Cystic FibrosisPhase 2NCT01002534
Does a Nasal Instillation of Vardenafil Normalize the Nasal Potential Difference in Cystic Fibrosis Patients?
A Phase 2 study testing Vardenafil in people with Cystic Fibrosis, run by Cliniques universitaires Saint-Luc- Université Catholique de Louvain at 1 site in Belgium.
Trial summary
The purpose of this study is to investigate the effect of a nasal instillation of Vardenafil on nasal potential difference in cystic fibrosis patients homozygous for the F508del mutation Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Vardenafil
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 1 siteBelgium
- Study dates
- Oct 2011to Mar 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Cystic fibrosis patients homozygous for the F508del mutation as confirmed by a genetic test
- Aged 14 years and older
- Male and female
- FEV1 >50% of predicted normal
Who cannot join
- Acute respiratory tract infection or pulmonary exacerbation requiring antibiotic intervention within 2 weeks of visit 1
- Any condition prohibiting the correct measurement of the NPD
- Active or passive smoking
- Planned treatment or treatment with another investigational drug or therapy within 1 month prior to randomisation
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Does a Nasal Instillation of Vardenafil Normalize the Nasal Potential Difference in Cystic Fibrosis Patients Homozygous for the F508del Mutation? A Randomized, Double Blind, Placebo-controlled Study.
The purpose of this study is to investigate the effect of a nasal instillation of Vardenafil on nasal potential difference in cystic fibrosis patients homozygous for the F508del mutation
Read the detailed description
CFTR protein has been shown to be an ohmic, small conductance channel regulated by cAMP intracellular levels that are balanced by degradation through cyclic nucleotide phosphodiesterases (PDE). Several families of PDEs with varying selectivities for cAMP and/or cGMP have been identified.PDE5 is highly specific for cGMP and is involved in the regulation of the intracellular concentration of cGMP in various tissues. Recently, it has been shown, in a preclinical model of transgenic mice, that pharmacological doses of sildenafil and vardenafil, two clinically approved PDE5 inhibitors, stimulate chloride transport activity of the mutant F508del-protein (Lubamba et al, 2008); this parameter has been assessed by means of the nasal potential difference (NPD). An increasing effect of sildenafil on the expression of F508del-CFTR protein (Dormer et al,2005) was originally reported in nasal epithelial cells harvested from patients with cystic fibrosis and cultured on impermeable supports, a configuration that allows interaction of drugs with the apical side of epithelia.
This study aims at investigating the effect of a single local administration of vardenafil on NPD measurements in CF patients homozygous for the F508del mutation.
- Conditions
- Cystic Fibrosis
- Being tested
- Vardenafil, Placebo
- Keywords
- Cystic Fibrosis, Vardenafil, Nasal instillation, Nasal Potential difference
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VardenafilDrug
Nasal instillation of Vardenafil
PlaceboDrug
Nasal instillation of placebo matching in appearance with the Vardenafil instillation
| Study group | Role | What they receive |
|---|---|---|
| baseline | No intervention | visit 1 |
| Vardenafil | Active comparator | nasal instillation of Vardenafil ( visit 2 or 3) Receives: Drug: Vardenafil |
| Placebo | Placebo comparator | Nasal instillation of placebo (visit 3 or 2) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulated changes in response to Chloride-free solution and isoproterenol (reflecting chloride transport)
Measured: Change from baseline (visit 1) and placebo to Vardenafil instillation
Secondary outcomes (1)
Change in basal voltage value and in amiloride response (reflecting sodium transport)
Measured: Change from baseline (visit1) and placebo to Vardenafil instillation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2009 (estimated)
- Study startOct 2011
- Primary completionJun 2012
- Study completionMar 2018
Why it stopped: not yet started
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Cliniques universitaires Saint-Luc- Université Catholique de LouvainThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Patrick LEBECQUE, MD, PhD, Principal investigator, Cliniques universitaires St.Luc (Université catholique de Louvain)Teresinha LEAL, MD, PhD, Principal investigator, Cliniques universitaires St.Luc ( Université Catholique de Louvain)Anissa LEONARD, MD, Principal investigator, Cliniques universitaires St.Luc (Université Catholique de Louvain)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01002534
- Sponsor's protocol ID
- VARD-99
- Status last verified
- Feb 2019
- First submitted
- 26 Oct 2009
- Last updated
- 25 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
Dormer RL, Harris CM, Clark Z, Pereira MM, Doull IJ, Norez C, Becq F, McPherson MA. Sildenafil (Viagra) corrects DeltaF508-CFTR location in nasal epithelial cells from patients with cystic fibrosis. Thorax. 2005 Jan;60(1):55-9. doi: 10.1136/thx.2003.019778.
PubMed 15618584Lubamba B, Lecourt H, Lebacq J, Lebecque P, De Jonge H, Wallemacq P, Leal T. Preclinical evidence that sildenafil and vardenafil activate chloride transport in cystic fibrosis. Am J Respir Crit Care Med. 2008 Mar 1;177(5):506-15. doi: 10.1164/rccm.200703-344OC. Epub 2007 Nov 15.
