Inherited Ophthalmic DiseasesPhase 1/2NCT02338973
Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)
A Phase 1/2 study testing Interferon gamma-1b in people with Inherited Ophthalmic Diseases and Inherited Retinal Degeneration, run by National Eye Institute (NEI) at 1 site in United States.
Trial summary
Background: People with rod-cone dystrophy (RCD) or enhanced S-cone syndrome (ESCS) have excess fluid under the retina of their eye. This can cause vision loss. The medicine interferon gamma-1b may help people with these diseases. Objectives: To see if interferon gamma-1b eyedrops are safe for people with RCD or ESCS. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Inherited Ophthalmic Diseases, Inherited Retinal Degeneration
- What is being tested?
- Interferon gamma-1b
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Jan 2015to 26 Jul 2018
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- To be eligible, the following inclusion criteria must be met, where applicable.
- Participant must be 12 years of age or older.
- Participant (or legal guardian or legal representative) must understand and sign the protocol informed consent.
- Participant is willing to comply with the study procedures and is expected to be able to return for all study visits.
- Participant must carry a clinical diagnosis of RCD or ESCS.
- ESCS participant must have molecular confirmation with two alleles for NR2E3 gene mutations
Show all 12Show fewer
- Female participant of childbearing potential must not be pregnant or breast-feeding, must have a negative pregnancy test at screening and must be willing to undergo pregnancy tests at scheduled study visits.
- Female participant of childbearing potential, and any male participant able to father children must have (or have a partner who has) had a hysterectomy or vasectomy, be completely abstinent from intercourse or must agree to practice two reliable methods of contraception while taking the IP and six weeks after completion. Acceptable methods of contraception include:
- Hormonal contraception (i.e., birth control pills, injected hormones, dermal patch or vaginal ring);
- Intrauterine device;
- Barrier methods (diaphragm, condom) with spermicide; or
- Surgical sterilization (tubal ligation).
Who cannot join
- A participant is not eligible if any of the following exclusion criteria are present.
- Participant has a history of other ocular disease likely to contribute significantly to visual disruption (e.g., optic neuropathy, glaucoma, uveitis, or other retinal disease).
- Participant has had diagnosis or treatment of a malignancy (excluding non-melanoma skin cancer) within the previous five years.
- Participant has received investigational treatment in another clinical study related to an ocular condition in the last six months.
- Participant is pregnant, lactating, planning to become pregnant (or father a child) during the study follow-up period.
- Participant is allergic to fluorescein dye.
Show all 22Show fewer
- Participant has a systemic condition that, in the opinion of the investigator, would preclude participation in the study (e.g., multiple sclerosis (MS), as IFN gamma may cause MS exacerbations).
- Study Eye Eligibility Criteria
- A participant must have at least one eye meeting all inclusion criteria and none of the exclusion criteria listed below.
- Study Eye Inclusion Criteria
- The study eye must retain adequate fixation to allow for completion of protocol assessments.
- The study eye must have macular cystic changes (>275 microns and/or disruption of foveal contour on OCT).
- Study Eye Exclusion Criteria
- The study eye has lens, cornea, or other media opacities that preclude adequate visualization and testing of the retina.
- The study eye has undergone intraocular surgery within 6 months prior to enrollment.
- The study eye has a disease that may confound the outcome of the study [e.g., choroidal neovascularization (CNV) in the fovea or parafoveal area].
- Participant is unwilling to discontinue wearing a contact lens in the study eye during IP administration.
- Study Eye Selection Criteria in Cases of Bilateral Disease
- RCD and ESCS usually affect both eyes to a similar degree. In case both eyes of a participant meet the study eye eligibility criteria, the following criteria will be used to select the study eye:
- The eye with more intraretinal fluid will be selected as the study eye;
- If both eyes have similar levels of intraretinal fluid, the eye with worse visual acuity will be selected as the study eye;
- If both eyes have the similar levels of intraretinal fluid and visual acuities, the right eye will be selected as the study eye.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Pilot Phase I/II Study of the Evaluation of Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)
Background:
- People with rod-cone dystrophy (RCD) or enhanced S-cone syndrome (ESCS) have excess fluid under the retina of their eye. This can cause vision loss. The medicine interferon gamma-1b may help people with these diseases.
