- Conditions
- XIAP Deficiency, NLRC4-MAS
- Interventions
- Tadekinig alfa
- Drug
- Lead sponsor
- AB2 Bio Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 8
- States / cities
- La Jolla, California • Gainsville, Florida • Atlanta, Georgia + 5 more
Search Results
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Showing 25–29
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- 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
- Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
- Interventions
- DISC-1459
- Drug
- Lead sponsor
- Disc Medicine, Inc
- Industry
- Eligibility
- 12 Years and older
- U.S. locations
- 5
- States / cities
- Miami, Florida • Boston, Massachusetts • New York, New York + 1 more
- Conditions
- Erythropoietic Protoporphyria (EPP), X-Linked Protoporphyria (XLP)
- Interventions
- Dersimelagon, Placebo
- Drug
- Lead sponsor
- Tanabe Pharma America, Inc.
- Industry
- Eligibility
- 12 Years to 75 Years
- Enrollment
- 165 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 13
- States / cities
- Huntington Beach, California • San Francisco, California • Miami, Florida + 10 more
- Conditions
- Erythropoietic Protoporphyria, EPP, X-Linked Protoporphyria, XLP, XLPP, X-Linked Dominant Erythropoietic Protoporphyria, XLEPP, XLDP
- Interventions
- Not listed
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Other
- Eligibility
- Not listed
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2019
- U.S. locations
- 6
- States / cities
- Birmingham, Alabama • San Francisco, California • New York, New York + 3 more