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Conditions: Pachyonychia Congenita
Interventions: QTORIN 3.9% rapamycin anhydrous gel, Vehicle; Drug
Lead sponsor: Palvella Therapeutics, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 87 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2023
U.S. locations: 7
States / cities: Palo Alto, California • New Haven, Connecticut • Orange Park, Florida + 4 more

Conditions: Kartagener Syndrome
Interventions: Not listed
Lead sponsor: University of North Carolina, Chapel Hill; Other
Eligibility: Not listed
Enrollment: 1,800 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2004 – 2027
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Friedreich Ataxia
Interventions: TAK-831, TAK-831 Placebo; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 6
States / cities: Los Angeles, California • Gainesville, Florida • Tampa, Florida + 3 more

Conditions: B Acute Lymphoblastic Leukemia, B Lymphoblastic Lymphoma, Down Syndrome
Interventions: Asparaginase Erwinia chrysanthemi, Blinatumomab, Cyclophosphamide, Cytarabine, Dexamethasone, Doxorubicin Hydrochloride, Leucovorin Calcium, Mercaptopurine, Mercaptopurine Oral Suspension, Methotrexate, Pegaspargase, Prednisolone, Prednisone, Radiation Therapy, Thioguanine, Vincristine Sulfate; Drug · Biological · Radiation
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 365 Days to 31 Years
Enrollment: 6,720 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2027
U.S. locations: 201
States / cities: Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 153 more

Conditions: Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
Interventions: cyclosporine ophthalmic emulsion, placebo; Drug · Other
Lead sponsor: Vanderbilt-Ingram Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 164 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2016
U.S. locations: 5
States / cities: Palo Alto, California • Chicago, Illinois • Nashville, Tennessee + 2 more

Conditions: Cytomegalovirus Infections, HIV Infections
Interventions: Ganciclovir; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Esophageal Atresia With Tracheo-esophageal Fistula, Esophageal Atresia, Esophageal Strictures
Interventions: Endoscopic Balloon Dilation; Procedure
Lead sponsor: Boston Children's Hospital; Other
Eligibility: Up to 18 Years
Enrollment: 128 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 2
States / cities: Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Cystic Fibrosis
Interventions: Exercise Intervention; Behavioral
Lead sponsor: Wuerzburg University Hospital; Other
Eligibility: 12 Years and older
Enrollment: 155 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 2
States / cities: Birmingham, Alabama • Pittsburgh, Pennsylvania

Conditions: Sickle Cell Anemia
Interventions: ACZ885, Placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 8 Years to 20 Years
Enrollment: 49 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 3
States / cities: Atlanta, Georgia • Augusta, Georgia • Greenville, North Carolina

Conditions: Hypothalamic Obesity
Interventions: Placebo, Tesomet; Other · Drug
Lead sponsor: Saniona; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Lansing, Michigan

Conditions: Cockayne Syndrome, Skin Neoplasms, Xeroderma Pigmentosum, Trichothiodystrophy Syndromes, Genodermatosis
Interventions: Not listed
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 6 Weeks to 100 Years
Enrollment: 709 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1999
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Autosomal Dominant Polycystic Kidney Disease, ADPKD, Polycystic Kidney, Autosomal Dominant
Interventions: RGLS8429, Placebo; Drug
Lead sponsor: Regulus Therapeutics Inc.; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 21
States / cities: Mesa, Arizona • Los Angeles, California • New Haven, Connecticut + 17 more

Conditions: Cystinuria
Interventions: Bucillamine; Drug
Lead sponsor: Revive Therapeutics, Ltd.; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 5
States / cities: Birmingham, Alabama • Boston, Massachusetts • New York, New York + 2 more

Conditions: Hypotonia, Muscle, Obstructive Sleep Apnea, Nasal Airway Obstruction
Interventions: Self-Supporting Nasopharyngeal Airway (ssNPA); Device
Lead sponsor: University of Michigan; Other
Eligibility: 3 Years to 23 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Ann Arbor, Michigan

Conditions: Spinocerebellar Ataxia Type 3
Interventions: BIIB132, BIIB132-Matching Placebo; Drug
Lead sponsor: Biogen; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 10
States / cities: Los Angeles, California • San Francisco, California • Gainesville, Florida + 7 more

