A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia (NCT03692052)
๐ฉบ Plain-English Summary (8th-Grade Level)
Study AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT). This study includes a core period (up to 24 weeks) followed by an extension period (up to 10 years) for eligible participants. 20 participants with NTDT were enrolled. The initial dose of AG-348 was 50 milligrams (mg) twice daily (BID) with one potential dose-level increase to 100 mg BID at the Week 6 visit based on the participant's safety and hemoglobin (Hb) concentrations.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (9 items) and avoid the listed exclusions (16 items).
Location: UCSF Benioff Children's Hospital Oakland โ Oakland, California
๐ "Do I Qualify?" โ 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- Known history of diagnosis of Hb S or Hb C forms of thalassemia;
- Significant medical condition that confers an unacceptable risk to participating in the study, and/or could confound the interpretation of the study data;
- Splenectomy scheduled during the study treatment period or having undergone splenectomy within 12 months prior to signing informed consent;
- Currently enrolled in another therapeutic clinical trial involving ongoing therapy with any investigational or marketed product or placebo;
- Exposure to any investigational drug, device, or procedure within 3 months prior to the first day of study drug;
- Prior exposure to sotatercept (ACE-011), luspatercept (ACE-536), ruxolitinib, or gene therapy;
- Prior bone marrow or stem cell transplant;
- Currently pregnant or breastfeeding;
- History of major surgery within 6 months of signing informed consent;
- Currently receiving medications that are strong inhibitors of cytochrome P450 (CYP)3A4, strong inducers of CYP3A4, strong inhibitors of P-glycoprotein (P-gp), or digoxin (a P-gp sensitive substrate medication) that have not been stopped for a duration of at least 5 days or a timeframe equivalent to 5 half-lives (whichever is longer) prior to the first day of study drug;
- Currently receiving chronic anticoagulant therapy, unless started and on a stable dose for at least 28 days prior to first day of study drug;
๐ Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
๐ All Participating Trial Locations (4 Sites)
- UCSF Benioff Children's Hospital Oakland โ Oakland, California
- Massachusetts General Hospital โ Boston, Massachusetts
- University Health Network (Toronto General Hospital) โ Toronto, Ontario
- Imperial College Healthcare NHS Trust (Hammersmith Hospital) โ London,
๐ฐ Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.