- Conditions
- Thyroid Eye Disease
- Interventions
- VRDN-003, Autoinjector
- Drug · Device
- Lead sponsor
- Viridian Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 87 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 13
- States / cities
- Inglewood, California • Cape Coral, Florida • Kissimmee, Florida + 7 more
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Showing 1–24
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- Conditions
- Retinal Dystrophies
- Interventions
- Optical coherence tomography (OCT)
- Diagnostic Test
- Lead sponsor
- Institute of Molecular and Clinical Ophthalmology Basel
- Other
- Eligibility
- Not listed
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2026
- U.S. locations
- 3
- States / cities
- San Francisco, California • Miami, Florida • Pittsburgh, Pennsylvania
- Conditions
- Fuchs Endothelial Corneal Dystrophy, Fuchs
- Interventions
- DT-168
- Drug
- Lead sponsor
- Design Therapeutics, Inc.
- Industry
- Eligibility
- 30 Years and older
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2026
- U.S. locations
- 2
- States / cities
- Indianapolis, Indiana • Grand Rapids, Michigan
- Conditions
- Behcet Disease
- Interventions
- AIN457, Placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 118 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 3
- States / cities
- Baltimore, Maryland • Arlington, Texas • Houston, Texas
- 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 | Blood or Tissue | 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
- Thyroid Eye Disease
- Interventions
- Efgartigimod PH20 SC, Placebo PH20 SC
- Combination Product · Other
- Lead sponsor
- argenx
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 105 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Mission Hills, California • Rancho Cordova, California + 8 more
- Conditions
- Thyroid Eye Disease
- Interventions
- Teprotumumab
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 3
- States / cities
- Miami, Florida • St Louis, Missouri • Bellaire, Texas
- Conditions
- Down Syndrome (DS), Keratoconus, Pellucid Marginal Degeneration, Forme Fruste Keratoconus (FFK)
- Interventions
- Epi-ON corneal cross-linking (CXL)
- Combination Product
- Lead sponsor
- Woolfson Eye Institute
- Other
- Eligibility
- 8 Years and older
- Enrollment
- 225 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 12
- States / cities
- Atlanta, Georgia • Canton, Georgia • Cumming, Georgia + 8 more
- Conditions
- Stargardt's Disease
- Interventions
- Long term follow up in all patients who received SAR422459 in previous study TDU13583
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 6 Years and older
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2033
- U.S. locations
- 1
- States / cities
- Portland, Oregon
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer, Liver Cancer, Neuroblastoma, Ovarian Cancer, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
- Interventions
- O6-benzylguanine, temozolomide
- Drug
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- Up to 21 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Thyroid Associated Ophthalmopathies, Thyroid-Associated Ophthalmopathy
- Interventions
- teprotumumab, normal saline
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 88 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2017
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Aurora, Colorado • Atlanta, Georgia + 8 more
- Conditions
- Disaster, Vulnerable Populations
- Interventions
- Nurses Taking On Readiness Measures (N- TORM)
- Behavioral
- Lead sponsor
- Hunter College of City University of New York
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 400 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Autosomal Dominant Retinitis Pigmentosa, Eye Diseases, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Disease, Retinitis, Vision Tunnel, Vision Disorders
- Interventions
- QR-1123, Sham procedure
- Drug · Other
- Lead sponsor
- ProQR Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 5
- States / cities
- Aurora, Colorado • Gainesville, Florida • Lexington, Kentucky + 2 more
- Conditions
- Cancer
- Interventions
- entinostat
- Drug
- Lead sponsor
- National Institutes of Health Clinical Center (CC)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2008
- U.S. locations
- 3
- States / cities
- Bethesda, Maryland
- Conditions
- Albinism, Oculocutaneous Albinism
- Interventions
- Levodopa/carbidopa
- Drug
- Lead sponsor
- University of Wisconsin, Madison
- Other
- Eligibility
- 3 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2018
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Thyroid Eye Disease, Graves Orbitopathy, Endocrine System Diseases, Eye Diseases, Thyroid Associated Ophthalmopathy, Graves Ophthalmopathy, Thyroid Diseases, Orbital Diseases, Proptosis, IGF1R, Exophthalmos, Hashimoto
