NCT04964323 (PKD) is a clinical trial in Pyruvate Kinase Deficiency, sponsored by Agios Pharmaceuticals, Inc..

Who is sponsoring NCT04964323?

NCT04964323 is sponsored by Agios Pharmaceuticals, Inc..

What condition does NCT04964323 study?

NCT04964323 studies Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia, Anemia, Hemolytic.

Is NCT04964323 still recruiting?

NCT04964323 is currently terminated. Last updated 7 September 2023.

Last reviewed 2023-09-07 · How we verify

NCT04964323: PKD

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

Terminated Last updated 7 September 2023

What this trial tests

trial in Pyruvate Kinase Deficiency in 15 participants. Terminated before completion.

Timeline

2 July 2021

Primary endpoint
30 June 2023

30 June 2023

Quick facts

Lead sponsor	Agios Pharmaceuticals, Inc.
Status	Terminated
Study type	OBSERVATIONAL
Enrollment	15
Start date	2 July 2021
Primary completion	30 June 2023
Estimated completion	30 June 2023
Sites	5 locations across Italy, Netherlands, United Kingdom, United States, Spain

Conditions studied

Pyruvate Kinase Deficiency — all drugs for Pyruvate Kinase Deficiency →
Pyruvate Kinase Deficiency Anemia — all drugs for Pyruvate Kinase Deficiency Anemia →
Anemia — all drugs for Anemia →
Anemia, Hemolytic — all drugs for Anemia, Hemolytic →

Sponsor

Agios Pharmaceuticals, Inc. — full company profile →

Who can join

18 and older, any sex, with Pyruvate Kinase Deficiency or Pyruvate Kinase Deficiency Anemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The primary objective of this study is to understand and characterize the health-related quality of life (HRQoL) and disease burden of adult participants with PK deficiency receiving routine clinical care. This study is an observational (i.e., noninterventional), longitudinal, multicenter, global registry for participants with PK deficiency, a rare nonspherocytic hemolytic anemia. This study will be open for enrollment for 2 years and all enrolled participants will be followed prospectively for up to 96 weeks. Data will be collected from participants who have provided informed consent and authorization pursuant to applicable laws and regulations.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design.
Grace RF, van Beers EJ, Vives Corrons JL, Glader B, et al · · 2023 · cited 4× · PMID 36958777 · DOI 10.1136/bmjopen-2022-063605

Verify or expand the search:

Other recruiting trials for Pyruvate Kinase Deficiency

Currently open trials in the same condition.

NCT04902833 — Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms · active not recruiting
NCT03481738 — Pyruvate Kinase Deficiency Global Longitudinal Registry · active not recruiting

Other Agios Pharmaceuticals, Inc. trials

Trials by the same sponsor.

NCT07075640 — A Study to Determine the Safety and Tolerability of AG-236 and How it is Absorbed, Broken Down, and Eliminated From the · Phase 1 · recruiting
NCT06745271 — A Study to Determine How Tebapivat is Absorbed, Broken Down, and Removed From the Body and the Extent to Which Tebapivat · Phase 1 · completed
NCT06648824 — Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants · Phase 1 · completed
NCT06286033 — Single and Multiple Ascending Dose Study and Food Effect Study for AG181 · Phase 1 · completed
NCT05610657 — A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Contro · Phase 1 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04964323 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Agios Pharmaceuticals, Inc.
Last refreshed: 7 September 2023

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04964323.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing