A Study of Orally Administered JBPOS0101 in Refractory In... | NCT03976076

NCT03976076

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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.	Drug: JBPOS0101

Name	Type	Description	Arm Group Labels	Other Names
JBPOS0101	Drug	JBPOS0101 (investigational product)	JBPOS0101 (investigational product)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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.	Day 1 to Day 56

Secondary Outcomes

Measure	Description	Time Frame
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	0.5 to1.5 hours post morning (AM) dose on Day 1
JBPOS0101 Plasma Concentration 4-6 Hours Post Morning Dose, Day 1

Eligibility Module

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).

Contacts/Locations Module

Name	Affiliation	Role
Hee Jin Kim	Bio-Pharm Solutions	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Arkansas Children's Hospital		Little Rock	Arkansas	72202	United States
Children's Hospital LA

References Module

No data available

No data is available for this block.

IPD Sharing Statement Module

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6mg/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. IP was100 mg, white to off-white powder for reconstitution into an oral solution.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Sep 15, 2020	Dec 10, 2022

Derived Section

Miscellaneous Info Module

Submission Tracking

Condition Browse Module

Intervention Browse Module

No data available

No data is available for this block.

Masking Info

Type of Units Analyzed

Arm/Group Information

ID	Title	Description
BG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6mg/kg, PO, 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 IP (JBPOS0101) 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. IP was100 mg, white to off-white powder for reconstitution into an oral solution.

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

Months

Title

Denominators

Categories

Title	Measurements
	BG00019.93± 8.373

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0006
Male	BG0006

Ethnicity (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino	BG0002
Not Hispanic or Latino	BG00010

Race (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
American Indian or Alaska Native	BG0000
Asian	BG0001

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

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.

Safety Population: Included all patients who were treated with at least one dose of the IP.

Posted

Count of Participants

Participants

Day 1 to Day 56

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, PO, twice a 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 IP (JBPOS0101) 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. IP was100 mg, white to off-white powder for reconstitution into an oral solution.

Units	Counts
Participants	OG00012

Title

Denominators

Categories

Title	Measurements
	OG00012

Secondary

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

Posted

Mean

Standard Deviation

Nanograms per milliliter (ng/mL)

0.5 to1.5 hours post morning (AM) dose on Day 1

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, PO, 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 IP (JBPOS0101) 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. IP was100 mg, white to off-white powder for reconstitution into an oral solution.

Units	Counts
Participants

Secondary

JBPOS0101 Plasma Concentration 4-6 Hours Post Morning Dose, Day 1

Pharmacokinetics: JBPOS0101 plasma concentration 4 - 6 hours post morning dose on Day 1

Posted

Mean

Standard Deviation

ng/mL

4 to 6 hours post morning (AM) dose on Day 1

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, per oral, 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 IP (JBPOS0101) 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. IP was 100 mg, white to off-white powder for reconstitution into an oral solution.

Units	Counts
Participants

Secondary

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.

PK population: The PK population included all patients, without any major protocol deviation affecting the secondary endpoint analysis, who were treated with at least one dose of the IP and had at least one measurable PK concentration. Here N=12 is the overall number of participants. n= 11 signifies number of participants with available data for specified time.

Posted

Mean

Standard Deviation

ng/mL

8 hours post morning (AM) dose and pre-PM dose on Day 1

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, per oral, 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 IP (JBPOS0101) 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. IP is 100 mg, white to off-white powder for reconstitution into an oral solution.

Units	Counts

Secondary

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

Posted

Mean

Standard Deviation

ng/mL

0.5 to1.5 hours post morning (AM) dose on Day 21

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, PO, 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 IP (JBPOS0101) 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. IP was100 mg, white to off-white powder for reconstitution into an oral solution.

Units	Counts

Secondary

JBPOS0101 Plasma Concentration 4 - 6 Hours Post Morning Dose, Day 21

Pharmacokinetics: JBPOS0101 plasma concentration 4 - 6 hours post morning dose on Day 21

PK population: PK Population: The PK population included all patients, without any major protocol deviation affecting the secondary endpoint analysis, who were treated with at least one dose of the IP and had at least one measurable PK concentration.

Here N=12 is the overall number of participants. n= 10 signifies number of participants with available data for specified time.

Posted

Mean

Standard Deviation

ng/mL

4 to 6 hours post morning (AM) dose on Day 21

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, PO, 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 IP (JBPOS0101) 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. IP was 100 mg, white to off-white powder for reconstitution into an oral solution.

