Activated PI3K delta Syndrome (APDS)
Conditions
Sponsors
UCB BioPharma SPRL
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 2. Subject/legal representative is considered reliable and capable of adhering to the protocol (eg, able to understand and complete diaries), visit schedule, and medication intake according to the judgment of the investigator; and be able to give written informed consent to participate in the study. 5. Subject must complete the full Treatment Period of APD001 and must be able to expect a reasonable benefit from the long-term administration of UCB5857 in the medical judgment of the Investigator following review of the subject?s overall response in APD001. The SMC from APD001 will confirm each subject?s eligibility based on an evaluation of individual benefit-risk balance. 8. Contraception methods for male subjects and their female partners: - Male subjects with a partner of childbearing potential must be willing to use a condom when sexually active during the study and for 3 months after the last administration of study drug (anticipated 5 half lives). - In addition the female partner of childbearing potential of a male subject must be willing to use a highly effective method of contraception (as above) during the study period and for 3 months after the last administration of study drug. Additional inclusion criteria are outlined in section 6.1 of the clinical trial protocol. Are the trial subjects under 18? yes Number of subjects for this age range: 3 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 3 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject has participated in a clinical study with an IMP within 3 months of randomization into the current study (excluding participation in APD001) or subject is currently participating in another study of an IMP (or a medical device). 2. Subject has a history of chronic alcohol or drug abuse within the previous 6 months. 3. Subject has a history of allogenic bone marrow transplant. 4. Subject has any medical or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the subject?s ability to participate in this study. 5. Subject has any new significant uncontrolled condition or an ongoing SAE from the previous study (APD001) that was assessed as related to IMP. 6. Subject has a known hypersensitivity to any components of the IMP or comparative drugs as stated in this protocol. 7. Subject is female and is breast-feeding, pregnant, or plans to become pregnant or to start breastfeeding during the study or within 3 months following last dose of IMP. 11. Subject has >=3x upper limit of normal (ULN) alanine aminotransferase (ALT) or alkaline phosphatase (ALP), or >ULN bilirubin (>=1.5xULN bilirubin if known Gilbert?s syndrome). If subject only has >1.5xULN bilirubin, fractionate bilirubin to identify possible undiagnosed Gilbert?s syndrome (ie, direct bilirubin ULN, a baseline diagnosis and/or the cause of any clinically meaningful elevation must be understood and recorded in eCRF . If subject has >ULN that does not meet the exclusion limit for, ALT, or ALP at screening, repeat, if possible, prior to dosing to ensure there is no further ongoing clinically relevant increase. In case of a clinically relevant increase, inclusion of the patient must be discussed with the Medical Monitor[IS18]. Serum ALT results up to 25% above the exclusion limit may be repeated once for confirmation. This includes re-screening. Additional exclusion criteria are outlined in section 6.2 of the clinical trial protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: The secondary objective is to assess the long-term PK profile of UCB5857 in subjects with APDS through sparse sampling.; Primary end point(s): - The total number of subjects experiencing at least one Treatment Emergent Adverse Event during the study - The total number of subjects experiencing at least one Serious Adverse Event during the study ;Timepoint(s) of evaluation of this end point: Time Frame: Baseline until the end of study;Main Objective: The primary objective of the study is to evaluate the long-term safety and tolerability of UCB5857 in subjects with APDS. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Plasma Concentration of UCB5857;Timepoint(s) of evaluation of this end point: Time Frame: Baseline until the end of study | — |
Countries
France, Germany, Italy, Spain
Contacts
Public ContactClin Trial Reg & Results Disclosure
UCB BIOSCIENCES GmbH
Outcome results
None listed