- Conditions
- Diabetes, Type 1, Diabetes, Type 2
- Interventions
- Technosphere® Insulin Inhalation Powder, Usual Care
- Drug
- Lead sponsor
- Mannkind Corporation
- Industry
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 2,053 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005 – 2008
- U.S. locations
- 82
- States / cities
- Phoenix, Arizona • Tempe, Arizona • Tucson, Arizona + 64 more
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 1–24
of 2,421
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
- Interventions
- NTLA-2001, Placebo
- Biological · Drug
- Lead sponsor
- Intellia Therapeutics
- Industry
- Eligibility
- 18 Years to 90 Years
- Enrollment
- 1,200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 38
- States / cities
- Tucson, Arizona • Los Angeles, California • Palo Alto, California + 29 more
- Conditions
- MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
- Interventions
- Glycerol Tributyrate
- Drug
- Lead sponsor
- George Washington University
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2027 – 2028
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Wilson Disease
- Interventions
- NCC-Sp Assay
- Diagnostic Test
- Lead sponsor
- Orphalan
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 50 participants
- Timeline
- 2023 – 2025
- U.S. locations
- 9
- States / cities
- Sacramento, California • New Haven, Connecticut • Miami, Florida + 6 more
- Conditions
- Friedreich Ataxia
- Interventions
- CTI-1601, Placebo
- Biological
- Lead sponsor
- Larimar Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Eatontown, New Jersey
- Conditions
- Urea Cycle Disorders, Organic Acidemias
- Interventions
- Therapeutic Hypothermia, Standard of Care Therapy
- Other
- Lead sponsor
- Uta Lichter-Konecki
- Other
- Eligibility
- 1 Day to 30 Days
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2015
- U.S. locations
- 3
- States / cities
- Washington D.C., District of Columbia • New York, New York • Milwaukee, Wisconsin
- Conditions
- Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
- Interventions
- sample collection
- Other
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- 1 Month and older
- Enrollment
- 230 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2024
- U.S. locations
- 3
- States / cities
- Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida
- Conditions
- Iron Overload, Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, 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(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Atypical Chronic Myeloid Leukemia, BCR-ABL Negative, Blastic Phase Chronic Myelogenous Leukemia, Chronic Eosinophilic Leukemia, Chronic Myelomonocytic Leukemia, Chronic Neutrophilic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, de Novo Myelodysplastic Syndromes, Disseminated Neuroblastoma, Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable, Nodal Marginal Zone B-cell Lymphoma, Noncontiguous Stage II Adult Burkitt Lymphoma, Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma, Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma, Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma, Noncontiguous Stage II Adult Lymphoblastic Lymphoma, Noncontiguous Stage II Grade 1 Follicular Lymphoma, Noncontiguous Stage II Grade 2 Follicular Lymphoma, Noncontiguous Stage II Grade 3 Follicular Lymphoma, Noncontiguous Stage II Mantle Cell Lymphoma, Noncontiguous Stage II Marginal Zone Lymphoma, Noncontiguous Stage II Small Lymphocytic Lymphoma, Poor Prognosis Metastatic Gestational Trophoblastic Tumor, Previously Treated Myelodysplastic Syndromes, Primary Myelofibrosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Malignant Testicular Germ Cell Tumor, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Mycosis Fungoides/Sezary Syndrome, Recurrent Neuroblastoma, Recurrent Ovarian Epithelial Cancer, Recurrent Ovarian Germ Cell Tumor, Recurrent Small Lymphocytic Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Hairy Cell Leukemia, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Splenic Marginal Zone Lymphoma, Stage I Multiple Myeloma, Stage II Multiple Myeloma, Stage II Ovarian Epithelial Cancer, Stage III Adult Burkitt Lymphoma, Stage III Adult Diffuse Large Cell Lymphoma, Stage III Adult Diffuse Mixed Cell Lymphoma, Stage III Adult Diffuse Small Cleaved Cell Lymphoma, Stage III Adult Hodgkin Lymphoma, Stage III Adult Immunoblastic Large Cell Lymphoma, Stage III Adult Lymphoblastic Lymphoma, Stage III Chronic Lymphocytic Leukemia, Stage III Grade 1 Follicular Lymphoma, Stage III Grade 2 Follicular