AMO-01 to Treat Adolescents and Adults With Phelan-McDerm... | NCT03493607

NCT03493607

Protocol Section

Identification Module

Results Section

Participant Flow Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module

Status Module

Description Module

Label	Type	Description	Intervention Names
AMO-01	Experimental	Intravenous Infusion	Drug: AMO-01

Name	Type	Description	Arm Group Labels	Other Names
AMO-01	Drug	Subjects will receive a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.	AMO-01

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Adverse Events	An adverse event is defined as any untoward medical occurrence in a study subject, temporally associated with the use of the experimental medication, whether or not considered related to the medication. An adverse event can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of the experimental medication. Adverse events will be monitored throughout all 8 weeks of study participation.	8 weeks

Secondary Outcomes

Measure	Description	Time Frame
Number of Weekly Seizure Counts	Seizure frequency was measured by a caregiver-completed seizure diary throughout the duration of the trial. A seizure diary was provided to each family at the screening visit to record each seizure event, its date/time, duration, and type. The study team reviewed the diary at each visit with the caregiver and weekly seizure frequency was calculated. The week 1 seizure count was the number of seizures in the 7 days following the infusion day. Similarly, the week 2 seizure count was the number of seizures in the 7 days between weeks 1 and 2. Lastly, the week 4 seizure count was the sum of seizures in the 14 days between weeks 2 and 4, divided by 2.

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Subjects under study must have a diagnosis of Phelan McDermid syndrome (PMS) with genetic confirmation of pathogenic SHANK3 deletion or mutation.
Subjects must be post pubertal males or females aged ≥12 years and ≤45 years at Screening.
Subject must have a diagnosis of epilepsy.
Subjects must have a syndrome-specific Clinical Global Impression- Severity Score of 4 or greater at Screening
Subject's parent or legally authorized representative (LAR) must provide written informed consent before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations).
Subject's caregiver must be willing and able to support the subject's participation for the duration of the study.
Subject's caregiver is able and willing to maintain an accurate and complete daily written seizure diary for the entire duration of the study.

Exclusion Criteria:

Receiving medications/therapies not stable (i.e. changed) within 4 weeks prior to Screening. For each enrollee, every effort should be made to maintain stable regimens of allowed concomitant medications and allowed non -medicine based therapies throughout the course of the study, from Screening until the last study assessment.
Known hypersensitivity to farnesylated dibenzodiazepinone or any of the formulation components.
Subjects with a history of uncontrolled hypotension or hypertension (Polysorbate 80 is a major constituent of AMO-01 and can cause hypotension).
Subjects that have received Coumadin or heparin in the 2 weeks preceding Screening.
Medical illness or other concern which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessments.
Females who are pregnant, lactating or not willing to use a protocol-defined acceptable contraception method if sexually active and not surgically sterile.
Males, engaged in sexual relations with a female of child bearing potential, not using an acceptable contraception method if sexually active and not surgically sterile.
Clinically significant abnormalities in safety laboratory tests, vital signs or ECG, as measured at Screening (may repeat to confirm).
Current clinically significant (as determined by the investigator) neurological, cardiovascular, renal, hepatic, endocrine or respiratory disease that may impact the interpretability of the study results.
Current clinically significant (as determined by the investigator) lymphedema that may compromise venous access and/or may have an adverse impact on study drug distribution and clearance.
Judged clinically to be at risk of suicide by the investigator.
Average QTcF value of >450 msec at Screening (may repeat to confirm).
Subjects in whom an indwelling intravenous line could not be established or maintained.

Name	Affiliation	Role
Alexander Kolevzon, MD	Icahn School of Medicine at Mount Sinai	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Seaver Autism Center for Research and Treatment at Mount Sinai		New York	New York	10029	United States

ID	Title	Description
FG000	AMO-01	Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.

ID	Title	Description
BG000	AMO-01	Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.

Units	Counts
Participants	BG000

Title	Description	Population Description	Parameter Type	Dispersion Type	Unit of Measure	Calculate Percentage	Denominator Units Selected	Denominators	Classes
Age, Continuous			Mean

Outcome Measures Module

Outcome Measures

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 Adverse Events

An adverse event is defined as any untoward medical occurrence in a study subject, temporally associated with the use of the experimental medication, whether or not considered related to the medication. An adverse event can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of the experimental medication. Adverse events will be monitored throughout all 8 weeks of study participation.

Posted

Number

events

8 weeks

ID	Title	Description
OG000	AMO-01	Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.

Units	Counts

Adverse Events Module

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	AMO-01	Subjects received a single 6-hour intravenous infusion for a total dose administration or 120 mg/m2 of AMO-01.

ID	Term
C536801	Telomeric 22q13 Monosomy Syndrome
D004827	Epilepsy

ID	Term
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases