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Evaluation of Efficacy and Safety for Single Dose of E004 in Children With Asthma

11. februar 2016 oppdatert av: Amphastar Pharmaceuticals, Inc.

Evaluation of Efficacy and Safety for Single Dose of E004 in Children With Asthma (A Randomized, Double-Blind, Placebo-Controlled, Crossover, Single Dose Study in 4 - 11 Year Old Children With Asthma)

This is a multi-center, randomized, double-blinded, placebo-controlled, crossover, single dose study in 24 pediatric patients (4-11 years old) with asthma.

The entire study consists of (i) a Screening Visit and (ii) a Study Period with two (2) Study Visits. All study subjects must be properly consented, under adult supervision, and screened against the inclusion and exclusion criteria, at the Screening Visit.

Studieoversikt

Status

Fullført

Forhold

Detaljert beskrivelse

This is a randomized, double-blinded, placebo-controlled, crossover, single dose study to be conducted in pediatric patients (4 - 11 years) with asthma.

The main features of the study design are:

The entire study consists of a Screening Visit, and a Study Period consisting of two (2) crossover Study Visits separated by a 2 to 14-day interval. All study subjects must be properly consented, under adult supervision, and screened against the inclusion and exclusion criteria at the Screening Visit and confirmed for enrollment on Visit 1. Efficacy and safety evaluations of E004 are conducted at each Study Visit.

This study employs two (2) double-blinded treatment arms as outlined in Table 2. A double-blinded design will be applied to E004 (Arm T) and Placebo-HFA (Arm P) since they are identical in all physical attributes and share a comparable formulation.

The enrolled subjects will be randomized into two sequences (as follows) to participate in two (2) crossover Study Visits with a 2 - 14 day interval between visits. Randomization is achieved using a ratio of 1:1.

Use E004 (T) and Placebo (P) in Visits 1 and 2, respectively or use Placebo (P) and E004 (T) in Visits 1 and 2, respectively

Subjects will be trained at the screening visit and each Study Visit for the correct dosing and spirometry methods. Under the supervision of dosing monitor, subjects will self-administer two (2) inhalations of the randomized study treatment, with a ~1 min interval at each Study Visit.

For the Screening and Study Visits, the subjects will be required to be at the site for a 30 minute "resting period". This resting period is designed to maintain a stable and consistent physical status of the subjects prior to the start of the baseline FEV1 procedures. For the Screening Visit, this period will begin upon subject arrival. For the Study Visits, the period will begin at the end of the option breakfast (or upon arrival if the breakfast is declined).

For each Study Visit, subjects will need to arrive at the study site early enough to complete all necessary baseline evaluations. The study site will provide an optional breakfast but it must be eaten at least 30 minutes prior to the pre-dose baseline FEV1 measurements. The optional breakfast will be light, and contain no added sugar. If the subjects decline the breakfast (i.e. they have already eaten a light breakfast prior to arriving), they are required to remain at the site for at least 30 minutes prior to the start of the Baseline FEV1 measurements, in order to maintain a stable physical status.

Baseline vital signs and safety evaluations will be taken prior to the pre-dose baseline FEV1 measurement. These can be performed during the 30 minute "resting period". Efficacy of the treatments at each visit will be evaluated based on spirometric measurements of serial FEV1 determined at the Pre-dose Baseline, and the seven (7) serial Post-dose FEV1 responses at 5, 30, 60, 120, 150, 180) and 240 minutes.

This study will be conducted with a double-blinded technique. This means neither the subject nor the site staff will be aware of the identity of the treatment arm since both study treatments are in identical looking containers.

