Activated PI3K delta Syndrome (APDS)
Conditions
Interventions
Product Name: UCB5857
Product Code: UCB5857
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: UCB5857
CAS Number: 1362850-20-1
Current Sponsor code: UCB5857
Concentration unit: mg milligram(s)
C
Sponsors
UCB Celltech, UK Registered Branch of UCB Pharma SA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Subject/legal representative is considered reliable and capable of adhering to the protocol (e.g. able to understand and complete diaries), visit schedule, or medication intake. - Subject must have a confirmed genotypic diagnosis of APDS (i.e. proven pathogenic mutation in either the PIK3R1 or PIK3CD gene). - Subjects must have had clinical findings and manifestations compatible with APDS such as nodal and/or extranodal lymphoproliferation, history of repeated oto-sinopulmonary infections and/or organ dysfunction (e.g., lung, liver) 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 another study of an investigational medicinal product (IMP) (or a medical device) within the previous 30 days or is currently participating in another study of an IMP (or a medical device). 2. Subject has a history of allogeneic bone marrow transplantation. 3. Subject has no history of any clinical manifestations in last 12 months. 4. Subject tests positive for human immunodeficiency virus Type 1 or Type 2, hepatitis B surface antigen, or hepatitis C virus antibody. 5. Subject whose only clinical manifestations in the last 12 months are due to advanced bronchiectasis (declining lung function, 3 or more recurrent exacerbations, lung abscess, pneumothorax for repeating cough). 6. Subject who has a severe (life-threatening) infection during the screening period. 7. Subject who have lymphoma at the time of the screening or have been treated for lymphoma in the previous 5 years prior Screening (Visit 1). 8. 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. 9. Subject has WBC<2000/mm3, or absolute neutrophil count <1000/mm3 at Screening (Visit 1). 10. Subject has a history of chronic alcohol or drug abuse within the previous 6 months. 11. Subject has any relevant medical or psychiatric condition that could jeopardize or would compromise the subject’s ability to participate in this study. 12. Subject has a known hypersensitivity to any components of the IMP or comparative drugs as stated in this protocol. 13. Subject has been treated with any mTOR inhibitors (unless washed out, approximately 6 weeks). 14. Subject has a history of inflammatory bowel disease, peptic ulcers, or recurrent colitis including rectal bleeding (within 1 year prior to Screening [Visit 1]). 15. Subject has a body weight of less than 20 kg at Screening (Visit 1).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the safety and tolerability of UCB5857 in subjects with Activated phosphoinositide-3 kinase (PI3K) delta Syndrome (APDS).;Secondary Objective: The secondary objective is to assess the pharmacokinetic (PK) profile of UCB5857 in subjects with Activated phosphoinositide-3 kinase (PI3K) delta Syndrome (APDS).;Primary end point(s): Safety variables The safety variables will include: • Incidence of AEs • Incidence of SAEs • Changes from Baseline in safety laboratory tests (serum chemistry, haematology, and urinalysis) at the time points measured • Change from Baseline in vital sign parameters (systolic and diastolic blood pressure, temperature, pulse rate and respiratory rate) and body weight at the time points measured • Change from Baseline in ECG parameters at the time points measured • Physical examination findings. Pharmacokinetic variable The PK variable will be the plasma concentration of UCB5857.;Timepoint(s) of evaluation of this end point: At the time points measured. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Please refer to the study variables of the Clinical Trial Protocol.;Timepoint(s) of evaluation of this end point: Please refer to the study variables of the Clinical Trial Protocol. | — |
Countries
France, Germany, Italy, Spain
Contacts
Public ContactClin Trial Reg & Results Disclosure
UCB BIOSCIENCES GmbH
Outcome results
None listed