Methotrexate For The Prevention and Treatment of Proliferative Vitreoretinopathy in Pediatric Patients

NCT04830878 | Withdrawn Phase 1 FDA Drug

• 1 other identifier: IRB-59989
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

Rhegmatogenous retinal detachment (RRD) is a sight-threatening condition. Children with RRD usually present late with clinical features of longstanding RRD, specifically a serious condition named: proliferative vitreoretinopathy (PVR). Therefore, children with RRD often have poorer outcomes. The objective of this study is to investigate the efficacy and safety of methotrexate in the treatment and prevention of PVR. Methotrexate is a medication that has been used to treat inflammatory conditions in children and adults for a long time and it has been recently used to treat PVR in adults.

Conditions

Proliferative Vitreoretinopathy

Outcome Measures

Primary Outcomes (1)

• Recurrence of Retinal Detachment

  Number of Participants with Recurrent Retinal Detachment

  6 months

Study Arms (1)

Treatment

EXPERIMENTAL

Methotrexate treatment

Drug: Methotrexate

Interventions

Methotrexate DRUG

Systemic and intraoperative intravitreal injection of methotrexate

Treatment

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Ability to get informed consent from a parent or a legal guardian of the child and attend all study visits.
• Males or females 18 years old or younger.
• Subject is undergoing vitrectomy for either i. initial retinal detachment with or without PVR ii. recurrent retinal detachment due to grade A or higher of proliferative vitreoretinopathy.
• Female subjects of childbearing potential must not be pregnant or breast-feeding, must have a negative serum pregnancy test at screening, and must be willing to undergo pregnancy tests throughout the study.
• Female subjects of childbearing potential and male subjects able to father children must (a) abstain from intercourse throughout the course of the study or (b) agree to practice acceptable methods of contraception throughout the course of the study (i.e., intrauterine device, oral contraceptives, barrier method, or other contraception deemed adequate by the investigator).

You may not qualify if:

• Ocular or periocular infection in either eye including (but not limited to):
• History of herpetic infection in the study eye(s) or adnexa.
• Presence of known active or inactive toxoplasmosis or toxoplasmosis scar in either eye.
• History of cytomegalovirus infection or clinical evidence of active cytomegalovirus infection at screening and/or Day 1.
• Pupillary dilation inadequate for quality stereoscopic fundus photography in the study eye(s).
• Media opacity that would limit clinical visualization in the study eye(s) and, in the opinion of the investigator, could not be repaired or improved during the RD surgery.
• Other planned eye surgery during the course of the trial
• Corneal opacity in the study eye(s) that would preclude reliable assessment of the posterior segment.
• Uncontrolled glaucoma in the study eye(s), evidenced by an intraocular pressure (IOP) \> 21 mmHg while on maximum medical therapy, or chronic hypotony (unmeasurable eye pressure).
• Subjects should not be currently undergoing treatment with one of the following at the time of RD diagnosis: systemic steroids, methotrexate, azathioprine, or mycophenolate mofetil (or an equivalent drug, e.g., mycophenolic acid) or other immunomodulatory therapies.
• Malignancy in remission for less than 5 years prior to study participation.
• Allergy or hypersensitivity to investigational product or other study related procedures/medications.
• Any recent systemic infection (excluding common cold) within 30 days of Day 1.
• Known to be immunocompromised.
• History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease condition that contraindicates the use of an investigational drug, might affect the interpretation of the results of the study, or renders the subject at high risk for treatment complications.

Sponsors & Collaborators

• Stanford University: lead

Study Sites (1)

Byers Eye Institute at Stanford University and Lucille Packard Children Hospital

Palo Alto, California, 94303, United States

Location

MeSH Terms

Conditions

Vitreoretinopathy, Proliferative

Interventions

Methotrexate

Condition Hierarchy (Ancestors)

Retinal Diseases; Eye Diseases

Intervention Hierarchy (Ancestors)

Aminopterin; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Edward Wood, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

• Quan Dong Nguyen, MD, MSc

  Stanford University

  PRINCIPAL INVESTIGATOR

• Ahmad Al-Moujahed, MD, PhD, MPH

  Stanford University

  PRINCIPAL INVESTIGATOR

• Hashem Ghoraba, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

• Darius Moshfeghi, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2021
• First Posted: April 5, 2021
• Study Start: April 1, 2021
• Primary Completion: April 1, 2023
• Study Completion: April 1, 2024
• Last Updated: April 20, 2023

Record last verified: 2023-04

Locations

US (1)