Studietype

Intervensjonell

Registrering (Faktiske)

28

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

    • California
      • Cypress, California, Forente stater, 90630
        • West Coast Clinical Trials Global
    • Oregon
      • Medford, Oregon, Forente stater, 97504
        • The Clinical Research Institute of Southern Oregn, PC
      • Portland, Oregon, Forente stater, 97202
        • Transitional Clinical Research, Inc. Allergy Associates Research Center
    • Texas
      • El Paso, Texas, Forente stater, 79903
        • Western Sky Medical
      • San Antonio, Texas, Forente stater, 78229
        • Sylvana Research Assocaites
    • Washington
      • Seattle, Washington, Forente stater, 98115
        • ASTHMA, Inc. Clinical Research Center

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

4 år til 11 år (Barn)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  • Generally healthy male, and premenarchal female, children ages 4 - 11 years upon Screening.
  • With documented asthma, requiring inhaled epinephrine or β2-agonist treatment, with or without concurrent anti-inflammatory therapies including orally inhaled corticosteroids, for at least 6-months prior to Screening.
  • Being capable of performing spirometry for FEV1 measurements.
  • Satisfying criteria of asthma stability, defined as no significant changes in asthma therapy (with the exception of switching LABA to SABA, adjustment of ICor SABA, etc, per investigator discretion) and no asthma-related hospitalization or emergency room visits, within 4 weeks prior to Screening.
  • Can tolerate withholding treatment with inhaled bronchodilators and other allowed medications for the minimum washout periods indicated in Appendix II prior to the Screening Baseline FEV1 testing.
  • Demonstrating a Mean Screening Baseline FEV1 (MSBF) that is 50.0% - 90% of Polgar predicted normal value.
  • Demonstrating an Airway Reversibility, i.e., FEV1 values ≥12% increase based upon volume compared with MSBF, within 30 min after 2 inhalations (440 mcg, epinephrine base) of previously marketed Epinephrine CFC-MDI, labeled "For Investigational Use Only". There will be up to 5 reversibility time points, each with up to 5 maneuvers that can be conducted anytime within 30 min post-dose.
  • Demonstrating satisfactory techniques in the use of a metered-dose inhaler (MDI).
  • Has been properly consented to participate in this study.

Exclusion Criteria:

  • Any current or past medical conditions that, per investigator discretion, might significantly affect pharmacodynamic responses to the study drugs, such as significant systemic or respiratory diseases (e.g., cystic fibrosis, bronchiectasis, active tuberculosis, emphysema, nonreversible pulmonary diseases), other than asthma.
  • Concurrent clinically significant cardiovascular, hematological, renal, neurologic, hepatic, endocrine, psychiatric, or malignant diseases.
  • Known intolerance or hypersensitivity to any component of the study drugs (i.e., Epinephrine, HFA-134a, CFC-12, CFC-114, polysorbate-80, thymol, ethanol, ascorbic acid, nitric acid, and hydrochloric acid), as well as the rescue Albuterol HFA inhalers (i.e., Albuterol, HFA-134a, ethanol, and oleic acid).
  • Recent infection of the upper respiratory tract (within 2 weeks), or lower respiratory tract (within 4 weeks), before screening.
  • Use of prohibited medications per Appendix II.
  • Having been on other investigational drug/device studies in the last 30 days prior to screening.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Crossover-oppdrag
  • Masking: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Arm T
Experimental arm utilizing Epinephrine HFA-MDI (E004)
Single dose 125 mcg/inhalation, 2 inhalations
Placebo komparator: Arm P
Placebo comparator arm utilizing Placebo-HFA
Single dose 0 mcg/inhalation, 2 inhalations

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Bronchodilator effect
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
Bronchodilator effect expressed as AUC of FEV1's relative change from the same day baseline (pre-dose) versus time up to 3 hours, defined as AUC(0-3) of change in FEV1%. The difference of primary endpoints of E004 and Placebo will be evaluated statistically.
up to 30 min pre-dose, postdose up to 3 hours

