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Pulmonary SarcoidosisPhase 2NCT06205121

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

A Phase 2 study testing OATD-01 in people with Pulmonary Sarcoidosis, run by Molecure S.A. at 28 sites in 8 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Pulmonary Sarcoidosis
What is being tested?
OATD-01
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
96 participants
Where
28 sitesin 8 countries
Study dates
21 Mar 2024to Sep 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 16Show fewer
  • 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.

Trial locations and contacts

28 sites in 8 countries. Contact the study team or a site near you to ask about taking part.

28 sites in 8 countries

United States7 sites, 4 recruiting
  • Molecure Investigative Site

    Baltimore, Maryland, 21224

    Recruiting
  • Molecure Investigative Site

    Birmingham, Alabama, 35209

    Withdrawn
  • Molecure Investigative Site

    Charleston, South Carolina, 29425

    Recruiting
  • Molecure Investigative Site

    Cleveland, Ohio, 44195

    Recruiting
  • Molecure Investigative Site

    Kansas City, Kansas, 66062

    Withdrawn
  • Molecure Investigative Site

    Philadelphia, Pennsylvania, 19140

    Recruiting
  • Molecure Investigative Site

    Rochester, Minnesota, 55905

    Withdrawn

Other trials in United States

United Kingdom5 sites, 4 recruiting
  • Molecure Investigative Site

    Birmingham, B15 2GW

    Recruiting
  • Molecure Investigative Site

    Cambridge, CB2 0AY

    Withdrawn
  • Molecure Investigative Site

    Edinburgh, EH16 4SA

    Recruiting
  • Molecure Investigative Site

    London, SE5 9RS

    Recruiting
  • Molecure Investigative Site

    London, SW3 6JY

    Recruiting

Other trials in United Kingdom

France4 sites, 3 recruiting
  • Molecure Investigative Site

    Bobigny, 93000

    Withdrawn
  • Molecure Investigative Site

    Montpellier, 34295

    Recruiting
  • Molecure Investigative Site

    Paris, 75013

    Recruiting
  • Molecure Investigative Site

    Paris, 75015

    Recruiting

Other trials in France

Greece4 sites, 3 recruiting
  • Molecure Investigative Site

    Corfu, 49100

    Withdrawn
  • Molecure Investigative Site

    Heraklion, 71500

    Recruiting
  • Molecure Investigative Site

    Pátrai, 26500

    Recruiting
  • Molecure Investigative Site

    Thessaloniki, 57010

    Recruiting

Other trials in Greece

Germany3 sites, 3 recruiting
  • Molecure Investigative Site

    Essen, 45239

    Recruiting
  • Molecure Investigative Site

    Freiburg im Breisgau, 79106

    Recruiting
  • Molecure Investigative Site

    Mainz-GE, 55131

    Recruiting

Other trials in Germany

Netherlands2 sites, 2 recruiting
  • Molecure Investigative Site

    Nieuwegein, 3435 CM

    Recruiting
  • Molecure Investigative Site

    Rotterdam, 3015 GD

    Recruiting

Other trials in Netherlands

Norway2 sites, 2 recruiting
  • Molecure Investigative Site

    Bergen, 5009

    Recruiting
  • Molecure Investigative Site

    Oslo, 1478

    Recruiting

Other trials in Norway

Denmark1 site
  • Molecure Investigative Site

    Vejle, 7100

    Withdrawn

Other trials in Denmark

About this study

Official title: A Randomized, Double-blind, Placebo-controlled, Multicenter Study to Assess the Efficacy and Safety of a 12-week Administration of OATD-01, an Oral Inhibitor of Chitinase-1 (CHIT1), for the Treatment of Active Pulmonary Sarcoidosis (the KITE Study)

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.

Read the 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.

Being tested
OATD-01, Placebo
Keywords
Pulmonary Sarcoidosis, Sarcoidosis, Chitinase
Medical subject headings
Sarcoidosis, Pulmonary, Sarcoidosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • OATD-01Drug

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

  • PlaceboDrug

    Matching placebo tablets

Study groups in NCT06205121
Study groupRoleWhat they receive
Active ArmExperimentalSubjects will receive OATD-01 as 25mg film-coated tablets for oral administration once daily for 12 weeks
Receives: Drug: OATD-01
Placebo ArmPlacebo comparatorSubjects will receive placebo as film-coated tablets for oral administration once daily for 12 weeks
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

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

Secondary outcomes (17)
  1. Total granulomatous inflammation evaluation

    Evaluation by [18F]FDG PET/CT imaging, quantified as the percent change of maximum, mean, peak SUV (SUVmax, SUVmean, SUVpeak), and volume of the lesions in pulmonary parenchyma, mediastinal/hilar nodes, and extrathoracic locations.

