- Conditions
- MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
- Interventions
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Other · Biological
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- Not listed
- Enrollment
- 1,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2035
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
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 are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Hemophilia A
- Interventions
- ASC618
- Biological
- Lead sponsor
- ASC Therapeutics
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2026
- U.S. locations
- 1
- States / cities
- Little Rock, Arkansas
- Conditions
- Sickle Cell Disease
- Interventions
- Busulfan, Fludarabine, r-ATG, Hematopoietic Cell Transplant, Tacrolimus, Methotrexate, Standard of Care, Alemtuzumab, Total Body Irradiation (TBI), Sirolimus, Melphalan, G-CSF
- Drug · Procedure
- Lead sponsor
- Medical College of Wisconsin
- Other
- Eligibility
- 15 Years to 40 Years
- Enrollment
- 138 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 35
- States / cities
- Oakland, California • Washington D.C., District of Columbia • Gainesville, Florida + 25 more
- Conditions
- Thalassemia, Iron Overload
- Interventions
- Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
- Drug
- Lead sponsor
- Elliott Vichinsky
- Other
- Eligibility
- 8 Years and older
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2012
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Ewing's Sarcoma, Soft Tissue Sarcoma, Hepatoblastoma, Hodgkin's Disease, Germ Cell Tumor
- Interventions
- High-Dose Chemotherapy with Tandem PBSC Rescue.
- Drug
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2012
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Blastic Phase Chronic Myelogenous Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Chronic Phase Chronic Myelogenous Leukemia, de Novo Myelodysplastic Syndromes, Disseminated Neuroblastoma, Juvenile Myelomonocytic Leukemia, Previously Treated Childhood Rhabdomyosarcoma, Previously Treated Myelodysplastic Syndromes, Pulmonary Complications, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Neuroblastoma, Recurrent Wilms Tumor and Other Childhood Kidney Tumors, Recurrent/Refractory Childhood Hodgkin Lymphoma, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes
- Interventions
- etanercept, methylprednisolone
- Biological · Drug
- Lead sponsor
- Children's Oncology Group
- Network
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2011
- U.S. locations
- 26
- States / cities
- Birmingham, Alabama • Arcadia, California • Loma Linda, California + 22 more
- Conditions
- Sickle Cell Disease
- Interventions
- Sapropterin Dihydrochloride
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 15 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 12
- States / cities
- Washington D.C., District of Columbia • Augusta, Georgia • Savannah, Georgia + 9 more
- Conditions
- Pyruvate Kinase Deficiency
- Interventions
- Not listed
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 13 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Sickle Cell Disease, Virtual Reality, Anxiety, Depression, Cancer
- Interventions
- Relaxation response and virtual reality (VR) session
- Device
- Lead sponsor
- Duke University
- Other
- Eligibility
- 8 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2019
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Sickle Cell Disease
- Interventions
- Guided Relaxation, Acupuncture
- Behavioral · Other
- Lead sponsor
- University of Illinois at Chicago
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 379 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2026
- U.S. locations
- 5
- States / cities
- Gainesville, Florida • Atlanta, Georgia • Chicago, Illinois + 2 more
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Megakaryoblastic Leukemia (M7), 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 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), Adult Acute Myelomonocytic Leukemia (M4), Adult Acute Promyelocytic Leukemia (M3), Adult Erythroleukemia (M6a), Adult Pure Erythroid Leukemia (M6b), Blastic Phase Chronic Myelogenous Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes, Untreated Adult Acute Myeloid Leukemia
- Interventions
- ispinesib, laboratory biomarker analysis, pharmacological study
- Drug · Other
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2004
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- Conditions
- Pain, Sickle Cell Disease
- Interventions
- Morphine, Nubain
- Drug
- Lead sponsor
- Children's Healthcare of Atlanta
- Other
- Eligibility
- 6 Years to 19 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2016
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia
- Conditions
- Sickle Cell Disease
- Interventions
- Digital app and information system
- Other
- Lead sponsor
- Makerere University
- Other
- Eligibility
- Up to 5 Years
- Enrollment
- 24,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024 – 2028
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Hemophilia A
- Interventions
- BAY94-9027 + Recombinant Factor VIII (Kogenate FS, BAY14-2222))
- Biological
- Lead sponsor
- Bayer
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 4
- States / cities
- Davis, California • Boston, Massachusetts • Minneapolis, Minnesota + 1 more
- Conditions
- Congenital Bleeding Disorder, Glanzmann's Disease
- Interventions
- activated recombinant human factor VII
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- Not listed
- Enrollment
- 218 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2011
- U.S. locations
- 1
- States / cities
- Princeton, New Jersey
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Interventions
- Not listed
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Other
- Eligibility
- 16 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2013
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Sickle Cell Disease
- Interventions
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 13 Years to 55 Years
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Chronic Granulomatous Disease
- Interventions
- Stem cell transplantation
- Procedure
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- Not listed
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2000 – 2004
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hemophilia A
- Interventions
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII
- Biological
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- Up to 11 Years
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 12
- States / cities
- Phoenix, Arizona • Denver, Colorado • Gainesville, Florida + 9 more
- Conditions
- Sickle Cell Disease, Malnutrition
- Interventions
- Maternal Nutrition Education, Standard Care
- Behavioral
- Lead sponsor
- Vanderbilt University Medical Center
- Other
- Eligibility
- 6 Months to 18 Months
- Enrollment
- 146 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Sickle Cell Anemia
- Interventions
- cyclosporine, fludarabine, mycophenolate mofetil, Bone Marrow Transplantation
- Drug · Procedure
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- Up to 16 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1999
- U.S. locations
- 2
- States / cities
- Oakland, California • Seattle, Washington
- Conditions
- Primary Immune Deficiencies, Autoimmune Diseases, Inflammatory Diseases, Common Variable Immune Deficiencies, Hypogammaglobulinemia
- Interventions
- Not listed
- Lead sponsor
- O & O Alpan LLC
- Other
- Eligibility
- 1 Day and older
- Enrollment
- 343 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2018
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Sickle Cell Disease, Thalassemia
- Interventions
- Video recording, Medication Administration Log, Education
- Behavioral
- Lead sponsor
- Duke University
- Other
- Eligibility
- 5 Years and older
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2016
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- DiGeorge Syndrome, Hypoparathyroidism, Complete DiGeorge Syndrome
- Interventions
- Thymus/Parathyroid Transplantation
- Biological
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Up to 24 Months
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2019
- U.S. locations
- 1
- States / cities
- Durham, North Carolina