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Conditions: Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases
Interventions: Red Cell Transfusions, Iron Chelation, Hydroxyurea, Phlebotomy; Procedure · Drug
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 5 Years to 18 Years
Enrollment: 134 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2010
U.S. locations: 28
States / cities: Birmingham, Alabama • Washington D.C., District of Columbia • Jacksonville, Florida + 23 more

Conditions: Friedreich's Ataxia
Interventions: Interferon γ-1b; Drug
Lead sponsor: Amgen; Industry
Eligibility: 10 Years to 26 Years
Enrollment: 86 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 4
States / cities: Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more

Conditions: Adrenoleukodystrophy
Interventions: glyceryl trierucate/glyceryl trioleate; Drug
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 18 Months to 8 Years · Male only
Enrollment: 126 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2014
U.S. locations: 2
States / cities: Baltimore, Maryland

Conditions: Diabetes Mellitus Type 2, Non Alcoholic Fatty Liver Disease, Obesity
Interventions: Amlexanox; Drug
Lead sponsor: University of Michigan; Other
Eligibility: 18 Years to 75 Years
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2014
U.S. locations: 1
States / cities: Ann Arbor, Michigan

Conditions: Pompe Disease
Interventions: Albuterol; Drug
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Graft Versus Host Disease, Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions: beclomethasone dipropionate; Drug
Lead sponsor: Fred Hutchinson Cancer Center; Other
Eligibility: 5 Years to 75 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2002
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7), Childhood Acute Minimally Differentiated Myeloid Leukemia (M0), Childhood Acute Monoblastic Leukemia (M5a), Childhood Acute Monocytic Leukemia (M5b), Childhood Acute Myeloblastic Leukemia With Maturation (M2), Childhood Acute Myeloblastic Leukemia Without Maturation (M1), Childhood Acute Myelomonocytic Leukemia (M4), Childhood Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Fanconi Anemia, Refractory Anemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Ringed Sideroblasts, Secondary Myelodysplastic Syndromes, Untreated Childhood Acute Myeloid Leukemia and Other Myeloid Malignancies
Interventions: laboratory biomarker analysis; Other
Lead sponsor: Children's Oncology Group; Network
Eligibility: Up to 21 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2016
U.S. locations: 1
States / cities: Monrovia, California

Conditions: Urea Cycle Disorder
Interventions: KB195; Drug
Lead sponsor: Kaleido Biosciences; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2021
U.S. locations: 6
States / cities: Washington D.C., District of Columbia • Tampa, Florida • Baltimore, Maryland + 3 more

Conditions: Congenital Adrenal Hyperplasia
Interventions: Chronocort; Drug
Lead sponsor: Immedica Pharma AB; Industry
Eligibility: 16 Years and older
Enrollment: 76 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 11
States / cities: Los Angeles, California • Orange, California • Jacksonville, Florida + 8 more

Conditions: Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
Interventions: BMN 110; Drug
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: Up to 5 Years
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 2
States / cities: Oakland, California • Manhasset, New York

Conditions: Prader-Willi Syndrome
Interventions: Interview; Other
Lead sponsor: Soleno Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 59 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2021
U.S. locations: 1
States / cities: Plymouth, Massachusetts

Conditions: Glycogen Storage Disease Type II
Interventions: Alglucosidase alfa GZ419829; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 0 Days to 6 Months
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2026
U.S. locations: 5
States / cities: Hawthorne, New York • Durham, North Carolina • Cincinnati, Ohio + 2 more

Conditions: Atherosclerosis, Coronary Artery Disease
Interventions: lovastatin; Drug
Lead sponsor: Merck Sharp & Dohme LLC; Industry
Eligibility: 21 Years to 67 Years
Enrollment: 270 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1985 – 1992
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Fanconi Anemia
Interventions: Etanercept; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 4 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2010
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Cytomegalovirus Retinitis, HIV Infections
Interventions: Fomivirsen sodium, Ganciclovir; Drug
Lead sponsor: Ionis Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 194 participants
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 18
States / cities: Los Angeles, California • Pasadena, California • San Diego, California + 14 more

Conditions: Mucopolysaccharidoses
Interventions: Not listed
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center; Other
Eligibility: 5 Years to 35 Years
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2019
U.S. locations: 3
States / cities: Oakland, California • Torrance, California • Minneapolis, Minnesota

Conditions: Non-transfusion Dependent Thalassemia
Interventions: deferasirox, placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 10 Years and older
Enrollment: 166 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2012
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • New York, New York

Conditions: X-linked Adrenoleukodystrophy
Interventions: exercise training; Behavioral
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Other
Eligibility: 21 Years to 70 Years · Female only
Enrollment: 31 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2014
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
Interventions: Acoramidis, Placebo oral tablet; Drug
Lead sponsor: Eidos Therapeutics, a BridgeBio company; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 587 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 40
States / cities: La Jolla, California • Los Angeles, California • San Francisco, California + 31 more

Conditions: Propionic Acidemia
Interventions: mRNA-3927; Biological
Lead sponsor: ModernaTX, Inc.; Industry
Eligibility: 1 Year and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2031
U.S. locations: 7
States / cities: Los Angeles, California • Palo Alto, California • Ann Arbor, Michigan + 4 more

Conditions: Bloating
Interventions: OPTI-BIOME™ Bacillus subtilis MB40, Placebo; Dietary Supplement · Other
Lead sponsor: BIO-CAT Microbials, LLC; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2017
U.S. locations: 2
States / cities: Foothill Ranch, California • Orlando, Florida

Conditions: Fabry Disease
Interventions: migalastat HCl, ERT; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 450 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 8
States / cities: Birmingham, Alabama • Little Rock, Arkansas • Atlanta, Georgia + 5 more

Conditions: Sanfilippo Syndrome Type A (MPS IIIA)
Interventions: SOBI003; Drug
Lead sponsor: Swedish Orphan Biovitrum; Industry
Eligibility: 12 Months to 72 Months
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 2
States / cities: Oakland, California • Chapel Hill, North Carolina

Conditions: Haemophilia A, Haemophilia B, Obesity
Interventions: No treatment given; Other
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years and older
Enrollment: 317 participants
Timeline: 2017 – 2018
U.S. locations: 1
States / cities: Plainsboro, New Jersey

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Stroke With Transfusions Changing to Hydroxyurea

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Amlexanox for Type 2 Diabetes and Obesity

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

Beclomethasone in Treating Patients With Graft-Versus-Host Disease of the Esophagus, Stomach, Small Intestine, or Colon

Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia

UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder

Long-term Safety Study of Chronocort in the Treatment of Participants With Congenital Adrenal Hyperplasia

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

PWS Outcomes Assessment Study

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

MARS - Monitored Atherosclerosis Regression Study

Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia

A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR )

Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

A Study to Evaluate OPTI-BIOME™ Bacillus Subtilis MB40 on Abdominal Discomfort, Gas and Bloating in a Healthy Population

A Study of Patients With Fabry Disease (US Specific)

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Awareness, Care, and Treatment In Obesity Management of Patients With Hemophilia (ACTION) to Inform Hemophilia Obesity Patient Empowerment (HOPE)