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

  2. Pulmonary function Forced Vital Capacity (FVC)

    Absolute change in FVC (% predicted).

    Measured: At Screening visit and over 12 weeks of treatment - at baseline (randomization) visit, at week 4 of treatment, week 8 and week 12 (EOT).

  3. Pulmonary function Forced Expiratory Volume in the first second (FEV1)

    Absolute change in FEV1 (% predicted).

    Measured: At Screening visit and over 12 weeks of treatment - at baseline (randomization) visit, at week 4 of treatment, week 8 and week 12 (EOT).

  4. Quality of life assessment

    Change in the quality of life measured by the Kings Sarcoidosis Questionnaire General and Lung (KSQ GENERAL and LUNG) scores. Range of each module 0-100, 100= best health status.

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

  5. Fatigue Assessment Scale (FAS)

    Change in FAS total score. Range: 10 (no fatigue) - 50 (extreme fatigue).

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

  6. Adverse events

    Occurrence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events, Adverse Events of Special Interest (AESIs), TEAEs leading to discontinuation and TEAEs leading to death.

    Measured: Recorded from the time of signature of informed consent and until 30 days after the last dose of OATD-01/Placebo.

  7. Laboratory tests

    Occurrence of clinically significant laboratory (hematology and biochemistry) parameter abnormalities (number of subjects with clinically significant abnormal laboratory values).

    Measured: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8 and week 12 (EOT).

  8. Vital signs - Systolic Blood Pressure

    Mean change in Systolic Blood Pressure.

    Measured: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any Follow-up (UP) visits

  9. Vital signs - Diastolic Blood Pressure

    Mean change in Diastolic Blood Pressure.

    Measured: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits.

  10. Vital signs

    Mean change in Heart Rate.

    Measured: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits.

  11. Vital signs - Respiratory Rate

    Mean change in Respiratory Rate

    Measured: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits

  12. Electrocardiography

    Occurrence of any clinically significant abnormalities (number of patients with any clinically significant Heart rhythm, PR Interval, QTcF interval or QRS width interval abnormalities) in 12-lead electrocardiography (ECG) or 24-h ECG.

    Measured: 12-lead-ECG measured at screening and over 12 weeks of treatment or study participation - at randomization visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits. 2-week 24-h-ECG recordings at weeks 0, 4 and 8 post randomization.

  13. Electrocardiography - specific parameters

    Occurrence of: a) QTcF >450 ms (male), >470 ms (female) and >500 ms (any sex) b) Change from baseline in QTcF >30 ms and >60 ms c) Heart rhythm abnormalities including supraventricular arrhythmias, ventricular arrhythmias, and non-sustained ventricular tachycardias.

    Measured: Measured at screening and over 12 weeks of treatment or study participation - at baseline (randomization) visit, at week 2 of treatment, week 4, week 8, week 12 (EOT) and any FUP visits.

  14. Male fertility

    Occurrence of a clinically significant abnormality of sperm parameters and/ or free testosterone concentration in males (optional testing)

    Measured: Measured at baseline (randomization) visit and at week 8 or week 12 (EOT) of treatment.

  15. Neurological status

    Occurrence of TEAEs of sensation abnormalities or ataxia.

    Measured: TEAEs detected during 12 weeks of treatment or study participation - from baseline (randomization) visit and up to FUP visit 7-10 days after last dose

  16. Thyroid and renal function

    Proportion of subjects with clinically significant thyroid parameters (Thyroid Stimulating Hormone [TSH], Free Triiodothyronine [FT3], and Free Thyroxine [FT4] and renal function parameters [blood urea nitrogen (BUN)/total urea, creatinine, creatinine clearance (CrCL)].

    Measured: Clinically significant abnormalities detected during 12 weeks of treatment - at baseline (randomization) visit, at week 4 of treatment, week 8 and week 12 (EOT).

  17. Pharmacokinetics assessment

    Mean plasma OATD-01 concentrations.

    Measured: Measured during 2 weeks of treatment - at baseline (randomization) visit, at week 4 of treatment, week 8 and week 12 (EOT).

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jan 2024
  2. Study start21 Mar 2024
  3. Primary completionAug 2027 (estimated)
  4. Study completionSep 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
96 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06205121
Sponsor's protocol ID
OATD-01-C-03
Other IDs
2023-506642-23, 2023
Status last verified
Oct 2026
First submitted
13 Dec 2023
Last updated
8 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing OATD-01 in people with Pulmonary Sarcoidosis, run by Molecure S.A. at 28 sites in 8 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 sites in United States, United Kingdom, France, Greece, Germany and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Molecure SA (+48 22 552 67 24, contact@molecure.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Response to treatment (time frame: After 12 weeks of treatment, i.e. from baseline (randomization) visit to End-of-Treatment (EOT) visit.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2026.

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