TC/RP: Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa

Sponsor

Red de Terapia Celular (Industry)

Overall Status

Completed

CT.gov ID

NCT02280135

Collaborator

Spanish National Health System (Other), Hospital Universitario Virgen de la Arrixaca (Other), Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia (Other), Public Health Service, Murcia (Other), Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca (Other)

Enrollment

Location

Arms

Duration (Months)

0.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to evaluate the safety of intravitreal injection of autologous bone marrow stem cells in patients with retinitis pigmentosa by a prospective, single-center, randomized, parallel, double-blind, phase I placebo-controlled clinical trial.

Condition or Disease	Intervention/Treatment	Phase
Retinitis Pigmentosa	Biological: Intravitreal injection of Autologous bone marrow Stem Cell Other: Subconjunctival injection of saline	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

8 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

Official Title:

Phase I Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa

Study Start Date :

Nov 1, 2014

Actual Primary Completion Date :

Mar 1, 2017

Actual Study Completion Date :

Mar 1, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Intravitreal injection of Autologous bone marrow Stem Cell Patients included in the trial will receive a pars plana intravitreal injection of autologous mononuclear cells (MNC) of bone marrow (BM) in one eye (experimental group A or group). The eye in which autologous BM MNCs were injected will be determined randomly. The average dose will be 30 million of cells (5-60 million) diluted in 0.1 ml. of saline.	Biological: Intravitreal injection of Autologous bone marrow Stem Cell We will proceed to the injection of 0.1 ml of the suspension with the autologous MNC of BM avoiding coincide with conjunctival and scleral hole and perpendicular to the sclera. After the injection, a dry, sterile dressing will be placed to prevent reflux. Immediately, the perception of light, the movement of the central retinal artery, the venous pulse and the color of the papilla will be evaluated. If necessary, anterior chamber paracentesis will be performed. After injection, the patient will remain with closed eyes and sitting, waiting for cells will be deposited on the bottom of the eyeball and for 3 days will instill tropicamide collyrium every 12 hours.
Placebo Comparator: Subconjunctival injection of saline Patients included in the trial will receive a subconjunctival injection of 0.1 ml of saline (SF) (placebo) in the fellow eye (group B or control group). In this way the patient will receive an injection in the control eye but avoid the risks of intraocular injection.	Other: Subconjunctival injection of saline In the control eye, will make a subconjunctival injection of 0.1 ml saline. To realize contralateral injection gloves and all surgical materials will change, trying extreme measures of sterility. After injection, the patient will remain with closed eyes and sitting, waiting for cells will be deposited on the bottom of the eyeball and for 3 days will instill tropicamide collyrium every 12 hours.

Arm

Intervention/Treatment

Experimental: Intravitreal injection of Autologous bone marrow Stem Cell

Patients included in the trial will receive a pars plana intravitreal injection of autologous mononuclear cells (MNC) of bone marrow (BM) in one eye (experimental group A or group). The eye in which autologous BM MNCs were injected will be determined randomly. The average dose will be 30 million of cells (5-60 million) diluted in 0.1 ml. of saline.

Biological: Intravitreal injection of Autologous bone marrow Stem Cell

We will proceed to the injection of 0.1 ml of the suspension with the autologous MNC of BM avoiding coincide with conjunctival and scleral hole and perpendicular to the sclera. After the injection, a dry, sterile dressing will be placed to prevent reflux. Immediately, the perception of light, the movement of the central retinal artery, the venous pulse and the color of the papilla will be evaluated. If necessary, anterior chamber paracentesis will be performed. After injection, the patient will remain with closed eyes and sitting, waiting for cells will be deposited on the bottom of the eyeball and for 3 days will instill tropicamide collyrium every 12 hours.

Placebo Comparator: Subconjunctival injection of saline

Patients included in the trial will receive a subconjunctival injection of 0.1 ml of saline (SF) (placebo) in the fellow eye (group B or control group). In this way the patient will receive an injection in the control eye but avoid the risks of intraocular injection.

