Pyruvate Kinase DeficiencyPhase 4NCT05777993
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
A Phase 4 study testing Mitapivat in people with Pyruvate Kinase Deficiency and Anemia, Hemolytic, run by Agios Pharmaceuticals, Inc. at 23 sites in 8 countries.
Trial summary
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease
- What is being tested?
- Mitapivat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 23 sitesin 8 countries
- Study dates
- 6 Jul 2023to Aug 2031
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participating in an Agios-sponsored study of mitapivat and deriving clinical benefit from ongoing mitapivat treatment, in the opinion of the Investigator.
- For women of childbearing potential (WOCBP), must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of providing informed consent/assent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can be an acceptable barrier method.
- Written informed consent/assent from the participant (or their legally authorized representative, parent[s], or legal guardian) before any study-related procedures are conducted, and participants must be willing to comply with all study procedures for the duration of the study.
- Agrees not to participate in other interventional clinical studies, including other studies of mitapivat, during participation in this study.
Who cannot join
- Known allergy or other contraindication to mitapivat or its inactive ingredients as defined in the current investigator brochure (IB).
- Any medical, hematologic, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data. Also excluded are:
- Participants who are institutionalized by regulatory or court order.
- Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor).
Trial locations and contacts
23 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
23 sites in 8 countries
Brazil7 sites
Hospital de Clínicas da Unicamp
Campinas, São Paulo, 13083-878
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS)
Porto Alegre, Rio Grande do Sul, 90619-900
HEMORIO Instituto Nacional de Hematologia
Rio de Janeiro, 20211-030
Praxis Pesquisa Medica
Santo André, 09090-790
GSH Banco de Sangue de São Paulo
São Paulo, 04006-002
Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo
São Paulo, 05403-010
Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP
São Paulo, 14051-030
France6 sites
CHU de Bordeaux - Hôpital Pellegrin
Bordeau, 33000
CHU Hôpital Henri Mondor
Créteil, 94010
Hopital Edouard Herriot, CHU de Lyon
Lyon, 69003
Hopitaux de La Timone
Marseille, Bouches-du-Rhône, 13005
AP-HP - Hôpital Européen Georges Pompidou
Paris, 75015
Hôpital Necker Enfants Malades
Paris, 75015
Japan3 sites
Kyoto Katsura Hospital
Kyoto, 615-8256
Toho University - Omori Medical Center
Ōta-ku, 143-8541
Tohoku University Hospital
Sendai, Miyagi, 980-8574
Canada2 sites
McMaster Centre for Transfusion Medicine McMaster University
Hamilton, Ontario, L8S3Z5
University Health Network
Toronto, Ontario, M5G2C4
Israel2 sites
Rambam Medical Center
Haifa, Ḥeifā, 31096
Ziv Medical Center
Safed, Ḥeifā, 13100
United Kingdom1 site
Manchester Royal Infirmary, Manchester University NHS Foundation Trust
Manchester, M13 9W
About this study
Official title: A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
Read the detailed description
Participants will continue the mitapivat dose regimen that they were receiving at the final visit of their antecedent study.
- Being tested
- Mitapivat
- Medical subject headings
- Pyruvate Kinase Deficiency of Red Cells, Anemia, Hemolytic, Anemia, Sickle Cell, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MitapivatDrug
Also called AG-348, Mitapivat sulfate
Tablets, granules
| Study group | Role | What they receive |
|---|---|---|
| Mitapivat | Experimental | Participants will continue to receive mitapivat at the same dose regimen they were receiving at the final visit of their antecedent study. Receives: Drug: Mitapivat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study
Measured: Not Applicable - Study Completion
Secondary outcomes (4)
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Up to approximately 6.2 years
Percentage of Participants With AEs and SAEs, Graded by Severity
The severity of all AEs will be graded by the Investigator according to Version 4.03 of the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) on a 5-point severity scale (Grade 1 through Grade 5) where Grade 1 is mild, Grade 2 is moderate, Grade 3 is severe, Grade 4 is life-threatening and Grade 5 is fatal.
Measured: Up to approximately 6.2 years
Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug
Measured: Up to approximately 6.2 years
Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug
Measured: Up to approximately 6.2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2023
- Study start6 Jul 2023
- Primary completionAug 2029 (estimated)
- Study completionAug 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pyruvate Kinase DeficiencyStudies of the same condition, open and under way.
- More trials run by Agios Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05777993
- Sponsor's protocol ID
- AG348-C-025
- Other IDs
- 2026-526243-35-00
- Status last verified
- Oct 2026
- First submitted
- 9 Mar 2023
- Last updated
- 28 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
- ClinicalTrials.gov ID
- NCT05777993
- Acronym / sponsor ID
- AG348-C-025
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Agios Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Jul 2023 (actual)
- Primary completion
- Aug 2029 (estimated)
- Study completion
- Aug 2031 (estimated)
- First posted
- 21 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.
Detailed description
Participants will continue the mitapivat dose regimen that they were receiving at the final visit of their antecedent study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mitapivat | Experimental | Drug: MitapivatParticipants will continue to receive mitapivat at the same dose regimen they were receiving at the final visit of their antecedent study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Participating in an Agios-sponsored study of mitapivat and deriving clinical benefit from ongoing mitapivat treatment, in the opinion of the Investigator.
- For women of childbearing potential (WOCBP), must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of providing informed consent/assent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can be an acceptable barrier method.
- Written informed consent/assent from the participant (or their legally authorized representative, parent[s], or legal guardian) before any study-related procedures are conducted, and participants must be willing to comply with all study procedures for the duration of the study.
- Agrees not to participate in other interventional clinical studies, including other studies of mitapivat, during participation in this study.
Key exclusion criteria (4 listed)
- Known allergy or other contraindication to mitapivat or its inactive ingredients as defined in the current investigator brochure (IB).
- Any medical, hematologic, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data. Also excluded are:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MitapivatAlso: AG-348, Mitapivat sulfateTablets, granules | Drug | Mitapivat |
Primary outcome
Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study
Time frame: Not Applicable - Study Completion
4 secondary outcomes
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to approximately 6.2 years
Percentage of Participants With AEs and SAEs, Graded by Severity
Time frame: Up to approximately 6.2 years
Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug
Time frame: Up to approximately 6.2 years
Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug
Time frame: Up to approximately 6.2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
- Individual participant dataSharing planned: No
Related trials for Pyruvate Kinase Deficiency
Active, not recruitingObservational study
Pyruvate Kinase Deficiency Global Longitudinal Registry
Pyruvate Kinase Deficiency
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
Not yet recruitingPhase 1
High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
RecruitingObservational study
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Sickle Cell Disease, Thalassemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Mitapivat in people with Pyruvate Kinase Deficiency and Anemia, Hemolytic, run by Agios Pharmaceuticals, Inc. at 23 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 28 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in Brazil, France, Japan, Canada, Israel and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study (time frame: Not Applicable - Study Completion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Oct 2026.