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Nonarteritic Anterior Ischemic Optic NeuropathyPhase 2NCT03547206

Efficacy & Safety of RPh201 Treatment in Patients With Previous Nonarteritic Anterior Ischemic Optic Neuropathy (NAION)

A Phase 2 study testing RPh201 Cohort A and RPh201 Cohort B in people with Nonarteritic Anterior Ischemic Optic Neuropathy, run by Regenera Pharma Ltd at 15 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This study is designed as a double-masked, randomized, placebo-controlled, clinical study to evaluate the efficacy and safety of subcutaneous (SC) administration of RPh201 in participants with previous NAION. Read more about this study

Who is it for?
Ages 50 years and older
What is being tested?
RPh201 Cohort A and RPh201 Cohort B
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
165 participants
Where
15 sitesUnited States
Study dates
10 Jul 2018to 27 Sep 2020
Phase
Phase 2
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

  • The participant must be 50 years of age or older at the time of the NAION episode in the study eye. This means that the participant's age at enrollment must be greater than or equal to the sum of 50 plus the number of years since NAION (e.g., at least 52 years of age if the episode was two years prior).
  • The participant must understand the nature of the procedure and provide written informed consent prior to any study procedure.
  • The participant has a definitive clinical diagnosis of NAION in the study eye that developed at least 12 months before randomization. Specifically, the disc swelling must have been observed and documented (by examination, OCT or photograph) by an ophthalmologist or neuro-ophthalmologist who previously examined the participant at the time of the NAION episode in the study eye during the acute episode.
  • The participant's study eye must have disc pallor (global or segmental) present.
  • The participant's study eye must have stable visual acuity (see Sections 5.3.3 and 5.3.4).
  • Using the study eye, the participant must read at least 20 and at most 66 EVA letters with best-corrected vision, at each Screening visit.
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  • The participant's study eye must have a HVF 24-2 SITA Standard visual field using spot size III with mean deviation -5 dB or worse and with a visual field defect compatible with NAION in the study eye (criteria in the MOP).
  • The participant must be 50 years of age or older at the time of the NAION episode in the study eye. This means that the participant's age at enrollment must be greater than or equal to the sum of 50 plus the number of years since NAION (e.g., at least 52 years of age if the episode was two years prior).
  • The participant must understand the nature of the procedure and provide written informed consent prior to any study procedure.
  • The participant has a definitive clinical diagnosis of NAION in the study eye that developed at least 6 months and no more than 3 years before randomization. Specifically, the disc swelling must have been observed and documented (by examination, OCT or photograph) by an ophthalmologist or neuro-ophthalmologist who previously examined the participant at the time of the NAION episode in the study eye during the acute episode.
  • The participant's study eye must have disc pallor (global or segmental) present.
  • The participant's study eye must have stable visual acuity (see Sections 5.3.3 and 5.3.4).
  • Using the study eye, the participant must read at least 20 and at most 66 EVA letters with best-corrected vision, at each Screening visit.

