Phase 1/2 Study of an Ocular Sirolimus (Rapamycin) Formulation in Patients With Age-Related Macular Degeneration

This study has been terminated.
MacuSight, Inc.
Information provided by (Responsible Party):
Santen Inc. Identifier:
First received: July 8, 2008
Last updated: January 8, 2013
Last verified: July 2010

The purpose of this study is to determine the safety and efficacy of an ocular sirolimus (rapamycin) formulation via different injection routes in patients with treatment-naive sub-foveal choroidal neovascularization secondary to age-related macular degeneration.

Condition Intervention Phase
Age-related Macular Degeneration
Choroidal Neovascularization
Drug: Sirolimus
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1/2, Randomized Clinical Study to Assess the Safety and Efficacy of Sirolimus in Patients With Newly Diagnosed, Treatment-Naive Sub-Foveal Choroidal Neovascularization Secondary to Age-Related Macular Degeneration

Resource links provided by NLM:

Further study details as provided by Santen Inc.:

Primary Outcome Measures:
  • Best-corrected visual acuity by ETDRS [ Time Frame: 180 days ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Best-corrected visual acuity by ETDRS [ Time Frame: 60 days, 120 days ] [ Designated as safety issue: No ]
  • Safety across injection routes [ Time Frame: Through 1 year ] [ Designated as safety issue: Yes ]

Enrollment: 20
Study Start Date: September 2008
Study Completion Date: March 2010
Primary Completion Date: November 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: Sirolimus
Three subconjunctival injections of 1320 micrograms sirolimus each.
Other Name: MS-R002, rapamycin
Experimental: 2 Drug: Sirolimus
Three intravitreal injections of 352 micrograms sirolimus each.
Other Name: MS-R001, rapamycin


Ages Eligible for Study:   50 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria include, but are not limited to:

  • Diagnosed with sub-foveal choroidal neovascularization secondary to age-related macular degeneration
  • Visual acuity of 20/50 to 20/200 in study eye

Exclusion Criteria:

  • Any other ocular disease that could compromise vision in the study eye
  • History of any prior treatment for choroidal neovascularization in the study eye
  • Presence of other causes of choroidal neovascularization other than secondary to age-related macular degeneration
  Contacts and Locations
Please refer to this study by its identifier: NCT00712491

United States, Arizona
Retinal Consultants of Arizona
Phoenix, Arizona, United States, 85044
Sponsors and Collaborators
Santen Inc.
MacuSight, Inc.
Study Director: Joel Naor, MD MacuSight, Inc.
  More Information

No publications provided

Responsible Party: Santen Inc. Identifier: NCT00712491     History of Changes
Other Study ID Numbers: AMD-002
Study First Received: July 8, 2008
Last Updated: January 8, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Santen Inc.:
wet AMD

Additional relevant MeSH terms:
Macular Degeneration
Neovascularization, Pathologic
Choroidal Neovascularization
Retinal Degeneration
Retinal Diseases
Eye Diseases
Pathologic Processes
Choroid Diseases
Uveal Diseases
Antibiotics, Antineoplastic
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
Antifungal Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
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