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A Study of FT-4202 in Patients With Thalassemia or Sickle Cell Disease

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ClinicalTrials.gov Identifier: NCT04987489
Recruitment Status : Not yet recruiting
First Posted : August 3, 2021
Last Update Posted : August 3, 2021
Sponsor:
Information provided by (Responsible Party):
Forma Therapeutics, Inc.

Brief Summary:
This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat (FT-4202) in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell transfusions required and increase hemoglobin.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Thalassemia Drug: Etavopivat tablets Phase 2

Detailed Description:
Etavopivat is a potent, selective, orally bioavailable, small-molecule activator of pyruvate kinase red blood cell (PKR) being developed by Forma Therapeutics, Inc and is intended for use as a treatment for patients with sickle cell disease (SCD) or other inherited hemoglobinopathies or refractory anemias. This study is a multicenter, Phase 2, open-label, multiple-cohort study examining the safety and efficacy of etavopivat 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of Etavopivat (FT-4202) in Patients With Thalassemia or Sickle Cell Disease
Estimated Study Start Date : September 30, 2021
Estimated Primary Completion Date : March 30, 2025
Estimated Study Completion Date : July 31, 2025


Arm Intervention/treatment
Experimental: Etavopivat 400 mg daily - SCD with transfusions
Patients with sickle cell disease on chronic red blood cell transfusions
Drug: Etavopivat tablets
Etavopivat 400 mg once daily
Other Name: FT-4202

Experimental: Etavopivat 400 mg daily - Thalassemia with transfusions
Patients with thalassemia on chronic red blood cell transfusions
Drug: Etavopivat tablets
Etavopivat 400 mg once daily
Other Name: FT-4202

Experimental: Etavopivat 400 mg daily - Thalassemia
Patients with thalassemia not on chronic red blood cell transfusions
Drug: Etavopivat tablets
Etavopivat 400 mg once daily
Other Name: FT-4202




Primary Outcome Measures :
  1. Cohorts A: Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history [ Time Frame: 12 weeks ]
  2. Cohorts B: Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history [ Time Frame: 12 weeks ]
  3. Cohort C: Hemoglobin response rate at Week 12 (increase of ≥ 1.0 g/dL from baseline) [ Time Frame: 12 weeks ]

Secondary Outcome Measures :
  1. Cohort A: Proportion of patients with ≥ 33% reduction in red blood cell transfusion over a continuous 12-week treatment period versus baseline red blood cell transfusion history [ Time Frame: 12 weeks ]
  2. Cohort B: Proportion of patients with ≥ 33% reduction in red blood cell transfusion over a continuous 12-week treatment period versus baseline red blood cell transfusion history [ Time Frame: 12 weeks ]
  3. Cohort A: Reduction in red blood cell transfusions over 12 weeks [ Time Frame: 12 weeks ]
  4. Cohort A: Reduction in red blood cell transfusions over 24 weeks [ Time Frame: 24 weeks ]
  5. Cohort A: Reduction in red blood cell transfusions over 48 weeks [ Time Frame: 48 weeks ]
  6. Cohort B: Reduction in red blood cell transfusions over 12 weeks [ Time Frame: 12 weeks ]
  7. Cohort B: Reduction in red blood cell transfusions over 24 weeks [ Time Frame: 24 weeks ]
  8. Cohort B: Reduction in red blood cell transfusions over 48 weeks [ Time Frame: 48 weeks ]
  9. Cohort C: Hemoglobin response rate at Week 24 (increase of ≥ 1.0 g/dL from baseline). [ Time Frame: 24 weeks ]
  10. Cohort C: Hemoglobin response rate at Week 48 (increase of ≥ 1.0 g/dL from baseline). [ Time Frame: 48 weeks ]
  11. Change from baseline in hemoglobin over 12 weeks [ Time Frame: 12 weeks ]
  12. Change from baseline in hemoglobin over 24 weeks [ Time Frame: 24 weeks ]
  13. Change from baseline in hemoglobin over 48 weeks [ Time Frame: 48 weeks ]
  14. Changes in serum ferritin levels at 12 weeks versus baseline. [ Time Frame: 12 weeks ]
  15. Changes in serum ferritin levels at 24 weeks versus baseline. [ Time Frame: 24 weeks ]
  16. Changes in serum ferritin levels at 48 weeks versus baseline. [ Time Frame: 48 weeks ]
  17. Changes in liver iron concentration at 48 weeks versus baseline [ Time Frame: 48 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Provision of consent
  • Female patients of childbearing potential must use highly effective methods of contraception, male patients are willing to use barrier methods of contraception

