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Open-label Study of Midazolam Hydrochloride Oromucosal Solution (MHOS/SHP615) in Children With Status Epilepticus (Convulsive) in a Healthcare Setting in Japan

15 juli 2020 bijgewerkt door: Shire

A Phase 3, Multicenter, Open-label Study to Determine the Efficacy, Safety, and Pharmacokinetics of Buccally Administered MHOS/SHP615 in Pediatric Patients With Status Epilepticus (Convulsive) in the Hospital or Emergency Room

The purpose of this study is to assess the efficacy, safety and pharmacokinetics of MHOS/SHP615 administered buccally in children with status epilepticus (convulsive) in a healthcare setting.

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

25

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • Japan
      • Fukuoka, Japan, 813-0017
        • Fukuoka Children's Hospital(NW)
      • Hokkaidō, Japan, 063-0005
        • NHO Hokkaido Medical Center
      • Kumamoto, Japan, 861-1196
        • Kumamoto Saishunso National Hospital
      • Nagasaki, Japan, 856-8562
        • NHO Nagasaki Medical Center
      • Niigata, Japan, 950-2085
        • NHO Nishi Niigata Chuo National Hospital
      • Obu, Japan, 474-8710
        • Aichi Children's Health and Medical Center(NW)
      • Okayama, Japan, 700-0914
        • Okayama University Hospital
      • Osaka, Japan, 535-0022
        • Nakano Children's Hospital
      • Osaka, Japan, 594-1101
        • Osaka Women's and Children's Hospital(NW)
      • Saitama, Japan, 330-8777
        • Saitama Children's Medical Center(NW)
      • Suita, Japan, 565-0871
        • Osaka University Hospital
      • Tokyo, Japan, 187-0031
        • National Center Hospital, NCNP
      • Tottori, Japan, 683-8504
        • Tottori University Hospital
      • Yamadaoka, Japan, 565-0871
        • Osaka University Hospital
      • Yokohama, Japan, 232-0066
        • Kanagawa Children's Medical Center(NW)
    • Fujimi
      • Kofu, Fujimi, Japan, 400-8506
        • Yamanashi Prefectural Central Hospital
    • Gifu Prefecture
      • Gifu, Gifu Prefecture, Japan, 500-8717
        • Gifu Prefectural General Medical Center
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 060-8648
        • Hokkaido University Hospital
    • Kawadacho
      • Tokyo, Kawadacho, Japan, 162-8666
        • Tokyo Women's Medical University Hospital
    • Okayama Prefecture
      • Okayama, Okayama Prefecture, Japan, 701-0304
        • NHO Minami-Okayama Medical Center
    • Owada Shinden
      • Yachiyo, Owada Shinden, Japan, 276-8524
        • Tokyo Women's Medical University Yachiyo Medical Center
    • Saitama-ken
      • Saitama-shi, Saitama-ken, Japan, 330-8503
        • Jichi Children's Medical Center Tochigi
    • Shizuoka Prefecture
      • Shizuoka, Shizuoka Prefecture, Japan, 420-8688
        • Shizuoka Institute of Epilepsy and Neurological Disorders

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

3 maanden tot 18 jaar (Kind, Volwassen)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria:

  • Male and female participants whose corrected gestational age is greater than or equal to (>=) 52 weeks (gestational weeks plus the number of weeks after birth) and less than (<) 18 years (and weight greater than [>] 5 kilogram [kg]), at the time of investigational product administration. If the participant's exact age is not known, the participant should be excluded.
  • Parent, guardian, or legally authorized representative (LAR) of the child provides informed consent (and assent, when applicable per Shire policy and country regulations) to participate in the study prior to participation in any protocol specific procedures. The participant may be prescreened by the investigator in their clinical practice and the parent, guardian, or LAR may sign informed consent before the participant presents to the healthcare setting for treatment of the seizure.

