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History of Changes for Study: NCT01344993
Safety and Tolerability of Sub-retinal Transplantation of hESC Derived RPE (MA09-hRPE) Cells in Patients With Advanced Dry Age Related Macular Degeneration (Dry AMD)
Latest version (submitted July 4, 2021) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 April 28, 2011 None (earliest Version on record)
2 November 9, 2011 Sponsor/Collaborators and Study Status
3 January 17, 2012 Contacts/Locations and Study Status
4 January 19, 2012 Contacts/Locations and Study Status
5 January 30, 2012 Contacts/Locations and Study Status
6 April 2, 2012 Contacts/Locations, Study Status and Eligibility
7 May 17, 2012 Contacts/Locations and Study Status
8 September 7, 2012 Contacts/Locations and Study Status
9 January 29, 2013 Study Status, Eligibility, Outcome Measures, Arms and Interventions, Study Design and Study Description
10 February 1, 2013 Study Status and Study Description
11 April 10, 2014 Study Status and Arms and Interventions
12 November 3, 2014 Study Status
13 April 27, 2015 Recruitment Status, Study Status, Contacts/Locations and Study Identification
14 October 21, 2015 Study Status
15 June 3, 2016 Recruitment Status, Study Status, Study Design, Sponsor/Collaborators, Study Identification and IPDSharing
16 June 10, 2016 Contacts/Locations and Study Status
17 August 17, 2016 Study Status
18 February 17, 2017 Study Status, Study Identification and Study Design
19 June 16, 2021 Study Status and References
20 July 4, 2021 IPDSharing and Study Status
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Study NCT01344993
Submitted Date:  April 28, 2011 (v1)

Open or close this module Study Identification
Unique Protocol ID: ACT MA09-hRPE AMD-001
Brief Title: Safety and Tolerability of Sub-retinal Transplantation of hESC Derived RPE (MA09-hRPE) Cells in Patients With Advanced Dry Age Related Macular Degeneration (Dry AMD)
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 Advanced Dry AMD
Secondary IDs:
Open or close this module Study Status
Record Verification: April 2011
Overall Status: Recruiting
Study Start: April 2011
Primary Completion: July 2013 [Anticipated]
Study Completion: July 2013 [Anticipated]
First Submitted: April 28, 2011
First Submitted that
Met QC Criteria:
April 28, 2011
First Posted: April 29, 2011 [Estimate]
Last Update Submitted that
Met QC Criteria:
April 28, 2011
Last Update Posted: April 29, 2011 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: Ocata Therapeutics
Responsible Party:
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: Yes
Open or close this module Study Description
Brief 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 dry Age Related Macular Degeneration (AMD) and to perform exploratory evaluation of potential efficacy endpoints to be used in future studies retinal pigment epithelium (RPE) cellular therapy.
Detailed Description:

This study will be a phase I/II, open-label, non randomized, sequential, multi-center safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with dry AMD and to perform exploratory evaluation of potential efficacy endpoints to be used in future studies RPE cellular therapy. Patients will be enrolled sequentially, and the clinical course of each patient treated will be reviewed by an independent Data Safety Monitoring Board (DSMB) before the next patient is enrolled.

Each eligible patient who signs a consent form and fulfills all (Inclusion/Exclusion) criteria will receive a single uniocular subretinal infusion of MA09-hRPE cells in one of four dose levels.

  1. Three patients - 50,000 cells transplanted
  2. Three patients - 100,000 cells transplanted
  3. Three patients - 150,000 cells transplanted
  4. Three patients - 200,000 cells transplanted

Six weeks after the first patient in each dose cohort receives the cell transplant, the DSMB will review the clinical data and recommend if the next two patients in the dose cohort may be treated. Each cohort of 3 patients will be reviewed by the DSMB when the 3rd patient completes 4 weeks of follow-up.

Open or close this module Conditions
Conditions: Dry Age Related Macular Degeneration
Keywords: Dry AMD
Geographic atrophy
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Other
Study Phase: Phase 1/Phase 2
Interventional Study Model: Single Group Assignment
Number of Arms:
Masking: None (Open Label)
Allocation: N/A
Enrollment: 12 [Anticipated]
Open or close this module Arms and Interventions
Intervention Details:
Biological: MA09-hRPE Cellular Therapy
  • Cohort 1- 50,000 MA09-hRPE cells transplanted
  • Cohort 2- 100,000 MA09-hRPE cells transplanted
  • Cohort 3- 150,000 MA09-hRPE cells transplanted
  • Cohort 4- 200,000 MA09-hRPE cells transplanted
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Safety of hESC derived RPE cells
[ Time Frame: 12 Months ]

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
Secondary Outcome Measures:
1. exploratory evaluations for potential efficacy endpoints.
[ Time Frame: 12 months ]

Secondary endpoints will be evaluated as exploratory evaluations for potential efficacy endpoints.

  • Change in the mean of BCVA
  • Autofluorescense photography
  • Reading speed

Evidence of successful engraftment will consist of:

  • Structural evidence (OCT imaging, fluorescein angiography, 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
Open or close this module Eligibility
Minimum Age: 55 Years
Maximum Age:
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

Inclusion Criteria:

  • Adult male or female over 55 years of age.
  • Patient should be in sufficiently good health to reasonably expect survival for at least four years after treatment
  • Clinical findings consistent with advanced dry AMD with evidence of one or more areas of >250microns of geographic atrophy (as defined in the Age-Related eye Disease Study [AREDS] study) involving the central fovea.
  • GA defined as attenuation or loss of RPE as observed by biomicroscopy, OCT, and FA.
  • No evidence of current or prior choroidal neovascularization
  • The visual acuity (BCVA) of the eye to receive the transplant will be no better than 20/400.
  • The visual acuity (BCVA) of the eye that is NOT to receive the transplant will be no worse than 20/400.
  • Electrophysiological findings consistent with advanced dry AMD.
  • 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:

Any laboratory value which falls slightly outside of the normal range will be reviewed by the Medical Monitor and Investigators to determine its clinical significance. If it is determined not to be clinically significant, the patient may be enrolled into the study.

  • 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. (NOTE:With the exception of abnormalities specifically identified in the exclusion criteria)
  • 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 (excised) basal cell carcinoma[skin cancer] or successfully treated 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 & 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; 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, 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 spermicidal 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

Exclusion Criteria:

  • Presence of active or inactive CNV.
  • Presence or history of retinal dystrophy, retinitis pigmentosa, chorioretinitis, central serious choroidopathy, diabetic retinopathy or other retinal vascular or degenerative disease other than ARMD.
  • History of optic neuropathy.
  • Macular atrophy due to causes other than AMD.
  • Presence of glaucomatous optic neuropathy in the study eye, uncontrolled IOP, or use of two or more agents to control IOP (acetazolamide, beta blocker, alpha-1-agonist, antiprostaglandins, anhydrous carbonic inhibitors).
  • Cataract of sufficient severity likely to necessitate surgical extraction within 1 year.
  • History of retinal detachment repair in the study eye.
  • Axial myopia of greater than -8 diopters
  • Axial length greater than 28 mm.
  • History of malignancy (with the exception of successfully treated [excised] basal cell carcinoma[skin cancer] or successfully treated squamous cell carcinoma of the skin).
  • History of myocardial infarction in previous 12 months.
  • History of diabetes mellitus.
  • History of cognitive impairments or dementia which may impact the patients ability participate in the informed consent process and to appropriately complete evaluations.
  • Any immunodeficiency.
  • Any current immunosuppressive therapy other than intermittent or low dose corticosteroids.
  • Alanine transaminase/aspartate aminotransferase (ALT/AST) >1.5 times the upper limit of normal or any known liver disease.
  • Renal insufficiency, as defined by creatine level >1.3 mg/dL.
  • A hemoglobin concentration of less than 10 gm/dL, a platelet count of less than 100k/mm3 or an absolute neutrophil count of less than 1000/mm3 at study entry.
  • Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
  • 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 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.
  • Ocular surgery in the study eye in the 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.
Open or close this module Contacts/Locations
Study Officials: Steven Schwartz, MD
Principal Investigator
Jules Stein Eye Institute-UCLA
Locations: United States, California
Jules Stein Eye Institute, UCLA School of Medicine
[Recruiting]
Los Angeles, California, United States, 90095
Contact:Contact: Logan Hitchcock 310-825-3046
Contact:Principal Investigator: Steven Schwartz, MD
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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