Search | Clinical Trials Finder

Conditions: Hemophilia B
Interventions: Adeno-Associated Viral with Human Factor IX; Genetic
Lead sponsor: Avigen; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2004
U.S. locations: 3
States / cities: Palo Alto, California • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania

Conditions: Muscular Dystrophy, Duchenne
Interventions: Eteplirsen; Drug
Lead sponsor: Sarepta Therapeutics, Inc.; Industry
Eligibility: 7 Years to 21 Years · Male only
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 9
States / cities: Los Angeles, California • Sacramento, California • Iowa City, Iowa + 6 more

Conditions: Fragile X Syndrome
Interventions: arbaclofen, placebo; Drug
Lead sponsor: Seaside Therapeutics, Inc.; Industry
Eligibility: 12 Years to 50 Years
Enrollment: 125 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2011
U.S. locations: 24
States / cities: Phoenix, Arizona • Long Beach, California • Sacramento, California + 20 more

Conditions: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 55 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Choroideremia
Interventions: 4D-110; Biological
Lead sponsor: 4D Molecular Therapeutics; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2027
U.S. locations: 2
States / cities: Dallas, Texas • Salt Lake City, Utah

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: Dyskeratosis Congenita, Telomere Biology Disorders
Interventions: Not listed
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years to 100 Years
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hemophilia B
Interventions: rFIXFc, FIX; Drug
Lead sponsor: Bioverativ Therapeutics Inc.; Industry
Eligibility: Up to 11 Years · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 7
States / cities: Phoenix, Arizona • Sacramento, California • Atlanta, Georgia + 4 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Hemophilia B standard half-life, Hemophilia B extended half-life, Hemophilia A standard half-life, Hemophilia A extended half-life; Drug
Lead sponsor: Pfizer; Industry
Eligibility: Not listed
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 3
States / cities: Gainesville, Florida • Las Vegas, Nevada • Fort Worth, Texas

Conditions: Immunologic Deficiency Syndromes, Chediak-Higashi Syndrome, Common Variable Immunodeficiency, Graft Versus Host Disease, X-Linked Lymphoproliferative Syndrome, Familial Erythrophagocytic Lymphohistiocytosis, Hemophagocytic Lymphohistiocytosis, X-linked Agammaglobulinemia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, X-linked Hyper IgM Syndrome, Severe Combined Immunodeficiency, Leukocyte Adhesion Deficiency Syndrome, Virus-Associated Hemophagocytic Syndrome
Interventions: anti-thymocyte globulin, busulfan, cyclophosphamide, cyclosporine, etoposide, methotrexate, methylprednisolone, prednisone, Allogeneic Bone Marrow Transplantation; Drug · Procedure
Lead sponsor: Fairview University Medical Center; Other
Eligibility: Up to 35 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2002
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Hemophilia A, Hemophilia B
Interventions: Ankle and Knee Ultrasound Joint Assessment, Ankle and Knee Magnetic Resonance Imaging; Other
Lead sponsor: University of Miami; Other
Eligibility: Up to 30 Months
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 4
States / cities: Gainesville, Florida • Miami, Florida • Lexington, Kentucky + 1 more

Conditions: X-linked Hypophosphatemia (XLH)
Interventions: No treatment given; Other
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years to 64 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 4
States / cities: New Haven, Connecticut • Indianapolis, Indiana • Columbus, Ohio + 1 more

Conditions: Reversal of Coagulopathy
Interventions: Beriplex® P/N (Kcentra), Fresh frozen plasma; Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older
Enrollment: 176 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2013
U.S. locations: 16
States / cities: Newark, Delaware • Lexington, Kentucky • Boston, Massachusetts + 13 more

Conditions: X-linked Hypophosphatemia
Interventions: KRN23; Drug
Lead sponsor: Kyowa Kirin Co., Ltd.; Industry
Eligibility: 18 Years and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 5
States / cities: San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more

