EUCTR2016-000468-41-FR进行中（未招募）1 期
An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d- activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - Extension to the study of safety and efficacy of CDZ173 in patients with APDS/PASLI
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 42
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
- •Patients must have participated in study CCDZ173X2201 or were treated previously with PI3Kd inhibitors other than CDZ173
- •Patients who are deemed by the Investigator to benefit from PI3K inhibitor therapy.
- •Patients or their legal representatives (for patients under the age of 18 years) must be able to communicate well with the Investigator, to understand and comply with the requirements of the study.
- •Documented APDS/PASLI-associated genetic PI3K delta mutation. Patients with mutations in either PIK3CD or PIK3R1 can be included.
- •Other protocol-defined inclusion criteria may apply
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 26
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1
排除标准
- •Patients who withdrew consent from the study CCDZ173X2201.
- •Use of other investigational drugs, except CDZ173, within 5 half -lives of enrollment, or within 30 days, whichever is longer.
- •Previous or concurrent use of immunosuppressive medication
- •Administration of any live vaccines (including any attenuated live vaccines) starting from 6 weeks before study entry, during the study and up to 7 days after the last dose of CDZ173 should be excluded.
- •Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation.
- •Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 2 days after last dose of study medication.
- •Uncontrolled chronic or recurrent infectious disease (with the exception of those that are considered to be characteristic of APDS/PASLI).
- •For patients who did not participate in study CCDZ173X2201 but were treated previously with PI3Kd inhibitors other than CDZ173, the following additional exclusion criteria apply:
- •Vital signs (systolic and diastolic blood pressure and pulse rate) will be assessed in the sitting position after the patient has rested for at least three minutes.
- •Patient must have a minimum body weight of 45 kg
- •Evidence of tuberculosis infection as defined by a positive QuantiFERON TB test (or comparable test) at screening. If presence of latent tuberculosis is established then treatment according to local country guidelines must have been completed before patients can be considered for enrollment.
- •History of acquired immunodeficiency diseases, or a positive HIV (ELISA and Western blot) test result at screening.
- •A positive Hepatitis B surface antigen or Hepatitis C test (by PCR) result at screening.
- •Other protocol-defined exclusion criteria may apply
研究者
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