Haematological Disorders
Conditions
Interventions
Matched Placebo
Mitapivat
Sponsors
Agios Pharmaceuticals
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Age =16 years; subjects age 16 or 17 years must physically have completed puberty. • Documented diagnosis of SCD (HbSS, HbSC, HbS/ß0-thalassemia, HbS/ß+ thalassemia, or other sickle cell syndrome variants). • At least 2 sickle cell pain crises (SCPCs) and no more than 10 SCPCs in the past 12 months. • Hemoglobin =5.5 and =10.5 g/dL. • If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before starting study drug. • Women capable of becoming pregnant and men with partners who are women that are capable of becoming pregnant must agree to use 2 forms of contraception. • Other protocol-defined inclusion criteria may apply.
Exclusion criteria
Exclusion criteria: • Pregnant or breastfeeding. • Receiving regularly scheduled transfusions. • Hepatobiliary disorders including but not limited to significant liver disease or gallbladder disease. • Severe kidney disease. • Prior exposure to gene therapy or prior bone marrow or stem cell transplantation. • Currently receiving treatment for SCD (eg, voxelotor, crizanlizumab, L glutamine), with the exception of hydroxyurea. The last dose of such therapies must have been administered at least 90 days before starting study drug. • Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before starting study drug. • Received treatment on another investigational trial within 90 days prior to start of study drug or plans to participate in another investigational drug trial. • Taking medications that are strong inhibitors of CYP3A4/5 or strong inducers of CYP3A4 that cannot be stopped in an acceptable timeframe before starting study drug (timeframe will be discussed with your doctor). • Other protocol defined exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Phase 2) To determine the recommended Phase 3 dose of Mitapivat by evaluating the effect of 2 dose levels of Mitapivat versus placebo on: • Anemia in subjects with sickle cell disease (SCD) • Safety Phase 3) To determine the effect of Mitapivat versus placebo on: • Anemia in subjects with sickle cell disease (SCD) • Sickle cell pain crises (SCPCs) in subjects with SCD | — |
Secondary
| Measure | Time frame |
|---|---|
| Phase 2) To evaluate the effect of 2 doses of Mitapivat versus placebo on: - Anemia - Markers of hemolysis and erythropoiesis - Patient-reported fatigue - Sickle cell pain crises (SCPCs). To evaluate the pharmacokinetic and pharmacodynamic effects of Mitapivat. Phase 3) To evaluate the effect of Mitapivat versus placebo on: • Anemia in subjects with SCD • Markers of hemolysis • Markers of erythropoiesis • Patient-reported fatigue • Additional clinical efficacy measures related to SCPC | — |
Countries
Ghana, Kenya, Nigeria
Contacts
Public ContactLucas Tina
Investigator
Outcome results
None listed