Geographic AtrophyPhase 1NCT01093170
A Phase I Open Label Dose Escalation Trial of RNA-144101 in the Treatment of Geographic Atrophy
A Phase 1 study testing RNA-144101 in people with Geographic Atrophy, run by Mark Kleinman at 1 site in United States.
Trial summary
This is a non-randomized, open-label, dose-escalating Phase Ia study performed at a single center designed to evaluate the safety, pharmacokinetics, and preliminary efficacy of intravitreous administration of RNA-144101 in patients with geographic atrophy (GA). Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Geographic Atrophy
- What is being tested?
- RNA-144101
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2011to Sep 2012
- 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
- Males and females of all races and ethnicities between the ages of 50-99
- Female subjects must be 1-year postmenopausal or surgically sterilized and must have negative serum pregnancy test
- Subjects must have GA from AMD in one or both eyes:
- The study eye will be the eye that meets all inclusion/exclusion criteria
- If both eyes meet criteria, then the eye with the smaller GA lesion will be studied
- If the GA lesions are equal in size, then the eye with the best-corrected visual acuity (BCVA) will chosen as the study eye
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- If both eyes have the same lesion size and same BCVA, the right eye will be chosen
- Subjects must have the following GA criteria for inclusion:
- GA lesions with an area > 2.5mm2 and ≤ 20mm2 as determine by fluorescein angiography and fundus autofluorescence
- A clear view to the fundus must be present in order to easily examine the study eye at baseline
- BCVA In the study eye between 20/20 and 20/100 as measured on Snellen chart or 85-50 letters by ETDRS
- Willing and able to provide signed informed consent prior to any study participation
Who cannot join
- GA due to a disease other than AMD
- Pregnancy or lactation
- Treatment of any systemic infection
- Autofluorescence pattern marked at none, focal or patchy
- Ocular surgery in the study eye in the previous 6 months.
- Presence or history of choroidal neovascularization (wet AMD) in the study eye
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- Any inflammatory (autoimmune, uveitis) or neovascular (diabetic retinopathy) disease of the retina
- Any history of glaucoma or disc cupping in the study eye
- Any history of severe dry eye disease
- High myopia > - 8D or high hyperopia > +8D in the study eye
- Presence of life-threatening disease
- Abnormal basal metabolic panel or liver function tests
- Current alcohol or other substance abuse
- Unwilling or unable to provide signed informed consent for any study procedures
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Kentucky / Dept of Ophthalmology
Lexington, Kentucky, 40536
About this study
This is a non-randomized, open-label, dose-escalating Phase Ia study performed at a single center designed to evaluate the safety, pharmacokinetics, and preliminary efficacy of intravitreous administration of RNA-144101 in patients with geographic atrophy (GA).
- Conditions
- Geographic Atrophy
- Being tested
- RNA-144101
- Medical subject headings
- Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RNA-144101Drug
Intravitreous administration of RNA-144101
| Study group | Role | What they receive |
|---|---|---|
| RNA-144101 | Experimental | Not described Receives: Drug: RNA-144101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of RNA-144101 as measured by vision testing, ophthalmic and physical exams, vital signs, clinical laboratory testing and AEs.
Measured: Through study completion or discontinuation
Secondary outcomes (2)
Pharmacokinetic studies measuring drug concentrations in serum over study period.
Measured: Through study completion or discontinuation
Measurement of total aggregate area of GA lesions (as determined by color fundus photography and autofluorescence imaging)
Measured: Through duration of study or discontinuation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2010 (estimated)
- Study startMar 2011
- Primary completionSep 2012
- Study completionSep 2012
Why it stopped: Study was not conducted, no subjects recruited.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Geographic AtrophyStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mark Kleinman
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Mark Kleinman, University of Kentucky
- Investigators
- Jayakrishna Ambati, MD, Principal investigator, University of Kentucky
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01093170
- Sponsor's protocol ID
- 144101a
- Status last verified
- Jan 2018
- First submitted
- 22 Mar 2010
- Last updated
- 17 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Open Label Dose Escalation Trial of RNA-144101 in the Treatment of Geographic Atrophy
- ClinicalTrials.gov ID
- NCT01093170
- Acronym / sponsor ID
- 144101a
- Conditions
- Geographic Atrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study was not conducted, no subjects recruited.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Mark KleinmanUniversity, hospital or other
- Study start
- Mar 2011
- Primary completion
- Sep 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 25 Mar 2010 (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.
This is a non-randomized, open-label, dose-escalating Phase Ia study performed at a single center designed to evaluate the safety, pharmacokinetics, and preliminary efficacy of intravitreous administration of RNA-144101 in patients with geographic atrophy (GA).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RNA-144101 | Experimental | Drug: RNA-144101 |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Males and females of all races and ethnicities between the ages of 50-99
- Female subjects must be 1-year postmenopausal or surgically sterilized and must have negative serum pregnancy test
- Subjects must have GA from AMD in one or both eyes:
- The study eye will be the eye that meets all inclusion/exclusion criteria
- If both eyes meet criteria, then the eye with the smaller GA lesion will be studied
Key exclusion criteria (14 listed)
- GA due to a disease other than AMD
- Pregnancy or lactation
- Treatment of any systemic infection
- Autofluorescence pattern marked at none, focal or patchy
- Ocular surgery in the study eye in the previous 6 months.
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 |
|---|---|---|
| RNA-144101Intravitreous administration of RNA-144101 | Drug | RNA-144101 |
Primary outcome
Safety and tolerability of RNA-144101 as measured by vision testing, ophthalmic and physical exams, vital signs, clinical laboratory testing and AEs.
Time frame: Through study completion or discontinuation
2 secondary outcomes
Pharmacokinetic studies measuring drug concentrations in serum over study period.
Time frame: Through study completion or discontinuation
Measurement of total aggregate area of GA lesions (as determined by color fundus photography and autofluorescence imaging)
Time frame: Through duration of study or discontinuation
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jan 2018.
Related trials for Geographic Atrophy
Active, not recruitingPhase 1/2
Phase 1/2 Study of VOY-101 in Patients With Advanced Non-Neovascular Age-Related Macular Degeneration
Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 3
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
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RecruitingPhase 1
Phase 1 Study of C.001 in Retinal Degeneration
Geographic Atrophy, Stargardt Disease
RecruitingPhase 2/3
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
Geographic Atrophy, Macular Degeneration
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.
Jayakrishna Ambati, MD principal investigator
University of Kentucky
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 RNA-144101 in people with Geographic Atrophy, run by Mark Kleinman at 1 site 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 12 inclusion and 14 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: withdrawn, as of the sponsor's last update on 17 Jan 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 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 was withdrawn before enrolling participants, so there are no results.
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?
Safety and tolerability of RNA-144101 as measured by vision testing, ophthalmic and physical exams, vital signs, clinical laboratory testing and AEs. (time frame: Through study completion or discontinuation). 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 Jan 2018.