Read more
Objectives:
- To see if interferon gamma-1b eyedrops are safe for people with RCD or ESCS. To see if the medicine can decrease retina fluid and help prevent vision loss.
Eligibility:
- People at least 12 years old with RCD or ESCS. Those with ESCS must have two mutations in the NR2E3 gene.
Design:
- Participants will be screened with medical history, physical exam, eye exam, and blood tests.
- Participants will stay at NIH for 3 days and get the first eyedrops.
- Participants will give themselves 4 study eyedrops 4 times daily for 2 weeks and keep a diary.
- Participants will have 5 outpatient visits over 8 weeks, 2 of which are telephone assessments. They may have:
- Repeats of screening tests.
- Questionnaires.
- Small piece of skin removed.
- Eye exams, including eye dilation and tasks on computer screens.
- Fluorescein angiography. A dye injected into an arm vein will travel to the blood vessels in the eyes. A camera will take pictures.
- Electroretinography. Participants will sit in the dark wearing eyepatches. A small electrode will be taped to the forehead. After 30 minutes, researchers will remove the eyepatches and put in numbing eyedrops and contact lenses. Participants will watch flashing lights.
- Electrooculography. Electrodes will be attached outside of the eyes and eye function will be measured in the dark and the light.
- Participants will have a follow-up visit after 52 weeks.
Read the detailed description
Objective:
Rod-cone dystrophy (RCD) is a term applied to a number of genetically heterogenous diseases presenting with night vision abnormalities, visual field defects and reduced rod electroretinography responses. Enhanced S-Cone syndrome (ESCS) is a rare autosomal recessive retinal disease with a developmental and a degenerative aspect. Macular cystic changes, often florid and usually resulting in a reduction of central acuity, are frequently associated with both diseases. The reason for this association is not well understood. Acetazolamide (Diamox) and Dorzolamide (Trusopt) have been reported to have variable success in reducing these cystic changes but the effect is frequently inadequate. The objective of this study is to evaluate the safety and potential efficacy of Interferon (IFN) gamma-1b administered topically for macular edema/retinal schisis cysts in RCD and ESCS. Possible disease-related pathophysiologic mechanisms will be explored using induced pluripotent stem cell (iPSC) protocols leading to iPSC-derived retinal pigment epithelium (RPE) and photoreceptor generation.
Study Population:
Up to five participants with RCD with significant macular cystic changes and up to five participants with ESCS with significant macular cystic changes will be enrolled to receive IFN gamma-1b administered topically in one eye. However, up to an additional two participants may be enrolled in order to obtain the five participants in each disease group to be included in the primary analysis if any participants withdraw from the study prior to receiving five days of treatment.
Design:
This is a single-center, prospective, uncontrolled, unmasked pilot Phase I/II study of the safety, tolerability and possible efficacy of IFN gamma-1b in participants with RCD and ESCS and macular cystic changes. One eye of up to five participants with RCD with significant macular cystic changes and up to five participants with ESCS with significant macular cystic changes [evidenced by optical coherence tomography (OCT) >275 microns central macular thickness and/or disruption of foveal contour] will receive topical IFN gamma-1b instilled as drops on the cornea. The initial stage of the study will include two participants from each disease category. Once all four participants have completed the 8-week visit, enrollment will be halted. Safety Adverse Event Review Committee members unaffiliated with the study will review the data as a preliminary assessment of safety and efficacy and to determine whether enrollment should continue. If the committee determines enrollment will continue, three additional participants with RCD and three participants with ESCS will be enrolled. The study will be completed once the final participant has received one year of follow-up.
Outcome Measures:
The primary outcome measure related to the safety and tolerability of IFN gamma-1b administered topically at the prescribed dosage for macular cystic changes in participants with RCD and ESCS will be assessed by the number and severity of adverse events related to the IP and the number of withdrawals at 52 weeks (one year) post-administration. Additional safety of IFN gamma-1b administered topically in participants with RCD and ESCS will be determined from the assessment of retinal function, ocular structure and occurrence of adverse events at all time points. Secondary outcomes include changes in visual function including visual acuity and microperimetry, and retinal imaging with OCT and fluorescein angiography.
