Trial Outcomes & Findings for Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon) (NCT NCT02230761)

NCT ID: NCT02230761

Last Updated: 2016-02-08

Results Overview

Photonumeric scale, range 0-4, 0 is absence of steatoblepharon, 4 is very severe steatoblepharon.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 90 participants
• Primary outcome timeframe: 11 weeks
• Results posted on: 2016-02-08

Participant Flow

Participant milestones

Measure	XOPH5 Ointment XOPH5 Ointment is the investigational drug to be studied. XOPH5 Ointment	Placebo Ointment Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient. Placebo
Overall Study STARTED	60	30
Overall Study COMPLETED	60	29
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Phase 2 Study of XAF5 (XOPH5) Ointment for Reduction of Excess Eyelid Fat (Steatoblepharon)

Baseline characteristics by cohort

Measure	XOPH5 Ointment n=60 Participants XOPH5 Ointment is the investigational drug to be studied. XOPH5 Ointment	Placebo Ointment n=30 Participants Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient. Placebo	Total n=90 Participants Total of all reporting groups
Age, Continuous	54.2 years STANDARD_DEVIATION 7.4 • n=5 Participants	55.1 years STANDARD_DEVIATION 7.5 • n=7 Participants	54.5 years STANDARD_DEVIATION 7.4 • n=5 Participants
Sex: Female, Male Female	40 Participants n=5 Participants	17 Participants n=7 Participants	57 Participants n=5 Participants
Sex: Female, Male Male	20 Participants n=5 Participants	13 Participants n=7 Participants	33 Participants n=5 Participants

PRIMARY outcome

Timeframe: 11 weeks

Population: intention-to-treat

Photonumeric scale, range 0-4, 0 is absence of steatoblepharon, 4 is very severe steatoblepharon.

Outcome measures

Measure	XOPH5 Ointment n=60 Participants XOPH5 Ointment is the investigational drug to be studied. XOPH5 Ointment	Placebo Ointment n=30 Participants Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient. Placebo
Lower Eyelid Steatoblepharon Severity (LESS) Score--Clinician-Reported	27 participants	6 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 0-11 weeks

Population: intention-to-treat

Change from Baseline to Week 11 in PSS1 score. PSS1 scale items use an ordinal scale with range 0-10, where 0 is the least favorable and 10 is the most favorable. The composite score is the sum of 6 items and has a possible response range of 0 to 60.

Outcome measures

Measure	XOPH5 Ointment n=60 Participants XOPH5 Ointment is the investigational drug to be studied. XOPH5 Ointment	Placebo Ointment n=30 Participants Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient. Placebo
Patient Satisfaction Scale 1 (PSS1)	17.9 units on a scale Standard Deviation 14.4	7.3 units on a scale Standard Deviation 13.0

OTHER_PRE_SPECIFIED outcome

Timeframe: 11 weeks

Population: intention-to-treat

Composite score on PSS2 scale. PSS2 is an ordinal scale with range 1-7, where 1 is least favorable and 7 is most favorable. The composite score is the sum of 3 items and has a possible response range of 3 to 21.

Outcome measures

Measure	XOPH5 Ointment n=60 Participants XOPH5 Ointment is the investigational drug to be studied. XOPH5 Ointment	Placebo Ointment n=30 Participants Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient. Placebo
Patient Satisfaction Scale 2 (PSS2)	16.3 units on a scale Standard Deviation 3.3	14.0 units on a scale Standard Deviation 3.0

Adverse Events

XOPH5 Ointment

Serious events: 0 serious events

Other events: 11 other events

Deaths: 0 deaths

Placebo Ointment

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	XOPH5 Ointment n=60 participants at risk XOPH5 Ointment is the investigational drug to be studied. XOPH5 Ointment	Placebo Ointment n=30 participants at risk Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient. Placebo
Nervous system disorders Guillan-Barre syndrome	0.00% 0/60	3.3% 1/30 • Number of events 1

Other adverse events

Measure	XOPH5 Ointment n=60 participants at risk XOPH5 Ointment is the investigational drug to be studied. XOPH5 Ointment	Placebo Ointment n=30 participants at risk Placebo is an ointment that matches XOPH5 Ointment but lacks the active ingredient. Placebo
Skin and subcutaneous tissue disorders Hyperemia eyelid (mild, transient)	15.0% 9/60 • Number of events 9	0.00% 0/30
Skin and subcutaneous tissue disorders Increase in vellus hair (mild)	6.7% 4/60 • Number of events 4	0.00% 0/30

Additional Information

Chief Scientific Officer

Topokine Therapeutics, Inc.

Phone: 617 203 2034

Email: CSO@topokine.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60