FT011 for Scleroderma

Phase 1

Conditions: diffuse systemic sclerosis
MedDRA version: 21.0Level: LLTClassification code 10012977Term: Diffuse systemic sclerosisSystem Organ Class: 100000004859
Therapeutic area: Body processes [G] - Immune system processes [G12]

Registration Number: EUCTR2020-005116-21-PL

Lead Sponsor: Certa Therapeutics Pty Ltd

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 30

Inclusion Criteria

1. Provide written informed consent prior to any study procedures and who agree to adhere to all
protocol requirements.
2. Aged 18 to 75 years inclusive at the time of consent.
3. Have a classification of systemic sclerosis, as defined by American College of Rheumatology (ACR)
and European League Against Rheumatism (EULAR) criteria with disease duration =5 years from
first non-Raynaud phenomenon manifestation.
4. Have a diagnosis of diffuse cutaneous SSc defined as systemic sclerosis with skin thickening on the
upper arms proximal to the elbows, on the upper legs proximal to the knees, or on the trunk.
5. Have skin thickening in a body area suitable for repeat biopsy.
6. Have a mRSS at Screening of =15 to =40.
7. FVC =50% of predicted at Screening.
8. If on azathioprine, mycophenolate mofetil, or hydroxychloroquine, have been on a stable dose for
at least 2 months prior to baseline.
9. Participants must agree to use contraception according to protocol section 5.4.4.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 18
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 12

Exclusion Criteria

1. Pregnant or breast-feeding, or plan to become pregnant during the study.
2. Have received any IMP within 30 days or 5 half-lives prior to randomisation (4 months if the
previous drug was a new chemical entity), whichever is longer.
3. Have known or suspected contraindications to the IMP.
4. Have severe or unstable SSc or end-stage organ involvement as evidenced by:
a. On an organ transplantation list or has received an organ transplant including autologous
stem cell transplant.
b. Renal crisis within 1 year prior to Baseline.
5. Interstitial lung disease or pulmonary hypertension requiring constant oxygen therapy. This
excludes oxygen used to aid sleep or exercise.
6. Gastrointestinal dysmotility requiring total parenteral nutrition or requiring hospitalisation within
the 6 months prior to Baseline.
7. Concomitant inflammatory myositis, rheumatoid arthritis, or systemic lupus erythematosus when
definite classification criteria for those diseases are met (Bohan and Peter criteria for polymyositis
and dermatomyositis)
8. SSc-like illnesses related to exposures or ingestions
9. The use of the following drugs within the specified periods:
a. Methotrexate in the 2 weeks prior to Day 1
b. Other anti-fibrotic agents including D-penicillamine or tyrosine kinase inhibitors (nilotinib,
imatinib, dasatinib) in the month prior to Screening.
c. Biologic drugs such as tumour necrosing factor (TNF) inhibitors, tocilizumab, or Janus
kinase (JAK) inhibitors, in the 3 months prior to Screening.
d. Rituximab in the 6 months prior to Screening.
e. Cyclophosphamide oral or IV in the 3 months prior to Screening.
f. Oral prednisolone >10 mg per day or IV steroids in the month prior to Screening.
10. Have any malignancy not considered cured (except basal cell or squamous cell carcinoma of the
skin, or carcinoma in situ of the cervix); a subject is considered cured if there has been no evidence
of cancer recurrence for the 6 years prior to randomisation.
11. Have aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase
(GGT), lactate dehydrogenase (LDH), or bilirubin values above the upper limit of normal (ULN) at
Screening or Baseline, or evidence of hepatic disease as determined by any one of the following:
history of hepatic encephalopathy, history of oesophageal varices, or history of portacaval shunt.
12. Estimated glomerular filtration rate (eGFR) <60mL/min, urinary albumin/creatinine ratio <30mg/g.
13. Haemoglobin < 80 g/L, platelets < 90 x 109/L, or neutrophil count < 1.4 x 109/L
14. Other than SSc, have any other medical condition or significant co-morbidities, clinically relevant
social or psychiatric conditions, or any finding during Screening, which in the investigator’s opinion
may put the subject at risk or interfere with the study objectives.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the PK of oral FT011 in participants with diffuse SSc;Secondary Objective: To assess the safety and tolerability of oral FT011 compared to placebo in participants with diffuse<br>SSc.<br><br>To evaluate the short-term efficacy of oral FT011 compared to placebo in improving disease<br>activity in participants with diffuse SSc.;Primary end point(s): • FT011 cmax, tmax, and AUC in plasma after a single dose and after 12-weeks of treatment.<br>• Measurement of steady state FT011 levels in plasma.<br><br>Additional calculations may be performed, and metabolites may also be measured.;Timepoint(s) of evaluation of this end point: FT011 cmax, tmax, and AUC in plasma after a single dose on day 1 and after last dose at week 12<br>Steady state concentrations will be at the 1, 2, and 3 month pre-dose timepoints

Secondary Outcome Measures

Name	Time	Method

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