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- Klinische proef NCT01543191
A Safety and Tolerability Study OF PUR118 In Subjects With Cystic Fibrosis
A Phase 1 Dose-escalation Study TO Evaluate the Safety and Tolerability of PUR118 and Placebo in Subjects With CF
Studie Overzicht
Studietype
Inschrijving (Werkelijk)
Fase
- Fase 1
Contacten en locaties
Studie Locaties
-
-
Northern Ireland
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Belfast, Northern Ireland, Verenigd Koninkrijk, BT9 6AD
- Celerion
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Beschrijving
Inclusion Criteria:
- Males or non-pregnant, non-lactating healthy female.
- Age 18 to 55 years of age (inclusive) on day of signing informed consent.
- Subject has a BMI of 17 or greater.
- Subject has a confirmed diagnosis of cystic fibrosis documented by a compatible clinical or radiographic presentation, and definitive accepted lab and clinical criteria (e.g., sweat chloride > 60 mEq/L or 2 disease causing CFTR mutations) OR is regularly attending a Consultant led clinic where the diagnosis of Cystic Fibrosis is not in doubt.
- Subject has an FEV1 ≥ 45% of predicted at screening.
- Subject has an oxygen saturation >92% on room air as determined by pulse oximetry at screening.
- Subject is a non-smoker or ex-smoker has not smoked for at least six months prior to screening.
- Subject is otherwise medically stable without clinically significant abnormal screening results that depart from their usual baseline level of health as a subject with CF.
- Must be willing and able to communicate in English and participate in the whole study.
- Must provide written informed consent.
Exclusion Criteria:
- Subject has unstable lung disease, as defined by a change in medical regimen during the preceding 2-weeks, or a significant new finding on chest radiograph (pneumothorax, lobar/segmental collapse), or in the opinion of the Investigator, has had a decline in pulmonary status within the last year not considered a part of the usual, chronic progression of CF lung disease and part of their baseline health condition as a subject with CF.
- Subject has had an exacerbation of respiratory symptoms within the past 30 days that required initiation of a new or altered respiratory therapy.
- Subject had either an upper or lower respiratory illness within the 30 days prior to dosing days, or has symptoms from such an illness that have not resolved.
- Subject has a history of lung transplantation.
- Females of child bearing potential who are pregnant, or lactating. Females who are sexually active and either not surgically sterile or not willing to use an acceptable form of contraception
- Clinically significant abnormal biochemistry, haematology or urinalysis not consistent with CF as judged by the Investigator
- Failure to satisfy the Investigator of fitness to participate for any other reason.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: Gerandomiseerd
- Interventioneel model: Crossover-opdracht
- Masker: Verdrievoudigen
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
|---|---|
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Placebo-vergelijker: Placebo
|
low dose and high dose for 3 doses, BID
|
|
Experimenteel: PUR118
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low dose and high dose for 3 doses, BID
|
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Safety and Tolerability
Tijdsspanne: 4-6 weeks
|
Changes in clinical signs and symptoms from physical examination and chest examination, changes in clinical safety laboratory tests, vital signs, spirometry, lung clearance index, 24-hour sputum weight and volume, oxygen saturation, ECG, telemetry, and adverse events.
|
4-6 weeks
|
Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
|
Pharmacodynamics
Tijdsspanne: 36 hours
|
To assess impact of PUR118 on mobilization and expectorated volume of airway mucus secretions. To evaluate impact of PUR118 on measurements of lung function, including ventilation inhomogeneity, by both spirometry and measurement of the lung clearance index (LCI). To evaluate an impact of PUR118 on sputum, serum and plasma biomarkers related to airway inflammation. |
36 hours
|
Medewerkers en onderzoekers
Sponsor
Medewerkers
Onderzoekers
- Hoofdonderzoeker: Stephen Smith, MD, Celerion
Studie record data
Bestudeer belangrijke data
Studie start
Primaire voltooiing (Werkelijk)
Studie voltooiing (Werkelijk)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Schatting)
Updates van studierecords
Laatste update geplaatst (Schatting)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- 601-0009
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
product vervaardigd in en geëxporteerd uit de V.S.
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