Clinical Trial Results:
A Randomized, Double Masked, Uncontrolled, Multicenter Phase I/II Study to Evaluate Safety and Tolerability of PAN-90806 Eye Drops, Suspension in Treatment-Naïve Participants with Neovascular Age-Related Macular Degeneration (AMD)
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Summary
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EudraCT number |
2016-004601-14 |
Trial protocol |
GB LV CZ HU |
Global end of trial date |
27 Jun 2019
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Results information
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Results version number |
v1(current) |
This version publication date |
26 Feb 2020
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First version publication date |
26 Feb 2020
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
PAN-01-102
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT03479372 | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
IND: 120693 | ||
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Sponsors
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Sponsor organisation name |
PanOptica, Inc
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Sponsor organisation address |
13 McGregor Avenue, Mt Arlington, United States, 07856
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Public contact |
Clinical Trial Information, PanOptica, Inc., +1 9087660899, clinical@panopticapharma.com
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Scientific contact |
Clinical Trial Information, PanOptica, Inc., +1 9087660899, clinical@panopticapharma.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
27 Aug 2019
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
27 Jun 2019
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Global end of trial reached? |
Yes
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Global end of trial date |
27 Jun 2019
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
Assess the safety and tolerability of topical ocular PAN-90806 Eye Drops, Suspension
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Protection of trial subjects |
This study was conducted according to the Declaration of Helsinki and the local laws and regulations relevant to the use of an investigational new drug. All participants signed the informed consent form prior to undergoing study-related procedures. All participants were informed fully of the nature and aims of the study Ample time was provided for the participants to read the informed consent document and ask any questions regarding the investigational drug and study requirements. Participants were informed that their participation was voluntary and that they could withdraw from the study at any time for any reason without incurring penalty or withholding treatment on the part of the investigator. Copies of the signed document were given to the participant and filed in the investigator’s study file.
An independent data monitoring committee reviewed subject safety data during the course of the study.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
27 Apr 2018
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
United Kingdom: 12
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Country: Number of subjects enrolled |
Czech Republic: 13
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Country: Number of subjects enrolled |
Hungary: 3
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Country: Number of subjects enrolled |
Latvia: 3
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Country: Number of subjects enrolled |
United States: 20
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Worldwide total number of subjects |
51
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EEA total number of subjects |
31
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
5
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From 65 to 84 years |
35
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85 years and over |
11
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Recruitment
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Recruitment details |
This study was conducted in 27 centers in 5 countries. Regulatory authority approval & IRB/IEC approvals were obtained prior to opening a center for recruitment. Participants were screened for the study between 27 April 2018 and 04 March 2019. Written informed consent was obtained prior to conducting any of the Screening procedures. | ||||||||||||||||||||
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Pre-assignment
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Screening details |
Treatment-naive patients with newly diagnosed, active, pathologic CNV associated with neovascular AMD were screened for inclusion into the study by assessing medical/ophthalmic history, visual acuity, ophthalmic examination, vital signs, ocular imaging, laboratory & pregnancy tests. Eligible participants were randomized to 1 of 3 doses of PAN-90806 | ||||||||||||||||||||
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Period 1
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Period 1 title |
Overall Trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Double blind | ||||||||||||||||||||
Roles blinded |
Subject, Investigator, Monitor, Carer, Assessor | ||||||||||||||||||||
Blinding implementation details |
The investigational product was coded and labeled in a manner that protected the masking of the study.
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Arms
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Are arms mutually exclusive |
Yes
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Arm title
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2 mg/mL PAN-90806 | ||||||||||||||||||||
Arm description |
- | ||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||
Investigational medicinal product name |
PAN-90806 Eye Drops
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Eye drops, suspension
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Routes of administration |
Ocular use
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Dosage and administration details |
Participants were instructed to administer the PAN-90806 eye drops once daily at approximately the same time every day (before bedtime was recommended) for 12 weeks.
