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Study of Safety and Clinical Activities of FT-4202 in Thalassemia or SCD

  • Research type

    Research Study

  • Full title

    A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassemia or Sickle Cell Disease



  • Contact name

    Frank Kelly

  • Contact email

  • Sponsor organisation

    Forma Therapeutics, Inc.

  • Eudract number


  • Identifier


  • Research summary

    This study is a multicenter, Phase 2, open-label, multiple-cohort study examining the safety and efficacy of FT-4202 for the treatment of patients, age 12 to 65 years, with SCD or thalassemia. Three treatment cohorts based on the patients hemoglobinopathy (SCD or thalassemia) and transfusion requirements will be evaluated.
    • Cohort A: Up to 20 patients with SCD on chronic RBC transfusion therapy to prevent stroke or recurrence of stroke.
    • Cohort B: Up to 20 patients with thalassemia on chronic RBC transfusion therapy to prevent symptomatic anemia.
    • Cohort C: Up to 20 patients with thalassemia not receiving chronic RBC transfusion therapy
    • Primary objective: To assess the erythroid response to FT-4202 in adolescents and adults with SCD or thalassemia
    • Secondary Objective:
    • To further assess the safety and clinical activity of FT-4202 in adolescents and adults with SCD or thalassemia
    • To measure the effects of FT-4202 on measures of iron overload in all patients
    Patients will be enrolled into a 48-week treatment period. The dose of FT-4202 is 400 mg daily (QD) administered continuously. The study will end when all patients who entered the treatment period complete study treatment and the End of Study (EOS) visit or discontinue study participation, approximately 52 weeks after the last patient is enrolled in the study. Duration of study for an individual patient may last up to 58 weeks and includes a Screening
    Period (up to 6 weeks before study treatment), the treatment period of the study for up to
    48 weeks, and an EOS visit at 4 weeks (± 1 week) after last dose of study drug.
    Up to 20 patients will be enrolled per cohort to achieve a minimum of 12 patients evaluable for an erythroid response after 12 weeks of FT-4202.

  • REC name

    West of Scotland REC 1

  • REC reference


  • Date of REC Opinion

    11 Aug 2022

  • REC opinion

    Further Information Favourable Opinion

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