Units	Counts

Secondary

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.

Here N=12 is the overall number of participants. n= 9 signifies number of participants with available data for specified time.

Posted

Mean

Standard Deviation

ng/mL

8 hours post morning (AM) dose and pre-PM dose on Day 21

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: 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. IP was100 mg, white to off-white powder for reconstitution into an oral solution.

Secondary

JBPOS0101 Urine Concentration at Day 1

Pharmacokinetics: JBPOS0101 urine concentration on Day 1. Urine samples were collected following the morning dose.

PK population: The PK population included all patients, without any major protocol deviation affecting the secondary endpoint analysis, who were treated with at least one dose of the IP and had at least one measurable PK concentration. Here N=12 is the overall number of participants. n= 10 signifies number of participants with available data for specified time.

Posted

Mean

Standard Deviation

ng/mL

Day 1

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, PO, 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 IP (JBPOS0101) 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. IP was 100 mg, white to off-white powder for reconstitution into an oral solution.

Units	Counts
Participants

Secondary

JBPOS0101 Urine Concentrations at Day 21

Pharmacokinetics: JBPOS0101 urine concentration on Day 21. Urine samples were collected following the morning dose.

PK population: The PK population included all patients, without any major protocol deviation affecting the secondary endpoint analysis, who were treated with at least one dose of the IP and had at least one measurable PK concentration. Here N=12 is the overall number of participants. n= 9 signifies number of participants with available data for specified time.

Posted

Mean

Standard Deviation

ng/mL

Day 21

ID	Title	Description
OG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, PO, 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 IP (JBPOS0101) 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. IP was 100 mg, white to off-white powder for reconstitution into an oral solution.

Units	Counts
Participants

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	JBPOS0101 (Investigational Product)	JBPOS0101: JBPOS0101 (investigational product) During Treatment Period 1, the IP was administered at 6 mg/kg, PO, 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) at a dose of 9 mg/kg orally BID. Starting on Day15, 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. IP was 100 mg, white to off-white powder for reconstitution into an oral solution.	0	12	3	12	12	12

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Liver function test increased	Investigations	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Seizure	Nervous system disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Aspiration Pneumonia	Respiratory, thoracic and mediastinal disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Somnolence	Nervous system disorders	MedDRA Version: 23.0	Systematic Assessment	EG0003 events3 affected12 at risk
Sedation	Nervous system disorders	MedDRA Version: 23.0	Systematic Assessment	EG0004 events2 affected12 at risk
Liver function test increased	Investigations	MedDRA Version: 23.0	Systematic Assessment	EG0002 events2 affected12 at risk
Electrocardiogram QT prolonged	Investigations	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Gamma-glutamyltransferase increased	Investigations	MedDRA Version: 23.0	Systematic Assessment	EG0002 events1 affected12 at risk
Abnormal behaviour	Psychiatric disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Irritability	Psychiatric disorders	MedDRA Version: 23.0	Systematic Assessment	EG0002 events2 affected12 at risk
Constipation	Gastrointestinal disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Fatigue	General disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Sedation complication	Injury, poisoning and procedural complications	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Urine odour abnormal	Renal and urinary disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Drooling	Nervous system disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Seizure	Nervous system disorders	MedDRA Version: 23.0	Systematic Assessment	EG0002 events1 affected12 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA Version: 23.0	Systematic Assessment	EG0002 events2 affected12 at risk
Pyrexia	General disorders	MedDRA Version: 23.0	Systematic Assessment	EG0002 events1 affected12 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA Version: 23.0	Systematic Assessment	EG0002 events2 affected12 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Aspiration pneumonia	Respiratory, thoracic and mediastinal disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Wheezing	Respiratory, thoracic and mediastinal disorders	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Gastroenteritis	Infections and infestations	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Nasopharyngitis	Infections and infestations	MedDRA Version: 23.0	Systematic Assessment	EG0002 events1 affected12 at risk
Otitis media acute	Infections and infestations	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA Version: 23.0	Systematic Assessment	EG0001 events1 affected12 at risk

No limitations reported for the study.

Certain Agreements

First MCP Posted Date

ID	Term
D013036	Spasms, Infantile

ID	Term
D004829	Epilepsy, Generalized
D004827	Epilepsy
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D000073376	Epileptic Syndromes