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Mitapivat for Pediatric Pyruvate Kinase Deficiency

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Pediatric Pyruvate Kinase Deficiency+1 MoreMitapivat - Drug
Eligibility
1 - 17
All Sexes
What conditions do you have?
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Study Summary

This trial will evaluate the efficacy and safety of a drug called mitapivat in children with a condition called pyruvate kinase deficiency. Children will be given either the drug or a placebo, and researchers will track how well they do.

Eligible Conditions
  • Pediatric Pyruvate Kinase Deficiency
  • Pediatric Hemolytic Anemia

Treatment Effectiveness

Effectiveness Progress

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

Similar Trials

1
RP-L301
1
Group A
1
Mitapivat

Study Objectives

1 Primary · 28 Secondary · Reporting Duration: Week 8: ≤30 minutes predose; Week 12: ≤30 minutes predose

Week 20
Maximum Change in Hb Concentration From Baseline During the Double-blind Period
Percentage of Participants Achieving a Hemoglobin (Hb) Response
Week 280
Change From Baseline in Serum Ferritin Concentration
Change From Baseline in Serum Iron Concentration
Change From Baseline in Total Iron-binding Capacity
Change From Baseline in Transferrin/Transferrin Saturation
Week 286
Change From Baseline in Body Mass Index (BMI)-for-age Z-score
Change From Baseline in Bone Mineral Density (BMD) Z-score
Change From Baseline in Estradiol Concentration
Change From Baseline in Estrone Concentration
Change From Baseline in Free Testosterone Concentration in Participants ≥7 Years of Age or Tanner Stage ≥2 (Whichever Occurs First)
Change From Baseline in Height-for-age Z-score
Change From Baseline in Luteinizing Hormone Concentration in Participants ≥6 Years of Age
Change From Baseline in Reticulocytes
Change From Baseline in Sexual Maturity Rating with Tanner Stage
Change From Baseline in Total Testosterone Concentration
Change From Baseline in Weight-for-age Z-score
Change From Baseline in the Size of Ovarian Cysts in Female Participants
Number of Female Participants With Development of Ovarian Cysts
Week 294
Change From Baseline in Pyruvate Kinase Deficiency Diary (PKDD) Score
Change From Baseline in Pyruvate Kinase Deficiency Impact Assessment (PKDIA) Score
Baseline, Week 20
Change From Baseline in Haptoglobin Concentration at Week 20
Week 12
Average Change From Baseline in Hb Concentration at Weeks 12, 16, and 20
Average Change From Baseline in Indirect Bilirubin Concentration at Weeks 12, 16, and 20
Average Change From Baseline in Lactose Dehydrogenase (LDH) Concentration at Weeks 12, 16, and 20
Week 16
Concentration at Steady State (Css) of Mitapivat
Week 12
Trough Concentration (Ctrough) of Mitapivat
Week 2
Population PK Model Parameter Estimate: Area Under the Concentration-time Curve (AUC) Derived From Plasma Concentrations of Mitapivat
Population Pharmacokinetic (PK) Model Parameter Estimate: Maximum Plasma Concentration (Cmax) of Mitapivat

Trial Safety

Safety Progress

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

Similar Trials

1
RP-L301
2
Group A
2
Mitapivat

Trial Design

3 Treatment Groups

Mitapivat
1 of 3
Mitapivat (OLE period)
1 of 3
Placebo
1 of 3

Experimental Treatment

Non-Treatment Group

30 Total Participants · 3 Treatment Groups

Primary Treatment: Mitapivat · Has Placebo Group · Phase 3

Mitapivat
Drug
Experimental Group · 1 Intervention: Mitapivat · Intervention Types: Drug
Mitapivat (OLE period)Experimental Group · 2 Interventions: Mitapivat, Mitapivat-matching placebo · Intervention Types: Drug, Drug
Placebo
Drug
PlaceboComparator Group · 1 Intervention: Mitapivat-matching placebo · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: week 8: ≤30 minutes predose; week 12: ≤30 minutes predose

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
49 Previous Clinical Trials
4,006 Total Patients Enrolled
1 Trials studying Pediatric Pyruvate Kinase Deficiency
45 Patients Enrolled for Pediatric Pyruvate Kinase Deficiency
Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
35 Previous Clinical Trials
8,315 Total Patients Enrolled
1 Trials studying Pediatric Pyruvate Kinase Deficiency
45 Patients Enrolled for Pediatric Pyruvate Kinase Deficiency

Eligibility Criteria

Age 1 - 17 · All Participants · 7 Total Inclusion Criteria

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References

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