- Being tested
- Interferon gamma-1b
- Keywords
- Macular Edema, Rod-Cone Dystrophy, Inherited Retinal Degeneration, Enhanced S-Cone Syndrome, Interferon Gamma
- Medical subject headings
- Macular Edema, Cone-Rod Dystrophies, Enhanced S-Cone Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Interferon gamma-1bDrug
| Study group | Role | What they receive |
|---|---|---|
| Interferon Gamma-1b | Experimental | Topical interferon (IFN) gamma-1b, 112 µg dose, administered in study eye daily for two weeks Receives: Drug: Interferon gamma-1b |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and Severity of IP-related AEs
The number and severity of adverse events related to the investigation product (IP).
Measured: Study duration, up to 52 weeks
Number of Participants Who Withdrew
The number of participants who withdrew early.
Measured: Study duration, up to 52 weeks
Secondary outcomes (29)
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 1
Change in BCVA from baseline as compared to Day 1 by participant in both study and fellow eyes.
Measured: Day 1
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 2
Change in BCVA from baseline as compared to Day 2 by participant in both study and fellow eyes.
Measured: Day 2
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 3
Change in BCVA from baseline as compared to Day 3 by participant in both study and fellow eyes.
Measured: Day 3
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 2
Change in BCVA from baseline as compared to Week 2 by participant in both study and fellow eyes.
Measured: Week 2
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 5
Change in BCVA from baseline as compared to Week 5 by participant in both study and fellow eyes.
Measured: Week 5
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 8
Change in BCVA from baseline as compared to Week 8 by participant in both study and fellow eyes.
Measured: Week 8
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 52
Change in BCVA from baseline as compared to Week 52 by participant in both study and fellow eyes.
Measured: Week 52
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 1
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 1 by participant in both study and fellow eyes.
Measured: Day 1
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 2
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 2 by participant in both study and fellow eyes.
Measured: Day 2
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 3
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Day 3 by participant in both study and fellow eyes.
Measured: Day 3
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 2
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Week 2 by participant in both study and fellow eyes.
Measured: Week 2
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 5
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Week 5 by participant in both study and fellow eyes.
Measured: Week 5
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 8
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Week 8 by participant in both study and fellow eyes.
Measured: Week 8
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 52
Change in maximum subretinal fluid volume as measured on OCT from baseline as compared to Week 52 by participant in both study and fellow eyes.
Measured: Week 52
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 1
Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Day 1 by participant in both study and fellow eyes.
Measured: Day 1
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 2
Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Day 2 by participant in both study and fellow eyes.
Measured: Day 2
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 3
Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Day 3 by participant in both study and fellow eyes.
Measured: Day 3
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 2
Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Week 2 by participant in both study and fellow eyes.
Measured: Week 2
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 5
Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Week 5 by participant in both study and fellow eyes.
Measured: Week 5
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 8
Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Week 8 by participant in both study and fellow eyes.
Measured: Week 8
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 52
Change in central retinal thickness as measured on Cirrus OCT from baseline as compared to Week 52 by participant in both study and fellow eyes.
Measured: Week 52
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 1
Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Day 1 by participant in both study and fellow eyes.
Measured: Day 1
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 2
Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Day 2 by participant in both study and fellow eyes.
Measured: Day 2
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 3
Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Day 3 by participant in both study and fellow eyes.
Measured: Day 3
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 2
Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 2 by participant in both study and fellow eyes.
Measured: Week 2
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 5
Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 5 by participant in both study and fellow eyes.
Measured: Week 5
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 8
Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 8 by participant in both study and fellow eyes.
Measured: Week 8
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52
Change in central retinal thickness as measured on Spectralis OCT from baseline as compared to Week 52 by participant in both study and fellow eyes.
Measured: Week 52
Change in Central Visual Field Sensitivity at Day 2 and Week 5 Compared to Baseline.
Change in central visual field sensitivity as measured by microperimetry testing at Day 2 and Week 5 compared to baseline in both study and fellow eyes.
