NL-OMON55020撤回3 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
Parexel Nederland0 个研究点目标入组 3 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 发起方
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
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
- •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
- •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 the screening visits,
- •and a negative urine pregnancy test at Baseline visit (Visit 3) and must not be
- •* Post-menopausal is defined as no menses for at least 12 months without an
- •alternative medical cause. A high follicle stimulating hormone (FSH) level in
- •the postmenopausal range may be used to confirm a post-menopausal state in
- •women not using hormonal contraception or hormonal replacement therapy.
- •However, in the absence of 12 months of amenorrhea, a single FSH measurement is
- •insufficient.
- •** A female is considered of childbearing potential following menarche and
- •until becoming post-menopausal unless permanently sterile. Permanent
- •sterilization methods include hysterectomy, bilateral salpingectomy and
- •bilateral oophorectomy.
- •***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.
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
研究者
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