OATD-01 for Sarcoidosis

Recruiting at 19 trial locations

Overseen By Theodoros Charitos, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Molecure S.A.

Must not be taking: QT prolongation drugs, thiazides

Disqualifiers: Cardiac sarcoidosis, Neuro-sarcoidosis, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 7

Duration: 12 weeks

98 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications before joining. You must not be on corticosteroids within 3 months or immunosuppressants/anti-TNF agents within 4 months before enrolling. Also, you cannot take medications that affect heart rhythm, certain diuretics, or specific enzyme inhibitors.

What makes the drug OATD-01 unique for treating sarcoidosis?

OATD-01 is unique because it is a novel treatment option for sarcoidosis, which currently relies heavily on glucocorticosteroids like prednisolone. Unlike traditional steroid treatments, OATD-01 may offer a different mechanism of action, potentially providing an alternative for patients who need a steroid-sparing option. ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Research Team

Piotr Iwanowski, MD

Principal Investigator

CMO

Eligibility Criteria

This trial is for men and women with active symptomatic pulmonary sarcoidosis, which means they have lung inflammation that can be seen on a special type of scan. It's open to those who haven't been treated before or have had previous treatments.

Inclusion Criteria

Parenchymal pulmonary involvement on [18F]FDG PET/CT

I have been diagnosed with active pulmonary sarcoidosis.

I have not received treatment or have been treated before.

Exclusion Criteria

I have or had Löfgren syndrome.

I need to start treatment for my lung condition right away.

I have a lung condition or systemic disease other than sarcoidosis.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive either OATD-01 or placebo for 12 weeks

12 weeks

5 visits (in-person) at baseline, week 2, week 4, week 8, and week 12

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person) 7-10 days after last dose

Treatment Details

Interventions

• OATD-01

Trial Overview The study tests OATD-01 against a placebo to see if it's effective and safe for treating pulmonary sarcoidosis. Participants won't know which one they're getting, and the effects on the body will be closely monitored.

Participant Groups

Group I: Active Arm Experimental Treatment 1 Intervention

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

Group II: Placebo Arm Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Molecure S.A.

Lead Sponsor

Trials

2

Recruited

140+

Findings from Research

In a study of 86 patients with pulmonary sarcoidosis, high-dose prednisolone (40 mg/day) did not show any significant improvement in relapse rates or treatment failure compared to the standard low-dose (20 mg/day) over an 18-month period.

Both dosage groups experienced similar changes in lung function, health-related quality of life, and adverse effects, indicating that increasing the dose of prednisolone does not provide additional benefits for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

High-dose (40 mg) versus low-dose (20 mg) prednisolone for treating sarcoidosis: a randomised trial (SARCORT trial). Dhooria, S., Sehgal, IS., Agarwal, R., et al.[2023]

Glucocorticosteroids, particularly prednisolone at doses of 30-60 mg/day, are the primary treatment for sarcoidosis, with various administration routes available, including oral and inhaled options for pulmonary cases.

Other steroid-sparing medications like methotrexate and azathioprine can be effective alternatives, especially for patients requiring long-term treatment, which may last from 6 months to a lifetime depending on disease activity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of sarcoidosis. Selroos, O.[2013]

Extrapulmonary sarcoidosis affects 30-50% of patients with sarcoidosis and can involve any organ, with symptoms and severity varying widely among individuals, making accurate diagnosis and treatment challenging.

There is a need for a personalized and holistic treatment approach that considers both extrapulmonary and pulmonary manifestations, as well as patient history and preferences, due to the lack of randomized trials specifically addressing extrapulmonary sarcoidosis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

How to Tackle the Diagnosis and Treatment in the Diverse Scenarios of Extrapulmonary Sarcoidosis. Valeyre, D., Jeny, F., Rotenberg, C., et al.[2022]

In a study of 156 Dutch sarcoidosis patients and 200 healthy controls, the TLR-4 polymorphism Asp299Gly was not associated with sarcoidosis, indicating that this genetic variant may not play a role in the disease for this population.

The research emphasizes the need to validate genetic associations in different populations, as the allelic distribution of the Asp299Gly polymorphism varied significantly between Dutch and German control groups, suggesting that genetic factors may differ across ethnicities.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Toll-like receptor (TLR) 4 polymorphism Asp299Gly is not associated with disease course in Dutch sarcoidosis patients. Veltkamp, M., Grutters, JC., van Moorsel, CH., et al.[2018]

In a study of 25 sarcoidosis patients, 15 showed a significant immune response to the Mycobacterium tuberculosis protein Antigen 85A, indicating a potential link between mycobacterial antigens and sarcoidosis.

The research identified four specific peptides from Antigen 85A that triggered Th-1 immune responses in sarcoidosis subjects, suggesting that these mycobacterial proteins may play a role in the disease's development.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mycobacterium tuberculosis Antigen 85A induces Th-1 immune responses in systemic sarcoidosis. Hajizadeh, R., Sato, H., Carlisle, J., et al.[2018]

References

1. England pubmed.ncbi.nlm.nih.gov

High-dose (40 mg) versus low-dose (20 mg) prednisolone for treating sarcoidosis: a randomised trial (SARCORT trial). [2023]

2. Italy pubmed.ncbi.nlm.nih.gov

Treatment of sarcoidosis. [2013]

3. United States pubmed.ncbi.nlm.nih.gov

How to Tackle the Diagnosis and Treatment in the Diverse Scenarios of Extrapulmonary Sarcoidosis. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Toll-like receptor (TLR) 4 polymorphism Asp299Gly is not associated with disease course in Dutch sarcoidosis patients. [2018]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Mycobacterium tuberculosis Antigen 85A induces Th-1 immune responses in systemic sarcoidosis. [2018]