Evidence map›Paper›PMID 30911578›Full record

Trial reportAnnals of clinical and translational neurology2019

Randomized, double-blind, placebo-controlled study of interferon-

David R Lynch, Lauren Hauser, Ashley McCormick, McKenzie Wells, Yi Na Dong, Shana McCormack, Kim Schadt, Susan Perlman, Sub H Subramony, Katherine D Mathews and 7 more

Open access · goldAbstract readMulticenter StudyRandomized Controlled Trial
In one paragraph

Trial report in Annals of clinical and translational neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed, 1 pooled it
2.5field-weighted citation impact, top 11% 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

27 citing papers in PubMed, 1 synthesis or guideline pooled it, 43 citations in OpenAlex.

  1. Pooled it
  2. Review
  3. Review
  4. Article
  5. Article
  6. Review
  7. Review
  8. Review
  9. Article
  10. Article
  11. Article
  12. Review
  13. Article
  14. Review
  15. Friedreich ataxia: clinical features and new developments.Neurodegenerative disease management · 2022
    Review
  16. Review
  17. Review
  18. Drug Repositioning in Friedreich Ataxia.Frontiers in neuroscience · 2022
    Review
  19. Review
  20. Emerging therapies in Friedreich's Ataxia.Expert review of neurotherapeutics · 2020
    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

17 authors at 7 institutions in 1 country.

David R LynchDivision of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318.
Lauren HauserDivision of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318.
Ashley McCormickDivision of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318.
McKenzie WellsDivision of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318.
Yi Na DongDivision of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318.
Shana McCormackDivision of Endocrinology & Diabetes Children's Hospital of Philadelphia Philadelphia 19104.ORCID 0000-0002-1143-4459
Kim SchadtDivision of Neurology Children's Hospital of Philadelphia 502 Abramson Research Center, 3615 Civic Center Blvd Philadelphia Pennsylvania 19104-4318.
Susan PerlmanDepartment of Neurology University of California Los Angeles Box 956975, 1-167 RNRC Los Angeles California 90095.
Sub H SubramonyDepartment of Neurology University of Florida Room L3-100, McKnight Brain Institute, 1149 Newell Drive Gainesville Florida 32611.
Katherine D MathewsDepartment of Pediatrics and Neurology University of Iowa Carver College of Medicine Iowa City Iowa.
Alicia BrochtDepartment of Neurology University of Rochester Rochester New York 14620.
Julie BallHorizon Pharma, Inc. Lake Forest Illinois 60045.
Renee PerdokHorizon Pharma, Inc. Lake Forest Illinois 60045.
Amy GrahnHorizon Pharma, Inc. Lake Forest Illinois 60045.
Tom VescioHorizon Pharma, Inc. Lake Forest Illinois 60045.
Jeffrey W ShermanHorizon Pharma, Inc. Lake Forest Illinois 60045.
Jennifer M FarmerFriedreich's Ataxia Research Alliance 533 W Uwchlan Ave Downingtown Pennsylvania 19335.
Children's Hospital of Philadelphia · USHorizon Therapeutics (United States) · USFriedreich's Ataxia Research Alliance · USUniversity of California, Los Angeles · USUniversity of Florida · USUniversity of Iowa · USUniversity of Rochester · US

Funding

Therapeutic Potential of ESC-Derived Myogenic PrecursorsU54NS053672 · NINDS · UNIVERSITY OF IOWA · PI CAMPBELL, KEVIN P. · 2005 to 2019
$22.0M
NINDS NIH HHS U54 NS053672
6 · The paper itself

Abstract

Objective: In vitro, in vivo, and open-label studies suggest that interferon gamma (IFN- Methods: Ninety-two subjects with FRDA between 10 and 25 years of age were enrolled. Subjects received either IFN- Results: No difference was noted between the groups after 6 months of treatment in the mFARS or secondary outcome measures. No change was noted in buccal cell or whole blood frataxin levels. However, during an open-label extension period, subjects had a more stable course than expected based on natural history data. Conclusions: This study provides no direct evidence for a beneficial effect of IFN-

Indexed as

AdolescentAdultChildDouble-Blind MethodFemaleFrataxinFriedreich AtaxiaHumansInterferon-gammaIron-Binding ProteinsMaleRecombinant ProteinsTreatment OutcomeYoung AdultFrataxinInterferon-gammainterferon gamma-1bIron-Binding ProteinsRecombinant Proteins

Identifiers

PMID30911578
PMCPMC6414489
OpenAlexW2917106030

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.