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A Safety Surveillance Study in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03167203
Recruitment Status : Enrolling by invitation
First Posted : May 25, 2017
Last Update Posted : May 1, 2019
Sponsor:
Information provided by (Responsible Party):
Astellas Pharma Inc ( Astellas Institute for Regenerative Medicine )

Brief Summary:
The purpose of this study is to is to evaluate the occurrence of late onset (i.e., greater than 5 years after treatment) safety events of special interest in participants who have received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in an AIRM-sponsored clinical trial. The events of special interest are adverse events (AEs) that are presumed to have a potential causal relationship to the hESC-RPE cells.

Condition or disease Intervention/treatment Phase
Macular Degenerative Disease Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells Phase 1 Phase 2

Detailed Description:

Participants will be contacted by the Patient Outreach Center (POC) on an annual basis to complete a questionnaire about the occurrence of safety events of special interest, as defined in the Outcome Measure section.

The questionnaire will be completed directly by the participant (via a secure web based platform) or will be administered via telephone by a POC staff member. In the event of the occurrence of a safety event of special interest, study participant will be encouraged to contact the POC as soon as possible, rather than wait for the annual questionnaire completion. Follow up data is inclusive of the 1 year in the core trial, 4 years in the long term follow up trial and 10 years in this long term safety follow up. Participation for United Kingdom participants will be life-long.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 36 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Safety Surveillance Study of Events of Special Interest Occurring in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy
Actual Study Start Date : January 8, 2018
Estimated Primary Completion Date : December 2029
Estimated Study Completion Date : December 2029

Arm Intervention/treatment
Experimental: hESC-RPE cells
Participants previously enrolled into an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial and treated with Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial (hESC-RPE) cells
Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells
Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial
Other Name: ASP7316




Primary Outcome Measures :
  1. Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic [ Time Frame: Up to 15 Years ]
    Adverse events (AEs) will be coded using Medical Dictionary for Regulatory Activities (MedDRA). Participant self-reported via an annual questionnaire of adverse events that are ophthalmologic, neurologic, infectious or hematologic.

  2. Number of new diagnoses of an immune-mediated disorder [ Time Frame: Up to 15 Years ]
    Any new diagnosis of an immune-mediated disorder will be participant self-reported via an annual questionnaire.

  3. Incidents of new cancer, irrespective of prior history [ Time Frame: Up to 15 Years ]
    Any new cancer, irrespective of prior history, will be participant self-reported via an annual questionnaire.

  4. Incidents of hESC-RPE cell proliferation [ Time Frame: Up to 15 Years ]
    Occurrence of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cell proliferation will be participant self-reported via an annual questionnaire.

  5. Incidents of ectopic tissue (RPE or non-RPE) formation [ Time Frame: Up to 15 Years ]
    Occurrence of ectopic tissue (retinal pigment epithelial [RPE] or non-RPE) will be participant self-reported via an annual questionnaire

  6. Number of participant reported pregnancies or pregnancy of participant's partner [ Time Frame: Up to 15 Years ]
    Occurrence of pregnancy will be participant self-reported via an annual questionnaire

  7. Number of reported pregnancy outcome(s) [ Time Frame: Up to 15 Years ]
    Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire

  8. All cause death [ Time Frame: Up to 15 Years ]
    All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participant was treated with hESC-RPE cell therapy in an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial in macular degenerative disease.
  • Participant is able to understand.

Exclusion Criteria:

  • There are no exclusion criteria.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03167203


Locations
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United States, Missouri
Private Practice
Kansas City, Missouri, United States, 64133
United Kingdom
Site GB44001
London, United Kingdom
Sponsors and Collaborators
Astellas Institute for Regenerative Medicine
Investigators
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Study Director: Medical Monitor Astellas Institute for Regenerative Medicine

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Responsible Party: Astellas Institute for Regenerative Medicine
ClinicalTrials.gov Identifier: NCT03167203     History of Changes
Other Study ID Numbers: 7316-CL-0007
2016-005245-23 ( EudraCT Number )
RPE-SSS-001 ( Other Identifier: Sponsor )
First Posted: May 25, 2017    Key Record Dates
Last Update Posted: May 1, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Astellas Pharma Inc ( Astellas Institute for Regenerative Medicine ):
hESC-RPE
Macular Degenerative Disease