jRCTs031230296招募中不适用
A multicenter, open-label, uncontrolled study investigating the safety and tolerability of sirolimus in patients with primary immunodeficiency
未提供0 个研究点目标入组 20 人开始时间: 2023年9月20日最近更新:
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 主要终点
- -
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 0age 2month old over 至 No limit（—）
- 性别
- All
入选标准
- •Patients who meet the following 1) or 2) and all other criteria are included in this clinical study.
- •1.Patients enrolled in a phase II clinical trial (RALPID trial) to evaluate the efficacy and safety of sirolimus in patients with primary immunodeficiency and who received sirolimus.
- •2.In this study, patients who will start administration of sirolimus will be selected from those who satisfy all of the following criteria.
- •(1) Patients enrolled in PIDJ*
- •Patients who have been diagnosed with a definitive diagnosis by appropriate genetic testing, etc., based on the diagnosis support of the Japanese Society for Immunodeficiency and Autoinflammation and the judgment of a doctor who has sufficient experience in diagnosing and treating primary immunodeficiency disease (PID). are treated in the same manner as PIDJ registered patients. In that case, we will allow enrollment in this study prior to PIDJ enrollment, but enroll this patient in PIDJ as soon as it becomes possible. This agreement is valid only during the period when PIDJ registration is not possible.
- •(2) Patients diagnosed with any of the following diseases by flow cytometry or genetic testing
- •a.Activated PI3K-delta syndrome (APDS)
- •b.CTLA4 haploinsufficiency
- •c.LRBA deficiency
- •d.IPEX syndrome, IPEX-like
- •e.Autoimmune lymphoproliferative syndrome (ALPS), ALPS-like
- •f.Unclassifiable immunodeficiency disease (CVID) **
- •g.Other primary immunodeficiencies**
- •*Only patients judged by each research institution to have mTOR pathway enhancement in the background are eligible.
- •(3) Patients who show any of the following symptoms, who are not sufficiently effective despite steroid therapy for 3 months or longer, or whose side effects make continuation of steroid therapy undesirable.a.Patients with evaluable lymphoproliferative symptoms
- •a. Patients with evaluable lymphoproliferative symptoms
- •Hepatomegaly 3.0 cm or more palpable below the costal arch
- •Splenomegaly palpable
- •Measurable lymph node lesions or extranodal lesions CT or MRI with a major axis of 2.0 cm or more
- •b.Patients with enteritis (16 years old or older CDAI score equal or greater 220, under 16 years old wPCDAI score equal or greater 40)
- •c.Patients with 2 or more autoimmune cytopenias* that fall under any of the following
- •Hemoglobin concentration less than 10.0g/dL
- •Neutrophil count less than 1500/micro L
- •Platelet count less than 75,000/micro L
- •Autoimmune cytopenia cytopenia without decreased blood cell production, with a history of response to treatment with immunosuppressants, or with autoantibodies
- •(4) Patients aged 0 years and 2 months or older at the time of informed consent
- •(5) Patients who can take granules or tablets
- •3.Patients who can comply with the provisions of the research plan
- •4.Patients who have obtained written consent from the patient or their legal representative regarding participation in this clinical study
排除标准
- •Patients who meet any of the following criteria will not be included in this clinical study.
- •1.Patients who are judged by the principal investigator or co-investigator (hereinafter referred to as principal investigator, etc.) not to continue administration of sirolimus from the viewpoint of efficacy and safety.
- •2.Patients who meet any of the following criteria for starting sirolimus administration in this study will not be included in this study.
