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Efficacy and Safety Study of OATD-01 in Patients With Active Pulmonary Sarcoidosis
Study Purpose
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.
Recruitment Criteria
Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
No
Study Type
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
Interventional
Eligible Ages
18 Years and Over
Gender
All
More Inclusion & Exclusion Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- Requirement for immediate start of standard of care therapy for pulmonary sarcoidosis.
- 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.
- Potentially effective systemic or inhaled pharmacological (including investigational)
therapy for sarcoidosis (whether pulmonary or other disease), with the exception of
any of the following:
1.
corticosteroids received not later than 3 months prior to enrolment. 2. immunosuppressants or anti-Tumor Necrosis Factor (TNF) agents (or other
anti-inflammatory/anti-fibrotic treatment) received not later than 4 months prior
to enrolment.
- Systemic treatment indication being an extrapulmonary location of sarcoidosis (e.g.,
neurological)
- Heart conditions: QTcF interval prolongation, cardiac arrhythmia (other than
non-sustained supraventricular arrhythmia), heart failure (New York Heart Association
class III or IV) and/or known myocardial hypertrophy or Left Ventricle Ejection
Fraction <50% in the cardiac MRI.
- Known neurosarcoidosis or small fiber neuropathy or medical conditions causing primary
ataxia.
- Uncontrolled diabetes at Screening with plasma glucose exceeding 8.3 mmol/L, or other
contraindication to [18F]FDG administration and/or PET procedure (including body
temperature >37°C and any metabolic disease affecting the energy metabolism of
muscles) as described in the PET protocol.
- Known positivity for Human Immunodeficiency Virus (HIV 1/2 antibodies), hepatitis B
virus (HBV), or hepatitis C virus (HCV), or detected at screening.
- Severe, uncontrolled systemic disease (e.g., cardiovascular, pulmonary, thyroid, renal
or metabolic disease) at Screening, or other condition, which in the opinion of the
investigator, would compromise the safety of the subject or the subject's ability to
participate in the study.
- Current smoker of >5 cigarettes or e-cigarettes per day or user of
nicotine-releasing alternatives (patches, chewing gums etc)
- Prohibited medications: Current treatment with drug with QT prolongation effect,
thiazide diuretics, strong CYP3A4 inhibitors and/or inducers, P-glycoprotein and/or
BCRP strong inhibitors, drugs that are sensitive substrates of OCT1, MATE1, MATE2K,
OAT3 with a narrow therapeutic index
Trial Details
Trial ID:
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Phase 2
Lead Sponsor
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
Molecure S.A.
Principal Investigator
The person who is responsible for the scientific and technical direction of the entire clinical study.
Samson Fung, MD
Principal Investigator Affiliation
CMO
Agency Class
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
Industry
Overall Status
Recruiting
Countries
United Kingdom, United States
Conditions
The disease, disorder, syndrome, illness, or injury that is being studied.
Pulmonary Sarcoidosis
Additional Details
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.
Arms & Interventions
Arms
Experimental: Active Arm
Subjects will receive OATD-01 as 25mg film-coated tablets for oral administration once daily for 12 weeks
Placebo Comparator: Placebo Arm
Subjects will receive placebo as film-coated tablets for oral administration once daily for 12 weeks
Interventions
Drug: - OATD-01
OATD-01 is an oral inhibitor of chitinase-1 (CHIT1)
Drug: - Placebo
Matching placebo tablets
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.
