Retinitis Pigmentosa (RP)Phase 1NCT01543906
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
A Phase 1 study testing QLT091001 in people with Retinitis Pigmentosa (RP), run by QLT Inc. at 2 sites in 2 countries.
Trial summary
The purpose of this study is: To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65. To evaluate duration of visual function improvement (if observed) in RP subjects with an autosomal dominant mutation in RPE65 after 7-day… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Retinitis Pigmentosa (RP)
- What is being tested?
- QLT091001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Feb 2012to Aug 2014
- 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
- Subjects will have RP caused by an autosomal dominant mutation in RPE65, as diagnosed by an ocular geneticist or ophthalmologist.
- Subjects who have a best-corrected standard ETDRS visual acuity of 3 letters or better (20/800 Snellen equivalent) or visible photoreceptor outer segments on OCT/FAF.
Who cannot join
- Subjects with any clinically important abnormal physical finding at Screening.
- Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane/Roaccutane® or Soriatane/Neotigason®) within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.
- Subjects with a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis.
- Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of Screening.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
Canada1 site
Montreal Children's Hospital, McGill University Health Centre
Montreal, Quebec
About this study
Official title: An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
The purpose of this study is:
- To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.
- To evaluate duration of visual function improvement (if observed) in RP subjects with an autosomal dominant mutation in RPE65 after 7-day treatment with oral QLT091001.
- To evaluate the safety of oral QLT091001 administered once daily for 7 days in RP subjects with an autosomal dominant mutation in RPE65.
- Conditions
- Retinitis Pigmentosa (RP)
- Being tested
- QLT091001
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QLT091001Drug
oral QLT091001 administered once daily for 7 days
| Study group | Role | What they receive |
|---|---|---|
| QLT091001 | Experimental | oral QLT091001 administered once daily for 7 days Receives: Drug: QLT091001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Visual field
Measured: 12 months
Secondary outcomes (1)
Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs
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 posted5 Mar 2012 (estimated)
- Study startFeb 2012
- Primary completionJul 2014
- Study completionAug 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Retinitis Pigmentosa (RP)Studies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- QLT Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sushanta Mallick, Study director, QLT Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01543906
- Sponsor's protocol ID
- RET RP 01
- Status last verified
- Dec 2014
- First submitted
- 17 Feb 2012
- Last updated
- 15 Dec 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Schwartz SG, Wang X, Chavis P, Kuriyan AE, Abariga SA. Vitamin A and fish oils for preventing the progression of retinitis pigmentosa. Cochrane Database Syst Rev. 2020 Jun 18;6(6):CD008428. doi: 10.1002/14651858.CD008428.pub3.
PubMed 32573764
Study at a glance
From the registry record, last updated by the sponsor on 15 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
- ClinicalTrials.gov ID
- NCT01543906
- Acronym / sponsor ID
- RET RP 01
- Conditions
- Retinitis Pigmentosa (RP)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- QLT Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2012
- Primary completion
- Jul 2014 (actual)
- Study completion
- Aug 2014 (actual)
- First posted
- 5 Mar 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 evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.
- To evaluate duration of visual function improvement (if observed) in RP subjects with an autosomal dominant mutation in RPE65 after 7-day treatment with oral QLT091001.
- To evaluate the safety of oral QLT091001 administered once daily for 7 days in RP subjects with an autosomal dominant mutation in RPE65.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| QLT091001 | Experimental | Drug: QLT091001oral QLT091001 administered once daily for 7 days |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Subjects will have RP caused by an autosomal dominant mutation in RPE65, as diagnosed by an ocular geneticist or ophthalmologist.
- Subjects who have a best-corrected standard ETDRS visual acuity of 3 letters or better (20/800 Snellen equivalent) or visible photoreceptor outer segments on OCT/FAF.
Key exclusion criteria (4 listed)
- Subjects with any clinically important abnormal physical finding at Screening.
- Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane/Roaccutane® or Soriatane/Neotigason®) within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.
- Subjects with a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis.
- Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of Screening.
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 |
|---|---|---|
| QLT091001oral QLT091001 administered once daily for 7 days | Drug | QLT091001 |
Primary outcome
Visual field
Time frame: 12 months
1 secondary outcome
Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs
Time frame: 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 15 Dec 2014.
- Derived from this study: PubMed 32573764Schwartz SG, Wang X, Chavis P, Kuriyan AE, Abariga SA. Vitamin A and fish oils for preventing the progression of retinitis pigmentosa. Cochrane Database Syst Rev. 2020 Jun 18;6(6):CD008428. doi: 10.1002/14651858.CD008428.pub3.
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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.
Sushanta Mallick study director
QLT Inc.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing QLT091001 in people with Retinitis Pigmentosa (RP), run by QLT Inc. at 2 sites in 2 countries.
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 2 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 15 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Canada, Ireland. 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?
Visual field (time frame: 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 NCT01543906, which also lists 1 linked publication.
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 15 Dec 2014.