Evidence map›Paper›PMID 30747463›Full record

ArticleJournal of intellectual disability research : JIDR2019

Fragile X syndrome clinical trials: exploring parental decision-making.

C S D'Amanda, H L Peay, A C Wheeler, E Turbitt, B B Biesecker

Open access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of intellectual disability research : JIDR, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 1 pooled it
1.6field-weighted citation impact, top 16% 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

9 citing papers in PubMed, 1 synthesis or guideline pooled it, 22 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Determinants of Infant Participation and Non-Participation in Primary Prevention Trials.Hormone and metabolic research = Hormon- und Stoffwechselforschung = Hormones et metabolisme · 2026
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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

5 authors at 2 institutions in 1 country.

C S D'AmandaSocial and Behavioral Research Branch, National Human Genome Research Institute, Bethesda, MD, USA.ORCID 0000-0001-5443-5040
H L PeayCenter for Newborn Screening, Ethics, and Disability Studies, RTI International, Research Triangle Park, NC, USA.ORCID 0000-0002-3053-7453
A C WheelerCenter for Newborn Screening, Ethics, and Disability Studies, RTI International, Research Triangle Park, NC, USA.ORCID 0000-0003-2693-5616
E TurbittSocial and Behavioral Research Branch, National Human Genome Research Institute, Bethesda, MD, USA.ORCID 0000-0002-6650-9702
B B BieseckerSocial and Behavioral Research Branch, National Human Genome Research Institute, Bethesda, MD, USA.ORCID 0000-0001-9665-8963
National Human Genome Research Institute · USRTI International · US

Funding

Interventions in Genetic CounselingZIAHG200317 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI BIESECKER, BARBARA BOWLES · 2009 to 2018
$7.6M
Intramural NIH HHS ZIA HG200317
6 · The paper itself

Abstract

backgroundThe objective of this research was to understand parental proxy decision-making for drug trial participation for children with Fragile X syndrome (FXS). Specifically, we aimed to capture preferences, motivations, influencing factors and barriers related to trial involvement among trial joiners and decliners and describe ease of trial decision-making and decisional regret.

methodsInterviews were conducted with parents from two groups: those who chose to enrol their child with FXS in a trial (N = 16; Joiners) and those who declined trial participation (N = 15; Decliners). Data were coded and interpreted through inductive content analysis.

resultsProminent decisional factors included attitudes about medicating FXS symptoms, potential for direct benefit (primarily evaluated through the degree of match between target outcomes and child symptomatology and drug mechanism), logistical convenience and perceived risks of side effects. The ultimate motivation for participation was potential for direct benefit. None of the parents reported decisional regret, and ease of decision-making ranged from easy to difficult for our participants.

conclusionsTherapeutic optimism was high among those who elected participation. Parents may benefit from an explanation of the rationale behind chosen outcome variables and may be more interested in trials that target or measure as an exploratory outcome the symptoms they find most concerning. Our findings reinforce the need for future trials to reduce participant inconvenience. Our results contrast with what has previously been observed in parents of children with life-threatening conditions; parents of children with FXS may be more trial risk averse and find trial decisions to be harder. Parents of children with FXS considering trials may benefit from a decisional intervention aimed at deliberating motivations and barriers.

Indexed as

Clinical Trials as TopicDecision MakingHealth Knowledge, Attitudes, PracticeMotivationParentsPatient Acceptance of Health CareAdultChildFemaleFragile X SyndromeHumansMaleQualitative Researchclinical trialsdecisional factorsFragile X syndromeparentsproxy decision-making

Identifiers

PMID30747463
PMCPMC6639141
OpenAlexW2911619051

What Socratic 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 Socratic graph.