Recruiting
Phase 2
Efficacy and Safety Study of OATD-01 in Patients With Active Pulmonary Sarcoidosis
Condition(s) studied
Pulmonary Sarcoidosis
Investigational drug(s) / intervention(s)
OATD-01Placebo
OATD-01: OATD-01 is an oral inhibitor of chitinase-1 (CHIT1)
Placebo: Matching placebo tablets
Study 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.
Eligibility
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.
Primary outcome measure(s)
- Response to treatment — After 12 weeks of treatment, i.e. from baseline (randomization) visit to End-of-Treatment (EOT) visit.
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.
Trial sites (28)
| Facility | City | Region | Status |
| Molecure Investigative Site |
Birmingham |
Alabama |
Withdrawn |
| Molecure Investigative Site |
Kansas City |
Kansas |
Withdrawn |
| Molecure Investigative Site |
Baltimore |
Maryland |
Recruiting |
| Molecure Investigative Site |
Rochester |
Minnesota |
Withdrawn |
| Molecure Investigative Site |
Cleveland |
Ohio |
Recruiting |
| Molecure Investigative Site |
Philadelphia |
Pennsylvania |
Recruiting |
| Molecure Investigative Site |
Charleston |
South Carolina |
Recruiting |
| Molecure Investigative Site |
Vejle |
Denmark |
Withdrawn |
| Molecure Investigative Site |
Bobigny |
France |
Withdrawn |
| Molecure Investigative Site |
Montpellier |
France |
Recruiting |
| Molecure Investigative Site |
Paris |
France |
Recruiting |
| Molecure Investigative Site |
Paris |
France |
Recruiting |
| Molecure Investigative Site |
Essen |
Germany |
Recruiting |
| Molecure Investigative Site |
Freiburg im Breisgau |
Germany |
Recruiting |
| Molecure Investigative Site |
Mainz-GE |
Germany |
Recruiting |
| Molecure Investigative Site |
Corfu |
Greece |
Withdrawn |
| Molecure Investigative Site |
Heraklion |
Greece |
Recruiting |
| Molecure Investigative Site |
Pátrai |
Greece |
Recruiting |
| Molecure Investigative Site |
Thessaloniki |
Greece |
Recruiting |
| Molecure Investigative Site |
Nieuwegein |
Netherlands |
Recruiting |
| Molecure Investigative Site |
Rotterdam |
Netherlands |
Recruiting |
| Molecure Investigative Site |
Bergen |
Norway |
Recruiting |
| Molecure Investigative Site |
Oslo |
Norway |
Recruiting |
| Molecure Investigative Site |
Birmingham |
United Kingdom |
Recruiting |
| Molecure Investigative Site |
Cambridge |
United Kingdom |
Withdrawn |
| Molecure Investigative Site |
Edinburgh |
United Kingdom |
Recruiting |
| Molecure Investigative Site |
London |
United Kingdom |
Recruiting |
| Molecure Investigative Site |
London |
United Kingdom |
Recruiting |
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