- Interventions
- linsitinib
- Drug
- Lead sponsor
- Sling Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 2
- States / cities
- Miami, Florida • Morgantown, West Virginia
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan and Epilepsy), Alpha-Dystroglycanopathy (Dystroglycanopathy, Congenital With or Without Mental Retardation (Formerly MDC1C)), Alpha-Dystroglycanopathy (Fukuyama CMD), Alpha-Dystroglycanopathy (LGMDR09 FKRP Related (Formerly LGMD2I)), Alpha-Dystroglycanopathy (LGMDR11 POMT1 Related (Formerly LGMD2K)), Alpha-Dystroglycanopathy (LGMDR13 FKTN Related (Formerly LGMD2M)), Alpha-Dystroglycanopathy (LGMDR14 POMT2 Related (Formerly LGMD2N)), Alpha-Dystroglycanopathy (LGMDR15 POMGnT1 Related (Formerly LGMD2O)), Alpha-Dystroglycanopathy (LGMDR19 GMPPB Related (Formerly LGMD2T)), Alpha-Dystroglycanopathy (LGMDR20 ISPD Related (Formerly LGMD2U)), Alpha-Dystroglycanopathy (LGMDR24 POMGnT2 Related), Alpha-Dystroglycanopathy (Muscle Eye Brain Disease (MEB)), Alpha-Dystroglycanopathy (Walker Warburg Syndrome (WWS)), Choline Kinase B Receptor - CHKB, Collagen VI Related Disorders, Collagen XII Related Disorders, Congenital Muscular Dystrophy Not Otherwise Specified (Including Merosin Positive), Congenital Muscular Dystrophy With Cataracts and Intellectual Disability (MDCCAID), Congenital Muscular Dystrophy With Joint Hyperlaxity, Congenital Muscular Dystrophy With Rigid Spine Related to ACTA1, Emery-Dreifuss Muscular Dystrophy, GOLGA2-related Congenital Muscle Dystrophy With Brain Involvement, LMNA Related Disorders, Merosin Deficient CMD (Full or Partial), Nesprin Related MD (SYNE1), SELENON Related Disorders (Previously Known as SEPN1), SELENON Related Myopathy (Aka SEPN1), Telethonin CMD, Congenital Myasthenic Syndrome, Limb-Girdle Muscular Dystrophy, LGMDD01 - DNAJB6 (Formerly LGMD1D), LGMDD05 - Collagen VI Related Bethlem Myopathy (Dominant), LGMDR07 - Telethonin (TCAP) Related (Formerly LGMD2G), LGMDR08 - TRIM Related (Formerly LGMD2H), LGMDR09 - FKRP Related (Formerly LGMD2I), LGMDR10 - Titin (TTN) Related (Formerly LGMD2J), LGMDR11 - POMT1 Related (Formerly LGMD2K), LGMDR13 - Fukutin (FKTN) Related (Formerly LGMD2M), LGMDR14 - POMT2 Related (Formerly LGMD2N), LGMDR15 - POMGnT1 Related (Formerly LGMD2O), LGMDR16 - DAG1 Related Dystroglycanopathy (Formerly LGMD2P), LGMDR17 - Plectin (PLEC) Related (Formerly LGMD2Q), LGMDR18 - TRAPPC11 Related (Formerly LGMD2S), LGMDR19 - GMPPB Related (Formerly LGMD2T), LGMDR20 - ISPD Related (Formerly LGMD2U), LGMDR22 - Collagen VI Related Bethlem Myopathy (Recessive), LGMDR23 - LAMA2 Related, LGMDR24 - POMGnT2 Related
- Interventions
- Not listed
- Lead sponsor
- Cure CMD
- Other
- Eligibility
- Not listed
- Enrollment
- 4,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2029
- U.S. locations
- 1
- States / cities
- Lakewood, California
- Conditions
- Neurofibromatosis Type 1, Recurrent Childhood Brain Stem Glioma, Recurrent Childhood Visual Pathway Glioma, Recurrent Primary Central Nervous System Neoplasm, Refractory Primary Central Nervous System Neoplasm
- Interventions
- Laboratory Biomarker Analysis, Pharmacological Study, Pomalidomide
- Other · Drug
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 3 Years to 20 Years
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2020
- U.S. locations
- 12
- States / cities
- Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 9 more
- Conditions
- Hermansky-Pudlak Syndrome (HPS)
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 115 Years
- Enrollment
- 600 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years and older
- Enrollment
- 180 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 22
- States / cities
- San Diego, California • San Francisco, California • San Mateo, California + 14 more
- Conditions
- Ocular Albinism (OA), Oculocutaneous Albinism (OCA)
- Interventions
- Lutein plus Zeaxanthin, Placebo
- Dietary Supplement
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Cytomegalovirus Retinitis, HIV Infections
- Interventions
- Foscarnet sodium, Ganciclovir
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 13 Years and older
- Enrollment
- 240 participants
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 9
- States / cities
- La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
- Conditions
- Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Neuroblastoma, Neutropenia, Sarcoma
- Interventions
- voriconazole
- Drug
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 2 Years to 11 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2004
- U.S. locations
- 7
- States / cities
- Orange, California • San Diego, California • Bethesda, Maryland + 4 more
- Conditions
- Bioavailability, Bioequivalence
- Interventions
- TEPEZZA, EDP
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Las Vegas, Nevada