Study of Midazolam Hydrochloride Oromucosal Solution (MHOS/SHP615) in Pediatric Patients With Status Epilepticus (Convulsive) in the Community Setting

Shire

Status and phase: Completed; Phase 3

Conditions: Nervous System Diseases

Treatments: Drug: SHP615; Drug: MHOS/SHP615

Study type: Interventional

Funder types: Industry

Identifiers

NCT03336450

SHP615-302

Details and patient eligibility

About

The purpose of this study is to determine if the investigational treatment, MHOS/SHP615, is safe and effective in children with status epilepticus (SE) (convulsive) in the community setting. This study is open-label extension for patients who completed the SHP615-301 study and who tolerated and responded to MHOS/SHP615 treatment in the hospital setting.

Enrollment: 3 patients

Sex: All

Ages: 6 to 216 months old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subjects who completed the SHP615-301 study and who tolerated and responded to treatment with MHOS/SHP615 in the hospital and/or emergency room, and are considered stable for discharge from the hospital.

• Subjects who are greater than (>) 6 months and less than (<) 18 years of age at the time of investigational product administration. If the subject's exact age is not known, the subject should be excluded.

• Parent, guardian, or legally authorized representative of the child who provides informed consent and assent (when applicable) to participate in the study after initial stabilization of the subject with SE in hospital or emergency room during the SHP615-301 study. The subject also provides informed consent prior to participation, where applicable.

• Parent, guardian, or legally authorized representative who have received appropriate training/education and are deemed qualified by the investigator and are willing to:

  1. Properly administer MHOS/SHP615.
  2. Record seizure information and dosing of MHOS/SHP615 in a subject diary (including time of seizure onset, type of seizure, time necessary to administer MHOS/SHP615, time between MHOS/SHP615 administration to seizure cessation, etc.)
  3. Follow the necessary instructions to secure the safety of the subject.

• Subjects who experience generalized tonic-clonic SE with seizures accompanied by loss of consciousness with any of the following characteristics persistent at the time of study drug administration:

  1. Currently presenting with seizure (convulsive) activity and 3 or more convulsions within the preceding hour
  2. Currently presenting with seizure (convulsive) and 2 or more convulsions in succession without recovery of consciousness.
  3. Currently presenting with a single seizure (convulsive) persisting greater than or equal to (>=) 5 minutes.

Exclusion criteria

• Female subjects who are pregnant, suspected to be pregnant, or nursing.
• Subjects with major trauma, not necessarily restricted to the head, as the cause of the seizure.
• Subjects with known or suspected recurrent seizures due to illegal drug or alcohol withdrawal.
• Subjects with seizures due to illegal drug or acute alcoholic intoxication.
• Subjects with seizures of psychogenic origin.
• Subjects with seizures due to severe encephalitis or meningitis, as determined by the PI
• Subjects with known history of hypersensitivities, nonresponsiveness or contraindications to benzodiazepines (that is (ie), clinically significant respiratory depression, severe acute hepatic failure, myasthenia gravis, syndrome of sleep apnea, glaucoma with closed angle, or use of concomitant drugs determined by the investigator to have a contraindication to the use of benzodiazepines.)
• Subjects with a known history of benzodiazepine abuse.
• Subjects who have not responded to previous administrations of midazolam systemic therapies, including MIDAFRESA and/or DORMICUM.
• Subjects who need emergent surgical intervention and general anesthesia/intubation.
• Subjects who have been receiving human immunodeficiency virus (HIV) protease inhibitors or HIV reverse transcriptase inhibitors.
• Subjects with severe cerebral anoxia (except cerebral palsy), in the judgment of the healthcare provider.
• Have used an investigational product or been enrolled in a clinical study (including vaccine studies) that, in the investigator's opinion, may impact this Shire-sponsored study.
• Subject has prior placement of a vagus nerve stimulator.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

SHP615

Experimental group

Description:

Participants will receive a single age-specific dose (approximately 0.25 to 0.5 milligram per kilogram \[mg/kg\] as midazolam) of SHP615 oromucosal solution through buccal route upon onset of seizures.

Treatment:

Drug: MHOS/SHP615

Drug: SHP615