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Stargardt's Macular DystrophyPhase 1/2NCT01345006

Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy

A Phase 1/2 study testing MA09-hRPE in people with Stargardt's Macular Dystrophy, run by Astellas Institute for Regenerative Medicine at 3 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

This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Stargardt's Macular Dystrophy
What is being tested?
MA09-hRPE
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
3 sitesUnited States
Study dates
16 Jun 2011to 10 Aug 2015
Phase
Phase 1/2
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Adult male or female over 18 years of age.
  • Clinical diagnosis of advanced SMD.
  • If known, the patient's genotype will be recorded in the medical history, if unknown, patient will allow for the submission of a sample for genotyping.Clinical findings consistent with SMD.
  • The visual acuity of the eye to receive the transplant will be no better than 20/400. The visual acuity of the eye in the better vision cohort to receive the transplant will be no better than 20/100.
  • The visual acuity of the eye that is not to receive the transplant will be no better than 20/400 for the worse vision patients and no worse than 20/100 for the better vision patients.
  • Peripheral visual field constriction documented on standard kinetic visual field testing.
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  • Electrophysiological findings consistent with SMD.
  • Medically suitable to undergo vitrectomy and subretinal injection.
  • Medically suitable for general anesthesia or waking sedation, if needed.
  • Medically suitable for transplantation of an embryonic stem cell line:
  • Normal serum chemistry (sequential multi-channel analyzer 20 [SMA- 20]) and hematology (complete blood count [CBC], prothrombin time [PT], and activated partial thromboplastin time [aPTT]) screening tests.
  • Negative urine screen for drugs of abuse.
  • Negative human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV) serologies.
  • No history of malignancy,with the exception of successfully treated basal cell or squamous cell carcinoma of the skin.
  • Negative cancer screening within previous 6 months:
  • complete history \& physical examination;
  • dermatological screening exam for malignant lesions;
  • negative fecal occult blood test \& if over age 50 years, negative colonoscopy within previous 7 years;
  • negative chest roentgenogram (CXR);
  • normal CBC \& manual differential;
  • negative urinalysis (U/A);
  • normal thyroid exam;
  • if male, normal testicular examination; if over age 40, digital rectal examination (DRE) and prostate specific antigen (PSA);
  • if female, normal pelvic examination with Papanicolaou smear; and
  • if female, normal clinical breast exam and if 40 years of age or older, negative mammogram.
  • If female and of childbearing potential, willing to use two effective forms of birth control during the study.
  • If male, willing to use barrier and spermicide contraception during the study.
  • Willing to defer all future blood, blood component or tissue donation. -Able to understand and willing to sign the informed consent.

Who cannot join

  • History of malignancy,with the exception of successfully treated basal cell or squamous cell carcinoma of the skin.
  • History of myocardial infarction in previous 12 months.
  • History of diabetes mellitus.
  • Any immunodeficiency.
  • Any current immunosuppressive therapy other than intermittent or low dose corticosteroids.
  • Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
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  • Current participation in any other clinical trial.
  • Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.
  • Any other sight-threatening ocular disease.
  • Any chronic ocular medications.
  • Any history of retinal vascular disease (compromised blood-retinal barrier.
  • Glaucoma.
  • Uveitis or other intraocular inflammatory disease.
  • Significant lens opacities or other media opacity.
  • Ocular lens removal within previous 3 months.
  • If female, pregnancy or lactation.
  • Any other medical condition, which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromises patient safety, or interferes with the interpretation of the study results.

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • 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: 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 Stargardt's Macular Dystrophy (SMD)

This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD).

Read the detailed description

This study is a Phase I/II, open-label, non randomized, sequential, multi-center clinical trial. There will be 5 cohorts, the 4 low vision cohorts will contain 3 patients, the better vision cohort will contain 4 patients. The enrolled cohorts will be as follows:

Three SMD patients- 50,000 MA09-hRPE cells transplanted

Three SMD patients- 100,000 MA09-hRPE cells transplanted

Four Better Vison SMD patients- 100,000 MA09-hRPE cells transplanted

Three SMD patients- 150,000 MA09-hRPE cells transplanted

Three SMD patients- 200,000 MA09-hRPE cells transplanted

Patients will be enrolled sequentially, and within each cohort of 3 patients, each patient's clinical course over the first 6 weeks following cell transplantation will be reviewed by an independent (DSMB) before enrollment is opened for the next 2 patients. A full safety assessment of all 3 patients in each cohort will be made by the DSMB when the 3rd patient in each cohort completes 4 weeks of follow-up, and before the first patient in the next cohort receives a cell transplant. The exception is the better vision group where all patients may be enrolled once DSMB approval has been received.

Each cohort will be enrolled sequentially in turn, with the exception of the better vision cohort which may be enrolled in parallel with the other cohorts.

The day of the cell implantation will be Day 0, and patients will remain in the study until the last visit at 12 months.

Being tested
MA09-hRPE
Keywords
juvenile macular dystrophy, SMD, fundus flavimaculatus
Medical subject headings
Stargardt Disease

What participants receive

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

  • MA09-hRPEBiological

    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 NCT01345006
Study groupRoleWhat they receive
MA09-hRPEExperimentalPatients will undergo subretinal injection of MA09-hRPE
Receives: Biological: MA09-hRPE

What is being measured

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

  1. The safety and tolerance of transplantation of hESC-derived RPE cells MA09-hRPE

    The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of:

    • Any grade 2 (NCI grading system) or greater adverse event related to the cell product
    • Any evidence that the cells are contaminated with an infectious agent
    • Any evidence that the cells show tumorigenic potential

    Measured: 12 months

  2. Safety Assessments

    • Adverse Event and Serious Adverse Event assessment
    • Clinical monitoring
    • Serial vital signs
    • Clinical laboratory tests
    • Directed ophthalmological monitoring
    • Monitoring of RPE cells acceptance/integrity/rejection
    • Monitoring of local and systemic infection
    • Monitoring of tumorigenic cell transformation

    Measured: 12 months

Secondary outcomes (1)

  1. Evidence of successful engraftment

    Evidence of successful engraftment will consist of:

    • Structural evidence (OCT imaging, fluorescein angiography, autofluorescence photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location
    • Electroretinographic evidence (mfERG) showing enhanced activity in the implant location

    Measured: 12 months

Timeline

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

  1. First posted29 Apr 2011 (estimated)
  2. Study start16 Jun 2011
  3. Primary completion10 Aug 2015
  4. Study completion10 Aug 2015

Resources

More details

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

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
13 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01345006
Sponsor's protocol ID
7316-CL-0001
Other IDs
ACT SMD 01 MA09-hRPE, ACT MA09-hRPE SMD-001
Status last verified
Oct 2024
First submitted
28 Apr 2011
Last updated
31 Oct 2024
Results posted
No
Data monitoring committee
Yes
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

  • Davis JL. The Blunt End: Surgical Challenges of Gene Therapy for Inherited Retinal Diseases. Am J Ophthalmol. 2018 Dec;196:xxv-xxix. doi: 10.1016/j.ajo.2018.08.038. Epub 2018 Sep 5.

    PubMed 30194931

  • 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

  • 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

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing MA09-hRPE in people with Stargardt's Macular Dystrophy, run by Astellas Institute for Regenerative Medicine at 3 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 28 inclusion and 17 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 3 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.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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?

The safety and tolerance of transplantation of hESC-derived RPE cells MA09-hRPE (time frame: 12 months). The study lists 2 primary outcomes in all. 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 NCT01345006, which also lists 3 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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