Trial Outcomes & Findings for Safety and Efficacy Study of RPh201 Treatment of Ischemic Optic Neuropathy (ION). (NCT NCT02045212)
NCT ID: NCT02045212
Last Updated: 2020-03-19
Results Overview
Best Corrected Visual Acuity (BCVA) was assessed at all the visits, with refraction as necessary. VA measurements were taken in a sitting position at a test distance of 4 meters using early treatment diabetic retinopathy study (ETDRS) charts.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
22 participants
Primary outcome timeframe
26/39 weeks
Results posted on
2020-03-19
Participant Flow
Participant milestones
| Measure |
RPh201
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
9
|
|
Overall Study
COMPLETED
|
11
|
8
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
RPh201
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Finished 13 weeks, chose not to extend
|
1
|
0
|
Baseline Characteristics
Safety and Efficacy Study of RPh201 Treatment of Ischemic Optic Neuropathy (ION).
Baseline characteristics by cohort
| Measure |
RPh201
n=13 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
n=9 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
Total
n=22 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Age, Continuous
|
61.46 years
STANDARD_DEVIATION 8.1 • n=5 Participants
|
60.33 years
STANDARD_DEVIATION 5.8 • n=7 Participants
|
61.00 years
STANDARD_DEVIATION 7.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Region of Enrollment
Israel
|
13 participants
n=5 Participants
|
9 participants
n=7 Participants
|
22 participants
n=5 Participants
|
|
Height
|
1.7 metres
STANDARD_DEVIATION 0.12 • n=5 Participants
|
1.7 metres
STANDARD_DEVIATION 0.12 • n=7 Participants
|
1.7 metres
STANDARD_DEVIATION 0.12 • n=5 Participants
|
|
Weight
|
82.8 kg
STANDARD_DEVIATION 13.58 • n=5 Participants
|
95.5 kg
STANDARD_DEVIATION 24.54 • n=7 Participants
|
88.0 kg
STANDARD_DEVIATION 19.38 • n=5 Participants
|
|
BMI
|
29.16 kg per metre squared
STANDARD_DEVIATION 4.24 • n=5 Participants
|
34.77 kg per metre squared
STANDARD_DEVIATION 5.52 • n=7 Participants
|
31.46 kg per metre squared
STANDARD_DEVIATION 5.46 • n=5 Participants
|
PRIMARY outcome
Timeframe: 26/39 weeksPopulation: Efficacy
Best Corrected Visual Acuity (BCVA) was assessed at all the visits, with refraction as necessary. VA measurements were taken in a sitting position at a test distance of 4 meters using early treatment diabetic retinopathy study (ETDRS) charts.
Outcome measures
| Measure |
RPh201
n=12 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
n=8 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
|---|---|---|
|
Mean Increase From Baseline in ETDRS Letters Read at 26 Weeks and Off-drug Follow-up Visit
Changes in BCVA from baseline to week 26
|
15 EDTRS Letters
Standard Error 4.6
|
7 EDTRS Letters
Standard Error 5.4
|
|
Mean Increase From Baseline in ETDRS Letters Read at 26 Weeks and Off-drug Follow-up Visit
changes in BCVA from baseline to 39 weeks
|
9 EDTRS Letters
Standard Error 5.6
|
6 EDTRS Letters
Standard Error 4.7
|
PRIMARY outcome
Timeframe: 26 weeksPopulation: Efficacy
Visual field mean deviation (MD) changes from the screening at week 26 using with the HVF 24-2 FASTPAC program using the size III or Size V test stimulus
Outcome measures
| Measure |
RPh201
n=11 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
n=8 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
|---|---|---|
|
Changes in Visual Field Observed Following the Treatment
Changes Visual Field size III
|
7.85 dB
Standard Error 5.8
|
0.84 dB
Standard Error 1.47
|
|
Changes in Visual Field Observed Following the Treatment
Changes Visual Field size V
|
0.41 dB
Standard Error 1.66
|
0.49 dB
Standard Error 2.59
|
PRIMARY outcome
Timeframe: 26 weeksPopulation: Efficacy
TMean RNFL thickness measured by OCT. The data are in microns, as measured with an Opko OCT machine.
