- Conditions
- Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
- Interventions
- Fludarabine, Cyclophosphamide, Busulfan, Alemtuzumab, Rabbit Anti-thymocyte Globulin, Horse Anti-thymocyte Globulin
- Drug
- Lead sponsor
- Columbia University
- Other
- Eligibility
- Up to 30 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2021
- U.S. locations
- 1
- States / cities
- New York, New York
Search Results
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Showing 1–18
of 18
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
- Interventions
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
- Biological · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 149 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2029
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Autosomal Dominant Osteopetrosis Type 2
- Interventions
- ACTIMMUNE
- Drug
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 3 Years to 65 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Osteopetrosis
- Interventions
- Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies
- Procedure · Device · Drug
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2009
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
- Interventions
- Hematopoietic stem cell transplantation
- Procedure
- Lead sponsor
- Children's Hospital Los Angeles
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2015
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Osteopetrosis
- Interventions
- rhPTH
- Drug
- Lead sponsor
- University of Chicago
- Other
- Eligibility
- 20 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2003 – 2011
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome, Lymphomas, Bone Marrow Failure, Hemoglobinopathy, Immune Deficiency, Osteopetrosis, Cytopenias, Leukocyte Disorders, Anemia Due to Intrinsic Red Cell Abnormality
- Interventions
- CD34+ enriched, T Cell Depleted donor stem cell product
- Biological
- Lead sponsor
- Christopher Dvorak
- Other
- Eligibility
- 2 Months to 30 Years
- Healthy volunteers
- Healthy volunteers not accepted
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome, Lymphomas, Bone Marrow Failure, Hemoglobinopathy, Immune Deficiency, Osteopetrosis
- Interventions
- CliniMACS® (T cell depletion)
- Device
- Lead sponsor
- Wake Forest University Health Sciences
- Other
- Eligibility
- Up to 30 Years
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2016
- U.S. locations
- 1
- States / cities
- Charlotte, North Carolina
- Conditions
- Disorder; Trance, Anxiety
- Interventions
- Standardized Hypnotic Susceptibility Testing
- Behavioral
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes, Primary Immune Deficiency Disorder, Osteopetrosis, Cytopenia, Hemoglobinopathy in Children, Anemia, Aplastic
- Interventions
- BPX-501 T cells, Rimiducid
- Biological · Drug
- Lead sponsor
- Bellicum Pharmaceuticals
- Industry
- Eligibility
- 1 Month to 26 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2034
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 7 more
- Conditions
- Infantile Malignant Osteopetrosis
- Interventions
- RP-L401
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 1 Month and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Osteopetrosis
- Interventions
- Interferon gamma-1b
- Drug
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Other
- Eligibility
- 1 Year and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 2
- States / cities
- Torrance, California • Minneapolis, Minnesota
- Conditions
- Osteopetrosis
- Interventions
- Actimmune Registry
- Drug
- Lead sponsor
- InterMune
- Industry
- Eligibility
- Not listed
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2005
- U.S. locations
- 1
- States / cities
- Brisbane, California
- Conditions
- Severe Osteopetrosis
- Interventions
- umbilical cord blood transplantation, Campath-1H, Total Lymphoid Irradiation, Cyclophosphamide, Busulfan, Fludarabine monophosphate, marrow graft transplantation
- Procedure · Drug · Radiation
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 1 Day to 45 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Osteopetrosis
- Interventions
- Stem Cell or Umbilical Cord Blood Transplantation, Campath, Busulfan, Clofarabine, Total Lymphoid Irradiation
- Procedure · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 45 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2008
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
- Interventions
- data collection
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 60 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2028
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- 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