NCT07055243 To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
| NCT ID | NCT07055243 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | University Health Network, Toronto |
| Condition | Anemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 9 participants |
| Start Date | 2025-06-26 |
| Primary Completion | 2026-06-30 |
Trial Parameters
Eligibility Fast-Check
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Brief Summary
This is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII, a rare sub type of anemia. Nine patients from Princess Margaret who are diagnosed with CDAII will be enrolled to the study. Patients will be in the trial for 57 weeks treatment weeks and a safety follow up week after 30 days from last dose. First 8 weeks will be dose escalating period followed by 48 weeks of fixed dose period. 57th week will be dose tapering week. Data collected from Princes Margaret will be incorporated to the main study conducted in EU for analysis. Overall, approximately 25 patients are expected to be enrolled: Approximately 16 patients at sites in the EU and approximately 9 patients in Canada.
Eligibility Criteria
Inclusion Criteria: 1. Male or female with RBC membranopathy or congenital dyserythropoietic anemia type II (CDAII). Diagnosis must be supported genetically by a ACMG class 3 (VUS), 4 or 5 variant. 2. Age ≥18 years. 3. Average hemoglobin (Hb) concentration (average of at least 2 Hb measurements separated by a minimum of 7 days the during screening period) must be less than 13.0 g/dL for males and 11.0 g/dL for females. Patients with average Hb \>10.0 g/dL for males and females must meet at least one of the following additional criteria: 1. Splenomegaly (length ≥12.5 cm) 2. Fatigue attributed to hemolysis 3. Active hemolysis as evaluated by one or more of the following: haptoglobin, bilirubin, LDH, reticulocytes 4. Subjects must start or continue taking at least the equivalent of daily 0.8 mg oral folic acid for the duration of the study. 5. Have adequate organ function, as defined by: 1. Serum aspartate aminotransferase (AST) ≤2.5 × ULN (unless the increased AST is assessed by the Inve