Retinitis PigmentosaPhase 1NCT01531348
Intravitreal Injection of MSCs in Retinitis Pigmentosa
A Phase 1 study testing BM-MSC in people with Retinitis Pigmentosa, run by Mahidol University at 1 site in Thailand.
Trial summary
The purpose of this study is to determine the feasibility and safety of adult human bone marrow-derived mesenchymal stem cells by intravitreal injection in patients with retinitis pigmentosa. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- BM-MSC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteThailand
- Study dates
- Apr 2012to Jul 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
- Retinitis pigmentosa patients diagnosed by ophthalmologists
- Age 18-65 years old
- Central visual field less than or equal to 20 degrees
- Best corrected visual acuity less than 6/120 by Snellen visual acuity chart
- Electroretinogram nonrecordable or the amplitudes were less than 25% of normal
Who cannot join
- Other eye conditions that could mask the interpretation of the results
- Unable to return for follow up
- Underlying diseases including asthma, heart failure, myocardial infarction, liver failure, renal failure
- Pregnant and lactating women
Trial locations and contacts
1 site in 1 country.
Thailand1 site
Siriraj Hospital Mahidol University
Bangkoknoi, Bangkok, 10700
About this study
Official title: Feasibility and Safety of Adult Human Bone Marrow-Derived Mesenchymal Stem Cells by Intravitreal Injection in Patients With Retinitis Pigmentosa
The purpose of this study is to determine the feasibility and safety of adult human bone marrow-derived mesenchymal stem cells by intravitreal injection in patients with retinitis pigmentosa.
Read the detailed description
Retinitis pigmentosa (RP) is an inherited disorder of the photoreceptor cells in the retina. Patients may lose vision since they were young or later in life. Currently, there are more than 60 genes identified as the cause of this condition, one of which, RPE65, has been studied in several gene therapy trials for Leber congenital amaurosis with promising results. Another treatment approach for RP is stem cell therapy. Studies in animal models of RP have shown that subretinal injection of bone marrow-derived mesenchymal stem cells may delay degenerative changes of photoreceptor cells.
- Conditions
- Retinitis Pigmentosa
- Being tested
- BM-MSC
- Keywords
- Retinitis pigmentosa, Bone marrow-derived mesenchymal stem cells, Intravitreal injection
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BM-MSCOther
Bone marrow-derived mesenchymal stem cells 1 million cells in balanced salt solution 100 microlitres will be injected into the vitreous cavity.
| Study group | Role | What they receive |
|---|---|---|
| BM-MSC | Experimental | Bone marrow-derived mesenchymal stem cells 1 million cells in balanced salt solution 100 microlitres will be injected into the vitreous cavity. Receives: Other: BM-MSC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in laser flare and cell measurements
Measured: up to 12 months
Secondary outcomes (1)
Change from baseline in visual function tests
Measured: up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2012 (estimated)
- Study startApr 2012
- Primary completionJul 2020
- Study completionJul 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- More trials run by Mahidol UniversityThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mahidol University
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health, Thailand
- Responsible party
- Principal investigator, La-ongsri Atchaneeyasakul, Mahidol University
- Investigators
- La-ongsri Atchaneeyasakul, MD, Principal investigator, Siriraj Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01531348
- Sponsor's protocol ID
- RP-001
- Status last verified
- Aug 2021
- First submitted
- 6 Feb 2012
- Last updated
- 8 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Hartong DT, Berson EL, Dryja TP. Retinitis pigmentosa. Lancet. 2006 Nov 18;368(9549):1795-809. doi: 10.1016/S0140-6736(06)69740-7.
PubMed 17113430Kociok N. Can the injection of the patient's own bone marrow-derived stem cells preserve cone vision in retinitis pigmentosa and other diseases of the eye? Graefes Arch Clin Exp Ophthalmol. 2005 Mar;243(3):187-8. doi: 10.1007/s00417-004-1062-0. Epub 2004 Nov 23. No abstract available.
PubMed 15565292Arnhold S, Absenger Y, Klein H, Addicks K, Schraermeyer U. Transplantation of bone marrow-derived mesenchymal stem cells rescue photoreceptor cells in the dystrophic retina of the rhodopsin knockout mouse. Graefes Arch Clin Exp Ophthalmol. 2007 Mar;245(3):414-22. doi: 10.1007/s00417-006-0382-7. Epub 2006 Aug 4.
PubMed 16896916Wang S, Lu B, Girman S, Duan J, McFarland T, Zhang QS, Grompe M, Adamus G, Appukuttan B, Lund R. Non-invasive stem cell therapy in a rat model for retinal degeneration and vascular pathology. PLoS One. 2010 Feb 15;5(2):e9200. doi: 10.1371/journal.pone.0009200.
PubMed 20169166Tuekprakhon A, Sangkitporn S, Trinavarat A, Pawestri AR, Vamvanij V, Ruangchainikom M, Luksanapruksa P, Pongpaksupasin P, Khorchai A, Dambua A, Boonchu P, Yodtup C, Uiprasertkul M, Sangkitporn S, Atchaneeyasakul LO. Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa. Stem Cell Res Ther. 2021 Jan 9;12(1):52. doi: 10.1186/s13287-020-02122-7.
PubMed 33422139
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility and Safety of Adult Human Bone Marrow-Derived Mesenchymal Stem Cells by Intravitreal Injection in Patients With Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT01531348
- Acronym / sponsor ID
- RP-001
- Conditions
- Retinitis Pigmentosa
- Keywords
- Retinitis pigmentosa, Bone marrow-derived mesenchymal stem cells, Intravitreal injection
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Mahidol UniversityUniversity, hospital or other
- Collaborators
- Ministry of Health, Thailand
- Study start
- Apr 2012 (actual)
- Primary completion
- Jul 2020 (actual)
- Study completion
- Jul 2020 (actual)
- First posted
- 10 Feb 2012 (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 determine the feasibility and safety of adult human bone marrow-derived mesenchymal stem cells by intravitreal injection in patients with retinitis pigmentosa.
Detailed description
Retinitis pigmentosa (RP) is an inherited disorder of the photoreceptor cells in the retina. Patients may lose vision since they were young or later in life. Currently, there are more than 60 genes identified as the cause of this condition, one of which, RPE65, has been studied in several gene therapy trials for Leber congenital amaurosis with promising results. Another treatment approach for RP is stem cell therapy. Studies in animal models of RP have shown that subretinal injection of bone marrow-derived mesenchymal stem cells may delay degenerative changes of photoreceptor cells.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BM-MSC | Experimental | Other: BM-MSCBone marrow-derived mesenchymal stem cells 1 million cells in balanced salt solution 100 microlitres will be injected into the vitreous cavity. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Retinitis pigmentosa patients diagnosed by ophthalmologists
- Age 18-65 years old
- Central visual field less than or equal to 20 degrees
- Best corrected visual acuity less than 6/120 by Snellen visual acuity chart
- Electroretinogram nonrecordable or the amplitudes were less than 25% of normal
Key exclusion criteria (4 listed)
- Other eye conditions that could mask the interpretation of the results
- Unable to return for follow up
- Underlying diseases including asthma, heart failure, myocardial infarction, liver failure, renal failure
- Pregnant and lactating women
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 |
|---|---|---|
| BM-MSCBone marrow-derived mesenchymal stem cells 1 million cells in balanced salt solution 100 microlitres will be injected into the vitreous cavity. | Other | BM-MSC |
Primary outcome
Change from baseline in laser flare and cell measurements
Time frame: up to 12 months
1 secondary outcome
Change from baseline in visual function tests
Time frame: up to 12 months
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 8 Sep 2021.
- Background: PubMed 17113430Hartong DT, Berson EL, Dryja TP. Retinitis pigmentosa. Lancet. 2006 Nov 18;368(9549):1795-809. doi: 10.1016/S0140-6736(06)69740-7.
- Background: PubMed 15565292Kociok N. Can the injection of the patient's own bone marrow-derived stem cells preserve cone vision in retinitis pigmentosa and other diseases of the eye? Graefes Arch Clin Exp Ophthalmol. 2005 Mar;243(3):187-8. doi: 10.1007/s00417-004-1062-0. Epub 2004 Nov 23. No abstract available.
- Results publication: PubMed 16896916Arnhold S, Absenger Y, Klein H, Addicks K, Schraermeyer U. Transplantation of bone marrow-derived mesenchymal stem cells rescue photoreceptor cells in the dystrophic retina of the rhodopsin knockout mouse. Graefes Arch Clin Exp Ophthalmol. 2007 Mar;245(3):414-22. doi: 10.1007/s00417-006-0382-7. Epub 2006 Aug 4.
- Results publication: PubMed 20169166Wang S, Lu B, Girman S, Duan J, McFarland T, Zhang QS, Grompe M, Adamus G, Appukuttan B, Lund R. Non-invasive stem cell therapy in a rat model for retinal degeneration and vascular pathology. PLoS One. 2010 Feb 15;5(2):e9200. doi: 10.1371/journal.pone.0009200.
- Derived from this study: PubMed 33422139Tuekprakhon A, Sangkitporn S, Trinavarat A, Pawestri AR, Vamvanij V, Ruangchainikom M, Luksanapruksa P, Pongpaksupasin P, Khorchai A, Dambua A, Boonchu P, Yodtup C, Uiprasertkul M, Sangkitporn S, Atchaneeyasakul LO. Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa. Stem Cell Res Ther. 2021 Jan 9;12(1):52. doi: 10.1186/s13287-020-02122-7.
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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.
La-ongsri Atchaneeyasakul, MD principal investigator
Siriraj Hospital
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 BM-MSC in people with Retinitis Pigmentosa, run by Mahidol University at 1 site in Thailand.
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 4 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 8 Sep 2021. 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 Thailand. 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. 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?
Change from baseline in laser flare and cell measurements (time frame: up to 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01531348, which also lists 5 linked publications.
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 8 Sep 2021.