Stem Cells Therapy in Degenerative Diseases of the Retina

Retinitis Pigmentosa Clinical Trial

Official title:

Stem Cells Therapy in Degenerative Diseases of the Retina

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT03772938
• Other study ID #: IKiKO-KB-0012/143/13
• Status: Enrolling by invitation
• Phase: Phase 1
• Start date: December 13, 2018
• Est. completion date: March 31, 2020

Study information

• Verified date: December 2018
• Source: Pomeranian Medical University Szczecin
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 30
• Est. completion date: March 31, 2020
• Est. primary completion date: February 28, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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,

Related Conditions & MeSH terms

• Age Related Macular Degeneration
• Macular Degeneration
• Retinal Degeneration
• Retinitis
• Retinitis Pigmentosa
• Stargardt Disease 1

Intervention

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.

Locations

Country	Name	City	State
Poland	I Department of Ophthalmology	Szczecin

Sponsors (1)

Lead Sponsor	Collaborator
Pomeranian Medical University Szczecin

Country where clinical trial is conducted

Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients.	Best corrected visual acuity, ETDRS chart [number of letters]	12 months
Secondary	Intraocular pressure	Pascal tonometer [mmHg]	12 months
Secondary	Optic disk retinal nerve fiber layer	optical coherence tomography [um]	12 months
Secondary	Central macular thickness	optical coherence tomography [um]	12 months
Secondary	Ganglion cell complex thickness	optical coherence tomography [um]	12 months
Secondary	Choroidal thickness	Enhanced depth imaging optical coherence tomography [um]	12 months
Secondary	Choroidal volume	Enhanced depth imaging optical coherence tomography [mm3]	12 months
Secondary	Computed perimetry (30-2 and 10-2 module)	mean deviation, pattern standard deviation [B]	12 months
Secondary	Goldmann perimetry with color filters	[degrees]	12 months
Secondary	Contrast sensitivity	Pelli-Robson chart [number of letters]	12 months
Secondary	Function of the photoreceptors (rods and cones), inner retinal cells (bipolar and amacrine cells), and ganglion cells.	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 months
Secondary	Function of the photoreceptors	Multifocal electroretinography (mfERG) examination: retinal response density [V/degree 2],; culmination time of P1 wave in 6 rings [s].	12 months
Secondary	Function of ganglion cells	Pattern electroretinography (PERG) examination: amplitude of P50 and N95 waves [V],; culmination time of P50 wave [s].	12 months