Evidence map›Paper›PMID 36462055›Full record

Trial reportJournal of neurology2023

Double blind trial of a deuterated form of linoleic acid (RT001) in Friedreich ataxia.

David R Lynch, Katherine D Mathews, Susan Perlman, Theresa Zesiewicz, Sub Subramony, Omid Omidvar, Adam P Vogel, Ana Krtolica, Nadia Litterman, Lex van der Ploeg and 3 more

Abstract readRandomized Controlled Trial
PubMed Publisher
In one paragraph

Trial report in Journal of neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
1.2field-weighted citation impact, top 23% of its field
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

8 citing papers in PubMed, 12 citations in OpenAlex.

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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 at 7 institutions in 2 countries.

David R LynchPerelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 19104, USA. lynchd@pennmedicine.upenn.edu.ORCID http://orcid.org/0000-0001-7168-214X
Katherine D MathewsDepartments of Pediatrics and Neurology, University of Iowa Carver College of Medicine, Iowa City, USA.
Susan PerlmanUniversity of California Los Angeles, Los Angeles, USA.
Theresa ZesiewiczUSF Ataxia Research Center, University of South Florida, James A. Haley Veteran's Hospital, Tampa, FL, USA.
Sub SubramonyNorman Fixel Center for Neurological Disorders, University of Florida College of Medicine, Gainesville, USA.
Omid OmidvarUniversity of California Los Angeles, Los Angeles, USA.
Adam P VogelUniversity of Melbourne, Parkville, Australia.
Ana KrtolicaRetrotope, Los Altos, CA, USA.
Nadia LittermanRetrotope, Los Altos, CA, USA.
Lex van der PloegRetrotope, Los Altos, CA, USA.
Frederic HeerinckxRetrotope, Los Altos, CA, USA.
Peter MilnerRetrotope, Los Altos, CA, USA.
Mark MideiRetrotope, Los Altos, CA, USA.
Retrotope (United States) · USUniversity of California, Los Angeles · USChildren's Hospital of Philadelphia · USThe University of Melbourne · AUUniversity of Florida · USUniversity of Iowa · USUniversity of South Florida · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectivesFriedreich ataxia is (FRDA) an autosomal recessive neurodegenerative disorder associated with intrinsic oxidative damage, suggesting that decreasing lipid peroxidation (LPO) might ameliorate disease progression. The present study tested the ability of RT001, a deuterated form of linoleic acid (D2-LA), to alter disease severity in patients with FRDA in a double-blind placebo-controlled trial.

methodsSixty-five subjects were recruited across six sites and received either placebo or active drug for an 11-month study. Subjects were evaluated at 0, 4, 9, and 11 months, with the primary outcome measure being maximum oxygen consumption (MVO2) during cardiopulmonary exercise testing (CPET). A key secondary outcome measure was a composite statistical test using results from the timed 1-min walk (T1MW), peak workload, and MVO2.

resultsForty-five subjects completed the protocol. RT001 was well tolerated, with no serious adverse events related to drug. Plasma and red blood cell (RBC) membrane levels of D2-LA and its primary metabolite deuterated arachidonic acid (D2-AA) achieved steady-state concentrations by 4 months. No significant changes in MVO2 were observed for RT001 compared to placebo. Similarly, no differences between the groups were found in secondary or exploratory outcome measures. Post hoc evaluations also suggested minimal effects of RT001 at the dosages used in this study. INTERPRETATIONS: The results of this study provide no evidence for a significant benefit of RT001 at the dosages tested in this Friedreich ataxia patient population.

Indexed as

Friedreich AtaxiaLinoleic AcidDouble-Blind MethodHumansLinoleic AcidsWalkingLinoleic AcidLinoleic AcidsRT001Clinical trialDeuteriumFerroptosisMitochondrion

Identifiers

PMID36462055
OpenAlexW4310640719

What OpenQuestion holds

Textmetadata
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.