An Efficacy Study of XTMAB-16 in Patients With Pulmonary Sarcoidosis With or Without Extrapulmonary Manifestations
Přehled studie
Postavení
Postavení
Podmínky
Podmínky
Intervence / Léčba
Intervence / Léčba
Detailní popis
Typ studie
Typ studie
Zápis (Odhadovaný)
Zápis
Fáze
Fáze
- Fáze 2
Kritéria účasti
Kritéria způsobilosti
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dospělý
- Starší dospělý
Přijímá zdravé dobrovolníky
Popis
Inclusion Criteria:
- Participant between 18 and 80 years (inclusive) of age.
- Weighs between 45 and 160 kg (99 to 353 lbs) at Screening.
- Diagnosis of pulmonary sarcoidosis (at least 6 months before Screening) using the 2020 American Thoracic Society (ATS) Clinical Practice Guideline (Crouser et al, 2020), the European Respiratory Society (ERS) or the World Association of Sarcoidosis and Other Granulomatous Disorders (WASOG) criteria including a compatible clinical and radiologic presentation with other causes of granulomatous disease ruled out (cutaneous and ocular involvement permitted).
- mMRC Dyspnea Scale of ≥1.
- Baseline pre-BD ppFVC of ≥ 50% during screening window, and confirmed at the Baseline visit prior to enrollment and dosing.
- Receiving corticosteroid treatment 5 to 25 mg/day of oral prednisone, or equivalent), during the screening period and, at the determination of the Investigator, is capable of undergoing the protocol specific corticosteroid taper regimen.
- Receiving treatment with methotrexate, azathioprine, mycophenolate, leflunomide, chloroquine, or hydroxychloroquine for at least 3 months before Screening that has been at a stable dose for 4 weeks before Screening. All efforts should be made to maintain stable background therapy at the Screening dose through the intervention period at the Investigator's discretion.
Radiographic evidence of disease in the lung parenchyma (at least Scadding stage II disease) without the presence of significant fibrosis.
Note: Significant fibrotic disease is defined as fibrosis > 20% as determined by central read.
- Able to provide written informed consent.
- In the opinion of the Investigator, the participant is capable of understanding and complying with protocol requirements.
Exclusion Criteria:
- Pregnant or breastfeeding women or women who are planning to become pregnant during the study.
- Known potentially significant fibrotic disease and/or active inflammation contained solely in the hilar region as shown by HRCT, confirmed by a central reader. Participants with current active inflammation in the hilar region with concurrent inflammation outside the hilar region may be included. A historical HRCT performed within 6 months of screening may be submitted for diagnostic confirmation by central review. If a participant's last HRCT was from >6 months of screening, an HRCT should be performed during screening for diagnostic confirmation by central review. Note: Significant fibrotic disease is defined as >20% fibrosis on HRCT.
- Clinically significant extra-pulmonary sarcoidosis requiring systemic therapy as determined by the Investigator.
- Any therapy with an anti-TNFα monoclonal antibody (e.g., infliximab, adalimumab, golimumab and their biosimilars) within 6 months.
- Baseline pre-BD ppFVC of >85%.
- Prior treatment with rituximab or repository corticotropin injection within the previous 12 months.
- Clinically significant CNS sarcoidosis requiring therapy, except history of isolated seventh cranial nerve palsy or evidence of demyelinating neurologic disease.
- Advanced congestive heart failure (New York Heart Association 3 or 4).
- Current disease presentation consistent with Lofgren's syndrome (i.e., presence of the triad of erythema nodosum, bilateral hilar lymphadenopathy on chest X-ray, and joint pain).
- Clinically significant pulmonary hypertension requiring treatment. Note: Clinically significant pulmonary hypertension requiring treatment would be defined as treatment with, i.e., prostacyclins, phosphodiesterase 5 inhibitors, and endothelin receptor antagonists.
- Known hypersensitivity to any component of the formulation of XTMAB-16.
- Evidence of active or latent tuberculosis (TB) by interferon-gamma release assay (IGRA) or invasive fungal infections at Screening.
- Known positive history of malignancy other than non-melanomatous skin cancer in the last 2 years, including in-situ carcinoma of the uterine cervix completely cured by radical surgery.