PubMed 18006891
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Does a Nasal Instillation of Vardenafil Normalize the Nasal Potential Difference in Cystic Fibrosis Patients Homozygous for the F508del Mutation? A Randomized, Double Blind, Placebo-controlled Study.
- ClinicalTrials.gov ID
- NCT01002534
- Acronym / sponsor ID
- VARD-99
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Vardenafil, Nasal instillation, Nasal Potential difference
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: not yet started
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de LouvainUniversity, hospital or other
- Study start
- Oct 2011
- Primary completion
- Jun 2012 (actual)
- Study completion
- Mar 2018 (actual)
- First posted
- 27 Oct 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to investigate the effect of a nasal instillation of Vardenafil on nasal potential difference in cystic fibrosis patients homozygous for the F508del mutation
Detailed description
CFTR protein has been shown to be an ohmic, small conductance channel regulated by cAMP intracellular levels that are balanced by degradation through cyclic nucleotide phosphodiesterases (PDE). Several families of PDEs with varying selectivities for cAMP and/or cGMP have been identified.PDE5 is highly specific for cGMP and is involved in the regulation of the intracellular concentration of cGMP in various tissues. Recently, it has been shown, in a preclinical model of transgenic mice, that pharmacological doses of sildenafil and vardenafil, two clinically approved PDE5 inhibitors, stimulate chloride transport activity of the mutant F508del-protein (Lubamba et al, 2008); this parameter has been assessed by means of the nasal potential difference (NPD). An increasing effect of sildenafil on the expression of F508del-CFTR protein (Dormer et al,2005) was originally reported in nasal epithelial cells harvested from patients with cystic fibrosis and cultured on impermeable supports, a configuration that allows interaction of drugs with the apical side of epithelia.
This study aims at investigating the effect of a single local administration of vardenafil on NPD measurements in CF patients homozygous for the F508del mutation.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| baseline | No intervention | –visit 1 |
| Vardenafil | Active comparator | Drug: Vardenafilnasal instillation of Vardenafil ( visit 2 or 3) |
| Placebo | Placebo comparator | Drug: PlaceboNasal instillation of placebo (visit 3 or 2) |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Cystic fibrosis patients homozygous for the F508del mutation as confirmed by a genetic test
- Aged 14 years and older
- Male and female
- FEV1 >50% of predicted normal
Key exclusion criteria (4 listed)
- Acute respiratory tract infection or pulmonary exacerbation requiring antibiotic intervention within 2 weeks of visit 1
- Any condition prohibiting the correct measurement of the NPD
- Active or passive smoking
- Planned treatment or treatment with another investigational drug or therapy within 1 month prior to randomisation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VardenafilNasal instillation of Vardenafil | Drug | Vardenafil |
| PlaceboNasal instillation of placebo matching in appearance with the Vardenafil instillation | Drug | Placebo |
Primary outcome
Cumulated changes in response to Chloride-free solution and isoproterenol (reflecting chloride transport)
Time frame: Change from baseline (visit 1) and placebo to Vardenafil instillation
1 secondary outcome
Change in basal voltage value and in amiloride response (reflecting sodium transport)
Time frame: Change from baseline (visit1) and placebo to Vardenafil instillation
Results and safety
The study was stopped early (reason given: not yet started). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2019.
- Results publication: PubMed 15618584Dormer RL, Harris CM, Clark Z, Pereira MM, Doull IJ, Norez C, Becq F, McPherson MA. Sildenafil (Viagra) corrects DeltaF508-CFTR location in nasal epithelial cells from patients with cystic fibrosis. Thorax. 2005 Jan;60(1):55-9. doi: 10.1136/thx.2003.019778.
- Results publication: PubMed 18006891Lubamba B, Lecourt H, Lebacq J, Lebecque P, De Jonge H, Wallemacq P, Leal T. Preclinical evidence that sildenafil and vardenafil activate chloride transport in cystic fibrosis. Am J Respir Crit Care Med. 2008 Mar 1;177(5):506-15. doi: 10.1164/rccm.200703-344OC. Epub 2007 Nov 15.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Patrick LEBECQUE, MD, PhD principal investigator
Cliniques universitaires St.Luc (Université catholique de Louvain)
Teresinha LEAL, MD, PhD principal investigator
Cliniques universitaires St.Luc ( Université Catholique de Louvain)
Anissa LEONARD, MD principal investigator
Cliniques universitaires St.Luc (Université Catholique de Louvain)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vardenafil in people with Cystic Fibrosis, run by Cliniques universitaires Saint-Luc- Université Catholique de Louvain at 1 site in Belgium.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Belgium. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: not yet started). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulated changes in response to Chloride-free solution and isoproterenol (reflecting chloride transport) (time frame: Change from baseline (visit 1) and placebo to Vardenafil instillation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01002534, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2019.