Evidence map›Paper›PMID 28869486›Full record

Trial reportJournal of neuromuscular diseases

Efficacy of Idebenone to Preserve Respiratory Function above Clinically Meaningful Thresholds for Forced Vital Capacity (FVC) in Patients with Duchenne Muscular Dystrophy.

Oscar H Mayer, Mika Leinonen, Christian Rummey, Thomas Meier, Gunnar M Buyse, DELOS Study Group

Registry-linked trialOpen access · bronzeAbstract readClinical Trial, Phase IIIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of neuromuscular diseases. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03603288 (A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy), which is not on this map. Cited by 18 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed, 3 pooled it
3.1field-weighted citation impact, top 8% 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.

NCT03603288 phase3terminatednot on this map

A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study

TypeinterventionalSponsorSanthera PharmaceuticalsRan2018 to 2020Enrolled161ConditionsDuchenne Muscular DystrophyArmsidebenone 150 mg film-coated tablets
3 · Its place in the literature

Who cites it

18 citing papers in PubMed, 3 syntheses or guidelines pooled it, 33 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

6 authors at 2 institutions in 2 countries.

Oscar H MayerThe Children's Hospital of Philadelphia, Philadelphia, USA.
Mika LeinonenSanthera Pharmaceuticals, Liestal, Switzerland.
Christian RummeyClinical Data Science, Basel, Switzerland.
Thomas MeierSanthera Pharmaceuticals, Liestal, Switzerland.
Gunnar M BuyseUniversity Hospitals Leuven, Leuven, Belgium.
DELOS Study Group
Santhera Pharmaceuticals (Switzerland) · CHChildren's Hospital of Philadelphia · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPatients with DMD experience progressive restrictive respiratory disease and eventual respiratory failure. Standard of care guidelines command changes in disease management when forced vital capacity percent of predicted (FVC% p) falls below clinically relevant thresholds. The Phase 3 DELOS trial in patients with DMD demonstrated that idebenone reduces the loss of peak expiratory flow and FVC compared to placebo (Buyse GM, et al.; Lancet 2015; 385 : 1748-57).

objectivePost-hoc analyses were conducted to assess whether treatment with idebenone could reduce the risk of patients dropping below clinically meaningful thresholds of FVC% p.

methodsThe DELOS trial enrolled DMD patients 10-18 years of age not using glucocorticoids to receive idebenone (N = 31) or placebo (N = 33) for 12 months. Change from baseline in FVC and FVC% p was assessed by hospital spirometry and analyzed by mixed model of repeated measures and slope analysis and proportions of patients falling below clinically meaningful thresholds of FVC% p were compared.

resultsThe change over 1 year in FVC and FVC% p showed a consistent pattern in favor of idebenone treatment across multiple analysis methods and fewer patients in the idebenone group declined by a margin of 10% or more in FVC and FVC% p compared to placebo. There were also fewer patients in the idebenone group (15%) with a decline below FVC% p of 50% compared to the placebo group (25%) and fewer patients in the idebenone group (28%) showed a decline below FVC% p of 50% or 40% or 30% compared to the placebo group (43%).

conclusionsThese data added to the consistency and clinical meaningfulness of findings from the DELOS trial showing that idebenone can slow the loss of pulmonary function in patients with DMD.

Indexed as

AdolescentAntioxidantsChildHumansMaleMuscular Dystrophy, DuchenneRespirationUbiquinoneVital CapacityAntioxidantsidebenoneUbiquinoneDuchenne muscular dystrophyforced vital capacityidebenonePulmonary functionrespiration

Identifiers

PMID28869486
PMCPMC5611828
OpenAlexW2750730558

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.