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Age-Related Macular DegenerationNCT02463344

Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Patients With AMD

An observational study of Age-Related Macular Degeneration, run by Astellas Institute for Regenerative Medicine at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of his study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry Age-Related Macular Degeneration (AMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells. Read more about this study

Who is it for?
Ages 55 years and older
Conditions the study is for
Age-Related Macular Degeneration
What is being tested?
MA09-hRPE
Type of treatment
Injection or infusion
How many people
11 participants
Where
4 sitesUnited States
Study dates
25 Feb 2013to 19 Aug 2019
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 55 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Study population: The study population is by invitation only for previous participants in the core protocol

Who may be able to join

  • Must have been treated with hESC-RPE cell transplant in the core protocol.
  • Able to understand and willing to sign the informed consent to participate in the follow up study.

Who cannot join

  • There are no exclusion criteria.

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Mass Eye and Ear

    Boston, Massachusetts, 02114-3002

  • Jules Stein Eye Institute, UCLA School of Medicine

    Los Angeles, California, 90095

  • Bascom Palmer Eye Institute

    Miami, Florida, 33136

  • Wills Eye Institute-Mid Atlantic Retina

    Philadelphia, Pennsylvania, 19107

Other trials in United States

About this study

Official title: Long Term Follow up to A Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-hRPE) Cells in Patients With Advanced Dry AMD

The purpose of his study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry Age-Related Macular Degeneration (AMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells.

Read the detailed description

This study is a long term, follow up to the ACT MA09-hRPE 001 phase I/II trial. The phase I/II trial (referred to as the core protocol) was an open-label, non randomized, dose escalation, multi-center trial. Thirteen AMD patients were treated in this trial. Ten patients with profound vision loss (visual acuity <= 20/400) received a single subretinal injection of MA09-hRPE cells, starting at a dose of 50,000 MA09-hRPE cells transplanted (three patients), 100,000 MA09-hRPE cells transplanted (three patients), 150,000 MA09-hRPE cells (three patients) and increasing to a maximum dose of 200,000 MA09-hRPE cells transplanted (one patient). Three patients with severe to moderate loss (visual acuity <= 20/100) received a dose of 100,000 MA09-hRPE cells. All patients who participated in the core protocol are eligible for participation in this follow-up protocol. The first visit of this long term follow-up protocol will correspond to the last visit of the core protocol, and will take place at 12 months post cell implantation. Informed consent will be obtained at this visit. Patients will be evaluated at 18, 24, 36, 48 and 60 months post-transplant (or more frequently as clinically indicated). Follow-up will include obtaining information about ophthalmological findings and events of special interest as defined in the Primary Outcome. At the last visit of this follow-up study, whether at 60 months post-transplant or at early discontinuation, patients will be invited to participate in a safety surveillance study for an additional 10 years under a separate protocol which will continue to monitor the long term risks of MA09-hRPE cell transplantation.

Being tested
MA09-hRPE
Keywords
Dry AMD, Geographic atrophy, ASP7316
Medical subject headings
Macular Degeneration, Geographic Atrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MA09-hRPEBiological

    Also called ASP7316

    Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 2a Better Vision 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells

Study groups in NCT02463344
Study groupRoleWhat they receive
MA09-hRPEExperimental: Subretinal injection of MA09-hRPE * Cohort 1 50,000 cells * Cohort 2 100,000 cells * Cohort 2a Better Vision 100,000 cells * Cohort 3 150,000 cells * Cohort 4 200,000 cells
Receives: Biological: MA09-hRPE

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product

    This will include obtaining information about ophthalmological findings and Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, pregnancy in a female subject or the partner of a male subject and pregnancy outcome, any Adverse Event (AE) that causes a subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer, ectopic or proliferative cell growth (Retinal pigment epithelium (RPE) or non-RPE) with adverse clinical consequence, and unexpected, clinically significant AEs possibly related to the cell transplant procedure or the investigational product (MA09-hRPE cells).

    Measured: 4 years

Secondary outcomes (2)

  1. Incidence of graft failure or rejection

    Evidence of graft failure or rejection will consist of: Presence of retinal edema, cystoid macular edema, retinal white dots, retinal hemorrhage, serous retinal detachment, subretinal exudates, subretinal fibrosis, or vascular and/or optic disc leakage, elevated intraocular pressure or hypotony. Evidence of unanticipated and persistent or increasing non-infectious ocular inflammation (e.g., vasculitis, retinitis, choroiditis, vitritis, pars planitis, anterior segment inflammation/uveitis).

    Measured: 4 years

  2. Number of patients with changes in ocular examinations or images

    The number of patients with clinically significant absolute values or changes from baseline in Intra-ocular pressure (IOP) and Best Corrected Visual Acuity (BCVA) will be summarized.

    Measured: 4 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Jun 2015 (estimated)
  2. Study start25 Feb 2013
  3. Primary completion19 Aug 2019
  4. Study completion19 Aug 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02463344
Sponsor's protocol ID
7316-CL-0005
Other IDs
ACT MA09-hRPE AMD -001 LTFU
Status last verified
Oct 2024
First submitted
13 May 2015
Last updated
31 Oct 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Publications

  • Schwartz SD, Hubschman JP, Heilwell G, Franco-Cardenas V, Pan CK, Ostrick RM, Mickunas E, Gay R, Klimanskaya I, Lanza R. Embryonic stem cell trials for macular degeneration: a preliminary report. Lancet. 2012 Feb 25;379(9817):713-20. doi: 10.1016/S0140-6736(12)60028-2. Epub 2012 Jan 24.

    PubMed 22281388

  • Schwartz SD, Regillo CD, Lam BL, Eliott D, Rosenfeld PJ, Gregori NZ, Hubschman JP, Davis JL, Heilwell G, Spirn M, Maguire J, Gay R, Bateman J, Ostrick RM, Morris D, Vincent M, Anglade E, Del Priore LV, Lanza R. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies. Lancet. 2015 Feb 7;385(9967):509-16. doi: 10.1016/S0140-6736(14)61376-3. Epub 2014 Oct 15.

    PubMed 25458728

Questions about this trial

What is this clinical trial studying?

An observational study of Age-Related Macular Degeneration, run by Astellas Institute for Regenerative Medicine at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 31 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product (time frame: 4 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02463344, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Oct 2024.

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