 18 Years · Male only
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Rett Syndrome, Family Caregivers, Social Support, Psychological Support
Interventions: Those who have patients prescribed Daybue, Caregiver Speaks + Daybue; Other
Lead sponsor: Colorado State University; Other
Eligibility: 18 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Fort Collins, Colorado

Conditions: Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication &#X7C; Blood or Tissue &#X7C; Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions: Not listed
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: Not listed
Enrollment: 12,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2030
U.S. locations: 23
States / cities: Los Angeles, California • Orange, California • Palo Alto, California + 18 more

Conditions: Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions: Assisted Standing Treatment Program; Other
Lead sponsor: Gillette Children's Specialty Healthcare; Other
Eligibility: 3 Years to 14 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Saint Paul, Minnesota

Conditions: Danon Disease
Interventions: RP-A501; Biological
Lead sponsor: Rocket Pharmaceuticals Inc.; Industry
Eligibility: 8 Years and older · Male only
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2025
U.S. locations: 3
States / cities: La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania

Conditions: Anxiety Disorders, Child Developmental Disorders, Pervasive
Interventions: Not listed
Lead sponsor: University of California, Davis; Other
Eligibility: 5 Years to 25 Years
Enrollment: 511 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2005 – 2010
U.S. locations: 1
States / cities: Sacramento, California

Conditions: Mucopolysaccharidosis II
Interventions: AGT-182; Drug
Lead sponsor: ArmaGen, Inc; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 3
States / cities: Oakland, California • Orange, California • Decatur, Georgia

Conditions: Fragile X Syndrome
Interventions: Baclofen, Memantine, Roflumilast, Placebo; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 18 Years to 45 Years
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Fragile X Syndrome, FXS, Fra(X) Syndrome
Interventions: BPN14770, Placebo; Drug
Lead sponsor: Tetra Discovery Partners; Industry
Eligibility: 18 Years to 45 Years · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: ALD (Adrenoleukodystrophy), Adrenoleukodystrophy, Cerebral Adrenoleukodystrophy
Interventions: Medical Record Abstraction, Biospecimen Sample Collection; Other
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2030
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Rett Syndrome
Interventions: Not listed
Lead sponsor: Rett Syndrome Research Trust; Other
Eligibility: Not listed
Enrollment: 5,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2031
U.S. locations: 1
States / cities: Trumbull, Connecticut

Conditions: Rett Syndrome
Interventions: dextromethorphan, placebo; Drug
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 1 Year to 10 Years
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Brain Disorders, Aicardi Syndrome
Interventions: Not listed
Lead sponsor: University of California, San Francisco; Other
Eligibility: Not listed
Enrollment: 2,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2027
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Fragile X Syndrome, FMR1 Premutation, Primary Ovairan Insufficiency, Premature Ovarian Failure, Premature Menopause
Interventions: Not listed
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 18 Years and older · Female only
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Rett Syndrome
Interventions: Dextromethorphan; Drug
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 2 Years to 15 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2010
U.S. locations: 1
States / cities: Baltimore, Maryland

Search by objective public record fields.

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

Analysis of the Glutathione Cycle in Children With Rett Syndrome

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome

Single Dose Pharmacokinetic (PK) Study

Speech-in-noise Perception in Autism and Fragile X

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

Adapting a Psychosocial Support Intervention for the Rett Syndrome Community

The Myelin Disorders Biorepository Project

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

Examining Social, Emotional, and Cognitive Functioning in People With Fragile X and Down Syndromes

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome

Adrenoleukodystrophy National Registry Study

The Rett Syndrome Global Registry

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Brain Development Research Program

The Natural History of Reproductive and Overall Health in Girls and Women With a Pre-Mutation in the FMR1 Gene; Creation of a Patient Registry

Trial of Dextromethorphan in Rett Syndrome