Efficacy and Safety Study of OATD-01 in Patients With Active Pulmonary Sarcoidosis
ConditionPulmonary Sarcoidosis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorMolecure S.A.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male and female subjects with active symptomatic pulmonary sarcoidosis, (definite diagnosis of active pulmonary sarcoidosis per ATS guidelines)
Treatment-naïve or previously treated (no recruitment cap)
Parenchymal pulmonary involvement on [18F]FDG PET/CT
Disqualifiers
Requirement for immediate start of standard of care therapy for pulmonary sarcoidosis
Active cardiac or neuro- sarcoidosis
History of/active Löfgren syndrome
Clinically significant lung disease other than sarcoidosis (e.g. tuberculosis, asthma, Chronic Obstructive Pulmonary Disease, interstitial lung disease, lung cancer) or any current inflammatory or immunological systemic disease other than sarcoidosis
Trial design
Design model
Parallel
Treatments tested in this trial
OATD-01
DrugOATD-01 is an oral inhibitor of chitinase-1 (CHIT1)
Placebo
DrugMatching placebo tablets
Treatment groups
96 Participants
are divided into 2 treatment groupsGroup A: Active ArmExperimental treatment 1 intervention
Group B: Placebo ArmPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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.Secondary outcomes
1
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.2
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).3
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).4
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).Sponsors and contacts
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Source ClinicalTrials.gov