AG-348 for Pyruvate Kinase Deficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests the safety and effectiveness of a drug called AG-348 (mitapivat) for individuals with pyruvate kinase (PK) deficiency, a rare blood disorder affecting red blood cells. Participants will receive either a low or high dose of AG-348 to evaluate its efficacy and potential side effects. Individuals with a known history of PK deficiency who have not required frequent blood transfusions in the past year might be suitable candidates for this trial. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.
Do I have to stop taking my current medications for the trial?
The trial requires that you stop taking certain medications that affect liver enzymes or are known to interact with the study drug within a specific time before starting the trial. Specifically, you must avoid medications that strongly affect cytochrome P450 (CYP) 3A4 or P-glycoprotein within 5 to 28 days before starting the trial.
Is there any evidence suggesting that AG-348 is likely to be safe for humans?
Research has shown that AG-348, also known as mitapivat, is generally safe for people with pyruvate kinase (PK) deficiency. In earlier studies, serious side effects such as hemolytic anemia (where red blood cells break down too quickly) and pharyngitis (throat inflammation) occurred in about 4% of patients. Overall, about half of the patients experienced some side effects, though these were not always serious.
Long-term research indicates that mitapivat is well-tolerated, meaning most people can take it without major problems. In a 32-week study with children, mitapivat was safe, with side effects similar to those seen in earlier studies. This ongoing research supports mitapivat as a promising treatment for PK deficiency, but individual experiences with the drug can vary.12345Why do researchers think this study treatment might be promising?
Researchers are excited about AG-348 for treating pyruvate kinase deficiency because it offers a novel approach by directly activating the pyruvate kinase enzyme, which is deficient in patients with this condition. Unlike current treatments that primarily focus on managing symptoms or require blood transfusions, AG-348 directly targets the root cause of the disease. This unique mechanism of action could lead to a more effective and sustainable management of the condition, potentially reducing the need for frequent blood transfusions and improving patients' quality of life.
What evidence suggests that AG-348 might be an effective treatment for PK deficiency?
Research has shown that mitapivat, also known as AG-348, may help treat pyruvate kinase (PK) deficiency. In earlier studies, mitapivat significantly increased hemoglobin levels in about half of the adults with this condition. Hemoglobin is crucial for carrying oxygen in the blood. Patients also experienced less breakdown of red blood cells, a common issue in PK deficiency. Additionally, mitapivat improved symptoms of anemia, making it the first potential treatment to directly target this disease. These findings suggest that mitapivat could be an effective treatment option for people with PK deficiency. Participants in this trial will receive either 50 mg or 300 mg of AG-348 twice daily to further evaluate its effectiveness.23678
Are You a Good Fit for This Trial?
Adults with Pyruvate Kinase (PK) Deficiency who are not pregnant or breastfeeding, have a hemoglobin level below set thresholds, and haven't had recent blood transfusions can join. They must be in stable health, not on certain medications, and agree to use contraception. Those with psychiatric disorders or other conditions that could affect study participation are excluded.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive multiple doses of AG-348 for up to 24 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Extension
Eligible participants may enter an Extension Period to receive AG-348 for up to 8 additional years
What Are the Treatments Tested in This Trial?
Interventions
- AG-348
Trial Overview
The trial is testing the safety and effectiveness of AG-348 (mitapivat) at various doses for treating PK deficiency. It's a multicenter study where participants receive different amounts of this medication to see how well it works and what side effects occur.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Participants with PK deficiency received AG-348, 50 milligrams (mg), as initial dose, twice daily (BID) for the Core Period (Week 24).
Participants with PK deficiency received AG-348, 300 mg, as initial dose, BID for the Core Period (Week 24).
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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 ...
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 ...
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.
4.
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 ...
NCT02476916 | A Study of AG-348 in Adult Participants ...
A multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.
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 ...
A Study to Evaluate Efficacy and Safety of AG-348 in Not ...
Study AG348-C-006 evaluated the efficacy and safety of orally administered AG-348 as compared with placebo in participants with pyruvate kinase (PK) deficiency.
8.
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 ...
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