Activated PI3K&delta
Conditions
Interventions
None listed
Sponsors
Pharming Technologies b.v.
Eligibility
Age
2 Years to 11 Years
Inclusion criteria
Inclusion criteria: • Patient is male or female and between the age of 4 to 11 years old at time of the first study procedure. Females should be of nonchildbearing potential at screening. • Patient weighs >=13 kg and
Exclusion criteria
Exclusion criteria: • Patient has previous or concurrent use of immunosuppressive medication such as: a. An mTOR inhibitor (eg, sirolimus, rapamycin, everolimus) or a PI3Kδ inhibitor (selective or non-selective PI3K inhibitors) within 6 weeks prior to first dose. i. Short-term use for up to a total of 5 days is allowed but only up to 1 month prior to enrollment in the study. b. B cell depleters (eg, rituximab) within 6 months prior to first dose of study medication. i. If patient has received prior treatment with a B cell depleter, absolute B lymphocyte counts in the blood must have regained normal values. c. Belimumab or cyclophosphamide within 6 months prior to first dose of study medication. d. Cyclosporine A, mycophenolate, 6-mercaptopurine, azathioprine, or methotrexate within 3 months prior to first dose of study medication. e. Systemic glucocorticoids above a dose equivalent to either >=2 mg/kg of body weight or >=20 mg/day of prednisone/prednisolone or equivalent. f. Other immunosuppressive medication where effects are expected to persist at start of dosing of study medication. • Patient has a history or current diagnosis of electrocardiogram (ECG) abnormalities indicating significant risk of safety for patients participating in the study such as: a. History of familial long QT syndrome or known family history of Torsades de Pointes. b. Concomitant clinically significant cardiac arrhythmias, eg, sustained ventricular tachycardia, and clinically significant second or third degree atrioventricular block without a pacemaker. c. Resting QTc (Fridericia preferred, but Bazett acceptable) >460 msec if the measurement is confirmed with an additional ECG repeated as soon as possible. d. Concomitant use of agents known to prolong the QT interval unless it can be permanently discontinued for the duration of the study. • Patient is currently using a medication known to be strong inhibitor or moderate or strong inducer of isoenzyme CYP3A (see Table 2), if treatment cannot be discontinued or switched to a different medication prior to starting study treatment. • Patient is currently using medications that are metabolized by isoenzyme CYP1A2 and have a narrow therapeutic index (drugs whose exposure- response indicates that increases in their exposure levels by the concomitant use of potent inhibitors may lead to serious safety concerns [eg, Torsades de Pointes]). e. Patient is currently using medications known to be organic anion transporter protein (OATP)1B1, OATP1B3, and breast cancer resistance protein (BCRP) substrates
Design outcomes
Primary
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Secondary
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Countries
Netherlands
Outcome results
None listed