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NCT06205121PHASE2Recruiting

Efficacy and Safety Study of OATD-01 in Patients With Active Pulmonary Sarcoidosis

Molecure S.A.

Start Date

3/21/2024

Completion Date

9/1/2027

Summary

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.

Detailed Description

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.

Eligibility Criteria

Age Range: 18 years to No maximum

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 * 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 * 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 * Lab abnormalities: Abnormal bilirubin, transaminases, alkaline phosphatase (ALP), Creatinine clearance (CrCL) Hypokalemia hypocalcemia (\<2.1 mmol/L), marked fasting hyperglycemia at screening * 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, pirfenidone and nintedanib.

Interventions

DRUG

OATD-01

DRUG

Placebo

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Conditions

Pulmonary Sarcoidosis

Locations

Molecure Investigative Site

Birmingham, Alabama 35209

United States

Molecure Investigative Site

Kansas City, Kansas 66062

United States

Molecure Investigative Site

Baltimore, Maryland 21224

United States

Molecure Investigative Site

Rochester, Minnesota 55905

United States

Molecure Investigative Site

Cleveland, Ohio 44195

United States

Molecure Investigative Site

Philadelphia, Pennsylvania 19140

United States

Molecure Investigative Site

Charleston, South Carolina 29425

United States

Molecure Investigative Site

Vejle, 7100

Denmark

Molecure Investigative Site

Bobigny, 93000

France

Molecure Investigative Site

Montpellier, 34295

France

Molecure Investigative Site

Paris, 75013

France

Molecure Investigative Site

Paris, 75015

France

Molecure Investigative Site

Essen, 45239

Germany

Molecure Investigative Site

Freiburg im Breisgau, 79106

Germany

Molecure Investigative Site

Mainz-GE, 55131

Germany

Molecure Investigative Site

Corfu, 49100

Greece

Molecure Investigative Site

Heraklion, 71500

Greece

Molecure Investigative Site

Pátrai, 26500

Greece

Molecure Investigative Site

Thessaloniki, 57010

Greece

Molecure Investigative Site

Nieuwegein, 3435 CM

Netherlands

Molecure Investigative Site

Rotterdam, 3015 GD

Netherlands

Molecure Investigative Site

Bergen, 5009

Norway

Molecure Investigative Site

Oslo, 1478

Norway

Molecure Investigative Site

Birmingham, B15 2GW

United Kingdom

Molecure Investigative Site

Cambridge, CB2 0AY

United Kingdom

Molecure Investigative Site

Edinburgh, EH16 4SA

United Kingdom

Molecure Investigative Site

London, SE5 9RS

United Kingdom

Molecure Investigative Site

London, SW3 6JY

United Kingdom