Evidence map›Paper›PMID 39079928›Full record

ArticleBMJ open2024

Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias - pilot study protocol.

Andreas Glenthøj, Eduard J van Beers, Richard van Wijk, Minke A E Rab, Evelyn Groot, Niels Vejlstrup, Nina Toft, Selma Kofoed Bendtsen, Jesper Petersen, Jens Helby and 3 more

Registry-linked trialAbstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in BMJ open, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05935202 (Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies), which is not on this map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT05935202 phase2not yet recruitingnot on this map

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies

TypeinterventionalSponsorEuroBloodNet AssociationRan2023 to 2026Enrolled25ConditionsHereditary Red Blood Cell Disorder (Disorder)ArmsMitapivat sulfate
3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors.

Andreas GlenthøjDepartment of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark andreas.glenthoej@regionh.dk.ORCID 0000-0003-2082-0738
Eduard J van BeersBenign Hematology Center, Van Creveldkliniek, University Medical Centre Utrecht, Utrecht, Netherlands.
Richard van WijkCentral Diagnostic Laboratory - Research, Division of Laboratories, Pharmacy and Biomedical Genetics, University Medical Center Utrecht, Urecht, Netherlands.
Minke A E RabCentral Diagnostic Laboratory - Research, Division of Laboratories, Pharmacy and Biomedical Genetics, University Medical Center Utrecht, Urecht, Netherlands.
Evelyn GrootBenign Hematology Center, Van Creveldkliniek, University Medical Centre Utrecht, Utrecht, Netherlands.
Niels VejlstrupDepartment of Cardiology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Nina ToftDepartment of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Selma Kofoed BendtsenDepartment of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Jesper PetersenDepartment of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Jens HelbyDepartment of Hematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark.
Fatiha ChermatEuroBloodNet Association, Université Paris Cité Faculté de Santé, Paris, France.
Pierre FenauxEuroBloodNet Association, Université Paris Cité Faculté de Santé, Paris, France.
Kevin H M KuoInstitute of Health Policy, Management and Evaluation, Dalla Lana School of Public Health, University of Toronto, Toronto, Ontario, Canada.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionMembranopathies encompass haemolytic disorders arising from genetic variants in erythrocyte membrane proteins, including hereditary spherocytosis and stomatocytosis. Congenital dyserythropoietic anaemia type II (CDA II) is associated with the METHODS AND ANALYSES: ETHICS AND DISSEMINATION: NCT05935202/CTIS:2023-503271-24-01. Findings will be published in peer-reviewed journals. TRIAL REGISTRATION NUMBER: Clinicaltrials.gov, NCT05935202. CTIS:2023-503271-24-01. Registered 07-July-2023. Protocol number: 2.1. https://clinicaltrials.gov/study/NCT05935202.

Indexed as

Pyruvate KinaseAdultAnemia, Dyserythropoietic, CongenitalAnemia, Hemolytic, Congenital NonspherocyticClinical Trials, Phase II as TopicFemaleHumansMaleMulticenter Studies as TopicPilot ProjectsProspective StudiesPyruvate Metabolism, Inborn ErrorsPyruvate KinaseAnaemiaClinical trialsHAEMATOLOGYQuality of Life

Identifiers

PMID39079928
PMCPMC11293418

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.