- Positive test result for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, coronavirus disease (COVID-19), TB, or a known history of human immunodeficiency virus (HIV) infection at Screening.
- Women of childbearing potential who are sexually active with a non-sterilized male partner and are not willing to adhere to highly effective birth control measures from the time of signing the informed consent, throughout the duration of the study, and for 90 days after 5 half-lives have elapsed since the last dose of study drug. See Appendix 4 for details on contraception requirements during the study.
- Male participants who are non-sterilized and sexually active with a female partner of childbearing potential and are not willing to use highly effective contraception from the time of signing the informed consent throughout the duration of the study, and for 90 days after 5 half-lives have elapsed since last dose of study drug. See Appendix 4 for details on contraception requirements during the study.
- Clinically significant hepatic or renal disease, including uncontrolled diabetes at the discretion of the Investigator.
- Any severe prior reaction to any type of biologics or human blood product such as albumin, immunoglobulin G (IgG), etc.
- Concurrent emphysema.
- Known hypercalcemia due to non-sarcoidosis conditions such as untreated hyperparathyroidism, at the discretion of the Investigator.
- Abnormal electrocardiogram (ECG): ventricular arrhythmias (non-sustained ventricular tachycardia (VT), multifocal or frequent premature ventricular contractions, bundle branch block, axis deviation, or abnormal Q waves). In the case of a corrected QT interval by Fredericia interval >450 ms (men) or >480 ms (women; participants with bundle branch block) or PR interval outside the range of 120 to 220 ms, the assessment may be repeated once for eligibility determination at Screening or Baseline.
- Donation or loss of 450 mL or more of his or her blood volume (including plasmapheresis) or transfusion of any blood product within 90 days prior to dosing.
- Known uncontrolled hypertension. Note: Uncontrolled hypertension is noted as blood pressure (BP) ≥160/100 mmHg despite antihypertensive therapy within 3 months of randomization.
- Clinical signs and symptoms consistent with COVID-19, e.g., fever, dry cough, dyspnea, sore throat, fatigue, new smell or taste disorder or confirmed infection by appropriate laboratory test within the last 4 weeks prior to Screening.
- In the opinion of the Investigator, inability to tolerate corticosteroid taper.
- Concurrent systemic steroid use for non-sarcoidosis conditions.
- Concurrent known auto-immune disease requiring treatment.
- Participation in another clinical trial of an investigational agent within 3 months (small molecule) / 6 months (biologics) or 5 half-lives (if known) of the agent, whichever is longer.
- Any condition that required hospitalization within the 3 months prior to Day 1 or is likely to require so during the study.
- Clinically significant abnormalities in the Screening physical exam, medical history, vital signs, ECG, or clinical laboratory tests that are not known to be due to concurrent sarcoidosis, and in the opinion of the Investigator and Medical Monitor should preclude the participant's participation in the clinical study.
Studijní plán
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: Randomizované
- Intervenční model: Paralelní přiřazení
- Maskování: Dvojnásobek
Počet zbraní
Zbraně a zásahy
Skupina účastníků / ArmSkupina účastníků / Arm |
Intervence / LéčbaIntervence / Léčba |
|---|---|
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Experimentální: XTMAB-16 4 mg/kg Q2W for 3 doses, then Q4W IV up to 52 weeks
|
Infusion
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Komparátor placeba: Placebo 4 mg/kg Q2W for 3 doses, then Q4W IV up to 52 weeks
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Infuze
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Co je měření studie?
