The Safety and Efficacy of Intravitreal Topotecan for the Treatment of Proliferative Vitreoretinopathy

November 24, 2025 updated by: Leo Am Kim, M.D., Massachusetts Eye and Ear Infirmary

The goal of this clinical trial is to evaluate the safety and efficacy of intravitreal topotecan for the treatment of patients with rhegmatogenous retinal detachment due to proliferative vitreoretinopathy (PVR) or resulting from an open globe injury, and compare the outcomes to those who do no receive intravitreal topotecan. The main objectives it aims to achieve are:

  • to study the safety profile of intravitreal topotecan in the treatment of PVR
  • to evaluate the efficacy of intravitreal topotecan in treating PVR.

Post-consent, participants will:

  • undergo vitrectomy (with or without scleral buckle) as part of standard treatment for retinal detachment.
  • receive intravitreal topotecan at the time of surgery, post-operative day 7 and post-operative day 28 (if randomized to receive the medication)
  • come in at post-operative day 1, 7, 28, 56, 84, 126 and 168 to undergo a complete ophthalmic exam along with a fundus photography and optical coherence tomography of the macula, have their intraocular pressure and visual acuity measured and their adverse events monitored, if any.

Researchers will compare participants who receive intravitreal topotecan for PVR to those who do not to see if there is a significant variability in recurrence of retinal detachment, rate of retinal reattachment and PVR grade 6 months after surgery.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Rhegmatogenous retinal detachment (RRD) is an acute, sight-threatening condition that occurs after separation of the neurosensory retina from the underlying retinal pigment epithelium (RPE) due to the presence of a retinal break. Proliferative vitreoretinopathy (PVR) represents growth of ectopic fibrocellular growth on the surface of, within and underneath the retina. PVR is hypothesized to occur secondary to the migration of RPE cells to the peri-retina, leading to a mesenchymal transition into contractile myofibroblasts. PVR affects 5-10% of RRDs and is the most common cause of surgical failure in RRD.

Given that PVR involves a pro-inflammatory and pro-fibrotic cellular response, adjuvants such as corticosteroids and antimetabolites such as 5-fluorouracil have been attempted in the treatment of this condition. Overall, the efficacy results of these treatments have been mixed, and no standard of care adjuvant therapy has emerged.

Topotecan is a chemotherapeutic agent that acts as a topoisomerase inhibitor. It is most commonly administered intravitreally for patients with refractory or recurrent vitreous seeds from retinoblastoma, and shows high efficacy in this setting. At the same time, intravitreal topotecan administered at doses of 5-30µg has been associated with no ocular or systemic complications or adverse electroretinogram changes. To the best of available knowledge, topotecan has not been administered for the treatment of proliferative vitreoretinopathy. Topotecan has anti-inflammatory, anti-proliferative and anti-fibrotic activity that is hypothesized to exhibit high efficacy for the treatment of PVR. In this study, the efficacy and safety of intravitreal topotecan for the treatment of PVR will be investigated.

Study Type

Interventional

Enrollment (Estimated)

50

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  • Patients > 18 years old
  • Patients presenting with retinal detachment due with PVR (grade C or higher) or retinal detachment associated with open globe trauma
  • Patients undergoing vitrectomy or vitrectomy with scleral buckle as part of standard care.

Exclusion Criteria:

  • Patient unable to give consent
  • Patient unable to follow-up
  • Females of childbearing age who are pregnant at the time of recruitment. A pregnancy test will be done to all women of ages 18-55 prior to surgery to ensure they are not pregnant at the time of recruitment.
  • Patients with a history of tractional or exudative retinal detachment.
  • Patients with other planned ocular surgery following PPV
  • Active or chronic or recurrent uncontrolled ocular or systemic disease
  • Active or history of chronic or recurrent inflammatory eye disease
  • Diagnosis of severe nonproliferative or proliferative diabetic retinopathy or vasoproliferative disease in the operative eye
  • Signs of ocular infection at presentation in either eye
  • Known or suspected sensitivity or allergy to any of the medications used in the operation or postoperatively
  • No Light Perception vision in the operative eye
  • Failure to achieve intraoperative reattachment
  • Patient with silicone oil instilled in the operative eye at time of presentation

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Participants who received intravitreal topotecan
These are the patients who will be receiving intravitreally 20 micrograms of topotecan in a 1cc syringe during surgery, at the post-operative day 7 and at the post-operative day 28.
Drug: Topotecan
20 micrograms of intravitreal topotecan given in a 1 cc tuberculin syringe at a concentration of 20 mcg/20mcL.
Other Names:
  • Hycamtin
No Intervention: Participants who did not received intravitreal topotecan
These patients will not be receiving any intervention.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Recurrence of rhegmatogenous retinal detachment secondary to PVR
Time Frame: 6 months after initial surgery, or last follow-up visit available
Investigators will be evaluating whether participant develop a recurrent retinal detachment throughout their follow up after their initial surgery.
6 months after initial surgery, or last follow-up visit available

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Best corrected visual acuity (BCVA)
Time Frame: at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Investigators will be measuring the BCVA of participants throughout their follow up and see if they notice any change from surgery time to final follow up
at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Variation of PVR grade
Time Frame: at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Investigators will be doing a dilated fundus exam and taking fundus photographies for all participants throughout their follow up time to see if the grade of the PVR changes over time. PVR grade goes from grade A to grade C-Anterior to equator/ C-Posterior to equator (grade C being worse than grade A)
at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Retinal reattachment rate at month 6 or last follow up
Time Frame: at post-operative day 168 (or last follow-up visit available if the participant did not show up at post-operative day 168)
Investigators will be doing a dilated fundus exam and taking fundus photographies for all participants throughout their follow up time to see if the retinal reattaches (whether completely or partially) after surgery
at post-operative day 168 (or last follow-up visit available if the participant did not show up at post-operative day 168)
Recurrence of rhegmatogenous retinal detachment due to any cause
Time Frame: at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Investigators will be measuring the BCVA of participants throughout their follow up and see if they notice any change from surgery time to final follow up
at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Number of participants and type of intraoperative or postoperative complications
Time Frame: at time of surgery, and each follow up visit (post-operative day1, 7, 28, 56, 84, 126, 168)
Any adverse event will be noted during surgery, and at each follow up visit for each participant if applicable.
at time of surgery, and each follow up visit (post-operative day1, 7, 28, 56, 84, 126, 168)

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Optical coherence tomography (OCT) of the macula
Time Frame: at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
an OCT of the macula will be taken to evaluate for any retinal changes throughout the study
at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Massachusetts Eye and Ear Infirmary

Investigators

  • Principal Investigator: Leo Kim, MD, PhD, Massachusetts Eye and Ear

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 1, 2026

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

March 1, 2027

Study Registration Dates

First Submitted

January 9, 2024

First Submitted That Met QC Criteria

May 20, 2024

First Posted (Actual)

May 22, 2024

Study Record Updates

Last Update Posted (Actual)

December 2, 2025

Last Update Submitted That Met QC Criteria

November 24, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2023P002259

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Rhegmatogenous Retinal Detachment

Clinical Trials on Topotecan

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Netherlands

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe