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NCT02445612

Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients

Completed Last updated 31 October 2024
What this trial tests

trial testing MA09-hRPE in Stargardt's Macular Dystrophy in 13 participants. Completed in 21 June 2019.

Timeline
11 July 2012
Primary endpoint
21 June 2019
21 June 2019

Quick facts

Lead sponsorAstellas Institute for Regenerative Medicine
StatusCompleted
Study typeOBSERVATIONAL
Enrollment13
Start date11 July 2012
Primary completion21 June 2019
Estimated completion21 June 2019
Sites3 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Astellas Institute for Regenerative Medicine — full company profile →

Who can join

18 and older, any sex, with Stargardt's Macular Dystrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced Stargardt's Macular Dystrophy (SMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Identification and Rescue of Splice Defects Caused by Two Neighboring Deep-Intronic ABCA4 Mutations Underlying Stargardt Disease.
    Albert S, Garanto A, Sangermano R, Khan M, et al · · 2018 · cited 113× · PMID 29526278 · DOI 10.1016/j.ajhg.2018.02.008
  2. Comparing the Therapeutic Potential of Stem Cells and their Secretory Products in Regenerative Medicine.
    Foo JB, Looi QH, Chong PP, Hassan NH, et al · · 2021 · cited 89× · PMID 34422061 · DOI 10.1155/2021/2616807
  3. Let There Be Light: Gene and Cell Therapy for Blindness.
    Dalkara D, Goureau O, Marazova K, Sahel JA. · · 2016 · cited 89× · PMID 26751519 · DOI 10.1089/hum.2015.147
  4. Therapeutic Application of Pluripotent Stem Cells: Challenges and Risks.
    Martin U. · · 2017 · cited 50× · PMID 29312943 · DOI 10.3389/fmed.2017.00229
  5. Recent Trends in Research with Human Pluripotent Stem Cells: Impact of Research and Use of Cell Lines in Experimental Research and Clinical Trials.
    Guhr A, Kobold S, Seltmann S, Seiler Wulczyn AEM, et al · · 2018 · cited 45× · PMID 30033087 · DOI 10.1016/j.stemcr.2018.06.012
  6. Two decades of embryonic stem cells: a historical overview.
    Eguizabal C, Aran B, Chuva de Sousa Lopes SM, Geens M, et al · · 2019 · cited 38× · PMID 30895264 · DOI 10.1093/hropen/hoy024
  7. Therapy Approaches for Stargardt Disease.
    Piotter E, McClements ME, MacLaren RE. · · 2021 · cited 33× · PMID 34439845 · DOI 10.3390/biom11081179
  8. Trends of Stem Cell Therapies in Age-Related Macular Degeneration.
    Maeda T, Sugita S, Kurimoto Y, Takahashi M. · · 2021 · cited 33× · PMID 33923985 · DOI 10.3390/jcm10081785

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Data sources for this page

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