Outcome measures
| Measure |
RPh201
n=11 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
n=8 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
|---|---|---|
|
Changes Mean RNFL Thickness in NAION Eyes Change From Screening to Week 26
|
-4.6 µm
Standard Error 1.58
|
-1.8 µm
Standard Error 2.08
|
SECONDARY outcome
Timeframe: 26/39 weeksSafety and tolerability multiple ascending SC doses as assessed by adverse events, vital signs, clinical laboratory and physical exam
Outcome measures
| Measure |
RPh201
n=13 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
n=9 Participants
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
|---|---|---|
|
Number of Participants With Adverse Events Assessed by Vital Signs, Clinical Laboratory and Physical Exam
Visual acuity ocular safety in fellow eye
|
0 participants
|
1 participants
|
|
Number of Participants With Adverse Events Assessed by Vital Signs, Clinical Laboratory and Physical Exam
Visual field ocular safety in fellow eye
|
0 participants
|
1 participants
|
Adverse Events
RPh201
Serious events: 4 serious events
Other events: 13 other events
Deaths: 0 deaths
Placebo
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
RPh201
n=13 participants at risk
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
n=9 participants at risk
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
|---|---|---|
|
Ear and labyrinth disorders
Vertigo
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Nervous system disorders
Dizziness
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Eye disorders
Visual acuity decreased
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Infections and infestations
Brucellosis
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Eye disorders
Ischemic optic neuropathy - AION right eye
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
Other adverse events
| Measure |
RPh201
n=13 participants at risk
3/6 month treatment schedule, consisting of bi-weekly SC administration of the RPh201
RPh201: SC injection twice a week during 13/26 weeks
|
Placebo
n=9 participants at risk
3/6 month treatment schedule, consisting of bi-weekly SC administration of the Placebo
Placebo: SC injection twice a week during 13/26 weeks
|
|---|---|---|
|
Cardiac disorders
Palpitations
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Eye disorders
Abnormal sensation in eye
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
Cataract
|
30.8%
4/13 • Number of events 5
|
0.00%
0/9
|
|
Eye disorders
Conjunctival irratation
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
Conjunctivitis
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
Corneal defect
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Eye disorders
Dry Eye
|
15.4%
2/13 • Number of events 2
|
22.2%
2/9 • Number of events 2
|
|
Eye disorders
Eye irritation
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
Eye Movement Disorder
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
Eye Pain
|
7.7%
1/13 • Number of events 1
|
22.2%
2/9 • Number of events 2
|
|
Eye disorders
Eye swelling
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Eye disorders
Eyelid oedema
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Eye disorders
Lacrimation increased
|
7.7%
1/13 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
Photopsia
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Eye disorders
Retinal haemorrhage
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Gastrointestinal disorders
Abdominal discomfort
|
7.7%
1/13 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
Gastrointestinal disorders
Diarrhoea
|
7.7%
1/13 • Number of events 2
|
11.1%
1/9 • Number of events 1
|
|
Gastrointestinal disorders
Haemorrhoids
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Gastrointestinal disorders
Nausea
|
7.7%
1/13 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
Gastrointestinal disorders
Toothache
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Gastrointestinal disorders
Vomitting
|
7.7%
1/13 • Number of events 1
|
11.1%
1/9 • Number of events 2
|
|
General disorders
Chest pain
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
General disorders
Facial pain
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
General disorders
Gait disturbance
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
General disorders
Hypothermia
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
General disorders
Injection site erythema
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
General disorders
Injection site mass
|
15.4%
2/13 • Number of events 2
|
11.1%
1/9 • Number of events 1
|
|
General disorders
Injection site pruritus
|
30.8%
4/13 • Number of events 8
|
0.00%
0/9
|
|
General disorders
Injection site warmth
|
7.7%
1/13 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
General disorders
Nodule
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
General disorders
Pyrexia
|
7.7%
1/13 • Number of events 1
|
33.3%
3/9 • Number of events 3
|
|
Infections and infestations
Influenza
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Infections and infestations
Rhinitis
|
0.00%
0/13
|
22.2%
2/9 • Number of events 2
|
|
Infections and infestations
Urinary tract infection
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Injury, poisoning and procedural complications
Foreign body in eye
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Injury, poisoning and procedural complications
Injury
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Injury, poisoning and procedural complications
Retinal injury
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Investigations
Intraocular pressure (IOP) increased
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Investigations
Weight decreased
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Arthalgia
|
7.7%
1/13 • Number of events 1
|
11.1%
1/9 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/13
|
22.2%
2/9 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Muscles spasms
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.7%
1/13 • Number of events 1
|
11.1%
1/9 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Upper extremity mass
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Nervous system disorders
Headache
|
30.8%
4/13 • Number of events 6
|
11.1%
1/9 • Number of events 2
|
|
Nervous system disorders
Parasthesia
|
7.7%
1/13 • Number of events 2
|
0.00%
0/9
|
|
Nervous system disorders
Visual field defect
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Psychiatric disorders
Insomnia
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Reproductive system and breast disorders
Nipple swelling
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.7%
1/13 • Number of events 1
|
44.4%
4/9 • Number of events 6
|
|
Respiratory, thoracic and mediastinal disorders
Orapharyngeal
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Blister
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Dermatitus
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Surgical and medical procedures
Removal of foreign body
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Vascular disorders
Haematoma
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Vascular disorders
Hypertension
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Vascular disorders
Hypotension
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
Ear and labyrinth disorders
Vertigo
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
Eye disorders
Visual acuity decreased (II)
|
0.00%
0/13
|
11.1%
1/9 • Number of events 1
|
|
General disorders
Fatigue
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
|
General disorders
Injection site inflammation
|
15.4%
2/13 • Number of events 5
|
0.00%
0/9
|
|
General disorders
Injection site pain
|
38.5%
5/13 • Number of events 23
|
0.00%
0/9
|
|
General disorders
Injection site rash
|
7.7%
1/13 • Number of events 1
|
0.00%
0/9
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60