- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving 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
Studieoversikt
Studietype
Registrering (Faktiske)
Fase
- Fase 1
Kontakter og plasseringer
Studiesteder
-
-
Northern Ireland
-
Belfast, Northern Ireland, Storbritannia, BT9 6AD
- Celerion
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
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.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Crossover-oppdrag
- Masking: Trippel
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Placebo komparator: Placebo
|
low dose and high dose for 3 doses, BID
|
|
Eksperimentell: PUR118
|
low dose and high dose for 3 doses, BID
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Safety and Tolerability
Tidsramme: 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
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Pharmacodynamics
Tidsramme: 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
|
Samarbeidspartnere og etterforskere
Sponsor
Samarbeidspartnere
Etterforskere
- Hovedetterforsker: Stephen Smith, MD, Celerion
Studierekorddatoer
Studer hoveddatoer
Studiestart
Primær fullføring (Faktiske)
Studiet fullført (Faktiske)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Anslag)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Anslag)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- 601-0009
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