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Stem Cells Therapy in Degenerative Diseases of the Retina

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ClinicalTrials.gov Identifier: NCT03772938
Recruitment Status : Enrolling by invitation
First Posted : December 12, 2018
Last Update Posted : December 17, 2018
Sponsor:
Information provided by (Responsible Party):
Marta P. Wiącek, Pomeranian Medical University Szczecin

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.

Condition or disease Intervention/treatment Phase
Retinal Degeneration Retinitis Pigmentosa Age Related Macular Degeneration Stargardt Disease 1 Biological: Stem/progenitor cells transplantation Phase 1

Detailed Description:
Degenerative diseases of the retina are challenging for ophthalmologists. This is a common term that covers heterogenous group of diseases, f.e. retinitis pigmentosa, Stargardt disease, Best's disease or age related macular degeneration. Undetermined etiology, wide range of factors that may trigger the onset of the disease and modulate it's course impede the implementation of an effective treatment. Currently, a stem cells therapy seems to be promising option in patients with degenerative diseases of the retina. The purpose of this prospective, nonrandomized, open label, pilot study is to conduct the investigation of the safety and efficacy of intravitreal injection of autologous bone marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects with degenerative diseases of the retina. Especially, this clinical trial is designated to test the therapeutic (pro-regenerative and neuro-protective) functions of different stem/progenitor cell populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a documented history of degenerative disease of the retina prior to study enrollment. Next, autologous bone marrow-isolated stem/progenitor cells intravitreous injection will be performed. Finally, treatment safety, adverse events and exploratory parameters, including best corrected visual acuity, visual field and electroretinography parameters, to establish disease progression rate will be recorded throughout the duration of the post-treatment follow up period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Stem Cells Therapy in Degenerative Diseases of the Retina
Actual Study Start Date : December 13, 2018
Estimated Primary Completion Date : February 28, 2020
Estimated Study Completion Date : March 31, 2020


Arm Intervention/treatment
Active Comparator: Stem/progenitor cells transplantation
Intervention: A single intravitreal injection of autologous bone marrow-derived stem/progenitor cells will be performed.
Biological: Stem/progenitor cells transplantation
Human autologous bone marrow-derived stem/progenitor cell transplantation administered as an intravitreal injection in patients with degenerative disease of retina.

Sham Comparator: Standard treatment of degenerative disease of retina
Symptomatic treatment of degenerative disease of retina without biologic cell-based treatment
Biological: Stem/progenitor cells transplantation
Human autologous bone marrow-derived stem/progenitor cell transplantation administered as an intravitreal injection in patients with degenerative disease of retina.




Primary Outcome Measures :
  1. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 12 months ]
    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.


Secondary Outcome Measures :
  1. Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients. [ Time Frame: 12 months ]
    Best corrected visual acuity, ETDRS chart [number of letters]

  2. Intraocular pressure [ Time Frame: 12 months ]
    Pascal tonometer [mmHg]

  3. Optic disk retinal nerve fiber layer [ Time Frame: 12 months ]
    optical coherence tomography [um]

  4. Central macular thickness [ Time Frame: 12 months ]
    optical coherence tomography [um]

  5. Ganglion cell complex thickness [ Time Frame: 12 months ]
    optical coherence tomography [um]

  6. Choroidal thickness [ Time Frame: 12 months ]
    Enhanced depth imaging optical coherence tomography [um]

  7. Choroidal volume [ Time Frame: 12 months ]
    Enhanced depth imaging optical coherence tomography [mm3]

  8. Computed perimetry (30-2 and 10-2 module) [ Time Frame: 12 months ]
    mean deviation, pattern standard deviation [B]

  9. Goldmann perimetry with color filters [ Time Frame: 12 months ]
    [degrees]

  10. Contrast sensitivity [ Time Frame: 12 months ]
    Pelli-Robson chart [number of letters]

  11. Function of the photoreceptors (rods and cones), inner retinal cells (bipolar and amacrine cells), and ganglion cells. [ Time Frame: 12 months ]

    Electroretinography (ERG) examination:

    • amplitude of a and b waves [V],
    • culmination time of a and b waves [s],
    • culmination time of q1-q3 waves [s].

  12. Function of the photoreceptors [ Time Frame: 12 months ]

    Multifocal electroretinography (mfERG) examination:

    • retinal response density [V/degree 2],
    • culmination time of P1 wave in 6 rings [s].

  13. Function of ganglion cells [ Time Frame: 12 months ]

    Pattern electroretinography (PERG) examination:

    • amplitude of P50 and N95 waves [V],
    • culmination time of P50 wave [s].



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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,

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03772938


Locations
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Poland
I Department of Ophthalmology
Szczecin, Poland, 70-111
Sponsors and Collaborators
Pomeranian Medical University Szczecin
Investigators
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Study Chair: Bogusław Machaliński, MD, PhD Pomeranian Medical University
Study Director: Anna Machalińska, MD, PhD Pomeranian Medical University

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Responsible Party: Marta P. Wiącek, MD, PhD candidate, principal investigator, Pomeranian Medical University Szczecin
ClinicalTrials.gov Identifier: NCT03772938     History of Changes
Other Study ID Numbers: IKiKO-KB-0012/143/13
First Posted: December 12, 2018    Key Record Dates
Last Update Posted: December 17, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Marta P. Wiącek, Pomeranian Medical University Szczecin:
stem cells
Retinitis Pigmentosa
Age Related Macular Degeneration
Stargardt Disease
electroretinography
Additional relevant MeSH terms:
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Macular Degeneration
Retinitis
Retinitis Pigmentosa
Retinal Degeneration
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Genetic Diseases, Inborn