NCT00376428

Brief Summary

Suppression of stop mutations in the CFTR gene with parenteral gentamicin can be predicted in vitro and is associated with clinical benefit and significant modification of the CFTR-mediated chloride transport in nasal and sweat gland epithelium.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2003

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2003

Completed
2.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2005

Completed
1.3 years until next milestone

First Submitted

Initial submission to the registry

September 13, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 14, 2006

Completed
Last Updated

February 25, 2015

Status Verified

September 1, 2006

First QC Date

September 13, 2006

Last Update Submit

February 24, 2015

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis Transmembrane Conductance RegulatorPremature termination codonGentamicin-induced readthrough

Outcome Measures

Primary Outcomes (1)

  • CFTR-dependant chlorate secretion

Secondary Outcomes (2)

  • CFTR expression in nasal cells

  • Clinical beneficial effects

Interventions

GentamicinDRUG

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • cystic fibrosis with CFTR codon stop mutations

You may not qualify if:

  • Rhinitis
  • nasal polyposis
  • passive or active smoking
  • modification of basal treatments within the previous month
  • treatments with aminoglycosides within three previous months

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Necker-Enfants malades

Paris, 75015, France

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Gentamicins

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

AminoglycosidesGlycosidesCarbohydrates

Study Officials

  • Aleksander Edelman, PhD

    Institut National de la Santé Et de la Recherche Médicale, France

    STUDY DIRECTOR

  • Isabelle Sermet, MD; PhD

    Assistance Publique - Hôpitaux de Paris

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

September 13, 2006

First Posted

September 14, 2006

Study Start

January 1, 2003

Study Completion

June 1, 2005

Last Updated

February 25, 2015

Record last verified: 2006-09

Locations

FR(1)