EUCTR2020-001263-85-DE进行中（未招募）1 期
A randomized, double-blind, placebo-controlled clinical trial of once-daily inhaled molgramostim nebulizer solution in adult subjects with autoimmune pulmonary alveolar proteinosis (aPAP). - IMPALA-2
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Savara ApS
- 入组人数
- 160
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Subject must be =18 years of age, at the time of signing the informed consent. Specific for Japan; Subject must be =20 years of age, at the time of signing the informed consent.
- •2. A serum anti-GM-CSF autoantibody test result confirming autoimmune PAP.
- •3. History of PAP, based on examination of a lung biopsy, bronchoalveolar lavage (BAL) cytology, or a high-resolution computed tomogram (HRCT) of the chest.
- •4. DLCO 70% predicted or lower at the first Screening and Baseline visits.
- •5. Change in % predicted DLCO of <15% points during the screening period.
- •6. Willing and able to come off supplemental oxygen use prior to and
- •during the treadmill exercise test, the DLCO assessment, and the arterial
- •blood gas sampling.
- •7. Resting SpO2 >85% during 15 minutes without use of supplemental
- •oxygen at the Screening visits.
- •8. Male or female
- •9. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those
- •participating in clinical studies.
- •a. Male subjects: Males agreeing to use condoms during and until 30
- •days after last dose of trial treatment, or males having a female partner
- •who is using adequate contraception as described below.
- •b. Female subjects: Females who have been post-menopausal for >1
- •year, or females of childbearing potential after a confirmed menstrual
- •period using a highly efficient method of contraception (i.e. a method
- •with <1% failure rate such as combined hormonal contraception,
- •progesterone-only hormonal contraception, intrauterine device,
- •intrauterine hormone-releasing system, bilateral tubal occlusion,
- •vasectomized partner, sexual abstinence*), during and until 30 days
- •after last dose of trial treatment. Females of childbearing potential must
- •have a negative serum pregnancy test at Screening (Visit 1) and a
- •negative urine pregnancy test at baseline (Visit 3) and must not be
- •*Sexual abstinence is considered a highly effective method only if
- •defined as refraining from heterosexual intercourse during the entire
- •period of risk associated with the trial treatments. The reliability of
- •sexual abstinence needs to be evaluated in relation to the duration of the
- •clinical trial and the preferred and usual lifestyle of the subject.
- •10. Capable of giving signed informed consent as described in Appendix
- •1 which includes compliance with the requirements and restrictions
- •listed in the informed consent form (ICF) and in this protocol.
- •11. Willing and able to comply with scheduled visits, treatment plan,
- •laboratory tests, and other trial procedures specified in the protocol as
- •judged by the Investigator.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 140
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 20
排除标准
- •1. Diagnosis of hereditary or secondary PAP, or a metabolic disorder of surfactant production.
- •2. WLL performed within 3 months prior to baseline.
- •3. Requirement for WLL at screening or baseline.
- •4. GM-CSF treatment within 6 months prior to baseline.
- •5. Treatment with rituximab within 6 months prior to baseline.
- •6. Treatment with plasmapheresis within 6 weeks months prior to baseline.
- •7. Treatment with any investigational medicinal product within 5 half-lives or 3 months (whichever is longer) prior to baseline.
- •8. Previously randomized in this trial.
- •9. History of allergic reactions to GM-CSF or any of the excipients in the nebulizer solution.
- •10. Inflammatory or autoimmune disease of a severity that necessitates significant (e.g. more than 10 mg/day systemic prednisolone) immunosuppression.
- •11. Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product.
- •12. History of, or present, myeloproliferative disease or leukemia.
- •13. Apparent pre-existing concurrent pulmonary fibrosis, or diagnosis of
- •interstitial lung disease other than aPAP.
- •14.Acute or unstable cardiac or pulmonary disease that may be
- •aggravated by exercise or confound assessment of the primary endpoint:
- •including presence of pulmonary edema, or diagnosis of chronic
- •obstructive pulmonary disease (COPD), pulmonary vasculitis, or
- •pulmonary hypertension.
- •15. Known active infection (viral, bacterial, fungal, or mycobacterial)
- •that may affect the efficacy evaluation in the trial.
- •16. Physical disability or other condition that precludes safe and
- •adequate exercise testing.
- •17. Any other serious medical condition which in the opinion of the
- •Investigator would make the subject unsuitable for the trial
- •18. Pregnant, planning to become pregnant during the trial, or
- •breastfeeding woman.
研究者
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