Primární výstupní opatření
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Absolute change from baseline in forced vital capacity (FVC) (mL) at Week 24
Časové okno: Baseline to Week 24
|
To establish efficacy of XTMAB-16 as measured by FVC (mL) in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations(mL) in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 24
|
Sekundární výstupní opatření
Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Absolute change from baseline in forced vital capacity (FVC) (mL) at Week 52
Časové okno: Baseline to Week 52
|
To further establish efficacy of XTMAB-16 as measured by disease control in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
|
Quality of Life King's Sarcoidosis Questionnaire-Lung (KSQ-L) mean change
Časové okno: Baseline to Week 24 and 52
|
To further establish efficacy of XTMAB-16 as measured by quality of life in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 24 and 52
|
|
Proportion of participants experiencing no independently adjudicated worsening (exacerbation) events through Week 52
Časové okno: Baseline to Week 52
|
To further establish efficacy of XTMAB-16 as measured by disease control in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
|
King's Sarcoidosis Questionnaire - General (KSQ-G)
Časové okno: Baseline to Week 24 and 52
|
To further establish efficacy of XTMAB-16 as measured by quality of life in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 24 and 52
|
|
Modified Medical Research Council (mMRC) Dyspnea Scale
Časové okno: Baseline to Week 24 and 52
|
To further establish efficacy of XTMAB-16 as measured by quality of life in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 24 and 52
|
|
Fatigue Assessment Scale (FAS)
Časové okno: Baseline to Week 24 and 52
|
To further establish efficacy of XTMAB-16 as measured by quality of life in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 24 and 52
|
|
Time to first adjudicated worsening (exacerbation) event through Week 52
Časové okno: Baseline to Week 52
|
To further establish efficacy of XTMAB-16 as measured by disease control in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
|
Annualized worsening-event rate
Časové okno: Baseline to Week 52
|
To further establish efficacy of XTMAB-16 as measured by disease control in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
|
Mean number of worsening events per participant
Časové okno: Baseline to Week 52
|
To further establish efficacy of XTMAB-16 as measured by disease control in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
|
Imaging Endpoints High-resolution computed tomography (HRCT; 'worse,' 'no change,' or 'better')
Časové okno: Baseline to Week 24 and 52
|
To further establish efficacy of XTMAB-16 as measured by imaging in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 24 and 52
|
|
Maximum observed concentration (Cmax)
Časové okno: Baseline to Week 52
|
To characterize the Pharmacokinetics (PK) of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
|
Clearence
Časové okno: Baseline to Week 52
|
To characterize the Pharmacokinetics (PK) of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
|
Volume Parameters
Časové okno: Baseline to Week 52
|
To characterize the Pharmacokinetics (PK) of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
|
Soluble tumor necrosis factor alpha (sTNFα)
Časové okno: Baseline to Week 12, 24 and 52
|
To characterize the Pharmacodynamics (PD) of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 12, 24 and 52
|
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Interleukin-1beta (IL-1β)
Časové okno: Baseline to Week 12, 24 and 52
|
To characterize the Pharmacodynamics (PD) of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 12, 24 and 52
|
|
Soluble IL-2 receptor (sIL-2R)
Časové okno: Baseline to Week 12, 24 and 52
|
To characterize the Pharmacodynamics (PD) of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 12, 24 and 52
|
|
Number and percentage of participants who test positive for anti-drug antibody (ADA) at Baseline and transient and persistent positive status at Weeks 12, 24, and 52.
Časové okno: Baseline to Week 12, 24 and 52
|
To characterize the immunogenicity of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 12, 24 and 52
|
|
Number and percentage of participants who test positive for neutralizing antibody (nAb) at Baseline and transient and persistent positive status at Weeks 12, 24, and 52.
Časové okno: Baseline to Week 12, 24 and 52
|
To characterize the immunogenicity of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 12, 24 and 52
|
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Adverse event (AE) assessments
Časové okno: Baseline to Week 52
|
To evaluate continued safety and tolerability of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations
|
Baseline to Week 52
|
Spolupracovníci a vyšetřovatelé
Sponzor
Sponzor
Termíny studijních záznamů
Hlavní termíny studia
Začátek studia (Odhadovaný)
Začátek studia
Primární dokončení (Odhadovaný)
Primární dokončení
Dokončení studie (Odhadovaný)
Dokončení studie
Termíny zápisu do studia
První předloženo
První předloženo
První předloženo, které splnilo kritéria kontroly kvality
První předloženo, které splnilo kritéria kontroly kvality
První zveřejněno (Aktuální)
První zveřejněno
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
Poslední zveřejněná aktualizace
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Naposledy ověřeno
Naposledy ověřeno
Více informací
Termíny související s touto studií
Další relevantní podmínky MeSH
Další identifikační čísla studie
Další identifikační čísla studie
- XTMAB-16-201 Part B
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Studuje produkt zařízení regulovaný americkým úřadem FDA
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