- Conditions
- Glycogen Storage Disease, Pompe Disease
- Interventions
- alglucosidase alfa
- Biological
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Female only
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2024
- U.S. locations
- 2
- States / cities
- Cambridge, Massachusetts • Grand Rapids, Michigan
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 169–192
of 10,330
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Thalassemia Major With Severe Transfusional Iron Overload
- Interventions
- Deferasirox and deferiprone
- Drug
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2017
- U.S. locations
- 2
- States / cities
- Chicago, Illinois • Philadelphia, Pennsylvania
- Conditions
- Retinitis Pigmentosa
- Interventions
- CNS10-NPC implantation
- Biological
- Lead sponsor
- Cedars-Sinai Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 1
- States / cities
- Beverly Hills, California
- Conditions
- Turner Syndrome, Hypogonadism, Premature Ovarian Failure
- Interventions
- 17 B estradiol orally, 17 B estradiol
- Drug
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- 13 Years to 20 Years · Female only
- Enrollment
- 41 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2012
- U.S. locations
- 2
- States / cities
- Jacksonville, Florida • Philadelphia, Pennsylvania
- Conditions
- Primary Insulin-like Growth Factor-1 Deficiency
- Interventions
- mecasermin [rDNA origin] injection
- Biological
- Lead sponsor
- Ipsen
- Industry
- Eligibility
- Not listed
- Enrollment
- 1,378 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2014
- U.S. locations
- 1
- States / cities
- Basking Ridge, New Jersey
- Conditions
- Sickle Cell Disease, Vaso-occlusive Crisis, Pain Crisis
- Interventions
- GMI-1070, Placebo
- Drug
- Lead sponsor
- GlycoMimetics Incorporated
- Industry
- Eligibility
- 12 Years to 60 Years
- Enrollment
- 81 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2013
- U.S. locations
- 21
- States / cities
- Birmingham, Alabama • Berkeley, California • Sacramento, California + 18 more
- Conditions
- Huntington Disease
- Interventions
- minocycline, Matching placebo
- Drug
- Lead sponsor
- Merit Cudkowicz
- Network
- Eligibility
- 18 Years and older
- Enrollment
- 114 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • Englewood, Colorado • Gainesville, Florida + 8 more
- Conditions
- Autosomal Dominant Polycystic Kidney Disease (ADPKD)
- Interventions
- VX-407, Placebo, Midazolam
- Drug
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 159 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- Lenexa, Kansas
- Conditions
- Cystic Fibrosis
- Interventions
- VX-659/TEZ/IVA, IVA
- Drug
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 6 Years to 11 Years
- Enrollment
- 18 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 6
- States / cities
- Aurora, Colorado • Chicago, Illinois • Kansas City, Missouri + 3 more
- Conditions
- Zellweger Spectrum Disorder
- Interventions
- Not listed
- Lead sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
- Other
- Eligibility
- Not listed
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2029
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
- Interventions
- No intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 3
- States / cities
- Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b), Adult Acute Myeloblastic Leukemia With Maturation (M2), Adult Acute Myeloblastic Leukemia Without Maturation (M1), Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Acute Myelomonocytic Leukemia (M4), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia
- Interventions
- alvocidib, daunorubicin hydrochloride, mitoxantrone hydrochloride, cytarabine
- Drug
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 172 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 11
- States / cities
- Scottsdale, Arizona • Tampa, Florida • Atlanta, Georgia + 7 more
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Interventions
- alemtuzumab, therapeutic allogeneic lymphocytes, fludarabine phosphate, melphalan, tacrolimus, thiotepa, peripheral blood stem cell transplantation
- Biological · Drug · Procedure
- Lead sponsor
- Yale University
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2008
- U.S. locations
- 1
- States / cities
- New Haven, Connecticut
- Conditions
- Atopic Dermatitis
- Interventions
- Lebrikizumab
- Drug
- Lead sponsor
- Eli Lilly and Company
- Industry
- Eligibility
- 12 Years and older
- Enrollment
- 90 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 36
- States / cities
- Birmingham, Alabama • Fountain Valley, California • Fremont, California + 30 more
- Conditions
- Acute Lymphoblastic Leukemia, Adult B Lymphoblastic Lymphoma, Ann Arbor Stage I B Lymphoblastic Lymphoma, Ann Arbor Stage II B Lymphoblastic Lymphoma, Childhood B Acute Lymphoblastic Leukemia, Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1, Childhood B Lymphoblastic Lymphoma, Down Syndrome, Hypodiploid B Acute Lymphoblastic Leukemia, Philadelphia Chromosome Positive
- Interventions
- Cyclophosphamide, Cytarabine, Dexamethasone, Doxorubicin Hydrochloride, Laboratory Biomarker Analysis, Leucovorin Calcium, Mercaptopurine, Methotrexate, Pegaspargase, Quality-of-Life Assessment, Questionnaire Administration, Thioguanine, Vincristine Sulfate
- Drug · Other
- Lead sponsor
- Children's Oncology Group
- Network
- Eligibility
- 1 Year to 30 Years
- Enrollment
- 9,350 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2026
- U.S. locations
- 213
- States / cities
- Birmingham, Alabama • Mobile, Alabama • Anchorage, Alaska + 153 more
- Conditions
- Hemophilia A
- Interventions
- efanesoctocog alfa
- Drug
- Lead sponsor
- Sanofi
- Industry
- Eligibility
- 12 Years to 50 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- Peoria, Illinois
- Conditions
- Mandible Tumor, Mandibular Injuries, Mandible; Deformity
- Interventions
- EB-CMF
- Combination Product
- Lead sponsor
- Epibone, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 3
- States / cities
- San Francisco, California • Cleveland, Ohio • San Antonio, Texas
- Conditions
- ABO Incompatibility, Hemolytic Disease of Newborn, Neonatal Jaundice, Neonatal Hyperbilirubinemia
- Interventions
- Not listed
- Lead sponsor
- State University of New York - Upstate Medical University
- Other
- Eligibility
- Up to 28 Days
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2010
- U.S. locations
- 2
- States / cities
- Syracuse, New York
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Idebenone
- Drug
- Lead sponsor
- Santhera Pharmaceuticals
- Industry
- Eligibility
- 8 Years and older
- U.S. locations
- 12
- States / cities
- Phoenix, Arizona • New Haven, Connecticut • Chicago, Illinois + 9 more
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Interventions
- SAT-3247
- Drug
- Lead sponsor
- Satellos Bioscience, Inc.
- Industry
- Eligibility
- 16 Years to 40 Years · Male only
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 4
- States / cities
- Los Angeles, California • St Louis, Missouri • Columbus, Ohio + 1 more
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
- Interventions
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Biological · Drug
- Lead sponsor
- Duke University
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Hemophilia B, Hemophilia B, Moderately Severe or Severe
- Interventions
- BE-101
- Drug
- Lead sponsor
- Be Biopharma
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 5
- States / cities
- Davis, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 2 more
- Conditions
- Beta Thalassemia
- Interventions
- 5-Azacytidine
- Drug
- Lead sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- NIH
- Eligibility
- Not listed
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Phenylketonuria (PKU)
- Interventions
- Pegvaliase
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2033
- U.S. locations
- 11
- States / cities
- Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 8 more