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A Study of Orally Administered JBPOS0101 in Refractory Infantile Spasms Patients

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ClinicalTrials.gov Identifier: NCT03976076
Recruitment Status : Recruiting
First Posted : June 5, 2019
Last Update Posted : March 5, 2021
Sponsor:
Information provided by (Responsible Party):
Bio-Pharm Solutions Co., Ltd.

Tracking Information
First Submitted Date  ICMJE May 31, 2019
First Posted Date  ICMJE June 5, 2019
Last Update Posted Date March 5, 2021
Actual Study Start Date  ICMJE March 1, 2019
Estimated Primary Completion Date November 30, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 4, 2019)
Incidence and severity of treatment emergent adverse events observed by the investigator at each visit or reported to the investigator [ Time Frame: 77 days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of Orally Administered JBPOS0101 in Refractory Infantile Spasms Patients
Official Title  ICMJE A Phase 2 Study to Assess the Safety, Tolerability, Exploratory Efficacy, and Pharmacokinetics of Orally Administered JBPOS0101 for Refractory Infantile Spasms Patients
Brief Summary This open label, multicenter study allows JBPOS0101 (investigational product) to be given as either add-on therapy or monotherapy for patients with refractory infantile spasms. The design and choice of study population of this Phase 2 clinical study is based on the need to provide initial safety, tolerability, pharmacokinetics (PK), and efficacy outcomes of the investigational product for future clinical studies.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
Patients will receive the investigational product at a dose of 6 mg/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 will be escalated and patients will receive the investigational product at a dose of 9 mg/kg orally twice daily. Starting on Day 15, the dose will be escalated again and patients will receive the investigational product at a dose of 15 mg/kg orally twice daily until the end of Treatment Period 1.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Refractory Infantile Spasms
Intervention  ICMJE Drug: JBPOS0101
JBPOS0101 (investigational product)
Study Arms  ICMJE Experimental: JBPOS0101 (investigational product)
Intervention: Drug: JBPOS0101
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 4, 2019)
35
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 7, 2022
Estimated Primary Completion Date November 30, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male or female between 6 months through 36 months of age at the time of informed consent
  • Has clinical diagnosis of 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 has no or partial response to at least 2 out of the 3 therapies of adrenocorticotrophic hormone (ACTH), vigabatrin, and glucocorticoids (i.e. prednisolone), or has no or partial response to at least 1 out of the 3 therapies of ACTH, vigabatrin, and glucocorticoids and is contraindicated to and/or refused by the patient's legal representative(s) for treatment with one or both other 2 therapies.
  • Patient has 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).

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 has known or suspected allergy to the investigational product or apple juice.
  • Patient has 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; has 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 has an ongoing or known history of human immunodeficiency virus infection, or chronic hepatitis B or C.
  • Patient has a clinically significant abnormality on ECG that, in the opinion of the investigator, increases the safety risks of participating in the study.
  • Patient has a neurodegenerative disorder as the underlying cause of IS.
  • Patient has a known history of aspiration pneumonia within the past year.
  • Patient has 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 has received therapy with felbamate, cannabinoids, ketogenic diet or vagus nerve stimulation within 14 days of screening.
  • Patient has received therapy with a medication known to be a CYP3A4 substrate and whose PK has been shown to be impacted in the presence of a CYP3A4 inhibitor within 14 days of screening.
  • Patient has 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 are not known to be CYP3A4 substrates and whose PK has not been shown to be impacted in the presence of a CYP3A4 inhibitor.
  • Patient has 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 has a body weight below 5 kg.
  • Patient has an underlying metabolic disease associated with glucose intolerance (e.g., glucose transporter deficiencies).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 36 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Brandon Choi 973-224-0567 bchoi@b-psol.com
Contact: Scott Wood 720-504-9454 scott.wood@iconplc.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03976076
Other Study ID Numbers  ICMJE CL-0101-WS01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Bio-Pharm Solutions Co., Ltd.
Study Sponsor  ICMJE Bio-Pharm Solutions Co., Ltd.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Brandon Choi Sponsor Management
PRS Account Bio-Pharm Solutions Co., Ltd.
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP