Trial Outcomes & Findings for Effects of FT011 in Systemic Sclerosis (NCT NCT04647890)

Last Updated: 2023-12-20

Results Overview

Measurement of maximum concentration (cmax) of FT011. First dose Cmax and last dose Cmax for each participant were averaged together to calculate a single mean Cmax for each treatment arm

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

Mean of cmax post first dose and cmax post last dose

Results posted on

2023-12-20

Participant Flow

Participant milestones

Participant milestones
Measure	FT011 200mg 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
Overall Study STARTED	10	10	10
Overall Study COMPLETED	9	9	10
Overall Study NOT COMPLETED	1	1	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Effects of FT011 in Systemic Sclerosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	FT011 200mg n=10 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=10 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks	Total n=30 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	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	9 Participants n=5 Participants	8 Participants n=7 Participants	9 Participants n=5 Participants	26 Participants n=4 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	4 Participants n=4 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	9 Participants n=7 Participants	6 Participants n=5 Participants	22 Participants n=4 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants	8 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	10 Participants n=5 Participants	10 Participants n=7 Participants	10 Participants n=5 Participants	30 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants

PRIMARY outcome

Timeframe: Mean of cmax post first dose and cmax post last dose

Population: Placebo participants not tested

Measurement of maximum concentration (cmax) of FT011. First dose Cmax and last dose Cmax for each participant were averaged together to calculate a single mean Cmax for each treatment arm

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
FT011 Levels in Plasma	5.45 ug/mL Standard Deviation 1.94	10.3 ug/mL Standard Deviation 4.27	—

PRIMARY outcome

Timeframe: Mean of time to cmax post first dose and time to cmax post last dose

Population: Placebo participants not tested

Measurement of time to cmax (tmax). First dose tmax and last dose tmax for each participant were averaged together to calculate a single mean tmax for each treatment arm

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
FT011 Levels in Plasma	4.19 hours Standard Deviation 1.75	4.20 hours Standard Deviation 1.61	—

PRIMARY outcome

Timeframe: Mean of AUC hours post first dose and AUC hours post last dose

Population: Placebo participants not tested

Measurement of area under the concentration time curve (AUC). First dose AUC and last dose AUC for each participant were averaged together to calculate a single mean AUC for each active arm

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
FT011 Levels in Plasma	29.8 h*ug/mL Standard Deviation 11.1	57.8 h*ug/mL Standard Deviation 29.0	—

SECONDARY outcome

Timeframe: Baseline to Week 16

TEAEs per arm during study treatment and follow up periods

Outcome measures

Outcome measures
Measure	FT011 200mg n=10 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=10 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) From Baseline to End of Study	7 Participants	4 Participants	6 Participants

SECONDARY outcome

Timeframe: End of treatment (week 12)

The mRSS is a validated physical evaluation of patient's skin thickness rated by clinical palpation using a 0-3 scale (0 = normal skin; 1 = mild thickness; 2 = moderate thickness; 3 = severe thickness with inability to pinch the skin into a fold) for each of 17 surface anatomic areas of the body: face, anterior chest, abdomen, and, with right and left sides of the body separately evaluated, the fingers, forearms, upper arms, thighs, lower legs, dorsum of hands and feet. Individual values are summed and defined as the total skin score. Total score is 0 to 51 with higher scores indicating worse symptomology

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=9 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
mRSS Change From Baseline	-3.7 score on a scale Standard Deviation 4.30	-3.1 score on a scale Standard Deviation 4.15	-2.7 score on a scale Standard Deviation 3.53

SECONDARY outcome

Timeframe: End of treatment (Week 12)

Percent predicted FVC is calculated using equations incorporating age, gender, and race. It is calculated as (FVC Observed / FVC predicted) x 100, where FVC predicted is calculated relative to a reference population

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=9 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
%FVC Change From Baseline	-2 percentage of FVC change Standard Deviation 2.24	4.7 percentage of FVC change Standard Deviation 8.68	-1.7 percentage of FVC change Standard Deviation 4.24

SECONDARY outcome

Timeframe: End of treatment (Week 12)

The physician's assessment of the patient's SSc status will be scored on a 100-mm horizontal VAS, ranging from 0 on the extreme left end of the scale indicating "no disease activity" (symptom free), and 100 on the extreme right end indicating "worst imaginable disease activity".

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=9 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
Physician Global Assessment Change From Baseline	-15.2 score on a scale Standard Deviation 19.78	-28.4 score on a scale Standard Deviation 22.89	-6.3 score on a scale Standard Deviation 18.29

SECONDARY outcome

Timeframe: End of treatment (Week 12)

The Patient's Global Assessment represents the patient's overall assessment of current SSc status on a 100-mm horizontal VAS, ranging from 0 on the extreme left end of the scale indicating "no disease activity" (symptom free), and 100 on the extreme right end indicating "worst imaginable disease activity".

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=9 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
Patient Global Assessment Change From Baseline	23 score on a scale Standard Deviation 9.53	10.8 score on a scale Standard Deviation 30.07	2.7 score on a scale Standard Deviation 19.99

SECONDARY outcome

Timeframe: End of treatment (Week 12)

The HAQ-DI consists of 20 questions referring to eight component sets consisting of dressing/grooming, arising, eating, walking, hygiene, reach, grip, and activities. Each item is scored from 0 to 3. The eight scores of the eight sections are summed and divided by 8. The total score indicates the patient's self-assessed level of disability - higher scores indicate worse symptomology. A negative change from baseline indicates improvement. The Scleroderma HAQ (SHAQ) includes an additional five scleroderma-specific visual analogue scales (VAS), addressing overall disease activity, Raynaud's phenomenon, finger ulcers, breathing, and intestinal problems. A composite VAS score is not created nor are the individual VAS scores incorporated into the HAQ DI score.

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=9 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
Scleroderma HAQ-DI Change From Baseline	-0.0694 score on a scale Standard Deviation 0.26598	-0.2625 score on a scale Standard Deviation 0.23162	0.0278 score on a scale Standard Deviation 0.24826

SECONDARY outcome

Timeframe: Week 12

CRISS components include modified Rodnan skin score (mRSS), forced vital capacity percent predicted (%FVC), Physician Global Assessment, Patient Global Assessment, and Health Assessment Questionnaire Disability-Index (HAQ-DI). The exponential algorithm determines the predicted probability of improvement from baseline, incorporating change in these 5 components. The outcome is a continuous variable between 0.0 and 1.0 (0 - 100%). A higher score indicates greater improvement

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=8 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (CRISS) at Week 12	0.282 score on a scale Standard Deviation 0.4073	0.542 score on a scale Standard Deviation 0.4535	0.131 score on a scale Standard Deviation 0.2397

SECONDARY outcome

Timeframe: End of treatment (Week 12)

The SCTC-DI is a 23-item composite damage index to quantify organ damage in systemic sclerosis (0-55 scale; moderate damage \>5, severe damage\>12)

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=9 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
Scleroderma Clinical Trial Consortium Damage Index (SCTC-DI) Change From Baseline	-0.3 score on a scale Standard Deviation 0.71	-0.3 score on a scale Standard Deviation 0.95	-1.2 score on a scale Standard Deviation 2.54

SECONDARY outcome

Timeframe: End of treatment (Week 12)

The 5-D itch scale is a 23-item validated instrument used to measure five domains of chronic itch: duration, degree, direction, disability, and distribution. Scores range from 5 to 25, with higher scores indicating a higher severity of chronic itch.

Outcome measures

Outcome measures
Measure	FT011 200mg n=9 Participants 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 Participants 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=9 Participants Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
5-D Itch Scale Change From Baseline	0.7 score on a scale Standard Deviation 2.55	-0.7 score on a scale Standard Deviation 4.47	0.2 score on a scale Standard Deviation 3.42

Adverse Events

FT011 200mg

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

FT011 400mg

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Prof Darren Kelly, CEO

Certa Therapeutics Pty Ltd

Phone: +61 3 9657 0700

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	FT011 200mg n=10 participants at risk 200mg once daily for 12 weeks FT011: Two x 100mg capsules once daily for 12 weeks	FT011 400mg n=10 participants at risk 400mg once daily for 12 weeks FT011: Two x 200mg capsules once daily for 12 weeks	Placebo n=10 participants at risk Placebo once daily for 12 weeks Placebo: Two placebo capsules once daily for 12 weeks
Infections and infestations Nasopharangytis	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	20.0% 2/10 • Number of events 3 • Over the duration of the study period (12 months)	30.0% 3/10 • Number of events 3 • Over the duration of the study period (12 months)
Infections and infestations COVID-19	20.0% 2/10 • Number of events 2 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	20.0% 2/10 • Number of events 2 • Over the duration of the study period (12 months)
Infections and infestations Upper respiratory tract infection	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 2 • Over the duration of the study period (12 months)
Infections and infestations Genital candidiasis	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)
Infections and infestations Hordeolum	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Infections and infestations Lower respiratory infection	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Gastrointestinal disorders Diarrhoea	0.00% 0/10 • Over the duration of the study period (12 months)	20.0% 2/10 • Number of events 4 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Gastrointestinal disorders Vomiting	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Gastrointestinal disorders Abdominal pain	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)
Gastrointestinal disorders Constipation	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Gastrointestinal disorders Dyspepsia	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)
Gastrointestinal disorders Mouth ulceration	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Gastrointestinal disorders Nausea	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Investigations Escherichia test positive	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Investigations Haemoglobin decreased	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Investigations Lymphocyte count decreased	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)
Musculoskeletal and connective tissue disorders Tenosynovitis stenosans	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)
Eye disorders Blepharitis	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Eye disorders Dry eye	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)
Nervous system disorders Headache	0.00% 0/10 • Over the duration of the study period (12 months)	20.0% 2/10 • Number of events 2 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Nervous system disorders Hypoaesthesia	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Respiratory, thoracic and mediastinal disorders Cough	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Respiratory, thoracic and mediastinal disorders Vocal cord disorder	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Blood and lymphatic system disorders Anaemia	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Injury, poisoning and procedural complications Limb injury	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Metabolism and nutrition disorders Hypokalaemia	0.00% 0/10 • Over the duration of the study period (12 months)	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Vascular disorders Raynaud's phenomenon	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)
Infections and infestations Sinusitis	10.0% 1/10 • Number of events 1 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)	0.00% 0/10 • Over the duration of the study period (12 months)