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

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Regenera Pharma Ltd

Intervention(s):

  • Drug: RPh201 Cohort A
  • Other: Placebo Cohort A
  • Drug: RPh201 Cohort B
  • Other: Placebo Cohort B

Phase:

Phase 2

Eligibility


Inclusion Criteria (Cohort A):

   1. 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).

   2. The participant must understand the nature of the procedure and provide written
   informed consent prior to any study procedure.

   3. 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.

   4. The participant's study eye must have disc pallor (global or segmental) present.

   5. The participant's study eye must have stable visual acuity (see Sections 5.3.3 and
   5.3.4).

   6. 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.

   7. 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).

Exclusion Criteria (Cohort A):

   1. 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.

   2. The participant had surgery, requiring general anesthesia with intubation, within 30
   days prior to enrollment.

   3. 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).

   4. The participant is breast-feeding or plans to breast-feed.

   5. 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.

   6. 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.

   7. 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.

   8. 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.

   9. The participant has a known allergy to cottonseed oil.

10. The participant is planning to move and not relocate near a study site and is
   unwilling to travel for appointments.

11. The participant cannot self-administer or arrange for administration of the IP.

12. 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)

13. The participant has symptoms, signs, and/or diagnosis of giant cell arteritis at any
   time.

14. 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).

15. The participant has systemic inflammatory or infectious disease associated with optic
   neuropathy or ocular disease.

16. The participant has a history of uveitis in the study eye within the last 10 years.

17. 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.

18. The participant has a visual field defect with homonymous non-altitudinal features or
   a defect that respects the vertical meridian.

Inclusion Criteria (Cohort B):

   1. 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).

   2. The participant must understand the nature of the procedure and provide written
   informed consent prior to any study procedure.

   3. 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.

   4. The participant's study eye must have disc pallor (global or segmental) present.

   5. The participant's study eye must have stable visual acuity (see Sections 5.3.3 and
   5.3.4).

   6. 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.

Exclusion Criteria (Cohort B):

   1. 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.

   2. The participant had surgery, requiring general anesthesia with intubation, within 30
   days prior to enrollment.

   3. 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).

   4. The participant is breast-feeding or plans to breast-feed.

   5. 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.

   6. 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.

   7. 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.

   8. 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.

   9. The participant has a known allergy to cottonseed oil.

10. The participant is planning to move and not relocate near a study site and is
   unwilling to travel for appointments.

11. The participant cannot self-administer or arrange for administration of the IP.

12. The participant has one or more of the following abnormal test results at screening:

      1. Erythrocyte Sedimentation Rate (ESR) above age/2 for men or [age + 10]/2 for
      women, as measured by Westergren method or equivalent.

      2. Platelets >400,000 mm3

      3. C-reactive protein (CRP) greater than two times the laboratory upper limit of
      normal.

      4. Severe anemia (Hgb < 10 g/dL)

13. The participant has symptoms, signs, and/or diagnosis of giant cell arteritis at any
   time.

14. 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).

15. The participant has systemic inflammatory or infectious disease associated with optic
   neuropathy or ocular disease.

16. The participant has a history of uveitis in the study eye within the last 10 years.

17. 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.

18. The participant has a visual field defect with homonymous non-altitudinal features or
   a defect that respects the vertical meridian.

Ages Eligible for Study

50 Years - N/A

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Mariana Nunez
650-497-7846
Not Recruiting