Evidence map›Paper›PMID 40978076›Full record

ArticleJournal of multidisciplinary healthcare2025

Clinical Research for Inherited Retinal Disease Related Pediatric Blindness: A Preliminary Descriptive Analysis Based on ClinicalTrials.gov.

Ahmed M Ashour, Maan H Harbi, Fahad S Alshehri, Saad M Wali, Mohammed M Aldurdunji, Nasser M Alorfi

Abstract read
In one paragraph

Article in Journal of multidisciplinary healthcare, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–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

1 citing paper in PubMed.

  1. Article
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.

Ahmed M AshourDepartment of Pharmacology and Toxicology, College of Pharmacy, Umm Al-Qura University, Makkah, Saudi Arabia.ORCID 0000-0002-2118-0499
Maan H HarbiDepartment of Pharmacology and Toxicology, College of Pharmacy, Umm Al-Qura University, Makkah, Saudi Arabia.ORCID 0000-0002-8892-5192
Fahad S AlshehriDepartment of Pharmacology and Toxicology, College of Pharmacy, Umm Al-Qura University, Makkah, Saudi Arabia.ORCID 0000-0001-6966-0128
Saad M WaliDepartment of Pharmacology and Toxicology, College of Pharmacy, Umm Al-Qura University, Makkah, Saudi Arabia.
Mohammed M AldurdunjiPharmaceutical Practices Department, College of Pharmacy, Umm Al-Qura University, Makkah, Saudi Arabia.ORCID 0000-0002-1134-4138
Nasser M AlorfiDepartment of Pharmacology and Toxicology, College of Pharmacy, Umm Al-Qura University, Makkah, Saudi Arabia.ORCID 0000-0002-0636-7685

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Childhood blindness is a significant global health concern, consistently identified in existing research as stemming from rare genetic and congenital disorders. With the technological advances of the 21 Methods: A cross-sectional analysis was conducted using data from ClinicalTrials.gov. The initial search for data in ClinicalTrials.gov yielded a total of 110 studies under blindness-related conditions. Upon further cross-examination of these studies based on the inclusion criteria, only five interventional trials (published between 2012 and 2023) specifically targeting childhood blindness met the inclusion criteria and were therefore included. Key trial characteristics studied conditions, intervention types, outcome measures, study phases, and enrollment sizes were extracted and analyzed descriptively. Results: Across the five included trials, a majority of trials investigated rare genetic conditions, including Leber Congenital Amaurosis, Wolfram Syndrome, and Osteoporosis Pseudoglioma. On interventions, the most commonly used approaches for handling childhood blindness include gene therapy vectors like AAV RPE65, antisense oligonucleotides like QR-110, and repurposed pharmacological agents such as lithium and dantrolene sodium. All studies included children within their target populations, and most were early-phase (Phase 1/2) trials with small sample sizes (11-26 participants). Primary outcomes focused on safety, while secondary outcomes assessed visual function and biochemical changes. Conclusion: Although limited in number, current clinical trials represent a promising shift toward targeted therapies for childhood blindness. The dominance of early-phase studies highlights the need for expanded, multicenter, and later-phase trials. Future research should aim to improve trial accessibility, standardize outcome measures, and ensure ethical conduct in pediatric populations.

Indexed as

achromatopsiachildhood blindnessclinical trialsgene therapyLeber Congenital AmaurosisLeber hereditary optic neuropathypediatric ophthalmologyUsher syndromeX-linked retinitis pigmentosa

Identifiers

PMID40978076
PMCPMC12449873

What Socratic holds

Textmetadata
LicenceCC BY-NC
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.