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

NCT06205121 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: OATD-01-C-032023-506642-23
• Study Type: interventional
• Target: 96
• Locations: 8 countries
• Sites: 28

Brief 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.

Conditions

Pulmonary Sarcoidosis

Keywords

Pulmonary Sarcoidosis; Sarcoidosis; Chitinase

Outcome Measures

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.

  After 12 weeks of treatment, i.e. from baseline (randomization) visit to End-of-Treatment (EOT) visit.

Secondary Outcomes (17)

• Total granulomatous inflammation evaluation

  After 12 weeks of treatment, i.e. from baseline (randomization) visit to EOT visit.

• Pulmonary function Forced Vital Capacity (FVC)

  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)

  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

  Assessed at baseline (randomization) visit and after 12 weeks of treatment (EOT visit) or study participation (week 12 visit).

• Fatigue Assessment Scale (FAS)

  Assessed at baseline (randomization) visit and after 12 weeks of treatment (EOT visit) or study participation (week 12 visit).

Study Arms (2)

Active Arm

EXPERIMENTAL

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

Drug: OATD-01

Placebo Arm

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

OATD-01 DRUG

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

Active Arm

Placebo DRUG

Matching placebo tablets

Placebo Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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:
• corticosteroids received not later than 3 months prior to enrolment
• 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)

Sponsors & Collaborators

• Molecure S.A.: lead

Study Sites (28)

Molecure Investigative Site

Birmingham, Alabama, 35209, United States

WITHDRAWN

Molecure Investigative Site

Kansas City, Kansas, 66062, United States

WITHDRAWN

Molecure Investigative Site

Baltimore, Maryland, 21224, United States

RECRUITING

Molecure Investigative Site

Rochester, Minnesota, 55905, United States

WITHDRAWN

Molecure Investigative Site

Cleveland, Ohio, 44195, United States

RECRUITING

Molecure Investigative Site

Philadelphia, Pennsylvania, 19140, United States

RECRUITING

Molecure Investigative Site

Charleston, South Carolina, 29425, United States

RECRUITING

Molecure Investigative Site

Vejle, 7100, Denmark

WITHDRAWN

Molecure Investigative Site

Bobigny, 93000, France

WITHDRAWN

Molecure Investigative Site

Montpellier, 34295, France

RECRUITING

Molecure Investigative Site

Paris, 75013, France

RECRUITING

Molecure Investigative Site

Paris, 75015, France

RECRUITING

Molecure Investigative Site

Essen, 45239, Germany

RECRUITING

Molecure Investigative Site

Freiburg im Breisgau, 79106, Germany

RECRUITING

Molecure Investigative Site

Mainz-GE, 55131, Germany

RECRUITING

Molecure Investigative Site

Corfu, 49100, Greece

WITHDRAWN

Molecure Investigative Site

Heraklion, 71500, Greece

RECRUITING

Molecure Investigative Site

Pátrai, 26500, Greece

RECRUITING

Molecure Investigative Site

Thessaloniki, 57010, Greece

RECRUITING

Molecure Investigative Site

Nieuwegein, 3435 CM, Netherlands

RECRUITING

Molecure Investigative Site

Rotterdam, 3015 GD, Netherlands

RECRUITING

Molecure Investigative Site

Bergen, 5009, Norway

RECRUITING

Molecure Investigative Site

Oslo, 1478, Norway

RECRUITING

Molecure Investigative Site

Birmingham, B15 2GW, United Kingdom

RECRUITING

Molecure Investigative Site

Cambridge, CB2 0AY, United Kingdom

WITHDRAWN

Molecure Investigative Site

Edinburgh, EH16 4SA, United Kingdom

RECRUITING

Molecure Investigative Site

London, SE5 9RS, United Kingdom

RECRUITING

Molecure Investigative Site

London, SW3 6JY, United Kingdom

RECRUITING

MeSH Terms

Conditions

Sarcoidosis, Pulmonary; Sarcoidosis

Interventions

OATD-01

Condition Hierarchy (Ancestors)

Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Hypersensitivity, Delayed; Hypersensitivity; Immune System Diseases

Study Officials

• Piotr Iwanowski, MD

  CMO

  STUDY CHAIR

Central Study Contacts

Theodoros Charitos, MD

CONTACT

+48789125928; t.charitos@molecure.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 13, 2023
• First Posted: January 12, 2024
• Study Start: March 21, 2024
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): September 1, 2027
• Last Updated: December 12, 2025

Record last verified: 2025-08

Locations

US (7); NO (2); DK (1); FR (4); GR (4); GB (5); DE (3); NL (2)