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

NCT ID: NCT06205121

Last Updated: 2025-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

96 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-21

Study Completion Date

2027-09-30

Brief Summary

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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

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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.

Conditions

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Pulmonary Sarcoidosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Active Arm

Subjects will receive OATD-01 as 25mg film-coated tablets for oral administration once daily for 12 weeks

Group Type EXPERIMENTAL

OATD-01

Intervention Type DRUG

OATD-01 is an oral inhibitor of chitinase-1 (CHIT1)

Placebo Arm

Subjects will receive placebo as film-coated tablets for oral administration once daily for 12 weeks

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Matching placebo tablets

Interventions

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OATD-01

OATD-01 is an oral inhibitor of chitinase-1 (CHIT1)

Intervention Type DRUG

Placebo

Matching placebo tablets

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Molecure S.A.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Piotr Iwanowski, MD

Role: STUDY_CHAIR

CMO

Locations

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Molecure Investigative Site

Birmingham, Alabama, United States

Site Status WITHDRAWN

Molecure Investigative Site

Kansas City, Kansas, United States

Site Status WITHDRAWN

Molecure Investigative Site

Baltimore, Maryland, United States

Site Status RECRUITING

Molecure Investigative Site

Rochester, Minnesota, United States

Site Status WITHDRAWN

Molecure Investigative Site

Cleveland, Ohio, United States

Site Status RECRUITING

Molecure Investigative Site

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Molecure Investigative Site

Charleston, South Carolina, United States

Site Status RECRUITING

Molecure Investigative Site

Vejle, , Denmark

Site Status WITHDRAWN

Molecure Investigative Site

Bobigny, , France

Site Status WITHDRAWN

Molecure Investigative Site

Montpellier, , France

Site Status RECRUITING

Molecure Investigative Site

Paris, , France

Site Status RECRUITING

Molecure Investigative Site

Paris, , France

Site Status RECRUITING

Molecure Investigative Site

Essen, , Germany

Site Status RECRUITING

Molecure Investigative Site

Freiburg im Breisgau, , Germany

Site Status RECRUITING

Molecure Investigative Site

Mainz-GE, , Germany

Site Status RECRUITING

Molecure Investigative Site

Corfu, , Greece

Site Status WITHDRAWN

Molecure Investigative Site

Heraklion, , Greece

Site Status RECRUITING

Molecure Investigative Site

Pátrai, , Greece

Site Status RECRUITING

Molecure Investigative Site

Thessaloniki, , Greece

Site Status RECRUITING

Molecure Investigative Site

Nieuwegein, , Netherlands

Site Status RECRUITING

Molecure Investigative Site

Rotterdam, , Netherlands

Site Status RECRUITING

Molecure Investigative Site

Bergen, , Norway

Site Status RECRUITING

Molecure Investigative Site

Oslo, , Norway

Site Status RECRUITING

Molecure Investigative Site

Birmingham, , United Kingdom

Site Status RECRUITING

Molecure Investigative Site

Cambridge, , United Kingdom

Site Status WITHDRAWN

Molecure Investigative Site

Edinburgh, , United Kingdom

Site Status RECRUITING

Molecure Investigative Site

London, , United Kingdom

Site Status RECRUITING

Molecure Investigative Site

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Denmark France Germany Greece Netherlands Norway United Kingdom

Central Contacts

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Theodoros Charitos, MD

Role: CONTACT

+48789125928

Other Identifiers

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2023-506642-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

OATD-01-C-03

Identifier Type: -

Identifier Source: org_study_id

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