- •(1) Patients with secondary immunodeficiency
- •(2) Patients with idiopathic interstitial pneumonia
- •(3) Patients with uncontrolled hyperlipidemia or uncontrolled diabetes
- •(4) Patients with clinically significant signs and symptoms of infection (infections requiring iv such as antibiotics)
- •(5) Patients with a serum creatinine level of 3 times or more, the upper limit of normal at screening (institutional reference values for adults, reference values shown in Appendix 2 for children)
- •(6) HBs antigen-positive patients, HBs antibody-positive or HBc antibody-positive patients with HBV-DNA detected, or active hepatitis C patients (HCV antibody-positive patients are inactive and have normal liver function values except)
- •(7) QFT/T-SPOT positive patients
- •(8) Patients who underwent surgery (invasion into the body cavity or hand requiring 3 or more stitches, including biopsy) within 2 weeks before registration
- •(9) Patients scheduled for surgery during study drug administration period
- •(10) Patients who underwent hematopoietic stem cell transplantation
- •(11) Patients who received blood transfusion or high-dose immunoglobulin therapy within 12 weeks prior to study entry
- •(12) Patients who received G-CSF formulation within 1 week before enrollment in this study (within 4 weeks for long-acting G-CSF formulation)
- •(13) Patients who have taken an angiotensin-converting enzyme inhibitor within 4 weeks prior to enrollment in this study
- •(14) Patients who have taken any other investigational drug within 4 weeks prior to enrollment in this study (excluding patients enrolled in the RALPID study)
- •(15) Patients who have been treated with the following drugs in the past
- •a.History of treatment with mTOR inhibitors (everolimus, temsirolimus) or PI3K delta inhibitors outside of the RALPID trial
- •b.reatment history of rituximab within 24 weeks before enrollment
- •c.History of treatment with abatacept or other biological immunosuppressants with monoclonal antibodies (etanercept, infliximab, tocilizumab, certolizumab, adalimumab, golimumab, canakinumab, etc.) within 8 weeks before registration
- •d.Other immunosuppressants (cyclosporine, tacrolimus, mycophenolate mofetil, azathioprine, mercaptopurine, methotrexate, JAK inhibitors <tofacitinib, baricitinib, etc.>, etc. within 2 weeks prior to enrollment. However, doses of 1 mg/kg or less history of treatment with systemic steroids and topical steroids (excluding topical steroids)
- •(16) Patients who received a live vaccine within 4 weeks prior to enrollment in this study
- •(17) Patients with NYHA class II-IV congestive heart failure
- •(18) Patients with a history of myocardial infarction within 6 months prior to enrollment
- •(19) Patients with symptomatic arrhythmias requiring treatment
- •3.If there is an active malignant tumor at the time of screening (However, depending on the disease, it is difficult to judge malignancy, so it will be judged comprehensively)
- •4.Patients who are of childbearing potential or have a partner of childbearing potential who cannot consent to adequate contraception from the time consent is obtained until 12 weeks after the last dose of study drug.
- •5.Patients who are pregnant, breastfeeding, or may be pregnant
- •6.Other patients who are deemed inappropriate for participation in this study by the principal investigator, etc
结局指标
主要结局
-
Frequency and rate of adverse events (or diseases, etc.)
次要结局
- Frequency and rate of improvement in main symptoms(12 weeks*, 52 weeks, and 2 years (104 weeks) of administration)
- Improvement frequency and rate of each symptom(12 weeks*, 52 weeks, and 2 years (104 weeks) of administration)
研究者
相似试验
已完成
1 期
A study to investigate the safety and tolerability of dostarlimab in adult participants with chronic HBV infection on nucleos(t)ide analogue (NA) treatment2023-509027-41-00Glaxosmithkline Research & Development Limited23
进行中（未招募）
不适用
A study investigating the safety and tolerability of deferiprone in patients with Friedreich's Ataxia - NDpatients affected by Friedreich AtaxiaMedDRA version: 9.1Level: LLTClassification code 10017374Term: Friedreich's ataxiaEUCTR2007-003331-23-ITAPOPHARMA INC., TORONTO, ONTARIO80
已完成
1 期
A programme to develop a skin patch containing two medicines (physostigmine and hyoscine), Study 6: Assessment of blood levels of the two medicines and any associated symptoms in healthy female participants.ISRCTN15358284Dstl36
已完成
不适用
A study to investigate safety, tolerability, and pharmacokinetics of AZD7503 in healthy Japanese participants.nonalcoholic fatty liver diseasejRCT2071230075Astrazeneca K.K12
已完成
不适用
A study to investigate the safety, tolerability and absorption and elimination of CCX507-B, a new drug for the treatment of patients with inflammatory bowel diseases, in healthy male and female subjects.Crohn's disease.Inflammatory bowel diseases10017969NL-OMON40895ChemoCentryx, Inc.30