- Conditions
- ALSP
- Interventions
- No intervention
- Other
- Lead sponsor
- Vigil Neuroscience, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 56 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 6
- States / cities
- San Francisco, California • Englewood, Colorado • Boca Raton, Florida + 3 more
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Showing 1–7
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- 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
- ALSP
- Interventions
- VGL101
- Drug
- Lead sponsor
- Vigil Neuroscience, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2025
- U.S. locations
- 5
- States / cities
- San Francisco, California • Englewood, Colorado • Jacksonville, Florida + 2 more
- 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
- CSF1R-related Leukoencephalopathy, ALSP, POLD, Hematopoietic Stem Cell Transplantation
- Interventions
- Not listed
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 1
- States / cities
- Jacksonville, Florida
- Conditions
- BRCA1 Gene Mutation, BRCA2 Gene Mutation, Locally Advanced Solid Neoplasm, Metastatic Malignant Solid Neoplasm, POLD1 Gene Mutation, POLE Gene Mutation, Recurrent Malignant Solid Neoplasm, Recurrent Ovarian Carcinoma, Stage III Breast Cancer AJCC v7, Stage III Ovarian Cancer AJCC v8, Stage IIIA Breast Cancer AJCC v7, Stage IIIA Ovarian Cancer AJCC v8, Stage IIIB Breast Cancer AJCC v7, Stage IIIB Ovarian Cancer AJCC v8, Stage IIIC Breast Cancer AJCC v7, Stage IIIC Ovarian Cancer AJCC v8, Stage IV Breast Cancer AJCC v6 and v7, Stage IV Ovarian Cancer AJCC v8, Stage IVA Ovarian Cancer AJCC v8, Stage IVB Ovarian Cancer AJCC v8
- Interventions
- Laboratory Biomarker Analysis, Pembrolizumab
- Other · Biological
- Lead sponsor
- Rutgers, The State University of New Jersey
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2027
- U.S. locations
- 2
- States / cities
- New Brunswick, New Jersey • New York, New York
- Conditions
- BRCA1 Mutation, POLD1 Gene Mutation, CDKN2A Mutation, BRCA2 Mutation, POLE Gene Mutation, APC Gene Mutation, ATM Gene Mutation, MLH1 Gene Mutation, BARD1 Gene Mutation, MSH2 Gene Mutation, BRIP1 Gene Mutation, MSH6 Gene Mutation, CHEK2 Gene Mutation, PMS2 Gene Mutation, PALB2 Gene Mutation, EPCAM Gene Mutation, RAD51C Gene Mutation, BMPR1A Gene Mutation, RAD51D Gene Mutation, SMAD4, PTEN Gene Mutation, GREM1
- Interventions
- Intervention Arm At-risk Relative/ARR Contacts, MyGene Portal, Standard of Care
- Behavioral
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Other
- Eligibility
- 25 Years and older
- Enrollment
- 1,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2022 – 2026
- U.S. locations
- 8
- States / cities
- Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more