Stem Cells Therapy in Degenerative Diseases of the Retina

Pomeranian Medical University Szczecin

Status and phase: Unknown; Phase 1

Conditions: Age Related Macular Degeneration; Retinal Degeneration; Retinitis Pigmentosa; Stargardt Disease 1

Treatments: Biological: Stem/progenitor cells transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT03772938

IKiKO-KB-0012/143/13

Details and patient eligibility

About

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.

Full 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.

Enrollment: 30 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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,

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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30 participants in 2 patient groups

Stem/progenitor cells transplantation

Active Comparator group

Description:

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

Treatment:

Biological: Stem/progenitor cells transplantation

Standard treatment of degenerative disease of retina

Sham Comparator group

Description:

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

Treatment:

Biological: Stem/progenitor cells transplantation