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
AUC analysis
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
The comparative analysis of AUC of FEV1 versus time
up to 30 min pre-dose, postdose up to 3 hours
Evaluation of FEV1 volume changes
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
Evaluation of AUC(0-3) and AUC(0-4) of FEV1 volume changes (AUC of ΔFEV1)
up to 30 min pre-dose, postdose up to 3 hours
Change in FEV1
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
Evaluation of Maximum of change in FEV1% (Fmax)
up to 30 min pre-dose, postdose up to 3 hours
Time response
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
Evaluation of time response curves of change in FEV1 and FEV1%
up to 30 min pre-dose, postdose up to 3 hours
Time to onset of bronchodilator effect
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
Determination of time to onset of bronchodilator effect (Tonset), determined by the time point (within 60 minutes) where FEV1 first reaches greater than or equal to 12% above Same-Day Pre-dose Baseline
up to 30 min pre-dose, postdose up to 3 hours
Time to peak FEV1 effect
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
The time to peak FEV1 effect (Tmax), defined as the time of Fmax
up to 30 min pre-dose, postdose up to 3 hours
Duration of efficacy
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
Evaluation of duration of efficacy (Tduration), defined as the total length of time when the change in FEV1% reaches and stays greater than or equal to 12% above Same-Day Pre-dose Baseline
up to 30 min pre-dose, postdose up to 3 hours
Percentage of positive responders
Tidsramme: up to 30 min pre-dose, postdose up to 3 hours
Evaluation of percentage of positive responders (R%), including all subjects whose Fmax reaches more than or equal to 12% above Same-Day Pre-dose Baseline
up to 30 min pre-dose, postdose up to 3 hours
Vital Signs
Tidsramme: Screening Visit: baseline up to 30 min predose and 30 min post dose; Visits 1-2: Baseline upt o 30 min predose and 3, 20, 60, and 240 min post-dose
Vital signs (SBP/DBP, and heart rate) will be monitored at the Screening Visit and at Study Visits 1 and 2
Screening Visit: baseline up to 30 min predose and 30 min post dose; Visits 1-2: Baseline upt o 30 min predose and 3, 20, 60, and 240 min post-dose
12-lead ECG (Routine and QT/QTc)
Tidsramme: Screening Visit: baseline up to 30 min predose and 30 min post dose; Visits 1-2: Baseline upt o 30 min predose and 3, 20, and 60 min post-dose
A 12-lead ECG (Routine and QT/QTc) will be recorded at Screening Visit and at the Study Visits 1 and 2
Screening Visit: baseline up to 30 min predose and 30 min post dose; Visits 1-2: Baseline upt o 30 min predose and 3, 20, and 60 min post-dose
Lab Tests
Tidsramme: prior to first dose
Lab tests for CBC, blood chemistry panel (8-hr fasted), and urinalysis will be performed at screening
prior to first dose
Albuterol HFA Usage
Tidsramme: up to 30 min predose
Albuterol HFA usage for rescue relief of acute asthma symptoms will be recorded at each visit
up to 30 min predose
Concomitant Medications
Tidsramme: up to 30 min predose
Concomitant medications will be reviewed and recorded
up to 30 min predose
Adverse Events
Tidsramme: predose and up to 3 hours postdose
All Adverse Events/side effects will be recorded and assessed
predose and up to 3 hours postdose

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Etterforskere

  • Studieleder: Selina Su, MPH, Amphastar Pharmaceuticals, Inc.

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart

1. oktober 2012

Primær fullføring (Faktiske)

1. mars 2013

Studiet fullført (Faktiske)

1. desember 2013

Datoer for studieregistrering

Først innsendt

20. november 2012

Først innsendt som oppfylte QC-kriteriene

28. november 2012

Først lagt ut (Anslag)

30. november 2012

Oppdateringer av studieposter

Sist oppdatering lagt ut (Anslag)

11. mars 2016

Siste oppdatering sendt inn som oppfylte QC-kriteriene

11. februar 2016

Sist bekreftet

1. februar 2016

Mer informasjon

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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