Cohort A (Sickle Cell Disease Transfusion Cohort)

  • Confirmed diagnosis of sickle cell disease
  • Chronically red blood cell transfused for primary stroke prevention or due to previous stroke. Chronic red blood cell transfusion is defined as: ≥ 6 red blood cell units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for > 35 days during that period
  • Receiving chronic red blood cell transfusion by straight transfusions
  • At least 24 months of chronic monthly red blood cell transfusions for primary stroke prevention or treatment of primary stroke (initial completed overt clinical stroke with document infarction on brain computed tomography [CT] or magnetic resonance imaging [MRI])
  • On iron chelation therapy for > 3 months prior to enrollment
  • Documented adequate monthly transfusions with average HbS ≤ 45% (the upper limit of the established academic community standard) for the previous 12 weeks of red blood cell transfusions before the first dose of study treatment

Cohort B (Thalassemia Transfusion Cohort)

  • Documented diagnosis of β-thalassemia, Hemoglobin E/ β-thalassemia or Hemoglobin H (α-thalassemia)
  • Chronically transfused, defined as: ≥ 6 red blood cell units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for > 35 days during that period
  • On iron chelation therapy for > 3 months prior to enrollment

Cohort C (Thalassemia Non-transfused Cohort)

  • Documented diagnosis of β-thalassemia, Hemoglobin E/ β-thalassemia or Hemoglobin H (α-thalassemia)
  • Hemoglobin ≤ 10 g/dL

Exclusion Criteria:

  • Female who is breast feeding or pregnant
  • Hepatic dysfunction characterized by:

    • Alanine aminotransferase (ALT) > 4.0 × upper limit of normal (ULN)
    • Direct bilirubin > 3.0 × ULN
    • History of cirrhosis
  • Known human immunodeficiency virus (HIV) positivity
  • Active hepatitis B or hepatitis C infection
  • Severe renal dysfunction or on chronic dialysis
  • History of malignancy within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation.

    • Patients with malignancy considered surgically cured are eligible (eg, non- melanoma skin cancer, cancer of the cervix in-situ, ductal carcinoma in situ [Stage 1], Grade 1 endometrial cancer)
  • History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following:

    • Unstable angina pectoris or myocardial infarction or elective coronary intervention
    • Congestive heart failure requiring hospitalization
    • Uncontrolled clinically significant arrhythmias
    • Symptomatic pulmonary hypertension

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04987489


Contacts
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Contact: Forma Therapeutics 617-679-1970 medicalinformation@formatherapeutics.com

Sponsors and Collaborators
Forma Therapeutics, Inc.
Investigators
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Study Director: Von Potter, MD Forma Therapeutics
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Responsible Party: Forma Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04987489    
Other Study ID Numbers: 4202-HEM-201
First Posted: August 3, 2021    Key Record Dates
Last Update Posted: August 3, 2021
Last Verified: July 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Forma Therapeutics, Inc.:
SCD
sickle cell disease
sickle cell
anemia
sickle cell anemia
hemolytic
hemoglobin
vaso-occlusive crisis
VOC
vaso-occlusive events
sickle cell crisis
pain crisis
pain episode
congenital anemia
hemolytic anemia
hematologic disease
hemoglobinopathy
hemoglobinopathies
genetic disease
inborn disease
sickle cell trait
pyruvate kinase
PKR
thalassemia
beta-thalassemia
alpha-thalassemia
transfusions
hemoglobin H
transfusion
transfusion-dependent
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn