Retinal DystrophiesRETIPSNCT03853252
iPS Cells of Patients for Models of Retinal Dystrophies
A study testing Skin biopsy in people with Retinal Dystrophies, run by University Hospital, Montpellier at 1 site in France.
Trial summary
The investigators are focused on inherited retinal dystrophies with an aim to further understand disease pathophysiology and to elaborate novel treatments, as, to date, there is no effective treatment to prevent blindness. Read more about this study
- Who is it for?
- Ages 5 to 70 years
- Conditions the study is for
- Retinal Dystrophies
- What is being tested?
- Skin biopsy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 150 participants
- Where
- 1 siteFrance
- Study dates
- 3 Nov 2014to 30 Oct 2023
- Phase
- Interventional
- 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 5 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Signed informed consent and
- Choroideremia :
- Males
- CHM mutation
- With multimodal Imaging anomalies in line with CHM
- All other presumed inherited retinal dystrophies with bilateral and symmetrical involvement with identified mutations in one of the Retnet gene
Show all 10Show fewer
- All presumed inherited optic neuropathy with bilateral and symmetrical involvement with identified mutations
- And in all cases or pattern
- Age from 5 to 70
- with appropriate health insurance
Who cannot join
- Patient under tutorship or curatorship
Trial locations and contacts
1 site in 1 country.
France1 site
CNMR Maolya, Genetic Sensory Diseases
Montpellier, Occitanie, 34294
About this study
The investigators are focused on inherited retinal dystrophies with an aim to further understand disease pathophysiology and to elaborate novel treatments, as, to date, there is no effective treatment to prevent blindness.
The main goal of this study is to generate human cellular models of healthy and disease retinas and perform studies to evaluate the efficiency of gene therapy approaches for different diseases.
Read more
Skin biopsies of volunteers are cultured to isolate fibroblasts that are then reprogrammed into iPS cells. Healthy and disease-specific iPS cells are then differentiated into retinal models.
This study should help to elucidate disease pathways and to provide proof-of-concept for various therapeutic approaches.
- Conditions
- Retinal Dystrophies
- Being tested
- Skin biopsy
- Medical subject headings
- Retinal Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Skin biopsyOther
Skin biopsy on a location preliminarily anesthetized Disinfection protocol Combined required blood tests (HIV, Hepatitis B)
| Study group | Role | What they receive |
|---|---|---|
| Skin biopsy | Other | Not described Receives: Other: Skin biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
number of human cell models obtained
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2019
- Study start3 Nov 2014
- Primary completion30 Oct 2023
- Study completion30 Oct 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinal DystrophiesStudies of the same condition, open and under way.
- More trials run by University Hospital, MontpellierThe 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 N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Montpellier
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03853252
- Sponsor's protocol ID
- 9366
- Acronym
- RETIPS
- Status last verified
- Jul 2025
- First submitted
- 22 Feb 2019
- Last updated
- 10 Jul 2025
- 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 10 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- iPS Cells of Patients for Models of Retinal Dystrophies
- ClinicalTrials.gov ID
- NCT03853252
- Acronym / sponsor ID
- RETIPS / 9366
- Conditions
- Retinal Dystrophies
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- University Hospital, MontpellierUniversity, hospital or other
- Collaborators
- Institut National de la Santé Et de la Recherche Médicale, France
- Study start
- 3 Nov 2014 (actual)
- Primary completion
- 30 Oct 2023 (actual)
- Study completion
- 30 Oct 2023 (actual)
- First posted
- 25 Feb 2019 (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 investigators are focused on inherited retinal dystrophies with an aim to further understand disease pathophysiology and to elaborate novel treatments, as, to date, there is no effective treatment to prevent blindness.
The main goal of this study is to generate human cellular models of healthy and disease retinas and perform studies to evaluate the efficiency of gene therapy approaches for different diseases.
Skin biopsies of volunteers are cultured to isolate fibroblasts that are then reprogrammed into iPS cells. Healthy and disease-specific iPS cells are then differentiated into retinal models.
This study should help to elucidate disease pathways and to provide proof-of-concept for various therapeutic approaches.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Skin biopsy | Other | Other: Skin biopsy |
Who may qualify
- Ages
- 5 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Signed informed consent and
- Choroideremia :
- All other presumed inherited retinal dystrophies with bilateral and symmetrical involvement with identified mutations in one of the Retnet gene
- All presumed inherited optic neuropathy with bilateral and symmetrical involvement with identified mutations
- And in all cases or pattern
Key exclusion criteria (1 listed)
- Patient under tutorship or curatorship
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 |
|---|---|---|
| Skin biopsySkin biopsy on a location preliminarily anesthetized Disinfection protocol Combined required blood tests (HIV, Hepatitis B) | Other | Skin biopsy |
Primary outcome
number of human cell models obtained
Time frame: 10 years
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 10 Jul 2025.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Skin biopsy in people with Retinal Dystrophies, run by University Hospital, Montpellier at 1 site in France.
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 10 inclusion and 1 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 10 Jul 2025. 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 France. 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.
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?
number of human cell models obtained (time frame: 10 years). 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 10 Jul 2025.