Age Related Macular DegenerationPhase 1GEMNCT01301443
Phase I Dose Escalation Safety Study of RetinoStat in Advanced Age-Related Macular Degeneration (AMD)
A Phase 1 study testing Subretinally injected RetinoStat in people with Age Related Macular Degeneration, run by Oxford BioMedica at 3 sites in United States.
Trial summary
The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, RetinoStat, designed to treat neovascular age-related macular degeneration. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Age Related Macular Degeneration
- What is being tested?
- Subretinally injected RetinoStat
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 3 sitesUnited States
- Study dates
- Feb 2011to May 2015
- 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 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinical diagnosis of AMD with active CNV that shows evidence of leakage.
- BCVA less than or equal to 20/200 in the study eye for dose escalation phase.
- BCVA less than or equal to 20/80 in the study eye for maximum tolerated dose phase.
Who cannot join
- Significant ocular abnormalities that prevent retinal assessment.
- Treatment with steroids within three months of screening.
- Treatment with anti-VEGF therapy to either eye within one month of screening.
- Clinically significant intercurrent illnesses, laboratory, ECG or chest XRay abnormalities.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Johns Hopkins University Hospital
Baltimore, Maryland, 21287-9277
The University of Iowa
Iowa City, Iowa, 52242-1098
Dr Andreas Lauer - Oregon Health & Science University
Portland, Oregon, 97239
About this study
Official title: A Phase I Dose Escalation Safety Study of Subretinally Injected RetinoStat, a Lentiviral Vector Expressing Endostatin and Angiostatin, in Patients With Advanced Neovascular Age-Related Macular Degeneration
The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, RetinoStat, designed to treat neovascular age-related macular degeneration.
Read the detailed description
There are two parts to the study. A dose-escalation phase looking at three doses of RetinoStat starting with the lowest dose, three patients will be recruited at each dose level. The escalation phase will be followed by a dose confirmation phase where the highest dose that is safe and well tolerated will be examined in 9 patients.
- Conditions
- Age Related Macular Degeneration
- Being tested
- Subretinally injected RetinoStat
- Keywords
- Age Related Macular Degeneration
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Subretinally injected RetinoStatDrug
Also called OXB-201
Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose.
| Study group | Role | What they receive |
|---|---|---|
| Subretinally Injected RetinoStat | Experimental | Subretinally injected RetinoStat Receives: Drug: Subretinally injected RetinoStat |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence of adverse events
The number and percentage of patients with treatment emergent adverse events.
Measured: 24 weeks
Secondary outcomes (1)
Change from baseline in subretinal and intraretinal fluid as measured by OCT
The change from baseline in the amount of subretinal and intraretinal fluid measured by Optical Coherence tomography
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2011 (estimated)
- Study startFeb 2011
- Primary completionSep 2014
- Study completionMay 2015
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 Age Related Macular 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oxford BioMedica
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Peter A Campochiaro, MD, Principal investigator, Johns Hopkins University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01301443
- Sponsor's protocol ID
- RS1/001/10
- Acronym
- GEM
- Status last verified
- Apr 2017
- First submitted
- 21 Feb 2011
- Last updated
- 5 Apr 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Campochiaro PA, Lauer AK, Sohn EH, Mir TA, Naylor S, Anderton MC, Kelleher M, Harrop R, Ellis S, Mitrophanous KA. Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study. Hum Gene Ther. 2017 Jan;28(1):99-111. doi: 10.1089/hum.2016.117. Epub 2016 Sep 26.
PubMed 27710144
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Dose Escalation Safety Study of Subretinally Injected RetinoStat, a Lentiviral Vector Expressing Endostatin and Angiostatin, in Patients With Advanced Neovascular Age-Related Macular Degeneration
- ClinicalTrials.gov ID
- NCT01301443
- Acronym / sponsor ID
- GEM / RS1/001/10
- Conditions
- Age Related Macular Degeneration
- Keywords
- Age Related Macular Degeneration
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Oxford BioMedicaIndustry (pharma, biotech, devices)
- Study start
- Feb 2011
- Primary completion
- Sep 2014 (actual)
- Study completion
- May 2015 (actual)
- First posted
- 23 Feb 2011 (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 first in man study is to examine the safety of an experimental gene transfer agent, RetinoStat, designed to treat neovascular age-related macular degeneration.
Detailed description
There are two parts to the study. A dose-escalation phase looking at three doses of RetinoStat starting with the lowest dose, three patients will be recruited at each dose level. The escalation phase will be followed by a dose confirmation phase where the highest dose that is safe and well tolerated will be examined in 9 patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subretinally Injected RetinoStat | Experimental | Drug: Subretinally injected RetinoStatSubretinally injected RetinoStat |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Clinical diagnosis of AMD with active CNV that shows evidence of leakage.
- BCVA less than or equal to 20/200 in the study eye for dose escalation phase.
- BCVA less than or equal to 20/80 in the study eye for maximum tolerated dose phase.
Key exclusion criteria (4 listed)
- Significant ocular abnormalities that prevent retinal assessment.
- Treatment with steroids within three months of screening.
- Treatment with anti-VEGF therapy to either eye within one month of screening.
- Clinically significant intercurrent illnesses, laboratory, ECG or chest XRay abnormalities.
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 |
|---|---|---|
| Subretinally injected RetinoStatAlso: OXB-201Single subretinal injections, with increasing doses. 9 patients with 3 patients at each dose followed, by 12 patients at maximum tolerated dose. | Drug | Subretinally Injected RetinoStat |
Primary outcome
The incidence of adverse events
Time frame: 24 weeks
The number and percentage of patients with treatment emergent adverse events.
1 secondary outcome
Change from baseline in subretinal and intraretinal fluid as measured by OCT
Time frame: 24 weeks
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 5 Apr 2017.
- Results publication: PubMed 27710144Campochiaro PA, Lauer AK, Sohn EH, Mir TA, Naylor S, Anderton MC, Kelleher M, Harrop R, Ellis S, Mitrophanous KA. Lentiviral Vector Gene Transfer of Endostatin/Angiostatin for Macular Degeneration (GEM) Study. Hum Gene Ther. 2017 Jan;28(1):99-111. doi: 10.1089/hum.2016.117. Epub 2016 Sep 26.
- Individual participant dataSharing planned: Undecided
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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.
Peter A Campochiaro, MD principal investigator
Johns Hopkins University Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Subretinally injected RetinoStat in people with Age Related Macular Degeneration, run by Oxford BioMedica at 3 sites in United States.
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 3 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 5 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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?
The incidence of adverse events (time frame: 24 weeks). 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 NCT01301443, 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 5 Apr 2017.