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

11. února 2016 aktualizováno: 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.

Přehled studie

Postavení

Dokončeno

Podmínky

Intervence / Léčba

Detailní popis

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.

Typ studie

Intervenční

Zápis (Aktuální)

28

Fáze

  • Fáze 3

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní místa

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

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

4 roky až 11 let (Dítě)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Všechno

Popis

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.

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: Randomizované
  • Intervenční model: Crossover Assignment
  • Maskování: Čtyřnásobek

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: Arm T
Experimental arm utilizing Epinephrine HFA-MDI (E004)
Single dose 125 mcg/inhalation, 2 inhalations
Komparátor placeba: Arm P
Placebo comparator arm utilizing Placebo-HFA
Single dose 0 mcg/inhalation, 2 inhalations

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Bronchodilator effect
Časové okno: 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

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
AUC analysis
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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)
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: up to 30 min predose
Concomitant medications will be reviewed and recorded
up to 30 min predose
Adverse Events
Časové okno: predose and up to 3 hours postdose
All Adverse Events/side effects will be recorded and assessed
predose and up to 3 hours postdose

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Vyšetřovatelé

  • Ředitel studie: Selina Su, MPH, Amphastar Pharmaceuticals, Inc.

Publikace a užitečné odkazy

Osoba odpovědná za zadávání informací o studiu tyto publikace poskytuje dobrovolně. Mohou se týkat čehokoli, co souvisí se studiem.

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia

1. října 2012

Primární dokončení (Aktuální)

1. března 2013

Dokončení studie (Aktuální)

1. prosince 2013

Termíny zápisu do studia

První předloženo

20. listopadu 2012

První předloženo, které splnilo kritéria kontroly kvality

28. listopadu 2012

První zveřejněno (Odhad)

30. listopadu 2012

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Odhad)

11. března 2016

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

11. února 2016

Naposledy ověřeno

1. února 2016

Více informací

Termíny související s touto studií

Tyto informace byly beze změn načteny přímo z webu clinicaltrials.gov. Máte-li jakékoli požadavky na změnu, odstranění nebo aktualizaci podrobností studie, kontaktujte prosím register@clinicaltrials.gov. Jakmile bude změna implementována na clinicaltrials.gov, bude automaticky aktualizována i na našem webu .