- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04707209
Sirolimus for Retinal Astrocytic Hamartoma
Intravitreal Injection of Sirolimus in the Treatment of Aggressive Retinal Astrocytic Hamartoma
Study Overview
Detailed Description
Retinal astrocytic hamartomas (RAH) are benign tumors of glial cells arising from astrocytes in the nerve-fiber layer of the retina. They are often associated with tuberous sclerosis complex (TSC) and, more rarely, neurofibromatosis type 1 (NF1). RAH may be caused by dysregulated tumor suppressors genes TSC1 or TSC2, which play a role in cell cycle regulation in retinal astrocytes via the PDGF-signalling pathway. Downregulation of TSC1 or TSC2 can result in hyperactivation of mTOR. Sirolimus is an inhibitor of mTOR, therefore inhibiting cell growth and proliferation of astrocytes. Systemic mTOR inhibitors (sirolimus and everolimus) have shown impressive reduction in the size of RAH in animal models and many human case series.
This single patient study is investigating the use of concurrent oral and intravitreal Sirolimus to treat a patient with multiple retinal astrocytic hamartomas (RAH) of both eyes to improve intraocular response and reduce duration of treatment. The patient has no other clinical features suggestive of neurofibromatosis and a presumed diagnosis of tuberous sclerosis is being considered, with only two hypopigmented skin lesions noted.
Study Type
Enrollment (Actual)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: Ashwin Mallipatna, MD
- Phone Number: 207403 416-813-7654
- Email: ashwin.mallipatna@sickkids.ca
Study Contact Backup
- Name: Kaitlyn Flegg, MSc
- Phone Number: 203287 416-813-7654
- Email: Kaitlyn.flegg@sickkids.ca
Study Locations
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Ontario
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Toronto, Ontario, Canada, M5G1X8
- The Hospital for Sick Children
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Intravitreal Sirolimus
|
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.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Size of Retinal Astrocytic Hamartomas compared to baseline
Time Frame: 21 months
|
Measured by ophthalmic imaging
|
21 months
|
Amount of retinal exudate compared to baseline
Time Frame: 21 months
|
Measured by ophthalmic imaging
|
21 months
|
Retinal detachment progression/regression compared to baseline
Time Frame: 21 months
|
Measured by ophthalmic imaging
|
21 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Visual acuity compared to baseline
Time Frame: 21 months
|
Measured using logMAR virtual activity test
|
21 months
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 1000072947
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
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.
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Clinical Trials on Sirolimus
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Aadi Bioscience, Inc.CompletedHigh Grade Recurrent Glioma and Newly Diagnosed GlioblastomaUnited States
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Stefan Schieke MDWithdrawnCutaneous T-cell Lymphoma (CTCL)United States
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Brigham and Women's HospitalNational Heart, Lung, and Blood Institute (NHLBI)CompletedLymphangioleiomyomatosisUnited States
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The University of Texas Health Science Center,...Society for Pediatric DermatologyCompletedTuberous Sclerosis | Neurofibromatoses | Neurofibroma | AngiofibromaUnited States
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Xijing HospitalAir Force Military Medical University, China; Shanghai MicroPort Medical (Group)... and other collaboratorsUnknown
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Shanghai Jiao Tong University School of MedicineUnknownCoronary Artery DiseaseChina
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Paul S Teirstein, MDCordis CorporationCompletedCoronary Artery Disease | Coronary Thrombosis | Coronary RestenosisUnited States
-
Meril Life Sciences Pvt. Ltd.Lifecare Institute of Medical Sciences and Research Ahmedabad Gujarat IndiaCompleted