Tebapivat for Healthy Subjects

Overseen By Agios Medical Affairs

Age: 18 - 65

Sex: Male

Trial Phase: Phase 1

Sponsor: Agios Pharmaceuticals, Inc.

Must not be taking: St. John's wort

Disqualifiers: Metabolic, Hepatic, Cardiovascular, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests how the body processes a new drug called tebapivat, using both oral and intravenous (IV) doses. It focuses on understanding the drug's metabolism and elimination in healthy male participants. The study seeks men who are generally healthy, have regular bowel movements, and weigh between 50 and 100 kilograms (about 110 to 220 pounds). Participants should not have significant health issues or a history of medication allergies. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new drug.

Will I have to stop taking my current medications?

Yes, you will need to stop taking any prescription medications at least 14 days before the trial, and any nonprescription medications, including vitamins and herbal supplements, 7 days before the trial. This is to ensure the medications do not affect the study results.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that tebapivat has been studied for its effects on anemia. While the safety of tebapivat in humans is still under investigation, some information about its tolerability is available. Earlier trials indicated that tebapivat generally caused mild side effects, which were manageable and expected for this type of treatment. This trial is in the early stages, focusing on how the body processes the drug. This phase often involves close monitoring to ensure safety and to gather more detailed information on any possible side effects. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about tebapivat because it offers a novel approach by using isotopically labeled compounds, [14C]-tebapivat and [13C2,15N3]-tebapivat, to study drug metabolism and pharmacokinetics. Unlike other treatments that target specific diseases, this investigation focuses on understanding how the body processes the drug, which could lead to more effective dosing strategies and safer use in the future. The use of radiolabeled compounds allows for precise tracking and measurement within the body, providing invaluable data that can inform the development of new therapies.

What evidence suggests that this trial's treatments could be effective?

This trial will evaluate the safety and effects of tebapivat in healthy subjects. Studies have shown that tebapivat may benefit individuals with certain blood disorders. Research indicates that tebapivat can improve anemia by raising hemoglobin levels in patients. Early findings suggest it is safe and well-tolerated. Tebapivat activates an enzyme in red blood cells, potentially enhancing their function and lifespan. This could benefit conditions like sickle cell disease, where healthy red blood cells are crucial. Although more research is needed, early results are promising for improving blood health. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for healthy individuals who can participate in a study to understand how the body absorbs, breaks down, and eliminates a drug called Tebapivat. The details on specific inclusion or exclusion criteria are not provided.

Inclusion Criteria

I am a man aged 18-55 and agree to use contraception.

Able to comprehend and willing to sign the informed consent form and abide by study restrictions

I have at least one bowel movement daily.

Exclusion Criteria

Use of tobacco- or nicotine-containing products within 3 months prior to check-in

I have not received or donated blood recently.

Participants deemed unsuitable by the investigator or designee

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive a single oral dose of 10 mg [14C]-tebapivat and a single intravenous microdose of 0.1 mg [13C2,15N3]-tebapivat on Day 1

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including quantification and identification of metabolites

6 weeks

Multiple visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• [13C2,15N3]-tebapivat
• [14C]-tebapivat

Trial Overview The study is testing how Tebapivat behaves in the body after a single oral dose by tracking its breakdown products and elimination. It also aims to compare the availability of Tebapivat when taken orally versus through an intravenous microdose.

How Is the Trial Designed?

Group I: [14C]-tebapivat and [13C2,15N3]-tebapivat Experimental Treatment 2 Interventions

Participants will receive a single oral dose of 10 milligrams (mg) \[14C\]-tebapivat containing 200 microcurie \[μCi\] of radiocarbon in a capsule followed by a single intravenous (IV) microdose of 0.1 mg \[13C2,15N3\]-tebapivat on Day 1.

Who Is Running the Clinical Trial?

Agios Pharmaceuticals, Inc.

Lead Sponsor

Trials

55

Recruited

4,200+

Citations

1. withpower.com withpower.com/trial/tebapivat-for-healthy-subjects-a999b

Tebapivat for Healthy Subjects · Info for Participants This trial is for healthy individuals who can participate in a study to understand how the body absorbs, breaks down, and eliminates a drug called Tebapivat.

2. investor.agios.com investor.agios.com/news-releases/news-release-details/agios-present-new-data-mitapivat-and-tebapivat-rare-blood

Agios to Present New Data on Mitapivat and Tebapivat in ... We will highlight the clinical progress of our two PK activators – mitapivat and tebapivat – in multiple rare blood disorders with high patient needs.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06924970

NCT06924970 | A Dose-Finding Study of Tebapivat to ... The main purpose of this study is to compare the effect of tebapivat versus placebo on anemia and to detect a dose-response for hemoglobin (Hb) response in ...

4. ash.confex.com ash.confex.com/ash/2024/webprogram/Paper203509.html

Paper: Results from a Phase 1 Study to Assess the Safety, ... 2496 Results from a Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Tebapivat (AG-946) in Patients with Sickle Cell ...

5. georgiacancerinfo.org georgiacancerinfo.org/clinical-trials/detail/5414

A Study of Tebapivat (AG-946) in Participants With Anemia ... Tests the safety of a drug in healthy volunteers or subjects with indications. The aim is to find the best dose of a new drug with the fewest side effects ...

6. medicine.yale.edu medicine.yale.edu/ycci/clinicaltrials/trial/a-study-of-ag-946-in-participants-with-anemia-due-to-lower-risk-myelodysplastic-syndromes-lr-mds/

Contact Information - Yale School of Medicine This purpose of this study is to establish proof of concept of tebapivat in participants with LR-MDS in Phase 2a and to evaluate the effect ...

7. thalassaemia.org.cy thalassaemia.org.cy/clinical-trial-updates-scd/tebapivat/

Tebapivat – TIF Clinical Trial/Study Information. Trial Name: A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)