Mitapivat for Non-Transfusion-dependent Alpha-Thalassemia

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Non-Transfusion-dependent Alpha-Thalassemia+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 placebo on anemia in people with either alpha- or beta-non-transfusion dependent thalassemia.

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

Treatment Effectiveness

Effectiveness Progress

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

Study Objectives

1 Primary · 23 Secondary · Reporting Duration: Pre-dose Day 1; pre-dose Week 12; pre-dose, 0.5, 1, 3, 5, 7 hours post-dose Week 20

Week 24
Change From Baseline in Average Functional Assessment of Chronic Illness Therapy (FACIT) -Fatigue Subscale Score from Week 12 through Week 24
Change From Baseline in Average Hb Concentration From Week 12 through Week 24
Percentage of Participants With Hb 1.5+ Response
Percentage of Participants With Hemoglobin (Hb) Response
Baseline, Week 24
Change From Baseline in Erythropoietin at Week 24
Change From Baseline in Haptoglobin at Week 24
Change From Baseline in Indirect Bilirubin at Week 24
Change From Baseline in Lactate Dehydrogenase (LDH) at Week 24
Change From Baseline in Reticulocytes at Week 24
Change From Baseline in Serum Ferritin at Week 24
Change From Baseline in Transferrin Saturation (TSAT) at Week 24
Change From Baseline in the 6-minute Walk Test (6MWT) Distance at Week 24
Week 12
Percentage of Participants With Improvement in the Patient Global Impression of Severity (PGIS) -Fatigue by at Least 1 Category at Weeks 12, 16, 20, and 24 Compared With Baseline, or "No Change" if No or Mild Fatigue at Baseline
Week 20
Blood Concentration of 2,3 - diphosphoglycerate (2,3-DPG)
Blood Concentration of Adenosine Triphosphate (ATP)
Week 20
Plasma or Blood Concentrations Over Time for Mitapivat
Week 20
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 293
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)
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
Week 12
Percentage of Participants With Improvement in the Patient Global Impression of Change (PGIC) -Fatigue at Weeks 12, 16, 20, and 24, or "No Change" if No or Mild Fatigue at Baseline

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

171 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, 0.5, 1, 3, 5, 7 hours post-dose week 20

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
49 Previous Clinical Trials
3,865 Total Patients Enrolled
Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
35 Previous Clinical Trials
8,174 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have documented thalassemia based on Hb electrophoresis, Hb high-performance liquid chromatography (HPLC), and/or DNA analysis.