Mitapivat for Pyruvate Kinase Deficiency
(ACTIVATE-Kids Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores the effectiveness and safety of mitapivat, a new oral treatment for children with pyruvate kinase deficiency (PKD), a rare blood disorder affecting red blood cell function. The goal is to determine if mitapivat can improve blood counts compared to a placebo. It targets children with PKD who are not receiving regular blood transfusions. This trial may suit children with PKD who have had no more than five blood transfusions in the past year and have low hemoglobin levels, impacting their energy and daily activities. Participants will initially receive either mitapivat or a placebo and may continue on mitapivat for up to five years. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to the development of a promising new treatment.
Will I have to stop taking my current medications?
The trial requires that you stop taking certain medications before participating. Specifically, you must stop taking strong inhibitors or inducers of CYP3A4, anabolic steroids, hematopoietic stimulating agents, and any pyruvate kinase activators for specified periods before joining the trial.
Is there any evidence suggesting that mitapivat is likely to be safe for humans?
Research has shown that mitapivat is generally safe and well-tolerated in people with pyruvate kinase deficiency (PKD). In earlier studies, serious side effects such as hemolytic anemia (a condition where red blood cells break down too quickly) and pharyngitis (throat inflammation) were rare, affecting only about 4% of patients. About half of the patients experienced some side effects, though these were not always serious. Overall, different studies have consistently demonstrated the safety of mitapivat.12345
Why do researchers think this study treatment might be promising for pyruvate kinase deficiency?
Mitapivat is unique because it targets the root cause of pyruvate kinase deficiency by activating the pyruvate kinase enzyme. Most treatments for this condition, like blood transfusions and chelation therapy, only manage symptoms without addressing the underlying enzyme issue. Researchers are excited because mitapivat has the potential to improve red blood cell function and survival, offering a more direct and effective approach to treating this rare blood disorder.
What evidence suggests that mitapivat might be an effective treatment for pyruvate kinase deficiency?
Research has shown that mitapivat can help treat pyruvate kinase deficiency (PKD). In studies, mitapivat significantly raised hemoglobin levels, which is important for reducing anemia, or low red blood cell count, in patients. It also reduced the breakdown of red blood cells. Patients reported feeling less tired and experiencing an overall improvement in well-being. In this trial, participants will receive either mitapivat or a mitapivat-matching placebo during the double-blind period. These findings suggest that mitapivat may help people with PKD manage their symptoms better.26789
Who Is on the Research Team?
Medical Affairs
Principal Investigator
Agios Pharmaceuticals, Inc.
Are You a Good Fit for This Trial?
This trial is for children and teenagers aged 1 to less than 18 with Pyruvate Kinase Deficiency (PKD) who aren't getting regular blood transfusions. They must have a certain level of hemoglobin, not had many blood transfusions recently, be on folic acid supplements, and agree to use contraception if applicable. Those with severe liver or heart issues, recent major surgery like splenectomy, or specific genetic mutations are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Double-blind Treatment
Participants receive either mitapivat or placebo during an 8-week dose titration period followed by a 12-week fixed-dose period
Open-label Extension
Participants who complete the double-blind period receive mitapivat for up to 5 years
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Mitapivat
- Mitapivat-matching placebo
Trial Overview
The study tests the oral drug Mitapivat against a placebo in young patients with PKD. Participants will randomly receive either the drug or placebo in a ratio of 2:1 and will be grouped by age for dosing. The trial includes an initial dose adjustment phase followed by a fixed-dose period before moving into a long-term extension phase lasting up to five years.
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Placebo Group
Participants who have completed the double-blind period will be eligible to receive mitapivat for up to 5 years in the OLE period. Participants entering the OLE period will first receive blinded mitapivat and placebo for 8 weeks to maintain the double-blind treatment assignment before being transitioned to only receive active, open-label drug (mitapivat).
Double-Blind Period: Participants will receive mitapivat orally, at doses based on age and weight, for 8 weeks in the dose titration period and for 12 weeks in the fixed-dose period.
Double-Blind Period: Participants will receive mitapivat-matching placebo orally for 8 weeks in the dose titration period and for 12 weeks in the fixed-dose period.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Agios Pharmaceuticals, Inc.
Lead Sponsor
Citations
Mitapivat versus Placebo for Pyruvate Kinase Deficiency
In patients with pyruvate kinase deficiency, mitapivat significantly increased the hemoglobin level, decreased hemolysis, and improved patient-reported ...
Clinically meaningful improvements in patient‐reported ...
Mitapivat, a first-in-class, oral activator of PK approved for adults with PK deficiency4 has demonstrated improvements in anemia and.
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
Conclusions: The administration of mitapivat was associated with a rapid increase in the hemoglobin level in 50% of adults with pyruvate kinase ...
4.
ashpublications.org
ashpublications.org/bloodrci/article/1/2/100014/546279/Long-term-mitapivat-treatment-is-safe-andLong-term mitapivat treatment is safe and efficacious in ...
Median of 2.53-year follow-up of mitapivat showed favorable safety and tolerability in patients with SCD. Sustained improvements in Hb, ...
Clinically meaningful improvements in patient‐reported ...
Treatment with mitapivat showed long-term, durable, and clinically meaningful improvements in signs, symptoms, and functional impacts of PK ...
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
The most common serious adverse events, hemolytic anemia and pharyngitis, each occurred in 2 patients (4%). A total of 26 patients (50%) had an ...
7.
investor.agios.com
investor.agios.com/news-releases/news-release-details/agios-announces-results-phase-3-activate-kidst-study-mitapivatAgios Announces Results from Phase 3 ACTIVATE-KidsT ...
In the 32-week double-blind treatment period, mitapivat was generally safe and well-tolerated, with safety results consistent with the safety ...
8.
ashpublications.org
ashpublications.org/blood/article/134/Supplement_1/3512/428360/Long-Term-Safety-and-Efficacy-of-Mitapivat-AG-348Long-Term Safety and Efficacy of Mitapivat (AG-348), a ...
These long-term data support the potential of mitapivat as the first disease-altering therapy for PK deficiency. Two phase 3 trials are underway ...
Mitapivat in adult patients with pyruvate kinase deficiency ...
Mitapivat was well tolerated across all doses studied in the ACTIVATE-T trial, and its safety profile was consistent across all previously reported studies.
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