AG-348 for Anemia, Hemolytic, Congenital Nonspherocytic

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Anemia, Hemolytic, Congenital Nonspherocytic+2 More
AG-348 - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a drug to see if it is safe and effective long-term for people who have already taken it.

Eligible Conditions
  • Anemia, Hemolytic, Congenital Nonspherocytic
  • Pyruvate Kinase Deficiency

Treatment Effectiveness

Effectiveness Progress

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

Study Objectives

2 Primary · 12 Secondary · Reporting Duration: Week 12: pre-dose, post-dose at 30 minutes, 1 h, 2 h, 4 h, 8 h

Week 198
Number of Participants with AEs Leading to Dose Reduction, Treatment Interruption and Treatment Discontinuation
Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Week 193
Change from Baseline in Bilirubin
Change from Baseline in HRQoL PRO Scores: Pyruvate Kinase Deficiency Impact Assessment (PKDIA)
Haptoglobins
Change from Baseline in Hb Concentration
Change from Baseline in Health-Related Quality of Life (HRQoL) Patient-Reported Outcome (PRO) Scores: Pyruvate Kinase Deficiency Diary (PKDD)
Change from Baseline in Lactate Dehydrogenase (LDH)
Change from Baseline in Number of Red Blood Cell (RBC) Units Transfused
Change from Baseline in Number of Transfusion Events
Change from Baseline in Reticulocyte Percentages
Week 12
Maximum Observed Concentration of AG-348 in Participants Who Previously Received Placebo in Study AG348-C-006
Week 12
Area Under the Concentration-Time Curve (AUC) of AG-348 in Participants Who Previously Received Placebo in Study AG348-C-006
Weeks 16, 20, 24
Percentage of Participants Achieving a Hemoglobin (Hb) Response in Participants Who Previously Received Placebo in Study AG348-C-006

Trial Safety

Safety Progress

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

Side Effects for

Experimental: AG-348, 50 mg
14%Headache
14%Nausea
14%Back pain
11%Fatigue
11%Dizziness
11%Abdominal pain
9%Hypertriglyceridaemia
9%Oropharyngeal pain
9%Middle insomnia
9%Nasopharyngitis
9%Hot flush
9%Cough
6%Arthralgia
6%Pain in extremity
6%Nasal congestion
6%Dyspnoea
6%Insomnia
6%Influenza like illness
6%Breast discomfort
6%Paraesthesia
6%Contusion
6%Abdominal distension
6%Gastroenteritis
6%Constipation
6%Diarrhoea
6%Presyncope
6%Dry skin
3%Alanine aminotransferase increased
3%Urinary tract infection
3%Epistaxis
3%Aspartate aminotransferase increased
3%Rhinitis allergic
3%Initial insomnia
3%Rib fracture
3%Atrial fibrillation
3%Hypertension
3%Dysmenorrhoea
This histogram enumerates side effects from a completed 2020 Phase 3 trial (NCT03548220) in the Experimental: AG-348, 50 mg ARM group. Side effects include: Headache with 14%, Nausea with 14%, Back pain with 14%, Fatigue with 11%, Dizziness with 11%.

Trial Design

3 Treatment Groups

Cohort 1
1 of 3
Cohort 2
1 of 3
Cohort 3
1 of 3

Experimental Treatment

90 Total Participants · 3 Treatment Groups

Primary Treatment: AG-348 · No Placebo Group · Phase 3

Cohort 1
Drug
Experimental Group · 1 Intervention: AG-348 · Intervention Types: Drug
Cohort 2
Drug
Experimental Group · 1 Intervention: AG-348 · Intervention Types: Drug
Cohort 3
Drug
Experimental Group · 1 Intervention: AG-348 · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Mitapivat
Not yet FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: week 12: pre-dose, post-dose at 30 minutes, 1 h, 2 h, 4 h, 8 h

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
49 Previous Clinical Trials
3,949 Total Patients Enrolled
10 Trials studying Anemia, Hemolytic, Congenital Nonspherocytic
1,196 Patients Enrolled for Anemia, Hemolytic, Congenital Nonspherocytic
Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
34 Previous Clinical Trials
8,238 Total Patients Enrolled
6 Trials studying Anemia, Hemolytic, Congenital Nonspherocytic
290 Patients Enrolled for Anemia, Hemolytic, Congenital Nonspherocytic

Eligibility Criteria

Age 18+ · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have signed written informed consent prior to participating in this study.
You are of reproductive potential.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 5th, 2021

Last Reviewed: November 26th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.