Who cannot join

  • The participant has received treatment for cancer within 12 months prior to enrollment (excluding localized basal cell carcinoma or localized squamous cell carcinoma) or had past diagnosis of cancer adjacent to the afferent visual pathway or had past diagnosis of metastatic cancer.
  • The participant had surgery, requiring general anesthesia with intubation, within 30 days prior to enrollment.
  • The participant is pregnant or a woman of child-bearing potential not using an acceptable method of contraception (per Section 4.1 of the protocol).
  • The participant is breast-feeding or plans to breast-feed.
  • The participant has had treatment with drugs that have potential neuroprotective or toxic effects on the optic nerve or retina (e.g., ethambutol, amiodarone, linezolid, hydroxychloroquine, fingolimod, brimonidine) within 6 months prior to enrollment.
  • The participant has participated in another interventional clinical trial within 60 days prior to enrollment, or previously participated in another clinical trial of RPh201 at any time.
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  • The participant has been receiving or has received within three months prior to enrollment, corticosteroids (except topical steroids, steroid inhalers or intermittent injections into a joint or back), or immunosuppressive drugs.
  • The participant has a medical condition, social or psychological issue, or other condition which, in the judgment of the investigator, could be a safety concern or preclude the individual from completing the protocol.
  • The participant has a known allergy to cottonseed oil.
  • The participant is planning to move and not relocate near a study site and is unwilling to travel for appointments.
  • The participant cannot self-administer or arrange for administration of the IP.
  • The participant has one or more of the following abnormal test results at screening:
  • Erythrocyte Sedimentation Rate (ESR) above age/2 for men or [age + 10]/2 for women, as measured by Westergren method or equivalent.
  • Platelets >400,000 mm3
  • C-reactive protein (CRP) greater than two times the laboratory upper limit of normal.
  • Severe anemia (Hgb < 10)
  • The participant has symptoms, signs, and/or diagnosis of giant cell arteritis at any time.
  • The participant has any other optic neuropathies (e.g., optic neuritis or glaucoma) in either or both eyes (other than self-limited optic neuropathies in the non-study eye, such as past traumatic optic neuropathy or past transient steroid-induced glaucoma due to localized steroid administration).
  • The participant has systemic inflammatory or infectious disease associated with optic neuropathy or ocular disease.
  • The participant has a history of uveitis in the study eye within the last 10 years.
  • The participant's study eye has an ocular condition that appears consistent with a reduction in visual acuity to <20/25, diabetic retinopathy beyond mild non-proliferative diabetic retinopathy not involving the macula, or vision-threatening macula disease.
  • The participant has a visual field defect with homonymous non-altitudinal features or a defect that respects the vertical meridian.
  • The participant has received treatment for cancer within 12 months prior to enrollment (excluding localized basal cell carcinoma or localized squamous cell carcinoma) or had past diagnosis of cancer adjacent to the afferent visual pathway or had past diagnosis of metastatic cancer.
  • The participant had surgery, requiring general anesthesia with intubation, within 30 days prior to enrollment.
  • The participant is pregnant or a woman of child-bearing potential not using an acceptable method of contraception (per Section 4.1 of the protocol).
  • The participant is breast-feeding or plans to breast-feed.
  • The participant has had treatment with drugs that have potential neuroprotective or toxic effects on the optic nerve or retina (e.g., ethambutol, amiodarone, linezolid, hydroxychloroquine, fingolimod, brimonidine) within 6 months prior to enrollment.
  • The participant has participated in another interventional clinical trial within 60 days prior to enrollment, or previously participated in another clinical trial of RPh201 at any time. Participants who were randomized into the placebo arm of Cohort A of this protocol are eligible for screening for Cohort B.
  • The participant has been receiving or has received within three months prior to enrollment, corticosteroids (except topical steroids, steroid inhalers or intermittent injections into a joint or back), or immunosuppressive drugs.
  • The participant has a medical condition, social or psychological issue, or other condition which, in the judgment of the investigator, could be a safety concern or preclude the individual from completing the protocol.
  • The participant has a known allergy to cottonseed oil.
  • The participant is planning to move and not relocate near a study site and is unwilling to travel for appointments.
  • The participant cannot self-administer or arrange for administration of the IP.
  • The participant has one or more of the following abnormal test results at screening:
  • Erythrocyte Sedimentation Rate (ESR) above age/2 for men or [age + 10]/2 for women, as measured by Westergren method or equivalent.
  • Platelets >400,000 mm3
  • C-reactive protein (CRP) greater than two times the laboratory upper limit of normal.
  • Severe anemia (Hgb < 10 g/dL)
  • The participant has symptoms, signs, and/or diagnosis of giant cell arteritis at any time.
  • The participant has any other optic neuropathies (e.g., optic neuritis or glaucoma) in either or both eyes (other than self-limited optic neuropathies in the non-study eye, such as past traumatic optic neuropathy or past transient steroid-induced glaucoma due to localized steroid administration).
  • The participant has systemic inflammatory or infectious disease associated with optic neuropathy or ocular disease.
  • The participant has a history of uveitis in the study eye within the last 10 years.
  • The participant's study eye has an ocular condition that appears consistent with a reduction in visual acuity to <20/25, diabetic retinopathy beyond mild non-proliferative diabetic retinopathy not involving the macula, or vision-threatening macula disease.
  • The participant has a visual field defect with homonymous non-altitudinal features or a defect that respects the vertical meridian.