Conditions: Hemophilia B
Interventions: CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua); Genetic
Lead sponsor: CSL Behring; Industry
Eligibility: 138 Months to 206 Months · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2033
U.S. locations: 5
States / cities: Orange, California • Gainesville, Florida • Atlanta, Georgia + 2 more

Conditions: Primary Hyperoxaluria, Dent Disease, APRT Deficiency, Cystinuria
Interventions: Not listed
Lead sponsor: Mayo Clinic; Other
Eligibility: Not listed
Enrollment: 2,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2030
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Fragile X Syndrome
Interventions: AFQ056, Placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years to 45 Years
Enrollment: 175 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 12
States / cities: Phoenix, Arizona • Sacramento, California • Decatur, Georgia + 9 more

Conditions: Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia, Carbamyl Phosphate Synthetase Deficiency, NAGS Deficiency
Interventions: Not listed
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Not listed
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 2
States / cities: Washington D.C., District of Columbia • Houston, Texas

Conditions: Rett Syndrome, MECP2 Duplication dIsorder, CDKL5 Disorder, FOXG1 Syndrome
Interventions: Not listed
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: Not listed
Enrollment: 1,044 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2021
U.S. locations: 14
States / cities: Birmingham, Alabama • Oakland, California • San Diego, California + 11 more

Conditions: Hemophilia A, Hemophilia B
Interventions: PF-06741086; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years to 74 Years · Male only
Enrollment: 188 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 4
States / cities: Tampa, Florida • Iowa City, Iowa • New Hyde Park, New York + 1 more

Conditions: Duchenne Muscular Dystrophy
Interventions: Not listed
Lead sponsor: University of Florida; Other
Eligibility: 18 Years to 99 Years
Enrollment: 57 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Intracranial Hemorrhage, Traumatic, Intracranial Hemorrhage, Spontaneous
Interventions: Four Factor Prothrombin Complex Concentrate, Fresh Frozen Plasma; Drug · Biological
Lead sponsor: University of Utah; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: Salt Lake City, Utah

Conditions: Duchenne Muscular Dystrophy
Interventions: HT-100; Drug
Lead sponsor: Akashi Therapeutics; Industry
Eligibility: 6 Years to 20 Years · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 5
States / cities: Sacramento, California • Baltimore, Maryland • St Louis, Missouri + 2 more

Conditions: X-Linked Retinitis Pigmentosa
Interventions: rAAV2tYF-GRK1-RPGR; Biological
Lead sponsor: Beacon Therapeutics; Industry
Eligibility: 8 Years to 50 Years · Male only
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 6
States / cities: Jacksonville, Florida • Boston, Massachusetts • Cincinnati, Ohio + 3 more

Search by objective public record fields.

Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia

Study of ANAVEX2-73 in Patients With Rett Syndrome

Needs Assessment for Individuals and Families Affected by Dyskeratosis Congenita (DC) and Related Telomere Biology Disorders (TBD)

Study of Recombinant Coagulation Factor IX Fc Fusion Protein, BIIB029, in Previously Treated Pediatric Participants With Hemophilia B

Understanding Hemophilia A and B Drug Dosage Administration Patterns

Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies

The Hemophilia Ultrasound Project

Treatment Patterns, Biochemical Profiles and Clinical Outcomes in Adults With X-Linked Hypophosphatemia

An Open-label, Randomized, Multicenter Phase IIIb Study to Assess the Efficacy, Safety and Tolerance of BERIPLEX® P/N (Kcentra) Compared With Plasma for Rapid Reversal of Coagulopathy Induced by Vitamin K Antagonists in Subjects Requiring an Urgent Surgical Procedure

An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

Rare Kidney Stone Consortium Biobank

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Hepatic Histopathology in Urea Cycle Disorders

Natural History of Rett Syndrome & Related Disorders

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

The Burden of Access in Duchenne Muscular Dystrophy in the US

FFP Versus PCC in Intracranial Hemorrhage

HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)