Conditions: Prader-Willi Syndrome, Obesity
Interventions: Group 1, Group 2; Other
Lead sponsor: University of Florida; Other
Eligibility: Up to 60 Years
Enrollment: 392 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2014
U.S. locations: 4
States / cities: Orange, California • Gainesville, Florida • Kansas City, Kansas + 1 more

Conditions: DiGeorge Syndrome, Velocardiofacial Syndrome, 22q11.2 Syndrome
Interventions: Not listed
Lead sponsor: National Institute of Mental Health (NIMH); NIH
Eligibility: 18 Years to 50 Years
Healthy volunteers: Accepts healthy volunteers
Timeline: 2005 – 2010
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Language Impairment, Down Syndrome
Interventions: Jasper-EMT with words and AAC; Behavioral
Lead sponsor: Vanderbilt University; Other
Eligibility: 30 Months to 54 Months
Enrollment: 78 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2018
U.S. locations: 2
States / cities: Los Angeles, California • Nashville, Tennessee

Conditions: Atopic Dermatitis
Interventions: Tralokinumab, Placebos; Drug
Lead sponsor: LEO Pharma; Industry
Eligibility: 12 Years to 17 Years
Enrollment: 301 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 29
States / cities: Birmingham, Alabama • Fort Smith, Arkansas • Fountain Valley, California + 25 more

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: Atopic Dermatitis
Interventions: DSXS, Placebo; Drug
Lead sponsor: Sun Pharmaceutical Industries, Inc.; Industry
Eligibility: 6 Months and older
Enrollment: 124 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 1
States / cities: Hawthorne, New York

Conditions: Angelman Syndrome
Interventions: obudanersen, Placebo; Drug
Lead sponsor: Ionis Pharmaceuticals, Inc.; Industry
Eligibility: 2 Years to 50 Years
Enrollment: 158 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 18
States / cities: Los Angeles, California • San Diego, California • Aurora, Colorado + 13 more

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions: cyclophosphamide, fludarabine phosphate, mycophenolate mofetil, tacrolimus, allogeneic bone marrow transplantation, radiation therapy; Drug · Procedure · Radiation
Lead sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins; Other
Eligibility: 6 Months to 74 Years
Enrollment: 210 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2015
U.S. locations: 3
States / cities: Atlanta, Georgia • Baltimore, Maryland • Philadelphia, Pennsylvania

Conditions: Dilated Cardiomyopathy (DCM), Ischemic Cardiomyopathy, Nonischemic Cardiomyopathy, Heart Failure
Interventions: Allogeneic Cardiosphere-Derived Cells (CDCs); Biological
Lead sponsor: Capricor Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 42 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 1
States / cities: Los Angeles, California

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A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita

Pathogenesis of Primary Ciliary Dyskinesia (PCD) Lung Disease

Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia

A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia

Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder

Ganciclovir: Compassionate Use in Patients With Serious or Life-Threatening Cytomegalovirus Infections

Balloon Inflation Time for Esophageal Strictures (BITES): A Randomized Multi-Center Study

Effects of a Partially Supervised Conditioning Program in CF

Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia

Study of Tesomet With Open-label Extension in Subjects With Hypothalamic Obesity (HO)

Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum, Cockayne Syndrome, or Trichothiodystrophy

A Study of RGLS8429 in Patients With Autosomal Dominant Polycystic Kidney Disease

Bucillamine Phase 2 Trial in Patients With Cystinuria

Long-term Evaluation of Nasopharyngeal Airway in Hypotonia

A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3

Characteristics of Prader-Willi Syndrome and Early-onset Morbid Obesity

Velocardiofacial (VCFS; 22q11.2; DiGeorge) Syndrome Study

Phenotypic Specific Communication Intervention for Children With Down Syndrome

Tralokinumab Monotherapy for Adolescent Subjects With Moderate to Severe Atopic Dermatitis - ECZTRA 6 (ECZema TRAlokinumab Trial no. 6).

The Myelin Disorders Biorepository Project

Clinical Study to Evaluate the Efficacy and Safety of Desoximetasone (DSXS) With Atopic Dermatitis

REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome

Fludarabine, Cyclophosphamide, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer

Dilated cardiomYopathy iNtervention With Allogeneic MyocardIally-regenerative Cells (DYNAMIC)