Retinitis PigmentosaPhase 1TC/RPNCT02280135
Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa
A Phase 1 study testing Intravitreal injection of Autologous bone marrow Stem Cell and Subconjunctival injection of saline in people with Retinitis Pigmentosa, run by Red de Terapia Celular at 1 site in Spain.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- Intravitreal injection of Autologous bone marrow Stem Cell and Subconjunctival injection of saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 1 siteSpain
- Study dates
- Nov 2014to Mar 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country.
Spain1 site
Clinical Universitary Hospital Virgen de la Arrixaca
El Palmar, Murcia, 30120
About this study
Official title: Phase I Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa
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
- Being tested
- Intravitreal injection of Autologous bone marrow Stem Cell, Subconjunctival injection of saline
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravitreal injection of Autologous bone marrow Stem CellBiological
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.
Subconjunctival injection of salineOther
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Other: Subconjunctival injection of saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of serious and non-serious adverse events related with the use of bone marrow mononuclear cells in patients with retinitis pigmentosa
Measured: 12 months from baseline
Secondary outcomes (11)
Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25).
Measured: 12 months from baseline
Visual acuity (VA): Test ETDRS (Early Treatment Diabetic Retinopathy Study).
Measured: 12 months from baseline
Color Vision: Ishihara Color Test.
Measured: 12 months from baseline
Contrast sensitivity: CSV-1000E.
Measured: 12 months from baseline
Intraocular pressure (IOP): measured in mm Hg with applanation tonometer Haag Streit AT 900.
Measured: 12 months from baseline
Examination of the anterior and posterior pole: Made with biomicroscopy (BMC).
Measured: 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).
Measured: 12 months from baseline
Visual field (VF) and macular sensitivity (The Humphrey perimeter).
Measured: 12 months from baseline
Study eye fundus: Made by Retinography and Angiography fluorescein.
Measured: 12 months from baseline
Electrical retinal function: electroretinogram (ERG) (altered / unaltered).
Measured: 12 months from baseline
Visual evoked potentials with Pattern Reversal (VEP) (altered / no altered).
Measured: 12 months from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2014 (estimated)
- Study startNov 2014
- Primary completionMar 2017
- Study completionMar 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Red de Terapia Celular
- Sponsor type
- Industry (pharma, biotech, devices)
- 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
- Responsible party
- Sponsor
- Investigators
- María Elena Rodriguez González-Herrero, MD, Principal investigator, Hospital Universitario Virgen de la Arrixaca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02280135
- Sponsor's protocol ID
- TC/RP
- Other IDs
- 2012-000618-12
- Acronym
- TC/RP
- Status last verified
- Mar 2017
- First submitted
- 22 Oct 2014
- Last updated
- 30 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT02280135
- Acronym / sponsor ID
- TC/RP / TC/RP
- Conditions
- Retinitis Pigmentosa
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Red de Terapia CelularIndustry (pharma, biotech, devices)
- 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
- Study start
- Nov 2014
- Primary completion
- Mar 2017 (actual)
- Study completion
- Mar 2017 (actual)
- First posted
- 31 Oct 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intravitreal injection of Autologous bone marrow Stem Cell | Experimental | Biological: Intravitreal injection of Autologous bone marrow Stem CellPatients 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. |
| Subconjunctival injection of saline | Placebo comparator | Other: Subconjunctival injection of salinePatients 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. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (14 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intravitreal injection of Autologous bone marrow Stem CellWe 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. | Biological | Intravitreal injection of Autologous bone marrow Stem Cell |
| Subconjunctival injection of salineIn 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. | Other | Subconjunctival injection of saline |
Primary outcome
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
11 secondary outcomes
Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25).
Time frame: 12 months from baseline
Visual acuity (VA): Test ETDRS (Early Treatment Diabetic Retinopathy Study).
Time frame: 12 months from baseline
Color Vision: Ishihara Color Test.
Time frame: 12 months from baseline
Contrast sensitivity: CSV-1000E.
Time frame: 12 months from baseline
Intraocular pressure (IOP): measured in mm Hg with applanation tonometer Haag Streit AT 900.
Time frame: 12 months from baseline
Examination of the anterior and posterior pole: Made with biomicroscopy (BMC).
Time frame: 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).
Time frame: 12 months from baseline
Visual field (VF) and macular sensitivity (The Humphrey perimeter).
Time frame: 12 months from baseline
Study eye fundus: Made by Retinography and Angiography fluorescein.
Time frame: 12 months from baseline
Electrical retinal function: electroretinogram (ERG) (altered / unaltered).
Time frame: 12 months from baseline
Visual evoked potentials with Pattern Reversal (VEP) (altered / no altered).
Time frame: 12 months from baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
María Elena Rodriguez González-Herrero, MD principal investigator
Hospital Universitario Virgen de la Arrixaca
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intravitreal injection of Autologous bone marrow Stem Cell and Subconjunctival injection of saline in people with Retinitis Pigmentosa, run by Red de Terapia Celular at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2017.