OATD-01 for Sarcoidosis

Recruiting at 23 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

96 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called OATD-01 for individuals with active pulmonary sarcoidosis, a condition that causes lung inflammation and can lead to breathing difficulties. The trial aims to assess the effectiveness and safety of OATD-01 compared to a placebo (a non-active substance). Participants will receive either the actual treatment or a placebo once daily for 12 weeks. Suitable candidates for the trial have been diagnosed with pulmonary sarcoidosis and have lung involvement confirmed by a PET/CT scan. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in sarcoidosis treatment.

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.

Is there any evidence suggesting that OATD-01 is likely to be safe for humans?

Research shows that OATD-01 has been tested for safety in treating conditions like pulmonary sarcoidosis. In earlier studies, adults received this treatment in controlled settings. The results indicated that OATD-01 was generally well-tolerated, meaning it did not cause any major side effects.

Moreover, OATD-01 demonstrated positive results in animal studies, further supporting its safety. While these findings are encouraging, ongoing research aims to fully understand its safety in humans. Prospective trial participants can feel reassured that earlier studies suggest the treatment is relatively safe. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for sarcoidosis?

OATD-01 is unique because it targets the enzyme chitotriosidase, which is thought to play a role in the inflammation and granuloma formation seen in sarcoidosis. Unlike current treatments like corticosteroids and immunosuppressants, which broadly dampen the immune system, OATD-01 offers a more targeted approach. Researchers are excited about this potential for precision, hoping it might reduce side effects and improve outcomes by focusing directly on a key part of the disease process.

What evidence suggests that OATD-01 might be an effective treatment for sarcoidosis?

Research has shown that OATD-01, a new medication, might help treat sarcoidosis, a lung disease. In studies, OATD-01 reduced lung tissue scarring, similar to the effects of pirfenidone, a known treatment. Tests demonstrated that OATD-01 lowered the Ashcroft score, which measures lung scarring, by about 32%. In this trial, participants in the active arm will receive OATD-01 to further evaluate its effectiveness in treating lung issues related to sarcoidosis. ^{1 2 3 4 6}

Who Is on the Research Team?

Piotr Iwanowski, MD

Principal Investigator

CMO

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

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]

Citations

1. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/451079

Efficacy and Safety Study of OATD-01 in Patients With ... This is a Phase 2, randomized, double-blind, placebo-controlled, adaptive, multicenter study to evaluate the efficacy, safety, tolerability, ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06205121

NCT06205121 | Efficacy and Safety Study of OATD-01 in ... In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical ...

3. atsjournals.org atsjournals.org/doi/pdf/10.1164/ajrccm-conference.2018.197.1_MeetingAbstracts.A4346?download=true

OATD-01, a Dual Chitinase Inhibitor, Significantly ... collected from patients with interstitial lung diseases (sarcoidosis and IPF). The therapeutic efficacy of OATD-01 in vivo was evaluated in the ...

4. synapse.patsnap.com synapse.patsnap.com/drug/93e0b92fef864b98a5b57756826ca597

OAT-889 - Drug Targets, Indications, Patents OATD-01 exhibited anti-fibrotic activity comparable to pirfenidone resulting in the reduction of the Ashcroft score by 32% and 31%, respectively. These studies ...

5. templehealth.org templehealth.org/services/lung/research-clinical-trials/efficacy-and-safety-study-of-oatd-01-in-patients-with-active-pulmonary-sarcoidosis

Efficacy and Safety Study of OATD-01 in Patients With ... A randomized, double-blind, placebo-controlled, multicenter study to assess the efficacy and safety of a 12-week administration of OATD-01.

6. delta.larvol.com delta.larvol.com/Products/?ProductId=6d1590bf-dec9-48b1-b33d-6c601331ef2d

OATD-01 / Molecure Efficacy and Safety Study of OATD-01 in Patients With Active Pulmonary Sarcoidosis ... Positive results of this clinical proof-of-concept study in sarcoidosis ...