Trial locations and contacts

15 sites in 1 country.

15 sites in 1 country

United States15 sites

  • Emory University

    Atlanta, Georgia, 30322

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114

  • University of Virginia

    Charlottesville, Virginia, 22908

  • NorthShore Medical Group

    Glenview, Illinois, 60026

  • Neuro-Eye Clinical Trials, Inc.

    Houston, Texas, 77005

  • Charleston Neuroscience Institute

    Ladson, South Carolina, 29456

  • Anne Bates Leach Eye Hospital/Bascom Palmer Eye Institute

    Miami, Florida, 33136

  • New York Eye and Ear Infirmary of Mount Sinai

    New York, New York, 10003

  • The Eye Care Group

    Orange, Connecticut, 06477

  • Byers Eye Institute at Stanford University

    Palo Alto, California, 94303

  • UCLA Doheny Eye Center

    Pasadena, California, 91105

  • Wills Eye Hospital

    Philadelphia, Pennsylvania, 19107

  • Bethesda Neurology, LLC

    Rockville, Maryland, 20852

  • Washington University Ophthalmology

    St Louis, Missouri, 63108

  • The Eye Care Group

    Waterbury, Connecticut, 06708

Other trials in United States

About this study

Official title: A Double-Masked Clinical Study Evaluating the Efficacy and Safety of RPh201 Treatment in Participants With Previous NAION

This study is designed as a double-masked, randomized, placebo-controlled, clinical study to evaluate the efficacy and safety of subcutaneous (SC) administration of RPh201 in participants with previous NAION. All participants enrolled in Cohort A of the study will have a documented history of NAION for at least 12 months and at most, five years prior to enrollment. Participants enrolled in Cohort B of the study will have a documented history of NAION for at least 6 months and at most, three years prior to enrollment.

Read the detailed description

This study is designed as a double-masked, randomized, placebo-controlled, clinical study to evaluate the efficacy and safety of SC administration of RPh201 in participants with previous NAION.

Following a screening phase of 1-8 weeks, participants will attend a baseline visit in which they will undergo testing and visual function assessments. Participants then will be randomized to receive RPh201 or control.

Cohort A After randomization, participants will begin a 26-week schedule consisting of twice-weekly treatment. Participants will return to the clinic for visits at Week 1, Week 4, Week 12 and Week 26 and Week 52

Cohort B After randomization, participants will begin a 12-week schedule consisting of four-times-per-week treatment. Participants will return to the clinic for visits at Week 4 and Week 12.

Safety and efficacy parameters will be recorded throughout the duration of the study.

Being tested
RPh201 Cohort A, Placebo Cohort A, RPh201 Cohort B, Placebo Cohort B
Keywords
NAION, ischemic optic neuropathy
Medical subject headings
Optic Neuropathy, Ischemic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RPh201 Cohort ADrug

    RPh201 is a proprietary, isolated botanical extract of gum mastic for treatment of nonarteritic anterior ischemic optic neuropathy (NAION).

  • Placebo Cohort AOther

    Also called Cottonseed oil

    The placebo is composed of RPh-201 excipients (cottonseed oil stabilized with butylated hydroxytoluene [BHT]).

  • RPh201 Cohort BDrug

    RPh201 is a proprietary, isolated botanical extract of gum mastic for treatment of nonarteritic anterior ischemic optic neuropathy (NAION).

