Retinal DegenerationPhase 1NCT03772938
Stem Cells Therapy in Degenerative Diseases of the Retina
A Phase 1 study testing Stem/progenitor cells transplantation in people with Retinal Degeneration and Retinitis Pigmentosa, run by Pomeranian Medical University Szczecin at 1 site in Poland.
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Retinal Degeneration, Retinitis Pigmentosa, Age Related Macular Degeneration
- What is being tested?
- Stem/progenitor cells transplantation
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 sitePoland
- Study dates
- 13 Dec 2018to 31 Mar 2020
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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,
Show all 11Show fewer
- 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,
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the detailed 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.
- Conditions
- Retinal Degeneration, Retinitis Pigmentosa, Age Related Macular Degeneration, Stargardt Disease 1
- Being tested
- Stem/progenitor cells transplantation
- Keywords
- stem cells, Retinitis Pigmentosa, Age Related Macular Degeneration, Stargardt Disease, electroretinography
- Medical subject headings
- Retinal Degeneration, Retinitis Pigmentosa, Macular Degeneration, Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Stem/progenitor cells transplantationBiological
Human autologous bone marrow-derived stem/progenitor cell transplantation administered as an intravitreal injection in patients with degenerative disease of retina.
| Study group | Role | What they receive |
|---|---|---|
| Stem/progenitor cells transplantation | Active comparator | Intervention: A single intravitreal injection of autologous bone marrow-derived stem/progenitor cells will be performed. Receives: Biological: Stem/progenitor cells transplantation |
| Standard treatment of degenerative disease of retina | Sham comparator | Symptomatic treatment of degenerative disease of retina without biologic cell-based treatment Receives: Biological: Stem/progenitor cells transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 months
Secondary outcomes (13)
Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients.
Best corrected visual acuity, ETDRS chart [number of letters]
Measured: 12 months
Intraocular pressure
Pascal tonometer [mmHg]
Measured: 12 months
Optic disk retinal nerve fiber layer
optical coherence tomography [um]
Measured: 12 months
Central macular thickness
optical coherence tomography [um]
Measured: 12 months
Ganglion cell complex thickness
optical coherence tomography [um]
Measured: 12 months
Choroidal thickness
Enhanced depth imaging optical coherence tomography [um]
Measured: 12 months
Choroidal volume
Enhanced depth imaging optical coherence tomography [mm3]
Measured: 12 months
Computed perimetry (30-2 and 10-2 module)
mean deviation, pattern standard deviation [B]
Measured: 12 months
Goldmann perimetry with color filters
[degrees]
Measured: 12 months
Contrast sensitivity
Pelli-Robson chart [number of letters]
Measured: 12 months
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].
Measured: 12 months
Function of the photoreceptors
Multifocal electroretinography (mfERG) examination:
- retinal response density [V/degree 2],
- culmination time of P1 wave in 6 rings [s].
Measured: 12 months
Function of ganglion cells
Pattern electroretinography (PERG) examination:
- amplitude of P50 and N95 waves [V],
- culmination time of P50 wave [s].
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2018
- Study start13 Dec 2018
- Primary completion28 Feb 2020 (estimated)
- Study completion31 Mar 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinal DegenerationStudies 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
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pomeranian Medical University Szczecin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Marta P. Wiącek, Pomeranian Medical University Szczecin
- Investigators
- Bogusław Machaliński, MD, PhD, Study chair, Pomeranian Medical UniversityAnna Machalińska, MD, PhD, Study director, Pomeranian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03772938
- Sponsor's protocol ID
- IKiKO-KB-0012/143/13
- Status last verified
- Dec 2018
- First submitted
- 3 Nov 2018
- Last updated
- 17 Dec 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Stem Cells Therapy in Degenerative Diseases of the Retina
- ClinicalTrials.gov ID
- NCT03772938
- Acronym / sponsor ID
- IKiKO-KB-0012/143/13
- Conditions
- Retinal Degeneration, Retinitis Pigmentosa, Age Related Macular Degeneration, Stargardt Disease 1
- Keywords
- stem cells, Retinitis Pigmentosa, Age Related Macular Degeneration, Stargardt Disease, electroretinography
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Pomeranian Medical University SzczecinUniversity, hospital or other
- Study start
- 13 Dec 2018 (actual)
- Primary completion
- 28 Feb 2020 (estimated)
- Study completion
- 31 Mar 2020 (estimated)
- First posted
- 12 Dec 2018 (actual)
- 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 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.
Detailed 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.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Stem/progenitor cells transplantation | Active comparator | Biological: Stem/progenitor cells transplantationIntervention: A single intravitreal injection of autologous bone marrow-derived stem/progenitor cells will be performed. |
| Standard treatment of degenerative disease of retina | Sham comparator | Biological: Stem/progenitor cells transplantationSymptomatic treatment of degenerative disease of retina without biologic cell-based treatment |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (11 listed)
- 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,
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 |
|---|---|---|
| Stem/progenitor cells transplantationHuman autologous bone marrow-derived stem/progenitor cell transplantation administered as an intravitreal injection in patients with degenerative disease of retina. | Biological | Standard treatment of degenerative disease of retina, Stem/progenitor cells transplantation |
Primary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 12 months
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.
13 secondary outcomes
Efficacy of autologous bone marrow stem/progenitor intravitreal injection in enrolled patients.
Time frame: 12 months
Intraocular pressure
Time frame: 12 months
Optic disk retinal nerve fiber layer
Time frame: 12 months
Central macular thickness
Time frame: 12 months
Ganglion cell complex thickness
Time frame: 12 months
Choroidal thickness
Time frame: 12 months
Choroidal volume
Time frame: 12 months
Computed perimetry (30-2 and 10-2 module)
Time frame: 12 months
Goldmann perimetry with color filters
Time frame: 12 months
Contrast sensitivity
Time frame: 12 months
Function of the photoreceptors (rods and cones), inner retinal cells (bipolar and amacrine cells), and ganglion cells.
Time frame: 12 months
Function of the photoreceptors
Time frame: 12 months
Function of ganglion cells
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2018.
- Individual participant dataSharing planned: No
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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.
Bogusław Machaliński, MD, PhD study chair
Pomeranian Medical University
Anna Machalińska, MD, PhD study director
Pomeranian Medical University
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 Stem/progenitor cells transplantation in people with Retinal Degeneration and Retinitis Pigmentosa, run by Pomeranian Medical University Szczecin at 1 site in Poland.
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 5 inclusion and 11 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: status not confirmed, as of the sponsor's last update on 17 Dec 2018. 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 Poland. 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?
Results are not available in the source record.
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?
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 (time frame: 12 months). 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 17 Dec 2018.