A Phase 2 interventional study of OATD-01 and Placebo in Pulmonary Sarcoidosis, sponsored by Molecure S.A.. Recruiting at 28 sites in 8 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-07-30.
Sponsored by Molecure S.A. · Phase 2, Interventional, and Treatment
This is a Phase 2, randomized, double-blind, placebo-controlled, adaptive, multicenter study to evaluate the efficacy, safety, tolerability, Pharmacodynamics (PD), and Pharmacokinetics (PK) of OATD-01 in the treatment of subjects with active pulmonary sarcoidosis.
Adult subjects (≥ 18 years of age) diagnosed with symptomatic pulmonary sarcoidosis and active granulomatous process captured by [18F]Fluorodeoxyglucose Positron emission tomography/computed tomography ([18F]FDG PET/CT) imaging, treatment-naïve or previously treated but currently untreated, will be enrolled in the study. The diagnosis of pulmonary sarcoidosis will be based on the diagnostic criteria for pulmonary sarcoidosis recommended by the American Thoracic Society (ATS, 2020).
Subjects will be randomized in a 1:1 ratio to receive either OATD-01 or placebo for 12 weeks. A stratification of the study population based on previous treatment status for sarcoidosis (previously treated/treatment-naïve) will be applied for statistical analysis without limitation for the ratio between the subject groups. Double-blind conditions will be kept for the whole treatment duration.
Exclusion Criteria:
Potentially effective systemic or inhaled pharmacological (including investigational) therapy for sarcoidosis (whether pulmonary or other disease), with the exception of any of the following:
Subjects will receive OATD-01 as 25mg film-coated tablets for oral administration once daily for 12 weeks
Drug: OATD-01
Subjects will receive placebo as film-coated tablets for oral administration once daily for 12 weeks
Drug: Placebo
OATD-01 is an oral inhibitor of chitinase-1 (CHIT1)
Matching placebo tablets
Response to treatment
Response classed as Complete response, Partial response, Stable disease and Progressive disease based on Standard Uptake Volume (SUV) changes in the uptake for {18F\]FDG-PET/CT above the background (pulmonary parenchyma /ascending aorta) in pulmonary target lesions and any new lesions.
Time frame: After 12 weeks of treatment, i.e. from baseline (randomization) visit to End-of-Treatment (EOT) visit.
Total granulomatous inflammation evaluation
Evaluation by \[18F\]FDG PET/CT imaging, quantified as the percent change of maximum, mean, peak SUV (SUVmax, SUVmean, SUVpeak), and volume of the lesions in pulmonary parenchyma, mediastinal/hilar nodes, and extrathoracic locations.
Time frame: After 12 weeks of treatment, i.e. from baseline (randomization) visit to EOT visit.
Pulmonary function Forced Vital Capacity (FVC)
Absolute change in FVC (% predicted).
Time frame: At Screening visit and over 12 weeks of treatment - at baseline (randomization) visit, at week 4 of treatment, week 8 and week 12 (EOT).
Pulmonary function Forced Expiratory Volume in the first second (FEV1)
Absolute change in FEV1 (% predicted).
Time frame: At Screening visit and over 12 weeks of treatment - at baseline (randomization) visit, at week 4 of treatment, week 8 and week 12 (EOT).
Quality of life assessment
Change in the quality of life measured by the Kings Sarcoidosis Questionnaire General and Lung (KSQ GENERAL and LUNG) scores. Range of each module 0-100, 100= best health status.
Time frame: Assessed at baseline (randomization) visit and after 12 weeks of treatment (EOT visit) or study participation (week 12 visit).
Fatigue Assessment Scale (FAS)
Change in FAS total score. Range: 10 (no fatigue) - 50 (extreme fatigue).
Time frame: Assessed at baseline (randomization) visit and after 12 weeks of treatment (EOT visit) or study participation (week 12 visit).
Adverse events
Occurrence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events, Adverse Events of Special Interest (AESIs), TEAEs leading to discontinuation and TEAEs leading to death.
Time frame: Recorded from the time of signature of informed consent and until 30 days after the last dose of OATD-01/Placebo.
Laboratory tests
Occurrence of clinically significant laboratory (hematology and biochemistry) parameter abnormalities (number of subjects with clinically significant abnormal laboratory values).
Time frame: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8 and week 12 (EOT).
Vital signs - Systolic Blood Pressure
Mean change in Systolic Blood Pressure.
Time frame: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any Follow-up (UP) visits
Vital signs - Diastolic Blood Pressure
Mean change in Diastolic Blood Pressure.
Time frame: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits.
Vital signs
Mean change in Heart Rate.
Time frame: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits.
Vital signs - Respiratory Rate
Mean change in Respiratory Rate
Time frame: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits
Electrocardiography
Occurrence of any clinically significant abnormalities (number of patients with any clinically significant Heart rhythm, PR Interval, QTcF interval or QRS width interval abnormalities) in 12-lead electrocardiography (ECG) or 24-h ECG.
Time frame: 12-lead-ECG measured at screening and over 12 weeks of treatment or study participation - at randomization visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits. 2-week 24-h-ECG recordings at weeks 0, 4 and 8 post randomization.
Electrocardiography - specific parameters
Occurrence of: a) QTcF \>450 ms (male), \>470 ms (female) and \>500 ms (any sex) b) Change from baseline in QTcF \>30 ms and \>60 ms c) Heart rhythm abnormalities including supraventricular arrhythmias, ventricular arrhythmias, and non-sustained ventricular tachycardias.
Time frame: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits.
Male fertility
Occurrence of a clinically significant abnormality of sperm parameters and/ or free testosterone concentration in males (optional testing)
Time frame: Measured at baseline (randomization) visit and at week 8 or week 12 (EOT) of treatment.
Neurological status
Occurrence of TEAEs of sensation abnormalities or ataxia.
Time frame: TEAEs detected during 12 weeks of treatment or study participation - from baseline (randomization) visit and up to FUP visit 7-10 days after last dose
Thyroid and renal function
Proportion of subjects with clinically significant thyroid parameters (Thyroid Stimulating Hormone \[TSH\], Free Triiodothyronine \[FT3\], and Free Thyroxine \[FT4\] and renal function parameters \[blood urea nitrogen (BUN)/total urea, creatinine, creatinine clearance (CrCL)\].
Time frame: Clinically significant abnormalities detected during 12 weeks of treatment - at baseline (randomization) visit, at week 4 of treatment, week 8 and week 12 (EOT).
Pharmacokinetics assessment
Mean plasma OATD-01 concentrations.
Time frame: Measured during 2 weeks of treatment - at baseline (randomization) visit, at week 4 of treatment, week 8 and week 12 (EOT).
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