OATD-01 for Sarcoidosis
Recruiting in Palo Alto (17 mi)
+19 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: Molecure S.A.
Must not be taking: QT prolongation drugs, thiazides
Disqualifiers: Cardiac sarcoidosis, Neuro-sarcoidosis, others
Prior Safety Data
Trial Summary
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.
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.12345
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.
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Trial 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 (Other)
Trial OverviewThe 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
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Active ArmExperimental Treatment1 Intervention
Subjects will receive OATD-01 as 25mg film-coated tablets for oral administration once daily for 12 weeks
Group II: Placebo ArmPlacebo Group1 Intervention
Subjects will receive placebo as film-coated tablets for oral administration once daily for 12 weeks
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Molecure Investigative SiteBirmingham, AL
Molecure Investigative SiteKansas City, KS
Molecure Investigative SiteBaltimore, MD
Molecure Investigative SiteCharleston, SC
More Trial Locations
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Who Is Running the Clinical Trial?
Molecure S.A.Lead Sponsor
References
High-dose (40 mg) versus low-dose (20 mg) prednisolone for treating sarcoidosis: a randomised trial (SARCORT trial). [2023]Label="BACKGROUND">Current guidelines recommend 20-40 mg·day-1 of oral prednisolone for treating pulmonary sarcoidosis. Whether the higher dose (40 mg·day-1) can improve outcomes remains unknown.
Treatment of sarcoidosis. [2013]Glucocorticosteroids represent the "drugs of choice" for treatment of sarcoidosis. Steroids can be given by all routes of administration. Daily therapy with oral steroids is most widely applied. Initial therapy should consist of prednisolone 30-60 mg/day or its equivalent. Alternate day therapy can be used during the maintenance phase. Inhaled steroids can also be tried during the maintenance phase for treatment of pulmonary sarcoidosis. Other drugs, which may be effective in sarcoidosis, and have a steroid-sparing capacity, are methotrexate, azathioprine, chlorambucil and cyclophosphamide. Chloroquine can be used for chronic skin lesions and potassium para-aminobenzoate may soften fibrotic lesions and keloids. Duration of treatment varies with the clinical situation; from between 6 and 18 months to lifetime. In principle, continuing signs of disease activity and functional impairment require continuing treatment. Determination of on-going activity may be a difficult task. Symptomatic patients with stage II-III pulmonary sarcoidosis, and many extrapulmonary manifestations of the disease, must be adequately treated. Symptom-free patients with deteriorating lung function and/or biochemical signs of disease activity also require treatment. Steroids are not indicated for pulmonary stage I disease (hilar lymphadenopathy) with or without erythema nodosum unless there are troublesome persistent chest symptoms (cough, pain, pressure symptoms) or arthralgia, oedema and pain of the legs.
How to Tackle the Diagnosis and Treatment in the Diverse Scenarios of Extrapulmonary Sarcoidosis. [2022]Extrapulmonary sarcoidosis occurs in 30-50% of cases of sarcoidosis, most often in association with pulmonary involvement, and virtually any organ can be involved. Its incidence depends according to the organs considered, clinical phenotype, and history of sarcoidosis, but also on epidemiological factors like age, sex, geographic ancestry, and socio-professional factors. The presentation, symptomatology, organ dysfunction, severity, and lethal risk vary from and to patient even at the level of the same organ. The presentation may be specific or not, and its occurrence is at variable times in the history of sarcoidosis from initial to delayed. There are schematically two types of presentation, one when pulmonary sarcoidosis is first discovered, the problem is then to detect extrapulmonary localizations and to assess their link with sarcoidosis, while the other presentation is when extrapulmonary manifestations are indicative of the disease with the need to promptly make the diagnosis of sarcoidosis. To improve diagnosis accuracy, extrapulmonary manifestations need to be known and a medical strategy is warranted to avoid both under- and over-diagnosis. An accurate estimation of impairment and risk linked to extrapulmonary sarcoidosis is essential to offer the best treatment. Most frequent extrapulmonary localizations are skin lesions, arthritis, uveitis, peripheral lymphadenopathy, and hepatic involvement. Potentially severe involvement may stem from the heart, nervous system, kidney, eye and larynx. There is a lack of randomized trials to support recommendations which are often derived from what is known for lung sarcoidosis and from the natural history of the disease at the level of the respective organ. The treatment needs to be holistic and personalized, taking into account not only extrapulmonary localizations but also lung involvement, parasarcoidosis syndrome if any, symptoms, quality of life, medical history, drugs contra-indications, and potential adverse events and patient preferences. The treatment is based on the use of anti-sarcoidosis drugs, on treatments related to organ dysfunction and supportive treatments. Multidisciplinary discussions and referral to sarcoidosis centers of excellence may be helpful for difficult diagnosis and treatment decisions.
Toll-like receptor (TLR) 4 polymorphism Asp299Gly is not associated with disease course in Dutch sarcoidosis patients. [2018]The aetiology of sarcoidosis, a systemic disorder characterized by the formation of non-caseating granulomas in variable organs, remains enigmatic. Clarification is hampered by heterogeneity in disease phenotypes and course, due partly to the influence of a variety of genetic and environmental factors. Multiple studies have pointed towards bacteria as possible causative agents. Toll-like receptors (TLR) are innate immunity receptors important in the immune response against pathogens. TLR-4, together with CD14 and MD-2, is an essential receptor for the recognition of lipopolysaccharide (LPS), unique to the cell wall of Gram-negative bacteria. Recently, an association between TLR-4 polymorphism Asp299Gly, leading to a change in the extracellular domain of the receptor and possible hyporesponsiveness to LPS, and a chronic course of sarcoidosis was found in German patients. In the present study this polymorphism was genotyped in 156 Dutch sarcoidosis patients and 200 healthy Dutch controls using dual-labelled fluorescent oligonucleotides. No differences were found in allelic distributions between patients and controls (P = 0.79) or within the different clinical entities of the sarcoidosis group (P = 0.44). Importantly, there were no differences between the Dutch and German sarcoidosis patients (P = 0.62). However, the allelic distribution of the Asp299Gly polymorphism differed significantly between both control groups (P = 0.04). This study highlights the importance of testing a reported gene association in a distinct population when performing genetic association studies.
Mycobacterium tuberculosis Antigen 85A induces Th-1 immune responses in systemic sarcoidosis. [2018]Sarcoidosis is a granulomatous disease of unknown etiology, characterized by a Th-1 immunophenotype. Although humoral immune responses by sarcoidosis subjects to mycobacterial proteins have been detected, mycobacterial antigens capable of inducing cellular immune responses in sarcoidosis subjects have not been reported. We used the enzyme-linked immunospot assay to assess for recognition of the Mycobacterium tuberculosis mycolyl transferase, Antigen 85A, by peripheral blood mononuclear cells from 25 sarcoidosis subjects, 22 PPD- (purified protein derivative) healthy volunteers, and 16 PPD+ healthy subjects. Reactivity to Ag85A whole protein was observed in 15 of 25 sarcoidosis subjects compared to 2 of 22 PPD- subjects (p=0.0006, Fisher's exact test) and to 14 of 16 PPD+ subjects (p=0.084, Fisher's exact test). Monoclonal antibody against HLA-DR inhibited recognition. In addition to immune recognition of Ag85A whole protein, peptide-mapping studies identified four immunogenic Ag85A peptides, which induced Th-1 immune responses in individual sarcoidosis subjects, suggesting that multiple epitopes from a mycobacterial protein may have a role in sarcoidosis immunopathogenesis.