NL-OMON54847招募中2 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/p110δ-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency) - CCDZ173X2201E1
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 5
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
- 12 至 64（—）
入选标准
- •Patients must have completed the study CCDZ173X2201 EOT/EOS visit, or were
- •treated previously with PI3Kδ inhibitors other than CDZ173.
- •Patients who are deemed by the Investigator to benefit from PI3K inhibitor
- •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
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.
- •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
- •PI3Kδ inhibitors other than CDZ173, the following additional exclusion criteria
- •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.
- •Use of unstable dosing regimen with i.v. Ig / s.c. Ig in the last 6 months
- •before screening. Stable maintenance immunoglobulin regimen, as per local
- •practice, such as regular injections with a consistent dosing interval (e.g.,
- •monthly) is acceptable
- •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
- •Other protocol-defined exclusion criteria may apply
研究者
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