  • Participant with 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) lasting >=5 mins

Exclusion Criteria:

  • Female participants 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 seizures due to illegal drug or acute alcoholic intoxication.
  • Subjects with known or suspected recurrent seizures due to illegal drug or alcohol withdrawal.
  • Subjects with history of seizures of psychogenic origin.
  • Subjects with seizures due to severe encephalitis or meningitis, as determined by the PI
  • Subjects with known history of hypersensitivities, non-responsiveness or contraindications to benzodiazepines (ie, clinically significant respiratory depression, severe acute hepatic failure, myasthenia gravis, syndrome of sleep apnea, glaucoma with closed angle, 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, in the judgment of the healthcare provider, 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 with significant hypotension and cardiac dysrhythmia (example [eg], atrioventricular [AV] block of second or third degree, VT [ventricular tachycardia]).
  • Subjects who have been receiving human immunodeficiency virus (HIV) protease inhibitors or HIV reverse transcriptase inhibitors.
  • Subjects with current hypoglycemia (glucose <60 milligram per deciliter [mg/dL]) upon presentation at the hospital or healthcare setting.
  • Subjects with severe cerebral anoxia (except cerebral palsy), in the judgment of the healthcare provider.
  • Subjects 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.
  • Subjects has received antiseizure medication prior to arrival in the healthcare setting.
  • Subjects has prior placement of a vagus nerve stimulator.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Opdracht voor een enkele groep
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: SHP615
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.
Geneesmiddel: SHP615
SHP615-oplossing voor oromucosaal gebruik wordt toegediend als een enkele leeftijdsspecifieke dosis (2,5, 5, 7,5 en 10 mg).
Andere namen:
  • MHOS
  • Midazolam hydrochloride oplossing voor oromucosaal gebruik

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Percentage of Participants With Response Rate
Tijdsspanne: From start of study drug administration up to 30 minutes post-dose
Response rate was defined as the percentage of participants with therapeutic success. Therapeutic success was defined as the cessation of visible seizure activity within 10 minutes with a sustained absence of visible seizure activity for 30 minutes following a single dose of MHOS/SHP615 without the need for additional rescue medication.
From start of study drug administration up to 30 minutes post-dose

