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

• ClinicalTrials.gov Identifier: NCT02280135
• Recruitment Status: Completed
• First Posted: October 31, 2014
• Last Update Posted: March 30, 2017
• Sponsor: Red de Terapia Celular
• Collaborators: 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
• Information provided by (Responsible Party): Red de Terapia Celular

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 (Clinical Trial)
• 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: November 2014
• Actual Primary Completion Date: March 2017
• Actual Study Completion Date: March 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.

Outcome Measures

Primary Outcome Measures :

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

Secondary Outcome Measures :

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

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 70 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Spain

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

More Information

• Responsible Party: Red de Terapia Celular
• ClinicalTrials.gov Identifier: NCT02280135
• Other Study ID Numbers: TC/RP; 2012-000618-12 ( EudraCT Number )
• First Posted: October 31, 2014
• Last Update Posted: March 30, 2017
• Last Verified: March 2017

Additional relevant MeSH terms:

Retinitis; Retinitis Pigmentosa; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration; Genetic Diseases, Inborn