NCT03772938

Brief Summary

The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells administered intravitreously in the subjects with degenerative diseases of the retina.

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
30

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Dec 2018

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

First Submitted

Initial submission to the registry

November 3, 2018

Completed
1 month until next milestone

First Posted

Study publicly available on registry

December 12, 2018

Completed
1 day until next milestone

Study Start

First participant enrolled

December 13, 2018

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 28, 2020

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2020

Completed
Last Updated

December 17, 2018

Status Verified

December 1, 2018

Enrollment Period

1.2 years

First QC Date

November 3, 2018

Last Update Submit

December 14, 2018

Conditions

Retinal DegenerationRetinitis PigmentosaAge Related Macular DegenerationStargardt Disease 1

Keywords

stem cellsRetinitis PigmentosaAge Related Macular DegenerationStargardt Diseaseelectroretinography

Outcome Measures

Primary Outcomes (1)

  • Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

    Confirm the safety of autologous bone marrow stem/progenitor cell intravitreal injection in enrolled patients by repeated follow-up over one year with clinical evaluations.

    12 months

Secondary Outcomes (13)

  • Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients.

    12 months

  • Intraocular pressure

    12 months

  • Optic disk retinal nerve fiber layer

    12 months

  • Central macular thickness

    12 months

  • Ganglion cell complex thickness

    12 months

  • +8 more secondary outcomes

Study Arms (2)

Stem/progenitor cells transplantation

ACTIVE COMPARATOR

Intervention: A single intravitreal injection of autologous bone marrow-derived stem/progenitor cells will be performed.

Biological: Stem/progenitor cells transplantation

Standard treatment of degenerative disease of retina

SHAM COMPARATOR

Symptomatic treatment of degenerative disease of retina without biologic cell-based treatment

Biological: Stem/progenitor cells transplantation

Interventions

Stem/progenitor cells transplantationBIOLOGICAL

Human autologous bone marrow-derived stem/progenitor cell transplantation administered as an intravitreal injection in patients with degenerative disease of retina.

Standard treatment of degenerative disease of retinaStem/progenitor cells transplantation

Eligibility Criteria

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

You may qualify if:

  • diagnosed degenerative disease of the retina,
  • age 18-65 years,
  • best corrected visual acuity max. 0,2 (Snellen letter chart),
  • good understanding of the protocol and willingness to consent,
  • signed informed consent.

You may not qualify if:

  • concomitant eye disease (glaucoma, etc.)
  • concomitant of other systemic disease or diseases,
  • inflammation (high protein or lymphocytosis in the CSF), active infections.
  • diabetes,
  • cardio-vascular disorders,
  • cancer,
  • autoimmune diseases,
  • renal failure,
  • impaired hepatic function,
  • subject unwilling or unable to comply with the requirements of the protocol,
  • patient has been treated previously with any cellular therapy,

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

I Department of Ophthalmology

Szczecin, 70-111, Poland

Location

MeSH Terms

Conditions

Retinal DegenerationRetinitis PigmentosaMacular DegenerationStargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DiseasesRetinal DystrophiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Bogusław Machaliński, MD, PhD

    Pomeranian Medical University

    STUDY CHAIR

  • Anna Machalińska, MD, PhD

    Pomeranian Medical University

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, PhD candidate, principal investigator

Study Record Dates

First Submitted

November 3, 2018

First Posted

December 12, 2018

Study Start

December 13, 2018

Primary Completion

February 28, 2020

Study Completion

March 31, 2020

Last Updated

December 17, 2018

Record last verified: 2018-12

Data Sharing

IPD Sharing
Will not share

Locations

PL(1)