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Percentage of Participants Who Had Sustained Absence of Seizure Activity for at Least 1, 4 and 6 Hours
Tijdsspanne: From start of study drug administration up to 1, 4 and 6 hours post-dose
Percentage of participants whose seizure event stopped within 10 minutes of single dose administration of SHP615 and who had sustained absence of seizure activity for at least 1, 4, and 6 hours were reported.
From start of study drug administration up to 1, 4 and 6 hours post-dose
Number of Participants With Time to Resolution of Seizures (Convulsions)
Tijdsspanne: From start of study drug administration up to follow-up (Day 8)
Time to resolution of seizures (convulsions) was calculated as time from IP administration to the end of the initial seizure or administration of rescue anti-convulsant medication, whichever occurs first. Initial seizure referred to the seizure that triggered the use of the IP. Number of participants with time to resolution of seizures (convulsions) from the administration of SHP615 were reported.
From start of study drug administration up to follow-up (Day 8)
Number of Participants With Time to Recovery of Consciousness
Tijdsspanne: From start of study drug administration up to follow-up (Day 8)
Time to recovery of consciousness (in minutes) was calculated only for participants who lost consciousness pre-dose at time from investigational product administration to recovery of consciousness post-dose or administration of rescue anticonvulsant medication, whichever occurs first. Number of participants with time to recovery of consciousness were reported.
From start of study drug administration up to follow-up (Day 8)
Percentage of Participants Who Required Additional Anticonvulsant Medication for Ongoing Status Epilepticus (SE)
Tijdsspanne: 10 minutes post-dose
Percentage of participants who required additional anticonvulsant medication for ongoing SE, 10 minutes after a single dose of SHP615 were reported.
10 minutes post-dose
Percentage of Participants Who Failed to Respond to the Treatment With SHP615
Tijdsspanne: 10 minutes post-dose
Treatment failure/non-responder was defined as participants with continuing seizure activity and/or the need for any additional rescue medication according to the participating healthcare setting protocol or guideline, for 10 mins or more after a single dose of the IP.
10 minutes post-dose
Concentration of SHP615 in Plasma at 10 Minutes (C10)
Tijdsspanne: 10 minutes post-dose
Concentration of SHP615 in plasma at 10 minutes were reported.
10 minutes post-dose
Maximum Plasma Concentration (Cmax) of SHP615
Tijdsspanne: 1, 3, 6 hours post-dose
Cmax of SHP615 in plasma were reported.
1, 3, 6 hours post-dose
Area Under the Concentration-time Curve From Time Zero to 10 Minutes (AUC0-10) of SHP615 in Plasma
Tijdsspanne: Pre-dose, 10 minutes post-dose
AUC0-10 of SHP615 in plasma were reported. Here "min ng/mL" was minutes nanogram per milliliter.
Pre-dose, 10 minutes post-dose
Area Under the Concentration-time Curve From Time Zero to 60 Minutes (AUC0-60) of SHP615 in Plasma
Tijdsspanne: Pre-dose, 60 minutes post-dose
AUC0-60 of SHP615 in plasma were reported.
Pre-dose, 60 minutes post-dose
Area Under the Concentration-time Curve From Time Zero to 180 Minutes (AUC0-180) of SHP615 in Plasma
Tijdsspanne: Pre-dose, 180 minutes post-dose
AUC0-180 of SHP615 in plasma were reported.
Pre-dose, 180 minutes post-dose
Area Under the Concentration-time Curve From Time Zero to Infinity (AUC0-inf) of SHP615 in Plasma
Tijdsspanne: Pre-dose, 1, 3, and 6 hours post-dose
AUC(0-infinity) of SHP615 in plasma were reported.
Pre-dose, 1, 3, and 6 hours post-dose
Time at Maximum Concentration (Tmax) of SHP615 in Plasma
Tijdsspanne: 1, 3, and 6 hours post-dose
Tmax of SHP615 in plasma were reported.
1, 3, and 6 hours post-dose
Elimination Half-life (T1/2) of SHP615 in Plasma
Tijdsspanne: 1, 3, and 6 hours post-dose
T1/2 of SHP615 in plasma were reported.
1, 3, and 6 hours post-dose
Number of Participants With Respiratory Depression
Tijdsspanne: From start of study drug administration up to follow-up (Day 8)
Respiratory depression, included the following measures within 24 hours after administration of the IP: i) Persistent decrease in oxygen saturation to < 92 percent (%) measured at 10, 30 minutes, and 4, 6, and 24 hours post-dose (i.e, < 92 % on room air for 2 minutes or more after dosing while monitoring [per healthcare setting protocol and/or the clinical judgment of the physician]) ii) Increase in respiratory effort such that assisted ventilation is used (bag-valve-mask ventilation or endotracheal intubation). Number of participants with respiratory depression were reported.
From start of study drug administration up to follow-up (Day 8)
Number of Participants With Aspiration Pneumonia Reported as Treatment Emergent Adverse Events (TEAEs)
Tijdsspanne: From start of study drug administration up to follow-up (Day 8)
TEAEs was defined as AEs that start or deteriorate on or after the date of the first dose of investigational product and no later than 3 days following the last dose of IP. Number of participants with aspiration pneumonia identified as TEAEs were reported.
From start of study drug administration up to follow-up (Day 8)
Change From Baseline in Riker Sedation-Agitation Scale at 24 Hours Post-dose
Tijdsspanne: Baseline, 24 hours post-dose
Sedation-Agitation was assessed, using the "Riker Sedation-Agitation Scale" (SAS) by the following 7-point scale: 7. dangerous agitation; 6. very agitated; 5. agitated; 4. calm, cooperative; 3. sedated; 2. very sedated; 1. unarousable. Change from baseline in riker sedation-agition scale at 24 hours post-dose were reported.
Baseline, 24 hours post-dose
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Tijdsspanne: From start of study drug administration up to follow-up (Day 8)
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. TEAEs was defined as AEs that start or deteriorate on or after the date of the first dose of investigational product and no later than 3 days following the last dose of IP. Number of participants with TEAEs were reported.
From start of study drug administration up to follow-up (Day 8)
Change From Baseline in Oxygen Saturation Percentage at 24 Hours Post-dose
Tijdsspanne: Baseline, 24 hours post-dose
Oxygen saturation at baseline was measured and recorded on room air. The investigator had recorded the oxygen saturation, oxygen delivery system and amount of oxygen administered during the study. Change from baseline in oxygen saturation percentage at 24 hours post-dose were reported.
Baseline, 24 hours post-dose

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Shire

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

23 oktober 2017

Primaire voltooiing (Werkelijk)

19 augustus 2019

Studie voltooiing (Werkelijk)

19 augustus 2019

Studieregistratiedata

Eerst ingediend

6 november 2017

Eerst ingediend dat voldeed aan de QC-criteria

6 november 2017

Eerst geplaatst (Werkelijk)

8 november 2017

Updates van studierecords

Laatste update geplaatst (Werkelijk)

31 juli 2020

Laatste update ingediend die voldeed aan QC-criteria

15 juli 2020

Laatst geverifieerd

1 juni 2020

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • SHP615-301

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

JA

Beschrijving IPD-plan

Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.

IPD-toegangscriteria voor delen

IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

IPD delen Ondersteunend informatietype

  • LEERPROTOCOOL
  • SAP
  • ICF
  • MVO

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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Klinische onderzoeken op SHP615

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