A Study of Orally Administered JBPOS0101 in Refractory Infantile Spasms Patients
- Conditions
- Refractory Infantile Spasms
- Registration Number
- NCT03976076
- Lead Sponsor
- Bio-Pharm Solutions Co., Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Terminated
- Sex
- All
- Target Recruitment
- 16
Inclusion Criteria:<br><br> - Male or female between 6 months through 36 months of age at the time of informed<br> consent<br><br> - Had clinical diagnosis of Infantile spasms (IS), confirmed by<br> video-electroencephalogram (EEG) analysis, and hypsarrhythmia on EEG at screening<br> according to the Burden of Amplitudes and Epileptiform Discharges (BASED) scale<br> score.<br><br> - As assessed by the investigator had no or partial response to at least 2 out of the<br> 3 therapies of adrenocorticotrophic hormone (ACTH), vigabatrin, and glucocorticoids<br> (i.e. prednisolone), or had no or partial response to at least 1 out of the 3<br> therapies of ACTH, vigabatrin, and glucocorticoids and was contraindicated to and/or<br> refused by the patient's legal representative(s) for treatment with one or both<br> other 2 therapies.<br><br> - Patient had general good health (defined as the absence of any clinically relevant<br> abnormalities as determined by the investigator) based on physical and neurological<br> examinations, medical history, normal renal function and electrocardiogram (ECG),<br> and clinical laboratory values completed during the Screening Period visit (Visit<br> 1).<br><br> - Parent(s)/caregiver(s) were willing and able to comply with the study procedures and<br> visit schedules in the opinion of the investigator.<br><br> - Parent(s)/caregiver(s) fully comprehend and sign the ICF in accordance with<br> applicable laws, regulations, and local requirements, understand all study<br> procedures, and can communicate satisfactorily with the investigator and study<br> coordinator.<br><br>Exclusion Criteria:<br><br> - Patient considered by the investigator, for any reason (including, but not limited<br> to, the risks described as precautions and warnings in the current version of the<br> investigator's brochure for investigational product) to be an unsuitable candidate<br> to receive the investigational product.<br><br> - Patient had known or suspected allergy to the investigational product or apple<br> juice.<br><br> - Patient had clinically significant renal impairment, defined as creatinine >1.5<br> mg/dL or blood urea nitrogen >2 × upper limit of normal (ULN);<br><br> - Clinically significant liver dysfunction, defined as total bilirubin =2 × ULN, or<br> aspartate aminotransferase or alanine aminotransferase =3 × ULN;<br><br> - Patient had clinically significant abnormal laboratory values; the investigator may<br> deem the patient eligible if he/she judges the laboratory values to be not<br> clinically significant.<br><br> - Patient had an ongoing or known history of human immunodeficiency virus infection,<br> or chronic hepatitis B or C.<br><br> - Patient had a clinically significant abnormality on ECG that, in the opinion of the<br> investigator, increases the safety risks of participating in the study.<br><br> - Patient had a neurodegenerative disorder as the underlying cause of IS.<br><br> - Patient had a known history of aspiration pneumonia within the past year.<br><br> - Patient had previously participated in another clinical study of the investigational<br> product or received any investigational drug or device or investigational therapy<br> within 30 days of study entry.<br><br> - Patient had received therapy with felbamate, cannabinoids, ketogenic diet or vagus<br> nerve stimulation within 14 days of screening.<br><br> - Patient had received therapy with a medication known to be a CYP3A4 substrate and<br> whose PK had been shown to be impacted in the presence of a CYP3A4 inhibitor within<br> 14 days of screening.<br><br> - Patient had not remained at stables doses of all drugs used for treating epileptic<br> seizures for at least 14 days prior to screening (except for rescue medications used<br> for acute treatment of breakthrough seizures which were not known to be CYP3A4<br> substrates and whose PK had not been shown to be impacted in the presence of a<br> CYP3A4 inhibitor.<br><br> - Patient had a lethal or potentially lethal condition other than infantile spasms,<br> with a significant risk of death before 18 months of age such as non-ketotic<br> hyperglycinemia.<br><br> - Patient had a body weight below 5 kg.<br><br> - Patient had an underlying metabolic disease associated with glucose intolerance<br> (e.g., glucose transporter deficiencies).
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Participants With Treatment Emergent Adverse Events (TEAEs)
- Secondary Outcome Measures
Name Time Method JBPOS0101 Plasma Concentration 0.5 - 1.5 Hours Post Morning Dose, Day 1;JBPOS0101 Plasma Concentration 4-6 Hours Post Morning Dose, Day 1;JBPOS0101 Plasma Concentration 8 Hours Post Morning Dose and Pre-PM Dose, Day 1;JBPOS0101 Plasma Concentration 0.5 -1.5 Hours Post Morning Dose, Day 21;JBPOS0101 Plasma Concentration 4 - 6 Hours Post Morning Dose, Day 21;JBPOS0101 Plasma Concentration 8 Hours Post Morning Dose and Pre-PM Dose, Day 21;JBPOS0101 Urine Concentration at Day 1;JBPOS0101 Urine Concentrations at Day 21