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Conditions: Rett Syndrome
Interventions: Bionetide, Placebo; Drug
Lead sponsor: Biomed Industries, Inc.; Industry
Eligibility: 5 Years to 20 Years · Female only
Enrollment: 210 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 14
States / cities: Phoenix, Arizona • La Jolla, California • Los Angeles, California + 11 more

Conditions: Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: RTI International; Other
Eligibility: 1 Day to 31 Days
Enrollment: 30,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: Rett Syndrome
Interventions: Intravenous infusion; Procedure
Lead sponsor: The Cleveland Clinic; Other
Eligibility: 5 Years to 25 Years · Female only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Cleveland, Ohio

Conditions: Rett Syndrome
Interventions: Recombinant Human Insulin Growth Factor 1 (rhIGF-1), Placebo; Drug
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 2 Years to 10 Years · Female only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Rett Syndrome
Interventions: Drug: Bionetide, Placebo; Drug
Lead sponsor: Biomed Industries, Inc.; Industry
Eligibility: 5 Years to 20 Years · Female only
Enrollment: 187 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2025
U.S. locations: 12
States / cities: La Jolla, California • Los Angeles, California • Sacramento, California + 9 more

Conditions: Rett Syndrome
Interventions: dextromethorphan, donepezil hydrochloride; Drug
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 1 Year to 15 Years
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2008
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Autism, Asperger's Syndrome, Rett's Disorder
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years and older
Enrollment: 324 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2015
U.S. locations: 1
States / cities: Bethesda, Maryland

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: Rett Syndrome, MECP2 Duplication, CDKL5, FOXG1 Disorders
Interventions: Not listed
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: Not listed
Enrollment: 752 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 12
States / cities: Birmingham, Alabama • Oakland, California • San Diego, California + 9 more

Conditions: Tuberous Sclerosis Complex
Interventions: Not listed
Lead sponsor: Translational Genomics Research Institute; Other
Eligibility: Not listed
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2023
U.S. locations: 1
States / cities: Phoenix, Arizona

Conditions: Telomeric 22Q13 Monosomy Syndrome, Tuberous Sclerosis, Hamartoma Syndrome, Multiple, Fragile X Syndrome, Angelman Syndrome, Rett Syndrome, Chromosome 15Q, Partial Deletion, Creatine Deficiency, X-linked
Interventions: Functional Behavioral Training (FBT), Positive Parenting Strategies-Treatment as Usual; Behavioral · Other
Lead sponsor: Rush University Medical Center; Other
Eligibility: 2 Years to 12 Years
Enrollment: 92 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Rett Syndrome
Interventions: TSHA-102; Genetic
Lead sponsor: Taysha Gene Therapies, Inc.; Industry
Eligibility: 6 Years to 21 Years · Female only
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2031
U.S. locations: 8
States / cities: Duarte, California • La Jolla, California • Oakland, California + 5 more

Conditions: Rett Syndrome, RTT, Rett Syndrome, Atypical
Interventions: EEG and Auditory and Visual Evoked Potentials (AEP and VEP), Clinical assessment; Other
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: 1 Year to 18 Years · Female only
Enrollment: 202 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2029
U.S. locations: 6
States / cities: Los Angeles, California • Aurora, Colorado • Brookline, Massachusetts + 3 more

Conditions: Rett Syndrome
Interventions: Modified Virtual Reality Gaming; Behavioral
Lead sponsor: Georgetown University; Other
Eligibility: 4 Years to 60 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Rett Syndrome
Interventions: ANAVEX2-73, Placebo; Drug
Lead sponsor: Anavex Life Sciences Corp.; Industry
Eligibility: 18 Years to 45 Years · Female only
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2020
U.S. locations: 8
States / cities: Birmingham, Alabama • Sacramento, California • Chicago, Illinois + 5 more

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: Rett Syndrome
Interventions: fenfluramine hydrochloride, Placebo; Drug · Other
Lead sponsor: UCB BIOSCIENCES, Inc.; Industry
Eligibility: 5 Years to 35 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 4
States / cities: Little Rock, Arkansas • Orlando, Florida • Houston, Texas + 1 more

Conditions: Rett Syndrome
Interventions: rhIGF-1; Drug
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 2 Years to 12 Years · Female only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Rett Syndrome
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 18 Years and older
Enrollment: 426 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2016
U.S. locations: 3
States / cities: Birmingham, Alabama • Baltimore, Maryland • Houston, Texas

Conditions: Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome, Duchenne Muscular Dystrophy, Phelan-McDermid Syndrome, Rett Syndrome, Smith Magenis Syndrome, Williams Syndrome, Turner Syndrome, Klinefelter Syndrome, Chromosome 22q11.2 Deletion Syndrome, Tuberous Sclerosis, Down Syndrome
Interventions: Parent-Infant Inter(X)action Intervention (PIXI); Behavioral
Lead sponsor: RTI International; Other
Eligibility: Up to 99 Years
Enrollment: 120 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2026
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

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

Conditions: Rett Syndrome
Interventions: Electroencephalogram (EEG) Recording; Procedure
Lead sponsor: University of Rochester; Other
Eligibility: 2 Years and older
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Rochester, New York

Conditions: Rett Syndrome, Rett Syndrome, Atypical, Genetic Disease, Genetic Diseases, X-Linked, Intellectual Disability, Neurobehavioral Manifestations, Neurologic Manifestations, Neurologic Disorder, Neurodevelopmental Disorders, Nervous System Diseases
Interventions: Not listed
Lead sponsor: International Rett Syndrome Foundation; Other
Eligibility: 0 Years to 99 Years
Enrollment: 3,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2028
U.S. locations: 19
States / cities: Birmingham, Alabama • Los Angeles, California • Oakland, California + 16 more

Conditions: Rett Syndrome
Interventions: Emerald; Device
Lead sponsor: Rett Syndrome Research Trust; Other
Eligibility: Female only
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Boston, Massachusetts

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To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome

Early Check: Expanded Screening in Newborns

An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome

Treatment of Rett Syndrome With Recombinant Human IGF-1

To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome

Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome

Predictors of Caregiver Adaptation to Pervasive Developmental Disorders

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

Biobanking of Rett Syndrome and Related Disorders

Studies in Patients With Tuberous Sclerosis Complex

Web Intervention for Parents of Youth With Genetic Syndromes (WINGS)

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

Neurophysiologic Biomarkers in Rett Syndrome

Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome

Study of ANAVEX2-73 in Patients With Rett Syndrome

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

A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)

The Role of Family Functioning in Adaptation to Being a Caregiver of an Individual With Rett Syndrome

Parent and Infant Inter(X)Action Intervention (PIXI)

Trial of Dextromethorphan in Rett Syndrome

Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome

Rett Syndrome Registry

ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME