A phase II, randomised, double blind, placebo-controlled study of the pharmacokinetics, pharmacodynamic effects, and safety, of oral FT011 in participants with diffuse systemic sclerosis
- Conditions
- Diffuse systemic sclerosisSclerodermaSystemic sclerosis (SSc)
- Registration Number
- NL-OMON51103
- Lead Sponsor
- Certa Therapeutics Pty Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
Participants must meet all the following criteria:
• Provide written informed consent prior to any study procedures and who agree
to adhere to all protocol requirements.
• Aged 18 to 75 years inclusive at the time of consent.
• 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.
• 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.
• Have skin thickening in a body area suitable for repeat biopsy.
• Have a mRSS at Screening of >=15 to <=40.
• FVC >=50% of predicted at Screening.
• If on azathioprine, mycophenolate mofetil, or hydroxychloroquine, have been
on a stable dose for at least 2 months prior to baseline.
• Participants must agree to use contraception according to protocol section
5.4.4.
Participants must not meet any of the following criteria:
• Pregnant or breast-feeding, or plan to become pregnant during the study.
• 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.
• Have known or suspected contraindications to the IMP.
• Have severe or unstable SSc or end-stage organ involvement as evidenced by:
o On an organ transplantation list or has received an organ transplant
including autologous stem cell transplant.
o Renal crisis within 1 year prior to Baseline.
• Interstitial lung disease or pulmonary hypertension requiring constant oxygen
therapy. This excludes oxygen used to aid sleep or exercise.
• Gastrointestinal dysmotility requiring total parenteral nutrition or requiring
hospitalisation within the 6 months prior to Baseline.
• 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)
• SSc-like illnesses related to exposures or ingestions
• The use of the following drugs within the specified periods:
o Methotrexate in the 2 weeks prior to Day 1
o Other anti-fibrotic agents including D-penicillamine or tyrosine
kinase inhibitors (nilotinib, imatinib, dasatinib) in the month prior to
Screening.
o Biologic drugs such as tumour necrosing factor (TNF) inhibitors, tocilizumab,
or Janus kinase (JAK) inhibitors, in the 3 months prior to Screening.
o Rituximab in the 6 months prior to Screening.
o Cyclophosphamide oral or intravenous (IV) in the 3 months prior to Screening.
o Oral prednisolone >10 mg per day or IV steroids in the month prior to
Screening.
• 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.
• 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.
• Estimated glomerular filtration rate (eGFR) <60mL/min, urinary
albumin/creatinine ratio <30mg/g.
• Haemoglobin < 80 g/L, platelets < 90 x 109/L, or neutrophil count < 1.4 x
109/L
• 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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary Outcome Measures<br /><br>• FT011 maximum concentration (cmax), time to maximum concentration (tmax), and<br /><br>area under the curve (AUC) in plasma after a single dose and after 12-weeks of<br /><br>treatment.<br /><br>• Measurement of steady state FT011 levels in plasma.</p><br>
- Secondary Outcome Measures
Name Time Method