Valproate for the Treatment of Residual Amblyopia

Part of paid clinical trials in Waltham, Massachusetts.

Sponsor
Boston Children's Hospital
Study ID
NCT07226141
Phase
PHASE1/PHASE2
Status
Not Yet Recruiting

Notify me when recruiting opens

Save your spot on the interest list for this study. We'll keep your details with this study so our team can follow up when recruiting opens.

Not yet recruiting

Add your contact details and location so we can keep your interest tied to this study.

Conditions

  • Amblyopia
  • Amblyopia Strabismic
  • Amblyopia Unilateral
  • Amblyopia, Anisometropic

Eligibility Criteria

Sex
ALL
Age
8 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • 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.

Study Details

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.

Key Dates

Start date
Jan 1, 2026
Status verified
Sep 2025
Primary completion
Sep 30, 2027
Completion
Sep 30, 2027

Study Design

Enrollment
28 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Placebo Comparator: Arm 1 - Placebo first
    Participants randomized to Arm 1 will receive oral placebo (250 mg twice daily) in combination with 2 hours of prescribed daily patching for 8 weeks. At the 8-week visit, participants will cross over to oral valproate at an initial dose of 15 mg/kg/day divided twice daily, while continuing the prescribed 2 hours of daily patching for an additional 8 weeks. Dose escalation of valproate up to 30 mg/kg/day (or placebo to 500 mg mice daily) will be permitted at interim visits if visual acuity has not improved and no adverse effects are present. All study medication will be discontinued after 16 weeks, with patching also discontinued at that time.
  • Experimental: Arm 2 - Valproate first
    Participants randomized to Arm 2 will receive oral valproate at an initial dose of 15 mg/kg/day divided twice daily, together with 2 hours of prescribed daily patching for 8 weeks. At the 8-week visit, participants will cross over to oral placebo (250 mg twice daily) while continuing 2 hours of daily patching for an additional 8 weeks. If visual acuity has not improved at interim visits and no adverse effects are observed, the valproate dose may be escalated up to 30 mg/kg/day during the initial treatment phase, or placebo increased to 500 mg twice daily during the cross-over phase. After 16 weeks, both study medication and patching will be discontinued.

Primary Outcome Measure

Amblyopic Eye Best-Corrected Visual Acuity (BCVA) [ Time Frame: The comparison will be made between the randomized groups at the 8-week visit, prior to cross-over. ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Boston Children's WalthamWalthamMassachusetts02453
Eric Gaier, MD
[email protected]
617-355-6401
Peter Casey-Caplan, BA
[email protected]
Eric Gaier, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Waltham, MA

By research site
Boston Children's Waltham· Waltham, MA

Related Studies