A Study of Orally Administered JBPOS0101 in Refractory Infantile Spasms Patients

NCT ID: NCT03976076

Last Updated: 2024-08-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-04-15
• Study Completion Date: 2021-12-10

Brief Summary

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

Detailed Description

This open label, multicenter study allowed 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 was based on the need to provide initial safety, tolerability, pharmacokinetics (PK), and efficacy outcomes of the investigational product for future clinical studies.

Conditions

Refractory Infantile Spasms

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

JBPOS0101 (investigational product)

During Treatment Period 1, the IP was administered at 6 mg/kg, per oral (PO), twice a day (BID), 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 Visit 3, the dose was escalated and patients received the Investigational Product (JBPOS0101) (IP) at a dose of 9 mg/kg orally BID. Starting on Day 15, the dose was escalated again and patients received the IP at a dose of 15 mg/kg orally BID until the end of Treatment Period 1 (Day 28). Each dose of the IP was administered after at least a 2-hour fast. Food was given 2 hours after dosing.

Group Type: EXPERIMENTAL

JBPOS0101

Intervention Type: DRUG

JBPOS0101 (investigational product)

Interventions

JBPOS0101

JBPOS0101 (investigational product)

Intervention Type: DRUG

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).

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 36 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bio-Pharm Solutions Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Hee Jin Kim

Role: STUDY_DIRECTOR

Bio-Pharm Solutions

Locations

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Children's Hospital LA

Los Angeles, California, United States

UCLA - David Geffen School of Medicine

Los Angeles, California, United States

UCSF Epilepsy Center

San Francisco, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nicklaus Children's Hospital

Miami, Florida, United States

Pediatric Neurology, PA

Winter Park, Florida, United States

Center for Rare Neurological Diseases

Norcross, Georgia, United States

University of Louisville School of Medicine

Louisville, Kentucky, United States

Mayo Clinic

Rochester, Minnesota, United States

Duke University Medical Center

Durham, North Carolina, United States

Cleveland Clinic

Cleveland, Ohio, United States

Oregon Health and Science University

Portland, Oregon, United States

The Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, United States

Texas Children's Hospital

Houston, Texas, United States

Virginia Commonwealth University

Richmond, Virginia, United States

Multicare Institute for Research and Innovation

Tacoma, Washington, United States

Pusan National University Yangsan Hospital

Pusan, Gyeongsangnam-do, South Korea

Kyungpook National University Chilgok Hospital (KNUH)

Daegu, , South Korea

Seoul National University Hospital

Seoul, , South Korea

Severance Hospital, Yonsei University Health System

Seoul, , South Korea

Asan Medical Center

Seoul, , South Korea

Samsung Medical Center

Seoul, , South Korea

Countries

United States; South Korea

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

CL-0101-WS01

Identifier Type: -

Identifier Source: org_study_id