Measured: Day 2 and Week 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2015 (estimated)
- Study start14 Jan 2015
- Primary completion1 Jun 2018
- Study completion26 Jul 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Inherited Ophthalmic DiseasesStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Eye Institute (NEI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Wadih Zein, M.D., Principal investigator, National Eye Institute (NEI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02338973
- Sponsor's protocol ID
- 150052
- Other IDs
- 15-EI-0052
- Status last verified
- 26 Jul 2018
- First submitted
- 14 Jan 2015
- Last updated
- 13 Aug 2019
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Phase I/II Study of the Evaluation of Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)
- ClinicalTrials.gov ID
- NCT02338973
- Acronym / sponsor ID
- 150052
- Keywords
- Macular Edema, Rod-Cone Dystrophy, Inherited Retinal Degeneration, Enhanced S-Cone Syndrome, Interferon Gamma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 4 participants (actual)
- Lead sponsor
- National Eye Institute (NEI)US National Institutes of Health
- Study start
- 14 Jan 2015
- Primary completion
- 1 Jun 2018 (actual)
- Study completion
- 26 Jul 2018 (actual)
- First posted
- 15 Jan 2015 (estimated)
- Results
- Posted 6 Aug 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
- People with rod-cone dystrophy (RCD) or enhanced S-cone syndrome (ESCS) have excess fluid under the retina of their eye. This can cause vision loss. The medicine interferon gamma-1b may help people with these diseases.
Objectives:
- To see if interferon gamma-1b eyedrops are safe for people with RCD or ESCS. To see if the medicine can decrease retina fluid and help prevent vision loss.
Eligibility:
- People at least 12 years old with RCD or ESCS. Those with ESCS must have two mutations in the NR2E3 gene.
Design:
- Participants will be screened with medical history, physical exam, eye exam, and blood tests.
- Participants will stay at NIH for 3 days and get the first eyedrops.
- Participants will give themselves 4 study eyedrops 4 times daily for 2 weeks and keep a diary.
- Participants will have 5 outpatient visits over 8 weeks, 2 of which are telephone assessments. They may have:
- Repeats of screening tests.
- Questionnaires.
- Small piece of skin removed.
- Eye exams, including eye dilation and tasks on computer screens.
- Fluorescein angiography. A dye injected into an arm vein will travel to the blood vessels in the eyes. A camera will take pictures.
- Electroretinography. Participants will sit in the dark wearing eyepatches. A small electrode will be taped to the forehead. After 30 minutes, researchers will remove the eyepatches and put in numbing eyedrops and contact lenses. Participants will watch flashing lights.
- Electrooculography. Electrodes will be attached outside of the eyes and eye function will be measured in the dark and the light.
- Participants will have a follow-up visit after 52 weeks.
Detailed description
Objective:
Rod-cone dystrophy (RCD) is a term applied to a number of genetically heterogenous diseases presenting with night vision abnormalities, visual field defects and reduced rod electroretinography responses. Enhanced S-Cone syndrome (ESCS) is a rare autosomal recessive retinal disease with a developmental and a degenerative aspect. Macular cystic changes, often florid and usually resulting in a reduction of central acuity, are frequently associated with both diseases. The reason for this association is not well understood. Acetazolamide (Diamox) and Dorzolamide (Trusopt) have been reported to have variable success in reducing these cystic changes but the effect is frequently inadequate. The objective of this study is to evaluate the safety and potential efficacy of Interferon (IFN) gamma-1b administered topically for macular edema/retinal schisis cysts in RCD and ESCS. Possible disease-related pathophysiologic mechanisms will be explored using induced pluripotent stem cell (iPSC) protocols leading to iPSC-derived retinal pigment epithelium (RPE) and photoreceptor generation.
Study Population:
Up to five participants with RCD with significant macular cystic changes and up to five participants with ESCS with significant macular cystic changes will be enrolled to receive IFN gamma-1b administered topically in one eye. However, up to an additional two participants may be enrolled in order to obtain the five participants in each disease group to be included in the primary analysis if any participants withdraw from the study prior to receiving five days of treatment.
Design:
This is a single-center, prospective, uncontrolled, unmasked pilot Phase I/II study of the safety, tolerability and possible efficacy of IFN gamma-1b in participants with RCD and ESCS and macular cystic changes. One eye of up to five participants with RCD with significant macular cystic changes and up to five participants with ESCS with significant macular cystic changes [evidenced by optical coherence tomography (OCT) >275 microns central macular thickness and/or disruption of foveal contour] will receive topical IFN gamma-1b instilled as drops on the cornea. The initial stage of the study will include two participants from each disease category. Once all four participants have completed the 8-week visit, enrollment will be halted. Safety Adverse Event Review Committee members unaffiliated with the study will review the data as a preliminary assessment of safety and efficacy and to determine whether enrollment should continue. If the committee determines enrollment will continue, three additional participants with RCD and three participants with ESCS will be enrolled. The study will be completed once the final participant has received one year of follow-up.
