Loading clinical trials...

A Study of Orally Administered JBPOS0101 in Refractory Infantile Spasms Patients | Clinical Trials | Clareo Health

TERMINATEDPHASE2

A Study of Orally Administered JBPOS0101 in Refractory Infantile Spasms Patients

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

NCT03976076•Bio-Pharm Solutions Co., Ltd.

View on ClinicalTrials.gov

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.

Eligibility

Age

0 - 3 years

Sex

ALL

Healthy Volunteers

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.
•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.
+7 more criteria

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;

Locations (23)

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

Key Dates

Start Date

April 15, 2020

Primary Completion Date

December 10, 2021

Completion Date

December 10, 2021

Last Updated

August 22, 2024

Enrollment

ACTUAL participants

Conditions

Refractory Infantile Spasms

Interventions

JBPOS0101

DRUG