Health TrialsbyHealthPanel

Valproate for the Treatment of Residual Amblyopia

ConditionsAmblyopiaAmblyopia UnilateralAmblyopia, AnisometropicAmblyopia Strabismic
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age8-17
SponsorBoston Children's Hospital

About this trial

The goal of this clinical trial is to determine the efficacy of valproate as an adjunct therapy to treat amblyopia beyond the critical period in children aged 8-17 years who have amblyopia of ≥3 lines of interocular best-corrected (with glasses) visual acuity difference.

The main questions it aims to answer are:

* Does valproate enable clinically meaningful and durable visual recovery from amblyopia? * Do valproate-treated patients show a change in amblyopic eye visual acuity (lines)? Participants will undergo daily patching for 2 hours (standard of care) plus the addition of valproate or placebo for a total of 16 weeks.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age 8-17 years

Amblyopia associated with strabismus and/or anisometropia

Heterotropia at distance and/or near with or without spectacle correction

History of strabismus surgery

Disqualifiers

None

Trial design

Design model

Crossover

Treatments tested in this trial

  • Valproate

    Drug

    Valproate is an anti-epileptic medication used in this study to treat amblyopia. Participants will receive valproate in oral tablet form. The dosage will be determined based on the participant's weight and age, following standard dosing guidelines for valproate. The medication will be administered daily for a duration of 8 weeks. Participants will be monitored for any adverse effects, and dosage adjustments will be made if necessary to ensure safety and tolerability.

  • Placebo

    Drug

    The placebo intervention consists of an inert substance designed to mimic the appearance and administration of valproate tablets. Participants in the placebo group will receive the placebo tablets daily for the same duration of 8 weeks. This control group will help to assess the efficacy of valproate by comparing outcomes between the valproate and placebo groups. Participants receiving the placebo will also be monitored for any adverse effects to ensure the study's integrity and participant safety.

  • Patch

    Behavioral

    Patching is a standard treatment for amblyopia, involving the occlusion of the fellow eye to stimulate the amblyopic eye. Participants will be required to patch their fellow eye for 2 hours daily throughout the 16-week study period. The patching regimen aims to improve visual acuity in the amblyopic eye by encouraging its use. Compliance with the patching protocol will be monitored, and participants will be provided with instructions and support to ensure proper application and adherence to the treatment.

Treatment groups

28 Participants
are divided into 2 treatment groups
Group A: Arm 1 - Placebo firstPlacebo comparator 3 interventions
Group B: Arm 2 - Valproate firstExperimental treatment 3 interventions

Trial outcomes

Primary outcomes

1

Amblyopic Eye Best-Corrected Visual Acuity (BCVA)

Change in visual acuity of the amblyopic eye after 8 weeks of treatment with valproate plus patching versus placebo plus patching.

Time frame
The comparison will be made between the randomized groups at the 8-week visit, prior to cross-over.

Secondary outcomes

1

Proportion of patients with resolved amblyopia

Proportion of participants achieving resolution of amblyopia, defined as visual acuity in the amblyopic eye of 20/25 or better, following 8 weeks of treatment.

Time frame
8 weeks
2

Durability of amblyopic eye visual acuity response

Among those in the study arm receiving valproate followed by placebo, the visual acuity in the amblyopic eye will be compared between the 8 week visit (when valproate is discontinued) to the 16 week visit (after completion of placebo). This difference reflects the maintenance of visual acuity and will be analyzed and interpreted in the context of change at an individual level in response to valproate between weeks 0 and 8. The proportion of subjects from the valproate then placebo group maintaining a ≥ 10 letters (2 lines) improvement in the amblyopic eye visual acuity will be calculated and a 95% confidence interval on overall change will be computed.

Time frame
8 weeks
3

Change in stereoacuity

Change in log arcsec in stereoacuity assessed with the Randot Preschool Stereoacuity Test. The change in log arcseconds will be compared between groups. Aggregated results will inform how overall stereoacuity has changed, and the difference from baseline will be compared across treatment groups and timepoints.

Time frame
8 weeks and 16 weeks
4

Quality of life measures using the Pediatric Eye Questionnaire (PedEyeQ)

This measure assesses the quality of life deficits across four domains: functional vision, bothered by eyes/vision, social, and frustration/worry. It uses a 3-point frequency scale and Rasch-calibrated domain scores (0-100).

Time frame
Change from baseline at 8 weeks and 16 weeks.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Boston Children's Hospital

Lead sponsor

Source ClinicalTrials.gov
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