A Study of Orally Administered JBPOS0101 in Refractory Infantile Spasms Patients
Condition: Refractory Infantile Spasms
MeSH terms: Muscle Cramp; Spasm; Spasms, Infantile
Intervention: JBPOS0101
Intervention description: JBPOS0101 (investigational product)
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Intervention model description: Patients received the investigational product at a dose of 6 milligram per kg orally twice daily; once in the morning and 12 hours following the morning dose during the first 7 days of Treatment Period 1. Starting from the PM dose on the day of Visit 3, the dose was escalated and patients received the investigational product at a dose of 9 mg/kg orally twice daily. Starting on Day 15, the dose was escalated again and patients received the investigational product at a dose of 15 mg/kg orally twice daily until the end of Treatment Period 1 (Day 28).
Masking: None (Open Label)
Enrollment: 16.0
Sex: All
Minimum age: 6 Months
Maximum age: 36 Months
Healthy volunteers: No
Keywords: JBPOS0101; Infantile Spasms; Electroencephalogram

Summary:

      A Phase 2 Study to Assess the Safety, Tolerability, Exploratory Efficacy, and
      pharmacokinetics of Orally Administered JBPOS0101 for Refractory Infantile Spasms Patients.
    

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