- Conditions
- Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
- Interventions
- Not listed
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 6 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2019
- U.S. locations
- 3
- States / cities
- Oakland, California • Minneapolis, Minnesota • New York, New York
Search Results
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Showing 25–41
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- Conditions
- Gas and Bloating, Diarrhea, Constipation
- Interventions
- GI Health Active Product 1, GI Health Product Placebo Control
- Dietary Supplement
- Lead sponsor
- Radicle Science
- Industry
- Eligibility
- 21 Years to 105 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2026
- U.S. locations
- 1
- States / cities
- Del Mar, California
- 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
- Conditions
- MPS I, MPS IV A, MPS - Mucopolysaccharidosis, MPS IVA
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Orange County
- Other
- Eligibility
- 0 Years to 99 Years
- Enrollment
- 240 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2030
- U.S. locations
- 3
- States / cities
- Orange, California • Minneapolis, Minnesota • St Louis, Missouri
- Conditions
- Achondroplasia
- Interventions
- Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 0 Years to 32 Months
- Enrollment
- 77 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2032
- U.S. locations
- 3
- States / cities
- Oakland, California • Baltimore, Maryland • Madison, Wisconsin
- Conditions
- Digestive Health, Abdominal Discomfort, Gas and Bloating
- Interventions
- Bloat Gummies, Placebo
- Dietary Supplement
- Lead sponsor
- Arrae
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026
- U.S. locations
- 1
- States / cities
- San Francisco, California
- Conditions
- Osteodysplasia
- Interventions
- Marrow stromal cell infusion
- Biological
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Not listed
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2008
- U.S. locations
- 1
- States / cities
- Memphis, Tennessee
- Conditions
- Osteogenesis Imperfecta
- Interventions
- Risedronate Oral Tablet
- Drug
- Lead sponsor
- Hospital for Special Surgery, New York
- Other
- Eligibility
- 6 Years to 100 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2019 – 2027
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Irritable Bowel Syndrome
- Interventions
- curcumin, placebo
- Dietary Supplement
- Lead sponsor
- Kaiser Permanente
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 17 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- IBS (Irritable Bowel Syndrome), Bloating, Abdominal Pain, Gut Health
- Interventions
- FODMAP-targeting digestive enzyme blend, Placebo
- Dietary Supplement · Other
- Lead sponsor
- Kiwi Health Inc
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 150 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Achondroplasia
- Interventions
- Recifercept
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 15 Months to 12 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 6
- States / cities
- Irvine, California • Long Beach, California • Torrance, California + 2 more
- Conditions
- MPS IV A
- Interventions
- BMN 110 Weekly, Placebo, BMN 110 Every Other Week
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 177 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 6
- States / cities
- Oakland, California • Wilmington, Delaware • Washington D.C., District of Columbia + 3 more
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- Albert Einstein College of Medicine
- Other
- Eligibility
- Up to 4 Weeks
- Enrollment
- 100,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2029
- U.S. locations
- 9
- States / cities
- Brooklyn, New York • Manhasset, New York • New York, New York + 2 more
- Conditions
- Morquio A Syndrome, Mucopolysaccharidosis IV A
- Interventions
- Not listed
- Lead sponsor
- Nadia Ali, PhD
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2018
- U.S. locations
- 1
- States / cities
- Decatur, Georgia
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Interventions
- Not listed
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- Not listed
- Enrollment
- 353 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2014
- U.S. locations
- 2
- States / cities
- Oakland, California • Chicago, Illinois
- Conditions
- Mucopolysaccharidosis IV A, Morquio A Syndrome, MPS IVA
- Interventions
- BMN 110 - Weekly, BMN 110 - Every Other Week
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 173 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • Oakland, California • Orange, California + 7 more
- Conditions
- Hypochondroplasia
- Interventions
- infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
- Drug
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Industry
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 8
- States / cities
- Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more