NCT04707209

Brief Summary

A single patient study using intravitreal Sirolimus to treat a patient with multiple retinal astrocytic hamartomas (RAH) of both eyes.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Nov 2020

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 4, 2020

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

December 16, 2020

Completed
28 days until next milestone

First Posted

Study publicly available on registry

January 13, 2021

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 22, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 22, 2024

Completed
Last Updated

March 25, 2024

Status Verified

March 1, 2024

Enrollment Period

3.4 years

First QC Date

December 16, 2020

Last Update Submit

March 22, 2024

Conditions

Retinal Astrocytic Hamartoma

Outcome Measures

Primary Outcomes (3)

  • Size of Retinal Astrocytic Hamartomas compared to baseline

    Measured by ophthalmic imaging

    21 months

  • Amount of retinal exudate compared to baseline

    Measured by ophthalmic imaging

    21 months

  • Retinal detachment progression/regression compared to baseline

    Measured by ophthalmic imaging

    21 months

Secondary Outcomes (1)

  • Visual acuity compared to baseline

    21 months

Study Arms (1)

Intravitreal Sirolimus

EXPERIMENTAL
Drug: Sirolimus

Interventions

SirolimusDRUG

An intravitreal injection of 20 µL of drug will be given through the pars plana of the eye. The injected eye will be treated with topical antibiotic and steroid eye drops prescribed for 7 to 10 days after the injection. The first injection will only be provided into the worse eye despite both eyes showing signs of exudation. Injection into the other eye will be considered only two months after the safety of this first injection is determined. Safety and efficacy of treatment will be determined by repeat eye examinations in the eye clinic one day, one week and one month after each injection. The interval between injections is roughly suggested to be between 3 to 8 weeks based on the drug deposit present in the eye. A total of 3-12 injections will be given per eye, over 6-24 months.

Also known as: DE-109
Intravitreal Sirolimus

Eligibility Criteria

Age15 Years - 16 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)
Single case diagnosed with multiple retinal astrocytic hamartomas (RAH) of both eyes with imminent threat to vision.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G1X8, Canada

Location

MeSH Terms

Interventions

Sirolimus

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Paediatric Ophthalmologist

Study Record Dates

First Submitted

December 16, 2020

First Posted

January 13, 2021

Study Start

November 4, 2020

Primary Completion

March 22, 2024

Study Completion

March 22, 2024

Last Updated

March 25, 2024

Record last verified: 2024-03

Data Sharing

IPD Sharing
Will not share

Locations

CA(1)