Topotecan Episcleral Plaque for Treatment of Retinoblastoma

Titre officiel

A Phase I Study of Sequestered Transscleral, Controlled-Release Topotecan Delivered From an Episcleral Reservoir in Retinoblastoma Eyes

Sommaire:

Cet essai clinique de phase I à groupe unique, à répartition non aléatoire et à dose croissante, évaluera principalement l’innocuité et secondairement l’efficacité du topotécan épiscléral chez les patients atteints d’un rétinoblastome intraoculaire actif de novo ou récurrent dans au moins un œil après la fin du traitement de première intention.

Description de l'essai

Primary Outcome:

• To determine the safety and tolerability of Episcleral Topotecan in participants with retinoblastoma-Maximum Tolerated Dose (MTD) and/or Recommended Phase II Dose (RP2D).

Secondary Outcome:

• To determine systemic exposure by measurement of Topotecan in plasma.
• To preliminarily define the antitumour activity as determine by assessments of tumour reponse;

Retinoblastoma is the most common pediatric malignant intraocular tumour and originates from the retina. Treatment of eyes with advanced intraocular retinoblastoma remains a challenge. The historic standard of care for patients with unilateral disease is enucleation and for those with bilateral disease, a variety of modalities have been tried. These include radiation therapy, systemic chemotherapy, periocular administration of chemotherapy, selective intra-arterial chemotherapy, and intravitreal chemotherapy. Unfortunately, all of these modalities are associated with significant morbidity and investigators are looking for new ways to treat these patients either with novel directed drug delivery methods or with new less toxic agents. This study will evaluate the safety and efficacy of topotecan delivered directly to the eye using a novel sustained-release topotecan episcleral delivery system (also referred to as a Chemoplaque) in patients with active de novo or recurrent intraocular retinoblastoma in at least one eye following completion of first-line therapy. The study intervention involves the insertion and removal of the Chemoplaque, examinations under anaesthesia (EUAs), visits to clinic to monitor for adverse events throughout, and post plaque removal toxicity evaluation. EUAs, clinic visits and laboratory tests are standard of care for retinoblastoma patients.

Voir cet essai sur ClinicalTrials.gov