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Allosteric Activator

Mitapivat for Sickle Cell Disease

Phase 2 & 3

Recruiting

Research Sponsored by Agios Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before starting study drug

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)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, weeks 24, 28, 40, and 52

Awards & highlights

Study Summary

This trial will test how well mitapivat works compared to placebo in treating sickle cell disease by increasing hemoglobin levels and reducing or preventing sickle cell pain crises. The long-term effect of mitapivat will also be explored.

Who is the study for?

Adults and teens aged 16 or older with sickle cell disease (SCD) who've had 2-10 pain crises in the past year can join. They need stable hemoglobin levels between 5.5 and 10.5 g/dL, possibly on a steady dose of hydroxyurea for at least three months, and must use two forms of contraception if they can have children. People with severe liver, gallbladder, kidney diseases, prior gene therapy or transplants, recent other SCD treatments except hydroxyurea, or certain drug interactions are excluded.Check my eligibility

What is being tested?

The trial is testing Mitapivat against a placebo to see if it raises hemoglobin levels and reduces sickle cell pain crises. Participants will be randomly assigned to receive either Mitapivat or a matching dummy pill (placebo). The study includes an initial phase to set the right dose followed by a longer-term evaluation of its effects.See study design

What are the potential side effects?

Possible side effects include but aren't limited to headaches, nausea, fatigue, insomnia and muscle pains. Since this is part of what the study is investigating; all potential side effects may not be known yet.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My hydroxyurea dose has been the same for the last 90 days.

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I have been diagnosed with sickle cell disease.

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I've had 2 to 10 sickle cell pain crises in the last year.

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I agree to use two forms of birth control.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, weeks 24, 28, 40, and 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, weeks 24, 28, 40, and 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, weeks 24, 28, 40, and 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Phase 2: Percentage of Participants With Hemoglobin (Hb) Response

Phase 2: Percentage of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious AEs (SAEs)

Phase 3: Annualized Rate of Sickle Cell Pain Crises (SCPCs)

+1 more

Secondary outcome measures

Phase 2: Annualized Rate of SCPCs

Phase 2: Change From Baseline in Absolute Reticulocytes Count

Phase 2: Change From Baseline in Erythropoietin

+33 more

Trial Design

7Treatment groups

Experimental Treatment

Placebo Group

Group I: Phase 3: Open-Label Extension PeriodExperimental Treatment1 Intervention

Participants may choose to receive mitapivat 100 mg BID for 216 weeks after the Double-blind Period. Participants who received mitapivat-matching placebo in the double-blind period, may choose to receive mitapivat 100 mg BID for 216 weeks after the Double-blind Period.

Group II: Phase 3: Mitapivat 100 mg BIDExperimental Treatment1 Intervention

Double-blind Period: Mitapivat 100 mg BID for 52 weeks.

Group III: Phase 2: Open-Label Extension PeriodExperimental Treatment1 Intervention

Participants who received mitapivat 50mg BID in the double-blind period may choose to receive mitapivat 50mg BID for 216 weeks after. Participants who received mitapivat 100mg BID in the double-blind period may choose to receive mitapivat 100 mg BID for 216 weeks after. Participants who received mitapivat-matching placebo in the double-blind period, may be randomized to receive either mitapivat 50 mg or 100 mg BID for 216 weeks after.

Group IV: Phase 2: Mitapivat 50 mg BIDExperimental Treatment1 Intervention

Double-blind Period: Mitapivat 50 milligrams (mg) twice daily (BID) for 12 weeks.

Group V: Phase 2: Mitapivat 100 mg BIDExperimental Treatment1 Intervention

Double-blind Period: Mitapivat 100 mg BID for 12 weeks.

Group VI: Phase 2: PlaceboPlacebo Group1 Intervention

Double-blind Period: Mitapivat-matching placebo for 12 weeks.

Group VII: Phase 3: PlaceboPlacebo Group1 Intervention

Double-blind Period: Mitapivat-matching placebo for 52 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mitapivat

2023

Completed Phase 1

~20

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor

52 Previous Clinical Trials

3,939 Total Patients Enrolled

Media Library

Mitapivat (Allosteric Activator) Clinical Trial Eligibility Overview. Trial Name: NCT05031780 — Phase 2 & 3

Sickle Cell Disease Research Study Groups: Phase 3: Mitapivat 100 mg BID, Phase 2: Mitapivat 50 mg BID, Phase 2: Open-Label Extension Period, Phase 2: Mitapivat 100 mg BID, Phase 2: Placebo, Phase 3: Placebo, Phase 3: Open-Label Extension Period

Sickle Cell Disease Clinical Trial 2023: Mitapivat Highlights & Side Effects. Trial Name: NCT05031780 — Phase 2 & 3

Mitapivat (Allosteric Activator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05031780 — Phase 2 & 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Where are the sites of this clinical trial?

"Presently, this study is enrolling patients from 13 different sites. While some of these sites are in major cities such as Boston and Houston, it is important to select the location closest to you to minimize travel requirements."

Answered by AI

Are we able to sign people up for this experiment currently?

"That is correct. The clinical trial is actively recruiting participants, as is stated on clinicaltrials.gov. The trial was originally posted on 2022-01-19 and was last updated on 2022-10-21. The trial is looking for 267 participants across 13 locations."

Answered by AI

Is this a novel clinical trial?

"There are 9 ongoing studies for Mitapivat in 51 cities and 25 countries. The first study was completed in 2015 and it reached Phase 2 drug approval. The sponsor of the original study was Agios Pharmaceuticals, Inc. and it had 52 participants. In the 5 years since the initial study, 12 more trials have been completed."

Answered by AI

Is this the first time Mitapivat has been researched?

"At present, there are 9 different clinical trials studying Mitapivat's efficacy. Of these 9 trials, 6 are in Phase 3. While the majority of these trials take place in Kuala Lumpur or Washington, there are a total of 210 trial sites for this medication."

Answered by AI

How many patients are included in this clinical trial?

"That is correct, the information available on clinicaltrials.gov confirms that this study is currently seeking patients. The trial was originally posted on January 19th, 2022 and was most recently updated on October 21st, 2022. A total of 267 patients are needed for the trial, which will be conducted at 13 different locations."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

2022. "A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05031780.

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