Outcome Measures:
The primary outcome measure related to the safety and tolerability of IFN gamma-1b administered topically at the prescribed dosage for macular cystic changes in participants with RCD and ESCS will be assessed by the number and severity of adverse events related to the IP and the number of withdrawals at 52 weeks (one year) post-administration. Additional safety of IFN gamma-1b administered topically in participants with RCD and ESCS will be determined from the assessment of retinal function, ocular structure and occurrence of adverse events at all time points. Secondary outcomes include changes in visual function including visual acuity and microperimetry, and retinal imaging with OCT and fluorescein angiography.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Interferon Gamma-1b | Experimental | Drug: Interferon gamma-1bTopical interferon (IFN) gamma-1b, 112 µg dose, administered in study eye daily for two weeks |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- To be eligible, the following inclusion criteria must be met, where applicable.
- Participant must be 12 years of age or older.
- Participant (or legal guardian or legal representative) must understand and sign the protocol informed consent.
- Participant is willing to comply with the study procedures and is expected to be able to return for all study visits.
- Participant must carry a clinical diagnosis of RCD or ESCS.
Key exclusion criteria (22 listed)
- A participant is not eligible if any of the following exclusion criteria are present.
- Participant has a history of other ocular disease likely to contribute significantly to visual disruption (e.g., optic neuropathy, glaucoma, uveitis, or other retinal disease).
- Participant has had diagnosis or treatment of a malignancy (excluding non-melanoma skin cancer) within the previous five years.
- Participant has received investigational treatment in another clinical study related to an ocular condition in the last six months.
- Participant is pregnant, lactating, planning to become pregnant (or father a child) during the study follow-up period.
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 |
|---|---|---|
| Interferon gamma-1b | Drug | Interferon Gamma-1b |
Primary outcome
Number and Severity of IP-related AEs
Time frame: Study duration, up to 52 weeks
The number and severity of adverse events related to the investigation product (IP).
Number of Participants Who Withdrew
Time frame: Study duration, up to 52 weeks
The number of participants who withdrew early.
29 secondary outcomes
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 1
Time frame: Day 1
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 2
Time frame: Day 2
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Day 3
Time frame: Day 3
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 2
Time frame: Week 2
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 5
Time frame: Week 5
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 8
Time frame: Week 8
Change in Best Corrected Visual Acuity (BCVA) From Baseline as Compared to Week 52
Time frame: Week 52
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 1
Time frame: Day 1
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 2
Time frame: Day 2
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Day 3
Time frame: Day 3
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 2
Time frame: Week 2
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 5
Time frame: Week 5
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 8
Time frame: Week 8
Change in Maximum Subretinal Fluid Volume From Baseline as Compared to Week 52
Time frame: Week 52
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 1
Time frame: Day 1
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 2
Time frame: Day 2
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Day 3
Time frame: Day 3
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 2
Time frame: Week 2
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 5
Time frame: Week 5
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 8
Time frame: Week 8
Change in Central Retinal Thickness as Measured on Cirrus OCT From Baseline as Compared to Week 52
Time frame: Week 52
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 1
Time frame: Day 1
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 2
Time frame: Day 2
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Day 3
Time frame: Day 3
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 2
Time frame: Week 2
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 5
Time frame: Week 5
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 8
Time frame: Week 8
Change in Central Retinal Thickness as Measured on Spectralis OCT From Baseline as Compared to Week 52
Time frame: Week 52
Change in Central Visual Field Sensitivity at Day 2 and Week 5 Compared to Baseline.
Time frame: Day 2 and Week 5
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Wadih Zein, M.D. principal investigator
National Eye Institute (NEI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Interferon gamma-1b in people with Inherited Ophthalmic Diseases and Inherited Retinal Degeneration, run by National Eye Institute (NEI) 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 22 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: stopped early, as of the sponsor's last update on 13 Aug 2019. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Aug 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 and Severity of IP-related AEs (time frame: Study duration, up to 52 weeks). The study lists 2 primary outcomes in all. 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 13 Aug 2019.