Other: Subconjunctival injection of saline

In the control eye, will make a subconjunctival injection of 0.1 ml saline. To realize contralateral injection gloves and all surgical materials will change, trying extreme measures of sterility. After injection, the patient will remain with closed eyes and sitting, waiting for cells will be deposited on the bottom of the eyeball and for 3 days will instill tropicamide collyrium every 12 hours.

Outcome Measures

Primary Outcome Measures

Rate of serious and non-serious adverse events related with the use of bone marrow mononuclear cells in patients with retinitis pigmentosa [12 months from baseline]

Secondary Outcome Measures

Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25). [12 months from baseline]
Visual acuity (VA): Test ETDRS (Early Treatment Diabetic Retinopathy Study). [12 months from baseline]
Color Vision: Ishihara Color Test. [12 months from baseline]
Contrast sensitivity: CSV-1000E. [12 months from baseline]
Intraocular pressure (IOP): measured in mm Hg with applanation tonometer Haag Streit AT 900. [12 months from baseline]
Examination of the anterior and posterior pole: Made with biomicroscopy (BMC). [12 months from baseline]
Width of retinal macula layer and nerve fiber: Measured with Optical Coherence Tomography Spectral domain (OCT)(Topcon 3D OCT-2000 Spectral Domain OCT). [12 months from baseline]
Visual field (VF) and macular sensitivity (The Humphrey perimeter). [12 months from baseline]
Study eye fundus: Made by Retinography and Angiography fluorescein. [12 months from baseline]
Electrical retinal function: electroretinogram (ERG) (altered / unaltered). [12 months from baseline]
Visual evoked potentials with Pattern Reversal (VEP) (altered / no altered). [12 months from baseline]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 70 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Retinitis Pigmentosa bilateral diagnosis.
Visual acuity (measured with ETDRS) less than or equal to 20/70 and visual field below 30° central in both eyes.
Signed informed consent
Warranty sufficient adherence to protocol. It must fulfill all inclusion criteria.

Exclusion Criteria:

Concurrence of any systemic or ocular disease that precludes or affects tracking study variables. Specifically retinal involvement with diabetes mellitus, glaucoma, macular degeneration or age.
Eye surgery in the previous 6 months.
Patients who are pregnant.
Patients with active lactation.
Physically fertile patients, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle or sexual orientation excludes sexual intercourse with a man and women whose partners have been sterilized by vasectomy or other methods, UNLESS tHEY are using a reliable contraceptive method.

This birth control method can be:

Complete abstinence from sexual intercourse
Surgical sterilization (tubal ligation)
Surgical sterilization partner (vasectomy)
Implanted or injectable hormonal contraceptives and oral.
Patients with cardiac disease, renal, hepatic, systemic, immune that might influence the survival of patients during the test.
Positive serology for hepatitis B, hepatitis C or HIV.
Clinical criteria or anesthetics that contraindicate the sedation or the extraction of BM (Altered coagulation system or anticoagulated patient with inability to withdraw anticoagulation, hemodynamic instability, altered skin in the puncture site, etc.)
Participation in other clinical trials.
Inability to sign informed consent or understanding.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Universitary Hospital Virgen de la Arrixaca	El Palmar	Murcia	Spain	30120

Sponsors and Collaborators

Red de Terapia Celular
Spanish National Health System
Hospital Universitario Virgen de la Arrixaca
Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
Public Health Service, Murcia
Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca

Investigators

Principal Investigator: María Elena Rodriguez González-Herrero, MD, Hospital Universitario Virgen de la Arrixaca

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Red de Terapia Celular

ClinicalTrials.gov Identifier:

NCT02280135

Other Study ID Numbers:

TC/RP
2012-000618-12

First Posted:

Oct 31, 2014

Last Update Posted:

Mar 30, 2017

Last Verified:

Mar 1, 2017

Additional relevant MeSH terms:

Retinitis

Retinitis Pigmentosa

Study Results

No Results Posted as of Mar 30, 2017