A Study Of Implantation Of Human Embryonic Stem Cell Derived Retinal Pigment Epithelium In Subjects With Acute Wet Age Related Macular Degeneration And Recent Rapid Vision Decline - Full Text View

Purpose

In this Phase 1 trial of retinal pigment epithelium (RPE) replacement, the aim will be to evaluate the safety and feasibility/efficacy of treating subjects with wet age-related macular degeneration (AMD) in whom there is rapidly progressing vision loss

Condition	Intervention	Phase
Age Related Macular Degeneration	Biological: PF-05206388	Phase 1

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Phase 1, Open-Label, Safety And Feasibility Study Of Implantation Of PF-05206388 (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE) Living Tissue Equivalent) In Subjects With Acute Wet Age Related Macular Degeneration And Recent Rapid Vision Decline

Resource links provided by NLM:

Genetics Home Reference related topics: age-related macular degeneration X-linked juvenile retinoschisis

MedlinePlus related topics: Macular Degeneration

U.S. FDA Resources

Further study details as provided by Pfizer:

Primary Outcome Measures:

Incidence and severity of adverse events. [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an improvement of 15 letters or more at Week 24. [ Time Frame: 24 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Change in baseline in ETDRS best corrected visual acuity (BCVA) - Proportion of subjects with an impovement of 15 letters or more [ Time Frame: at Weeks 1,2,4,8, 12,16, 36 & 52. ] [ Designated as safety issue: No ]
Mean change of best corrected visual acuity (BCVA) from baseline by study visit. [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Position of PF-05206388 by serial biomicroscopic evaluation. [ Time Frame: at Weeks 1, 2, 4,8, 10, 12, 16, 24, 36 and 52 ] [ Designated as safety issue: No ]
Position and presence of pigmented Retinal Pigment Epithelium cells by serial fundus photography and SLO autofluorescence. [ Time Frame: at Weeks 4, 6, 10, 12, 16, 24, 36 and 52 ] [ Designated as safety issue: No ]
Placement of PF-05206388 by serial spectral domain Ocular CoherenceTomography (OCT) scan [ Time Frame: at Weeks 4, 8, 12, 16, 24, 36 and 52. ] [ Designated as safety issue: No ]
Change in liver and renal function by blood tests and liver ultrasound . [ Time Frame: at Weeks 24 and 52 ] [ Designated as safety issue: Yes ]
Change in leakage or perfusion in normal fundal vasculature and presence of abnormal vasculature by fundus fluorescein angiography. [ Time Frame: at Weeks 4, 8, 12, 24 and 52 ] [ Designated as safety issue: Yes ]
Change in central 30 degree of visual function by Humphrey Field test. [ Time Frame: at Weeks 4, 8, 12, 24 and 52 ] [ Designated as safety issue: No ]
Evidence of diffuse retinal and RPE toxicity by full field ERG and EOG. [ Time Frame: at Weeks 12 and 52 ] [ Designated as safety issue: Yes ]
Photoreceptor survival (indirect indication of RPE survival) by Adaptive Optics. [ Time Frame: at Weeks 16, 24, 36 and 52 ] [ Designated as safety issue: No ]
Functional survival of retinal photoreceptors by Nidekmicroperimetry over PF-05206388. [ Time Frame: at Weeks 4, 16, 36 and 52 ] [ Designated as safety issue: No ]
Change in reading speed by Minnesota (MN) Read test. [ Time Frame: at Weeks 4, 8, 16, 24, 36 and 52 ] [ Designated as safety issue: No ]
Change in thickness of RPE layer by B-mode orbital ultrasound. [ Time Frame: at Weeks 4, 8, 16, 24, 36 and 52 ] [ Designated as safety issue: Yes ]
Detection of alloactive T-cells and alloreactive antibodies (blood) . [ Time Frame: at Weeks 2, 4, 8 12, 24 and 52 ] [ Designated as safety issue: No ]

Estimated Enrollment:	10
Study Start Date:	May 2013
Estimated Study Completion Date:	October 2015
Estimated Primary Completion Date:	March 2015 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Treatment	Biological: PF-05206388 PF-05206388 will be provided as a Retinal Pigment Epithelium living tissue equivalent for intraocular use in the form of a monolayer of Retinal Pigmented Epithelial (RPE) cells immobilized on a polyester membrane. The membrane is approximately 6 mm x 3 mm and will contain a confluent layer of RPE cells, at a nominal dose of 17 mm2. The implant is intended to be life-long.

Eligibility

Ages Eligible for Study:	61 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Male and /or post-menopausal female subjects aged 61 years or above.
Diagnosis of wet Age-related Macular Degeneration (AMD) plus rapid recent vision decline
An informed consent document signed and dated by the subject or a legal representative.

Exclusion Criteria:

Pregnant females; breastfeeding females; and females of childbearing potential.
Treatment with an investigational drug within 30 days (or as determined by the local requirement, whichever is longer) or 5 half-lives preceding the first dose of study medication.
Current or previous significant other ocular disease in the study eye, as determined by the investigator.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01691261

Contacts

Contact: Pfizer CT.gov Call Center

1-800-718-1021

Sponsors and Collaborators

Pfizer

University College, London

Investigators

Study Director:

Pfizer CT.gov Call Center

Pfizer

More Information

Additional Information:

To obtain contact information for a study center near you, click here. This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Pfizer
ClinicalTrials.gov Identifier:	NCT01691261 History of Changes
Other Study ID Numbers:	B4711001
Study First Received:	September 19, 2012
Last Updated:	May 12, 2013
Health Authority:	United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:

Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases

ClinicalTrials.gov processed this record on May 19, 2013