Lymphoma, Stage III Grade 3 Follicular Lymphoma, Stage III Malignant Testicular Germ Cell Tumor, Stage III Mantle Cell Lymphoma, Stage III Marginal Zone Lymphoma, Stage III Multiple Myeloma, Stage III Ovarian Epithelial Cancer, Stage III Small Lymphocytic Lymphoma, Stage IIIA Breast Cancer, Stage IIIB Breast Cancer, Stage IIIC Breast Cancer, Stage IV Adult Burkitt Lymphoma, Stage IV Adult Diffuse Large Cell Lymphoma, Stage IV Adult Diffuse Mixed Cell Lymphoma, Stage IV Adult Diffuse Small Cleaved Cell Lymphoma, Stage IV Adult Hodgkin Lymphoma, Stage IV Adult Immunoblastic Large Cell Lymphoma, Stage IV Adult Lymphoblastic Lymphoma, Stage IV Breast Cancer, Stage IV Chronic Lymphocytic Leukemia, Stage IV Grade 1 Follicular Lymphoma, Stage IV Grade 2 Follicular Lymphoma, Stage IV Grade 3 Follicular Lymphoma, Stage IV Mantle Cell Lymphoma, Stage IV Marginal Zone Lymphoma, Stage IV Ovarian Epithelial Cancer, Stage IV Small Lymphocytic Lymphoma
- Interventions
- deferasirox
- Drug
- Lead sponsor
- City of Hope Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 1
- States / cities
- Duarte, California
- Conditions
- Lipoidosis
- Interventions
- SCH-58235
- Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Inborn Errors of Metabolism
- Interventions
- N-carbamylglutamate
- Drug
- Lead sponsor
- Mendel Tuchman
- Other
- Eligibility
- 1 Day to 70 Years
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2020
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Cardiovascular Phenotype, Metabolic Phenotype, Hypertensive Disease, Psychological Phenotype, Audio
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 18 Years to 99 Years · Female only
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Familial Hypercholesterolemia
- Interventions
- Genetic testing
- Other
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 378 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2018
- U.S. locations
- 2
- States / cities
- Lancaster, Pennsylvania • Philadelphia, Pennsylvania
- Conditions
- Type 2 Diabetes
- Interventions
- Exercise program, Ascorbic Acid (Vitamin C)
- Behavioral · Drug
- Lead sponsor
- University of Colorado, Denver
- Other
- Eligibility
- 30 Years to 55 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2011
- U.S. locations
- 1
- States / cities
- Aurora, Colorado
- Conditions
- Pompe Disease
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 18 Years and older · Female only
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Friedreich Ataxia
- Interventions
- Rosuvastatin
- Drug
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Acidosis, Renal Tubular
- Interventions
- ADV7103
- Drug
- Lead sponsor
- Advicenne Pharma
- Industry
- Eligibility
- 4 Months to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
- Interventions
- No Intervention
- Other
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- Not listed
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2032
- U.S. locations
- 7
- States / cities
- Orange, California • Washington D.C., District of Columbia • Chicago, Illinois + 4 more
- Conditions
- Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
- Interventions
- Evuzamitide
- Drug
- Lead sponsor
- Lorena Saelices
- Other
- Eligibility
- Not listed
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • New York, New York • Dallas, Texas
- Conditions
- Pompe Disease
- Interventions
- Duvoglustat
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 74 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
- Interventions
- Not listed
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 2 Years to 18 Years
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2016
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Friedreich Ataxia, Cardiomyopathy, Secondary
- Interventions
- Low dose LX2006, Mid Dose LX2006, High Dose LX2006
- Genetic
- Lead sponsor
- Lexeo Therapeutics
- Industry
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2029
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Tampa, Florida • Rochester, Minnesota
- Conditions
- Fabry Disease
- Interventions
- Interview
- Other
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- Not listed
- Enrollment
- 23 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022
- U.S. locations
- 7
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
- Interventions
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2024
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Day to 99 Years
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2006
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia • Bethesda, Maryland