NCT04224207

Brief Summary

The aim of this study is to determine if umbilical cord Wharton's jelly derived mesenchymal stem cells implanted in sub-tenon space have beneficial effects on visual functions in retinitis pigmentosa patients by reactivating the degenerated photoreceptors in dormant phase.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
32

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Apr 2019

Shorter than P25 for phase_3

Geographic Reach
1 country

2 active sites

Status
completed

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

April 1, 2019

Completed
7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 30, 2019

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2020

Completed
5 days until next milestone

First Submitted

Initial submission to the registry

January 6, 2020

Completed
7 days until next milestone

First Posted

Study publicly available on registry

January 13, 2020

Completed
Last Updated

January 13, 2020

Status Verified

January 1, 2020

Enrollment Period

7 months

First QC Date

January 6, 2020

Last Update Submit

January 8, 2020

Conditions

Retinitis PigmentosaInherited Retinal Dystrophy

Keywords

Retinitis pigmentosaWharton's jelly derived mesenchimal stem cellSubtenon spaceUmbilical cord

Outcome Measures

Primary Outcomes (1)

  • ETDRS visual acuity

    The visual acuity scores obtained from the baseline testing and the final examination were analyzed and compared statistically to determine effectiveness.

    Change from baseline visual acuity at 6 months

Secondary Outcomes (1)

  • Outer retinal thickness

    Change from baseline outer retinal thickness at 6 months

Study Arms (2)

Before application

ACTIVE COMPARATOR

RP patients with progressive visual acuity and visual field loss: before stem cell application.

Biological: Wharton's jelly derived mesenchymal stem cell

After application

ACTIVE COMPARATOR

RP patients, after stem cell applications.

Biological: Wharton's jelly derived mesenchymal stem cell

Interventions

Wharton's jelly derived mesenchymal stem cellBIOLOGICAL

The mesenchymal cells that were used in this study were isolated from Wharton's jelly of the umbilical cord that was collected allogenicly from a single donor with the mother's consent. All cell preparation and cultivation procedures were conducted in a current Good Manufacturing Practice (cGMP) accredited laboratory (Onkim Stem Cell Technologies, Turkey).Cells were solubilized from cryopreservation before being made ready for injection. Average cell viability for each treatment was over 90.0% and each patient received cell numbers between 2-6x106 in a 1.5 ml saline solution .

After applicationBefore application

Eligibility Criteria

Age18 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64)

You may qualify if:

  • 18 years of age or older;
  • Diagnosis of any phenotypic or genotypic variation of RP, confirmed by clinical history, fundus appearance, visual field (VF), electroretinogram (ERG) and genetic mutation analysis;
  • Having experienced various degrees of VF loss;
  • BCVA from 50 letters to 110 letters in the ETDRS chart testing (Topcon CC-100 XP, Japan);
  • Mean deviation (MD) values ranging between -33.0 and -5.0 dB with Compass visual field analysis (threshold 24-2, Sita Standard, Stimulus 3-white);
  • Intraocular pressure (IOP) of \<22 mmHg.

You may not qualify if:

  • The presence of cataracts or other media opacity that might affect the VF, MD, or ERG recordings;
  • The presence of glaucoma, which causes visual field and optic disc changes;
  • The presence of any systemic disorder (e.g.,diabetes, neurological disease, or uncontrolled systemic hypertension) that may affect visual function;
  • The habit of smoking.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Ankara University Biotechnology Institute

Ankara, Türkiye, 06312, Turkey (Türkiye)

Location

Umut Arslan

Ankara, Türkiye, 06312, Turkey (Türkiye)

Location

Related Publications (8)

  • Musial-Wysocka A, Kot M, Sulkowski M, Badyra B, Majka M. Molecular and Functional Verification of Wharton's Jelly Mesenchymal Stem Cells (WJ-MSCs) Pluripotency. Int J Mol Sci. 2019 Apr 12;20(8):1807. doi: 10.3390/ijms20081807.

    PMID: 31013696RESULT

  • Leow SN, Luu CD, Hairul Nizam MH, Mok PL, Ruhaslizan R, Wong HS, Wan Abdul Halim WH, Ng MH, Ruszymah BH, Chowdhury SR, Bastion ML, Then KY. Safety and Efficacy of Human Wharton's Jelly-Derived Mesenchymal Stem Cells Therapy for Retinal Degeneration. PLoS One. 2015 Jun 24;10(6):e0128973. doi: 10.1371/journal.pone.0128973. eCollection 2015.

    PMID: 26107378RESULT

  • Rani S, Ryan AE, Griffin MD, Ritter T. Mesenchymal Stem Cell-derived Extracellular Vesicles: Toward Cell-free Therapeutic Applications. Mol Ther. 2015 May;23(5):812-823. doi: 10.1038/mt.2015.44. Epub 2015 Mar 19.

    PMID: 25868399RESULT

  • Canto-Soler V, Flores-Bellver M, Vergara MN. Stem Cell Sources and Their Potential for the Treatment of Retinal Degenerations. Invest Ophthalmol Vis Sci. 2016 Apr 1;57(5):ORSFd1-9. doi: 10.1167/iovs.16-19127.

    PMID: 27116661RESULT

  • Garg A, Yang J, Lee W, Tsang SH. Stem Cell Therapies in Retinal Disorders. Cells. 2017 Feb 2;6(1):4. doi: 10.3390/cells6010004.

    PMID: 28157165RESULT

  • Mohamed EM, Abdelrahman SA, Hussein S, Shalaby SM, Mosaad H, Awad AM. Effect of human umbilical cord blood mesenchymal stem cells administered by intravenous or intravitreal routes on cryo-induced retinal injury. IUBMB Life. 2017 Mar;69(3):188-201. doi: 10.1002/iub.1608. Epub 2017 Feb 5.

    PMID: 28164440RESULT

  • Limoli PG, Vingolo EM, Limoli C, Scalinci SZ, Nebbioso M. Regenerative Therapy by Suprachoroidal Cell Autograft in Dry Age-related Macular Degeneration: Preliminary In Vivo Report. J Vis Exp. 2018 Feb 12;(132):56469. doi: 10.3791/56469.

    PMID: 29553543RESULT

  • Ozmert E, Arslan U. Management of retinitis pigmentosa by Wharton's jelly-derived mesenchymal stem cells: prospective analysis of 1-year results. Stem Cell Res Ther. 2020 Aug 12;11(1):353. doi: 10.1186/s13287-020-01870-w.

    PMID: 32787913DERIVED

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DystrophiesRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Umut Arslan, MD

    Ankara Universitesi Teknokent

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: Prospective, open-label clinical trial; The statistical comparisons were made primarily between the baseline and final values from the same eye. The parametric results for visual functions and structural changes were analyzed.
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principle investigator, MD

Study Record Dates

First Submitted

January 6, 2020

First Posted

January 13, 2020

Study Start

April 1, 2019

Primary Completion

October 30, 2019

Study Completion

January 1, 2020

Last Updated

January 13, 2020

Record last verified: 2020-01

Data Sharing

IPD Sharing
Will not share

Locations

TU(2)