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Allosteric Activator

Continued Access to Mitapivat for Pyruvate Kinase Deficiency

Phase 4
Waitlist Available
Research Sponsored by Agios Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to approximately 6.2 years
Awards & highlights
No Placebo-Only Group
Pivotal Trial
Drug Has Already Been Approved

Summary

This trial will provide access to the med. mitapivat for those who completed a study & don't have it commercially.

Who is the study for?
This trial is for individuals who have completed a previous Agios-sponsored study on mitapivat and benefited from it, but can't get the drug commercially due to regulations or access issues. They must agree to use two forms of contraception if applicable, not join other studies, and comply with all procedures.
What is being tested?
The trial provides continued access to mitapivat for participants with hemolytic anemia or pyruvate kinase deficiency who previously responded well in an earlier study but lack commercial access to the medication.
What are the potential side effects?
While specific side effects are not listed here, participants should be aware that they may experience similar side effects as those encountered during their previous mitapivat trials. Known allergies or contraindications related to mitapivat's ingredients should be considered.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to approximately 6.2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to approximately 6.2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Percentage of Participants With AEs and SAEs, Graded by Severity

Side effects data

From 2020 Phase 3 trial • 80 Patients • NCT03548220
67%
Arthralgia
67%
Diarrhoea
67%
Nausea
67%
Nasopharyngitis
33%
Fatigue
33%
Headache
33%
Dyspnoea
33%
Hypertension
33%
Dyspepsia
33%
Musculoskeletal pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Experimental: AG-348, 20 mg
Experimental: AG-348, 5 mg
Experimental: AG-348, 50 mg
Placebo Comparator: Placebo

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Drug Has Already Been Approved
The FDA has already approved this drug, and is just seeking more data.

Trial Design

1Treatment groups
Experimental Treatment
Group I: MitapivatExperimental Treatment1 Intervention
Participants will receive mitapivat of 5, 20 or 50 milligrams (mg), orally, twice daily (BID), based on the last dose received by the participants in the antecedent study. Mitapivat will be administered from Day 1 up to end of treatment period of this study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Mitapivat
2023
Completed Phase 1
~20

Find a Location

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
53 Previous Clinical Trials
4,169 Total Patients Enrolled

Media Library

Mitapivat (Allosteric Activator) Clinical Trial Eligibility Overview. Trial Name: NCT05777993 — Phase 4
Hereditary Spherocytosis Research Study Groups: Mitapivat
Hereditary Spherocytosis Clinical Trial 2023: Mitapivat Highlights & Side Effects. Trial Name: NCT05777993 — Phase 4
Mitapivat (Allosteric Activator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05777993 — Phase 4
~4 spots leftby Aug 2029
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