Refractory Infantile SpasmsPhase 2NCT03976076
A Study of Orally Administered JBPOS0101 in Refractory Infantile Spasms Patients
A Phase 2 study testing JBPOS0101 in people with Refractory Infantile Spasms, run by Bio-Pharm Solutions Co., Ltd. at 23 sites in 2 countries.
Trial summary
A Phase 2 Study to Assess the Safety, Tolerability, Exploratory Efficacy, and pharmacokinetics of Orally Administered JBPOS0101 for Refractory Infantile Spasms Patients. Read more about this study
- Who is it for?
- Ages 6 to 36 months
- Conditions the study is for
- Refractory Infantile Spasms
- What is being tested?
- JBPOS0101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 23 sitesin 2 countries
- Study dates
- 15 Apr 2020to 10 Dec 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 36 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 16Show fewer
- 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).
Trial locations and contacts
23 sites in 2 countries.
23 sites in 2 countries
United States17 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Cleveland Clinic
Cleveland, Ohio, 44195
Duke University Medical Center
Durham, North Carolina, 27710
Texas Children's Hospital
Houston, Texas, 77030
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Children's Hospital LA
Los Angeles, California, 90027
UCLA - David Geffen School of Medicine
Los Angeles, California, 90095
University of Louisville School of Medicine
Louisville, Kentucky, 40202
Nicklaus Children's Hospital
Miami, Florida, 33155
Center for Rare Neurological Diseases
Norcross, Georgia, 30093
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23298
Mayo Clinic
Rochester, Minnesota, 55905
UCSF Epilepsy Center
San Francisco, California, 94143
Multicare Institute for Research and Innovation
Tacoma, Washington, 98405
Pediatric Neurology, PA
Winter Park, Florida, 32789
South Korea6 sites
Kyungpook National University Chilgok Hospital (KNUH)
Daegu, 41404
Pusan National University Yangsan Hospital
Pusan, Gyeongsangnam-do, 50612
Seoul National University Hospital
Seoul, 03080
Severance Hospital, Yonsei University Health System
Seoul, 03722
Asan Medical Center
Seoul, 05505
Samsung Medical Center
Seoul, 06351
About this study
Official title: A Phase 2 Study to Assess the Safety, Tolerability, Exploratory Efficacy, and Pharmacokinetics of Orally Administered JBPOS0101 for 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.
Read the 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
- Being tested
- JBPOS0101
- Keywords
- JBPOS0101, Infantile Spasms, Electroencephalogram
- Medical subject headings
- Spasms, Infantile
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JBPOS0101Drug
JBPOS0101 (investigational product)
| Study group | Role | What they receive |
|---|---|---|
| JBPOS0101 (investigational product) | Experimental | 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. Receives: Drug: JBPOS0101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
TEAEs were defined as any event that did not present before exposure to the investigational product (IP) or any event already present that worsened in either intensity or frequency after exposure to the IP.
Measured: Day 1 to Day 56
Secondary outcomes (8)
JBPOS0101 Plasma Concentration 0.5 - 1.5 Hours Post Morning Dose, Day 1
Pharmacokinetics: JBPOS0101 plasma concentration 0.5 - 1.5 hours post morning dose on Day 1
Measured: 0.5 to1.5 hours post morning (AM) dose on Day 1
JBPOS0101 Plasma Concentration 4-6 Hours Post Morning Dose, Day 1
Pharmacokinetics: JBPOS0101 plasma concentration 4 - 6 hours post morning dose on Day 1
Measured: 4 to 6 hours post morning (AM) dose on Day 1
JBPOS0101 Plasma Concentration 8 Hours Post Morning Dose and Pre-PM Dose, Day 1
Pharmacokinetics: JBPOS0101 plasma concentration 8 hours post morning dose and pre-PM dose on Day 1.
Measured: 8 hours post morning (AM) dose and pre-PM dose on Day 1
JBPOS0101 Plasma Concentration 0.5 -1.5 Hours Post Morning Dose, Day 21
Pharmacokinetics: JBPOS0101 plasma concentration 0.5 - 1.5 hours post morning dose on Day 21
Measured: 0.5 to1.5 hours post morning (AM) dose on Day 21
JBPOS0101 Plasma Concentration 4 - 6 Hours Post Morning Dose, Day 21
Pharmacokinetics: JBPOS0101 plasma concentration 4 - 6 hours post morning dose on Day 21
Measured: 4 to 6 hours post morning (AM) dose on Day 21
JBPOS0101 Plasma Concentration 8 Hours Post Morning Dose and Pre-PM Dose, Day 21
Pharmacokinetics: JBPOS0101 plasma concentration 8 hours post morning dose and pre-PM dose on Day 21.
