← Back to Search

Allosteric Activator

Long-Term Mitapivat for Sickle Cell Disease

Q: If a patient is 40 years old or younger, can they participate in this research study?

<p>This clinical trial is for adults aged 18-70. There are 149 other trials available for minors and 122 separate trials available for seniors.</p>

Q: How can I become a participant in this experiment?

<p>This clinical trial is looking for 15 patients with hemolytic anemia that meet the following requirements: between 18-70 years old, have not received a blood transfusion in the last 12 weeks, have Hb A on hemoglobin analysis, have a serum AST &#x3C;=2.5 x ULN, have a serum ALT &#x3C;=2.5 x ULN, have a serum creatinine &#x3C;=1.25 x UNL, and finally have an absolute neutrophil count >=1.0 x 10^9power/L..</p>

Phase 1 & 2

Waitlist Available

Led By Julia Z Xu, M.D.

Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria Checklist

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

Must have

1.6 For women of reproductive potential, have a negative serum pregnancy test during the screening period. Women of reproductive potential are defined as sexually mature women who have not undergone a hysterectomy, bilateral oophorectomy, or tubal occlusion; or who have not been naturally postmenopausal (i.e., who have not menstruated at all for at least the preceding 1 year prior to signing informed consent unrelated to hormonal contraception).

f. Activated partial thromboplastin time and international normalized ratio <=1.5 x ULN, unless the subject is receiving therapeutic anticoagulants.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 and 48 weeks

Awards & highlights

Study Summary

This trial is testing the long-term tolerability and safety of mitapivat (or AG-348) in people with sickle cell disease.

Who is the study for?

Adults aged 18-70 with Sickle Cell Disease (SCD) who previously participated in NIH study #19H0097 and benefited from it. They must have stable organ function, no recent blood transfusions, and for women of childbearing potential, a commitment to use two forms of contraception. Exclusions include significant heart rhythm issues, uncontrolled medical conditions like hypertension or diabetes, active infections or certain drug allergies.Check my eligibility

What is being tested?

The trial is testing the long-term safety and effectiveness of mitapivat tablets taken twice daily by people with SCD. It extends a previous study to see if benefits continue or improve over time. Participants will undergo various health assessments including physical exams, blood tests, heart checks (ECG), mobility tests (6-minute walk), echocardiograms for heart/lung function, bone scans (DXA), and health questionnaires.See study design

What are the potential side effects?

While specific side effects are not listed here, participants will monitor any symptoms they experience in a study diary which may include typical drug-related reactions such as digestive discomforts, potential liver enzyme changes due to medication metabolism challenges or other common medication-induced complications.

Eligibility Criteria

Inclusion Criteria

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

Select...

I am a woman who can have children and have a negative pregnancy test.

Select...

My blood clotting tests are within normal limits, unless I'm on blood thinners.

Select...

My kidney function, measured by creatinine levels or filtration rate, is within the normal range.

Select...

I am between 18 and 70 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 and 48 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 and 48 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 and 48 weeks for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

measure: Long-term safety and tolerability of mitapivat in subjects with stable sickle cell disease

Secondary outcome measures

To evaluate hemoglobin (Hb) response, changes in hemolytic markers, functional status, cardiopulmonary function, and health-related quality of life in SCD subjects maintained on mitapivat long-term.

To evaluate the pharmacokinetic

To monitor SCD-related safety endpoints in SCD subjects maintained on mitapivat long-term.

Trial Design

1Treatment groups

Experimental Treatment

Group I: 1Experimental Treatment1 Intervention

Subjects will be treated with a maintenance dose of mitapivat previously assessed for safety and tolerability in the Phase I study for an initial 48 weeks and undergo safety monitoring, evaluation of pharmacokinetics and pharmacodynamics, and assessment of secondary laboratory and clinical endpoints at pre-specified intervals during the study period.

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?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor

3,834 Previous Clinical Trials

47,310,620 Total Patients Enrolled

Julia Z Xu, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)

Swee Lay Thein, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)

12 Previous Clinical Trials

7,905 Total Patients Enrolled

Media Library

Mitapivat (Allosteric Activator) Clinical Trial Eligibility Overview. Trial Name: NCT04610866 — Phase 1 & 2

Sickle Cell Disease Research Study Groups: 1

Sickle Cell Disease Clinical Trial 2023: Mitapivat Highlights & Side Effects. Trial Name: NCT04610866 — Phase 1 & 2

Mitapivat (Allosteric Activator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04610866 — Phase 1 & 2

Frequently Asked Questions

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

If a patient is 40 years old or younger, can they participate in this research study?

"This clinical trial is for adults aged 18-70. There are 149 other trials available for minors and 122 separate trials available for seniors."

Answered by AI

How can I become a participant in this experiment?

"This clinical trial is looking for 15 patients with hemolytic anemia that meet the following requirements: between 18-70 years old, have not received a blood transfusion in the last 12 weeks, have Hb A on hemoglobin analysis, have a serum AST <=2.5 x ULN, have a serum ALT <=2.5 x ULN, have a serum creatinine <=1.25 x UNL, and finally have an absolute neutrophil count >=1.0 x 10^9power/L.."

Answered by AI

Are people with the relevant medical condition able to participate in this trial at this time?

"The clinical trial mentioned is not enrolling patients at the moment, based on the latest information available from clinicaltrials.gov. This study was originally posted on December 9th 2020 and was last edited on October 27th 2022. There are 249 other studies that are actively recruiting participants right now."

Answered by AI

What makes this research project unique compared to others in its field?

"Mitapivat has been the subject of clinical research since 2015. The first study, sponsored by Agios Pharmaceuticals, Inc., was conducted in 2015 and involved 52 patients. Following the successful completion of this initial trial, Mitapivat received approval for Phase 2 clinical trials. Today, there are 9 active studies being conducted across 54 cities in 28 countries."

Answered by AI

How many individuals are being monitored during this research?

"As of right now, this clinical trial is no longer recruiting patients for participation. The study was originally posted on December 9th, 2020 but the most recent update was on October 27th, 2022. If you are looking for other studies to participate in, there are 240 trials regarding hemolytic anemia and 9 involving Mitapivat that are actively searching for participants."

Answered by AI

Is there previous research to support the use of Mitapivat?

"There are currently 9 ongoing clinical studies investigating the effects of Mitapivat. The majority of these trials are in Phase 3 and most are based in Kyustendil, New york. However, there are a total of 330 research sites spread out across the globe."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat

2020. "Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04610866.

All > Sickle Cell Disease > Sickle Cell Anemia