  • Placebo Cohort BOther

    The placebo is composed of RPh-201 excipients (cottonseed oil stabilized with butylated hydroxytoluene [BHT]).

Study groups in NCT03547206
Study groupRoleWhat they receive
RPh201 Cohort AExperimentalA 26-week schedule consisting of twice-weekly subcutaneous administration of 400 μL of the Investigational Medical Product (IMP) (20 mg RPh201).
Receives: Drug: RPh201 Cohort A
Placebo Cohort APlacebo comparatorA 26-week schedule consisting of twice-weekly subcutaneous administration of 400 μL of the vehicle control.
Receives: Other: Placebo Cohort A
RPh201 Cohort BExperimentalA 12-week schedule consisting of four-times-per-week subcutaneous administration of 400 μL of the Investigational Medical Product (IMP) (20 mg RPh201).
Receives: Drug: RPh201 Cohort B
Placebo Cohort BPlacebo comparatorA 12-week schedule consisting of four-times-per-week subcutaneous administration of 400 μL of the vehicle control.
Receives: Other: Placebo Cohort B

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The change in number of best-corrected visual acuity (BCVA) letters from baseline to Week 26 (Cohort A) measured using electronic visual acuity (EVA).

    Visual acuity

    Measured: Week 26 or Week 12

  2. The change in number of best-corrected visual acuity (BCVA) letters from baseline to Week 12 (Cohort B) measured using electronic visual acuity (EVA).

    Visual acuity

    Measured: Week 12

Secondary outcomes (5)

  1. The proportion of study eyes improving by a 15-letter score or more in BCVA from baseline to Week 26 using EVA (Cohort A).

    Visual acuity

    Measured: Week 26

  2. The proportion of study eyes improving by a 15-letter score or more in BCVA from baseline to Week 12 (Cohort B).

    Visual acuity

    Measured: Week 12

  3. The proportion of study eyes improving from baseline in five or more locations of the Humphrey visual field (HVF) 24-2 full-threshold with the size V stimulus on the glaucoma change probability map (GCPM) at the 5% level by group.

    Humphrey visual field (HVF)

    Measured: Week 26

  4. The proportion of study eyes improving by a 10-letter score or more in BCVA from baseline to Week 26 (Cohort A) using EVA.

    Visual acuity

    Measured: Week 26

  5. The proportion of study eyes improving by a 10-letter score or more in BCVA from baseline to Week 12 (Cohort B) using EVA.

    Visual acuity

    Measured: Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Jun 2018
  2. Study start10 Jul 2018
  3. Primary completion27 Sep 2020
  4. Study completion27 Sep 2020

Why it stopped: Terminated by Sponsor

Resources

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
165 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03547206
Sponsor's protocol ID
RGN-ON-002
Status last verified
Oct 2020
First submitted
24 May 2018
Last updated
12 Oct 2020
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No: There is not a plan to make individual participant data available.

Publications

  • Rath EZ, Hazan Z, Adamsky K, Solomon A, Segal ZI, Levin LA. Randomized Controlled Phase 2a Study of RPh201 in Previous Nonarteritic Anterior Ischemic Optic Neuropathy. J Neuroophthalmol. 2019 Sep;39(3):291-298. doi: 10.1097/WNO.0000000000000786.

    PubMed 31430268

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing RPh201 Cohort A and RPh201 Cohort B in people with Nonarteritic Anterior Ischemic Optic Neuropathy, run by Regenera Pharma Ltd at 15 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 13 inclusion and 44 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 12 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 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. Some participants in this study may receive a placebo. 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 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?

The study was stopped early (reason given: Terminated by Sponsor). Results are not available in the source record.

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 change in number of best-corrected visual acuity (BCVA) letters from baseline to Week 26 (Cohort A) measured using electronic visual acuity (EVA). (time frame: Week 26 or Week 12). 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?

On the ClinicalTrials.gov record for NCT03547206, 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 12 Oct 2020.

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