NCT02749734

Brief Summary

The purpose of this study was to determine the safety and therapeutic effect of sub-retinal transplantation of human embryo stem cell derived retinal pigment epitheliums (hESC-RPE) in patients with macular degeneration diseases, and explore new treatment modalities for macular degeneration diseases (Age-related macular degeneration and Stargardt's macular dystrophy).

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2015

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2015

Completed
8 months until next milestone

First Submitted

Initial submission to the registry

January 7, 2016

Completed
4 months until next milestone

First Posted

Study publicly available on registry

April 25, 2016

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2018

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2019

Completed
Last Updated

January 31, 2018

Status Verified

January 1, 2018

Enrollment Period

3.6 years

First QC Date

January 7, 2016

Last Update Submit

January 29, 2018

Conditions

Macular DegenerationStargardt's Macular Dystrophy

Keywords

Human embryo stem cell derived retinal pigment epitheliumsSubretinal transplantationWet Age related Macular degenerationStargardt's macular dystrophy

Outcome Measures

Primary Outcomes (1)

  • Number of participants with Treatment-Related Adverse Events [Safety and Tolerability]

    Patients with Treatment-Related Adverse Events caused by local rejection of implanted cells or systemic immunosuppression treatment

    up to 12 months

Secondary Outcomes (5)

  • Number of Early Treatment Diabetic Retinopathy (ETDR ) letters participants can recognize

    up to 12 months

  • Visual Field as examined by Static perimetry

    up to 12 months

  • Flash Electroretinogram (FERG)

    up to 12 months

  • Amplitude and Latency of Flash Visual Evoked Potentials (FVEP)

    up to 12 months

  • Multifocal Electroretinogram (MFERG)

    up to 12 months

Study Arms (1)

hESC-RPE

EXPERIMENTAL

Subretinal transplantation of Human embryo stem cell derived retinal pigment epitheliums

Procedure: Subretinal transplantation

Interventions

Subretinal transplantationPROCEDURE

Transplant hESC-RPE into subretinal space of patients with macular degeneration

hESC-RPE

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Aging from 18 to 75 years
  • must have signed informed consent
  • At least one visually impaired eye caused by macular degeneration diseases
  • Can not be effectively treated with conventional therapies
  • Best corrected visual acuity scores between 19 and 73 letter in ETDRs (early treatment diabetic retinopathy ) eye chart , including 19 and 73 (or the equivalent of Snellen eyesight from 20/400 to 20/40)
  • Visual loss caused by macular degeneration diseases

You may not qualify if:

  • Eyes with concomitant diseases which will interfere the visual improvement of the study
  • Active intraocular inflammation regardless of the grade of severity
  • Active infection (e.g. conjunctivitis, keratitis, scleritis, uveitis, ophthalmia)
  • History of uveitis
  • Severe cataract, glaucoma, retinal blood vessels occlusion, retinal detachment, macular hole, vitreous-macula traction
  • Iris neovascularization
  • Patients who have only one functioning eye, or the best corrected vision of untreated eye scores less than 24 letters in ETDRS chart(corresponding to 20/320 in Snellen chart)
  • History of intraocular surgery
  • Severe systemic diseases: Stroke, coronary heart disease, angina pectoris, renal insufficiency needing dialysis
  • Allergic to sodium fluorescein
  • Uncontrolled hypertension (systolic pressure\>140mmHg,or diastolic pressure\>90mmHg)
  • Coagulative function disorder
  • System administration of drugs that are toxic to lens, retina, or optic nerve like hydroxychloroquine, phenothiazine, ethambutol, tamoxifen, etc.
  • Involved in other clinical trials of any medicine within 1 month (or within 5 half-life periods)
  • Have maternity plan in 6 months
  • +1 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Southwest Hospital

Chongqing, Chongqing Municipality, 400038, China

Location

Related Publications (1)

  • Lv YX, Li QY, Duan P, Zhang MF, Liu B, Li SY, Zhao TT, Wang H, Liu Y, Yin ZQ. Safe CNV removal is crucial for successful hESC-RPE transplantation in wet age-related macular degeneration. Stem Cell Reports. 2025 Mar 11;20(3):102424. doi: 10.1016/j.stemcr.2025.102424. Epub 2025 Feb 27.

    PMID: 40020685DERIVED

MeSH Terms

Conditions

Macular Degeneration

Condition Hierarchy (Ancestors)

Retinal DegenerationRetinal DiseasesEye Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

January 7, 2016

First Posted

April 25, 2016

Study Start

May 1, 2015

Primary Completion

December 1, 2018

Study Completion

December 1, 2019

Last Updated

January 31, 2018

Record last verified: 2018-01

Locations

CN(1)