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A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease
Overall Recruitment Status: Active, currently enrolling
 
Official Title
A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease
 
Region Sponsors
Mid-Atlantic
Agios Pharmaceuticals, Inc.
 
Acronym KP IRB No.
1781864
 
Study Type Phase
Clinical Trial Phase II
 
Study Population Description
Patients with Sickle Cell Disease
 
Purpose
The purpose of this research study is to find out if the drug mitapivat is safe and effective for treating sickle cell disease.
 
Detailed Description
The primary objective of Phase 2 is to determine the recommended dosage of mitapivat for Phase 3. Participants will be randomized 1:1:1 to receive 50mg BID mitapivat, 100mg BID mitapivat, or matched placebo. After 12 weeks of double-blind treatment, participants will have the option of receiving open-label mitapivat for up to an additional 216 weeks.
 
Gender Age Limit
Male & Female
 
Inclusion Criteria
  • Documented diagnosis of sickle cell disease (SCD) (HbSS, HbSC [combined heterozygosity for hemoglobins S and C], HbS/beta 0- thalassemia, HbS/ beta plus 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 at least 5.5 and 10.5 gram per deciliter (g/dL) at the most. Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by =7 days) collected during the Screening Period
  • 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.
 
Exclusion Criteria
  • 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

 
Keywords and/or Specific Medical Conditions
  • Hematology
  • Sickle Cell Disease
  • SCD
 
KP Clinical Facility
  • Largo Medical Center
 
Clinical Area
  • Hematology


Principal Investigator:
Bogdan Dumitriu, MD
Contact Information:
- Leslie Greenberg, MS
-Largo Medical Center


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