Measured: 8 hours post morning (AM) dose and pre-PM dose on Day 21
JBPOS0101 Urine Concentration at Day 1
Pharmacokinetics: JBPOS0101 urine concentration on Day 1. Urine samples were collected following the morning dose.
Measured: Day 1
JBPOS0101 Urine Concentrations at Day 21
Pharmacokinetics: JBPOS0101 urine concentration on Day 21. Urine samples were collected following the morning dose.
Measured: Day 21
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2019
- Study start15 Apr 2020
- Primary completion10 Dec 2021
- Study completion10 Dec 2021
Why it stopped: Difficulty with patient recruitment due to COVID-19
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Refractory Infantile SpasmsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bio-Pharm Solutions Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hee Jin Kim, Study director, Bio-Pharm Solutions
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03976076
- Sponsor's protocol ID
- CL-0101-WS01
- Status last verified
- Aug 2024
- First submitted
- 31 May 2019
- Last updated
- 22 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study to Assess the Safety, Tolerability, Exploratory Efficacy, and Pharmacokinetics of Orally Administered JBPOS0101 for Refractory Infantile Spasms Patients
- ClinicalTrials.gov ID
- NCT03976076
- Acronym / sponsor ID
- CL-0101-WS01
- Conditions
- Refractory Infantile Spasms
- Keywords
- JBPOS0101, Infantile Spasms, Electroencephalogram
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Difficulty with patient recruitment due to COVID-19
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Bio-Pharm Solutions Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Apr 2020 (actual)
- Primary completion
- 10 Dec 2021 (actual)
- Study completion
- 10 Dec 2021 (actual)
- First posted
- 5 Jun 2019 (actual)
- Results
- Posted 16 Feb 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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).
| Arm | Type | Receives |
|---|---|---|
| JBPOS0101 (investigational product) | Experimental | Drug: JBPOS0101During 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. |
Who may qualify
- Ages
- 6 to 36 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (16 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| JBPOS0101JBPOS0101 (investigational product) | Drug | JBPOS0101 (investigational product) |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: Day 1 to Day 56
TEAEs were defined as any event that did not present before exposure to the investigational product (IP) or any event already present that worsened in either intensity or frequency after exposure to the IP.
8 secondary outcomes
JBPOS0101 Plasma Concentration 0.5 - 1.5 Hours Post Morning Dose, Day 1
Time frame: 0.5 to1.5 hours post morning (AM) dose on Day 1
JBPOS0101 Plasma Concentration 4-6 Hours Post Morning Dose, Day 1
Time frame: 4 to 6 hours post morning (AM) dose on Day 1
JBPOS0101 Plasma Concentration 8 Hours Post Morning Dose and Pre-PM Dose, Day 1
Time frame: 8 hours post morning (AM) dose and pre-PM dose on Day 1
JBPOS0101 Plasma Concentration 0.5 -1.5 Hours Post Morning Dose, Day 21
Time frame: 0.5 to1.5 hours post morning (AM) dose on Day 21
JBPOS0101 Plasma Concentration 4 - 6 Hours Post Morning Dose, Day 21
Time frame: 4 to 6 hours post morning (AM) dose on Day 21
JBPOS0101 Plasma Concentration 8 Hours Post Morning Dose and Pre-PM Dose, Day 21
Time frame: 8 hours post morning (AM) dose and pre-PM dose on Day 21
JBPOS0101 Urine Concentration at Day 1
Time frame: Day 1
JBPOS0101 Urine Concentrations at Day 21
Time frame: Day 21
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hee Jin Kim study director
Bio-Pharm Solutions
23 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing JBPOS0101 in people with Refractory Infantile Spasms, run by Bio-Pharm Solutions Co., Ltd. at 23 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Feb 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment Emergent Adverse Events (TEAEs) (time frame: Day 1 to Day 56). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Aug 2024.