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Arm title
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6 mg/mL PAN-90806 | ||||||||||||||||||||
Arm description |
- | ||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||
Investigational medicinal product name |
PAN-90806 Eye Drops
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Eye drops, suspension
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Routes of administration |
Ocular use
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Dosage and administration details |
Participants were instructed to administer the PAN-90806 eye drops once daily at approximately the same time every day (before bedtime was recommended) for 12 weeks.
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Arm title
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10 mg/mL PAN-90806 | ||||||||||||||||||||
Arm description |
- | ||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||
Investigational medicinal product name |
PAN-90806 Eye Drops
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Eye drops, suspension
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Routes of administration |
Ocular use
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Dosage and administration details |
Participants were instructed to administer the PAN-90806 eye drops once daily at approximately the same time every day (before bedtime was recommended) for 12 weeks.
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Baseline characteristics reporting groups
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Reporting group title |
2 mg/mL PAN-90806
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
6 mg/mL PAN-90806
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
10 mg/mL PAN-90806
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
2 mg/mL PAN-90806
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Reporting group description |
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Reporting group title |
6 mg/mL PAN-90806
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Reporting group description |
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Reporting group title |
10 mg/mL PAN-90806
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Reporting group description |
- | ||
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End point title |
Number of participants with treatment-related adverse events [1] | ||||||||||||
End point description |
The number of participants with treatment-related adverse events was assessed on the Safety population which consisted of all randomized participants who took at least 1 dose of study treatment. Participants were analyzed according to the treatment they actually received.
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End point type |
Primary
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End point timeframe |
The reporting period for Adverse Events began with the signing of the informed consent document and continued until the last study visit.
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: This study was not powered for statistical analysis. Only descriptive statistics were performed on all endpoints |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Change from Baseline in Center Subfield Thickness | ||||||||||||||||
End point description |
Although the study was not designed nor powered to assess efficacy, potential biological responses to treatment were assessed as secondary endpoints by comparing the descriptive changes from baseline. Center Subfield Thickness (CST) was measured by the masked independent reading center. Change from baseline in CST was assessed on the ITT population which consisted of all randomized subjects who received at least one dose of study drug, irrespective of the dose actually received. Subjects were analyzed as per the dose group assigned at randomization.
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End point type |
Secondary
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End point timeframe |
At Week 12
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Number of Ranibizumab Rescue Injections | ||||||||||||||||
End point description |
Although the study was not designed nor powered to assess efficacy, potential biological responses to treatment were assessed as secondary endpoints by comparing the descriptive changes from baseline. A reduced number or lack of need for rescue therapy with intravitreal ranibizumab was viewed as a key indicator of potential anti-VEGF biological activity following topical ocular PAN-90806 treatment. Need for rescue therapy was assessed on the ITT population which consisted of all randomized subjects who received at least one dose of study drug, irrespective of the dose actually received. Subjects were analyzed as per the dose group assigned at randomization.
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End point type |
Secondary
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End point timeframe |
From Week 2 until final study visit
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| No statistical analyses for this end point | |||||||||||||||||
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End point title |
Change in Visual Acuity from Baseline | ||||||||||||||||
End point description |
Although the study was not designed nor powered to assess efficacy, potential biological responses to treatment were assessed as secondary endpoints by comparing the descriptive changes from baseline. Change in visual acuity was assessed on the ITT population which consisted of all randomized subjects who received at least one dose of study drug, irrespective of the dose actually received. Subjects were analyzed as per the dose group assigned at randomization.
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End point type |
Secondary
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End point timeframe |
Week 12
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| No statistical analyses for this end point | |||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
The evaluation period for adverse events began with the signing of the informed consent document and continued until the last study visit
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
21.0
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Reporting groups
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Reporting group title |
2mg/mL PAN-90806
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Reporting group description |
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Reporting group title |
6 mg/mL PAN-90806
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
10 mg/mL PAN-90806
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
