Evidence map›Paper›PMID 31669619›Full record

ReviewMitochondrion2020

The changing landscape of clinical trials for mitochondrial diseases: 2011 to present.

Delia Khayat, Tracie L Kurtz, Peter W Stacpoole

Abstract readReview
In one paragraph

Review in Mitochondrion, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

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

  1. Pooled it
  2. Review
  3. Article
  4. Review
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

3 authors.

Delia KhayatDepartments of Medicine (Division of Endocrinology, Diabetes and Metabolism), College of Medicine, University of Florida, United States.
Tracie L KurtzDepartments of Medicine (Division of Endocrinology, Diabetes and Metabolism), College of Medicine, University of Florida, United States.
Peter W StacpooleDepartments of Medicine (Division of Endocrinology, Diabetes and Metabolism), College of Medicine, University of Florida, United States; Biochemistry and Molecular Biology, College of Medicine, University of Florida, United States. Electronic address: pws@ufl.edu.

Funding

Personalized dosing of dichloroacetate for the treatment of rare and common diseasesR42HD089804 · NICHD · MEDOSOME BIOTEC, LLC · PI STACPOOLE, PETER WALLACE · 2016 to 2022
$4.7M
Phase 3 Trial of DCA in PDC Deficiency IND 028,625 (02/04/2015)R01FD005407 · FDA · UNIVERSITY OF FLORIDA · PI STACPOOLE, PETER WALLACE, THOMPSON, JOHN L · 2016 to 2022
$2.0M
FDA HHS R01 FD005407NICHD NIH HHS R42 HD089804
6 · The paper itself

Abstract

We reviewed the status of interventional clinical trials for primary mitochondrial diseases. Using national and international search engines, we found 48 randomized controlled trials (RCTs) registered as of May 15, 2019. Consilience between lay and professional mitochondrial disease communities to engage in RCTs has increased, as has progress in developing new disease and treatment biomarkers and potential therapies. The continued advancement of general knowledge of mitochondrial biology has fostered appreciation for the fundamental role mitochondria play in the etiopathology of other rare and common illnesses, emphasizing the therapeutic potential of mitochondrially-targeted small molecules for an increasing spectrum of human diseases.

Indexed as

Genetic TherapyOrphan Drug ProductionHumansMitochondrial DiseasesRare DiseasesResearch Design

Identifiers

PMID31669619
PMCPMC6934893

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

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.