An Extension Study of AG-348 in Adult Subjects with Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies

Phase 1

• Conditions: Pyruvate Kinase Deficiency Haemolytic anaemia; MedDRA version: 20.0 Level: PT Classification code 10037682 Term: Pyruvate kinase deficiency anaemia System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2018-003459-39-ES
• Lead Sponsor: Agios Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-04-12
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 96

Inclusion Criteria

1. Have signed written informed consent prior to participating in this extension study;

2. Have completed either antecedent study AG348-C-006 or AG348-C-007 through the Part 2 Week 24 Visit;

3. Cohorts 2 and 3: Have demonstrated clinical benefit from AG-348 treatment in the antecedent study, in the opinion of the Investigator;

4. For women of reproductive potential, have a negative pregnancy test at the Screening/Day 1 Visit;

5. For women of reproductive potential as well as men with partners who are women of reproductive potential, be abstinent as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of giving informed consent, during the study, and for 28 days (both men and women) following the last dose of study drug.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 96
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Have a significant medical condition (including clinically significant laboratory abnormality) that developed during his/her antecedent AG-348 study that confers an unacceptable risk to participating in this extension study, that could confound the interpretation of the study data, and/or that compromises the ability of the subject to complete study visits and procedures;

2. Are currently pregnant or breastfeeding;

3. Have a splenectomy scheduled during the study treatment period;

4. Meet the withdrawal criteria of his/her antecedent AG-348 study at the Screening/Day 1 Visit of this study;

5. Are currently receiving medications that are strong inhibitors of cytochrome P450 (CYP)3A4, strong inducers of CYP3A4, strong inhibitors of P-glycoprotein (P-gp), or digoxin (a P-gp sensitive substrate medication) that have not been stopped for a duration of at least 5 days or a timeframe equivalent to 5 half-lives (whichever is longer) prior to Screening/Day 1 of this extension study;

6. Have received anabolic steroids, including testosterone preparations, within 28 days prior to Screening/Day 1 of this extension study;

7. Have received hematopoietic stimulating agents (eg, erythropoietins, granulocyte colony stimulating factors, thrombopoietins) within 28 days prior to Screening/Day 1 of this extension study;

8. Have exposure to any investigational drug other than AG-348, device, or procedure within 3 months prior to Screening/Day 1 of this extension study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified