Clinical trial NCT03772938: Stem Cells Therapy in Degenerative Diseases of the Retina
Official title: Stem Cells Therapy in Degenerative Diseases of the Retina
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Retinal Degeneration
Retinitis Pigmentosa
Age Related Macular Degeneration
Stargardt Disease 1
Keywords: stem cells
Retinitis Pigmentosa
Age Related Macular Degeneration
Stargardt Disease
electroretinography
Lead sponsor: Pomeranian Medical University Szczecin
Sponsor class: OTHER
First posted: 2018-12-12
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

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.

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.

Eligibility 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,

Primary outcomes:
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: 12 months
Secondary outcomes:
measure: Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients.
timeFrame: 12 months
measure: Intraocular pressure
timeFrame: 12 months
measure: Optic disk retinal nerve fiber layer
timeFrame: 12 months
measure: Central macular thickness
timeFrame: 12 months
measure: Ganglion cell complex thickness
timeFrame: 12 months
measure: Choroidal thickness
timeFrame: 12 months
measure: Choroidal volume
timeFrame: 12 months
measure: Computed perimetry (30-2 and 10-2 module)
timeFrame: 12 months
measure: Goldmann perimetry with color filters
timeFrame: 12 months
measure: Contrast sensitivity
timeFrame: 12 months
measure: Function of the photoreceptors (rods and cones), inner retinal cells (bipolar and amacrine cells), and ganglion cells.
timeFrame: 12 months
measure: Function of the photoreceptors
timeFrame: 12 months
measure: Function of ganglion cells
timeFrame: 12 months
Other outcomes:
Locations:
city: Szczecin
country: Poland
facility: I Department of Ophthalmology
Study officials:
affiliation: Pomeranian Medical University
name: Bogusław Machaliński, MD, PhD
role: STUDY_CHAIR
affiliation: Pomeranian Medical University
name: Anna Machalińska, MD, PhD
role: STUDY_DIRECTOR
References: