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Conditions: Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes, Severe Congenital Neutropenia
Interventions: alemtuzumab, busulfan, cyclosporine, fludarabine phosphate, methotrexate, methylprednisolone, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, umbilical cord blood transplantation; Biological · Drug · Procedure
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 21 Years
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2011
U.S. locations: 2
States / cities: San Francisco, California • Madison, Wisconsin

Conditions: p47, Autosomal Recessive, Chronic Granulomatous Disease
Interventions: Cryopreserved Autologous CD34+ cells transduced with pCCLCHIM-p47; Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 3 Years to 65 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hepato/Renal Fibrocystic Disease, Autosomal Recessive Polycystic Kidney Disease, Joubert Syndrome, Bardet Biedl Syndrome, Meckel-Gruber Syndrome, Congenital Hepatic Fibrosis, Caroli Syndrome, Oro-Facial-Digital Syndrome Type I, Nephronophthisis, Glomerulocystic Kidney Disease
Interventions: Not listed
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: Up to 18 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2030
U.S. locations: 6
States / cities: Washington D.C., District of Columbia • Atlanta, Georgia • Boston, Massachusetts + 3 more

Conditions: Gyrate Atrophy
Interventions: Not listed
Lead sponsor: National Eye Institute (NEI); NIH
Eligibility: Not listed
Enrollment: 65 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1978 – 2004
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Sickle Cell Disease, Beta-Thalassemia
Interventions: alpha beta depletion; Drug
Lead sponsor: Mitchell Cairo; Other
Eligibility: 1 Day to 30 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 1
States / cities: Valhalla, New York

Conditions: TNNT1-associated Myopathy, Infantile-onset Nemaline Rod Myopathy, Myopathies, Nemaline, Myopathy, Myopathy, Rod, Myopathy; Hereditary, Amish Nemaline Myopathy, Nemaline Myopathy 5, NEM5, Genetic Muscle Disease, Recessive Hereditary Disorder (Autosomal), ANM
Interventions: Not listed
Lead sponsor: Clinic for Special Children; Other
Eligibility: Not listed
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2027
U.S. locations: 1
States / cities: Gordonville, Pennsylvania

Conditions: Alport Syndrome, X-Linked, Alport Syndrome, Autosomal Recessive
Interventions: Exaluren; Drug
Lead sponsor: Eloxx Pharmaceuticals, Inc.; Industry
Eligibility: 12 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2027
U.S. locations: 8
States / cities: Los Angeles, California • Stanford, California • Denver, Colorado + 5 more

Conditions: Chronic Granulomatous Disease (CGD)
Interventions: Not listed
Lead sponsor: Ensoma; Industry
Eligibility: Not listed
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 50
States / cities: Montgomery, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 47 more

Conditions: Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions: Abatacept; Drug
Lead sponsor: Emory University; Other
Eligibility: Up to 21 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy
Interventions: SB-007; Genetic
Lead sponsor: Splice Bio; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 5
States / cities: Aurora, Colorado • Miami, Florida • Boston, Massachusetts + 2 more

Conditions: Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA)
Interventions: OPGx-RDH12; Drug
Lead sponsor: Opus Genetics, Inc; Industry
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2034
U.S. locations: 3
States / cities: Phoenix, Arizona • Philadelphia, Pennsylvania • Houston, Texas

Conditions: Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions: Allogeneic stem cell transplant; Device
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 21 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2009
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Stargardt, Stargardt's Disease, Stargardt Disease, STGD1
Interventions: Not listed
Lead sponsor: Splice Bio; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 75 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 14
States / cities: San Diego, California • Aurora, Colorado • Gainesville, Florida + 10 more

Conditions: Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions: Hematopoietic stem cell transplantation; Procedure
Lead sponsor: Children's Hospital Los Angeles; Other
Eligibility: Up to 21 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Stargardt Disease-1
Interventions: STG-001; Drug
Lead sponsor: Stargazer Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 5
States / cities: Phoenix, Arizona • Gainesville, Florida • Portland, Oregon + 2 more

Conditions: Citrate Transporter Deficiency, Epilepsy, Rare Diseases, Movement Disorders, Genetic Disorder, SLC13A5 Deficiency, EIEE25, Kohlschutter-Tonz Syndrome (Non-ROGDI), Citrate Transporter Disorder, DEE25
Interventions: Not listed
Lead sponsor: TESS Research Foundation; Other
Eligibility: Not listed
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 3
States / cities: Palo Alto, California • Providence, Rhode Island • Dallas, Texas

Conditions: ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy
Interventions: OPGx-BEST1; Genetic
Lead sponsor: Opus Genetics, Inc; Industry
Eligibility: 18 Years and older
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 4
States / cities: Los Angeles, California • Gainesville, Florida • Cincinnati, Ohio + 1 more

Conditions: Ichthyosis, Autosomal Recessive Congenital Ichthyosis, Lamellar Ichthyosis, Congenital Ichthyosiform Erythroderma, Epidermolytic Ichthyosis, Netherton Syndrome
Interventions: Secukinumab, Placebo; Drug
Lead sponsor: Northwestern University; Other
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2020
U.S. locations: 2
States / cities: Chicago, Illinois • New York, New York

Conditions: Homozygous Familial Hypercholesterolemia
Interventions: Demographics, diagnosis type, genotype, lipid profile, treatment allocation, country of residence.; Other
Lead sponsor: University of Pennsylvania; Other
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2026
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

Conditions: Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency, Autosomal Recessive Hypophosphatemic Rickets, Generalized Arterial Calcification of Infancy, ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Interventions: INZ-701; Drug
Lead sponsor: Inozyme Pharma; Industry
Eligibility: Up to 1 Year
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 5
States / cities: San Diego, California • Boston, Massachusetts • Columbus, Ohio + 2 more

Conditions: Dysthymia, Anxiety Disorders, Familial Dysautonomia, Paroxysmal Hypertension, Autosomal Recessive Disease
Interventions: 8-week CBT Program; Behavioral
Lead sponsor: NYU Langone Health; Other
Eligibility: 18 Years and older
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: New York, New York

Conditions: Neutropenia
Interventions: Mavorixafor, Placebo; Drug
Lead sponsor: X4 Pharmaceuticals; Industry
Eligibility: 12 Years and older
Enrollment: 176 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 22
States / cities: Phoenix, Arizona • Irvine, California • La Jolla, California + 19 more

Conditions: Generalized Arterial Calcification of Infancy, Autosomal Recessive Hypophosphatemic Rickets Type2
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Year to 110 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
Interventions: ALK-001; Drug
Lead sponsor: Alkeus Pharmaceuticals, Inc.; Industry
Eligibility: 8 Years to 70 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 8
States / cities: Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 5 more

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Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders

pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

ARPKD Database Study

Gyrate Atrophy of the Choroid and Retina

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

WiTNNess - TNNT1 Myopathy Natural History Study

EXACT Study: A Blinded Study in Patients With Alport Syndrome to Evaluate Exaluren Efficacy and Safety

Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Study of STG-001 in Subjects With Stargardt Disease

SLC13A5 Deficiency Natural History Study - United States Only

Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)

The Efficacy and Safety of Secukinumab in Patients With Ichthyoses

HoFH, the International Clinical Collaborators Registry

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

A Trial of Cognitive Behavioral Therapy in Familial Dysautonomia

A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

Study of People With Generalized Arterial Calcification of Infancy (GACI) or Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)