Mitapivat for Beta Thalassemia Transfusion Dependent

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Beta Thalassemia Transfusion Dependent+1 MoreMitapivat - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial will compare the effect of the drug mitapivat versus a placebo on transfusion burden in people with transfusion-dependent alpha- or beta-thalassemia.

Eligible Conditions
  • Transfusion-dependent Alpha-Thalassemia
  • Beta Thalassemia Transfusion Dependent

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 19 Secondary · Reporting Duration: Pre-dose Day 1; pre-dose Week 12; pre-dose Week 24; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 36

Week 48
Percentage of Participants With Transfusion Reduction Response (TRR)
Percentage of Participants With ≥50% Reduction in Transfused RBC Units in Any Consecutive 24-week Period Through Week 48 Compared With Baseline
Week 48
Change From Baseline in Transfused RBC Units From Week 13 Through Week 48
Percentage of Participants With ≥33% Reduction in Transfused RBC Units From Week 13 Through Week 48 Compared With Baseline
Percentage of Participants With ≥50% Reduction in Transfused RBC Units From Week 13 Through Week 48 Compared With Baseline
Baseline, Week 48
Change From Baseline in Iron Through Week 48
Change From Baseline in Serum Ferritin Through Week 48
Change From Baseline in Total Iron Binding Capacity Through Week 48
Change From Baseline in Transferrin Saturation Through Week 48
Week 36
Blood Concentration of 2,3 - diphosphoglycerate (2,3-DPG)
Blood Concentration of Adenosine Triphosphate (ATP)
Week 36
Plasma Exchange
Week 36
Area Under the Concentration-time Curve From Time Zero to the Last Measurable Concentration (AUClast) of Mitapivat
Maximum Plasma Concentration (Cmax) of Mitapivat
Time of Maximum Plasma Concentration (Tmax) of Mitapivat
Up to Week 317
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Percentage of Participants with Adverse Events (AEs) Considered by the Investigator to be Related to Study Drug
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs), Graded by Severity
Percentage of Participants with Serious Adverse Events (SAEs) Considered by the Investigator to be Related to Study Drug
Up to Week 48
Percentage of Participants With Transfusion-Independence

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

2 Treatment Groups

Mitapivat
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

240 Total Participants · 2 Treatment Groups

Primary Treatment: Mitapivat · Has Placebo Group · Phase 3

Mitapivat
Drug
Experimental Group · 1 Intervention: Mitapivat · Intervention Types: Drug
PlaceboPlaceboComparator Group · 2 Interventions: Placebo Matching Mitapivat, Mitapivat · Intervention Types: Drug, Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: pre-dose day 1; pre-dose week 12; pre-dose week 24; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose week 36

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
49 Previous Clinical Trials
3,799 Total Patients Enrolled
Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
35 Previous Clinical Trials
8,108 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 5 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have documented a diagnosis of thalassemia based on DNA analysis.