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

NCT02280135 | Completed Phase 1 DMC

• 2 other identifiers: TC/RP2012-000618-12
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

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.

Conditions

Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (11)

• 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

Study Arms (2)

Intravitreal injection of Autologous bone marrow Stem Cell

EXPERIMENTAL

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

Subconjunctival injection of saline

PLACEBO COMPARATOR

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

Interventions

Intravitreal injection of Autologous bone marrow Stem Cell BIOLOGICAL

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.

Intravitreal injection of Autologous bone marrow Stem Cell

Subconjunctival injection of saline OTHER

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.

Subconjunctival injection of saline

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

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

Study Sites (1)

Clinical Universitary Hospital Virgen de la Arrixaca

El Palmar, Murcia, 30120, Spain

Location

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• María Elena Rodriguez González-Herrero, MD

  Hospital Universitario Virgen de la Arrixaca

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 22, 2014
• First Posted: October 31, 2014
• Study Start: November 1, 2014
• Primary Completion: March 1, 2017
• Study Completion: March 1, 2017
• Last Updated: March 30, 2017

Record last verified: 2017-03

Locations

ES (1)