Cystic FibrosisPhase 4NCT00680316

A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis

A Phase 4 study testing Dornase alfa in people with Cystic Fibrosis, run by Genentech, Inc..

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 3 to 5 years
Conditions the study is for: Cystic Fibrosis
What is being tested?: Dornase alfa
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 3 participants
Where: No sites listed yet
Study dates: Jun 2008to May 2009
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 3 to 5 years
All sexes
No healthy volunteers
Child

Who may be able to join

Signed Informed Consent Form
Aged 3-5 years
Diagnosis of cystic fibrosis

Who cannot join

Children taking scheduled inhaled Pulmozyme or hypertonic saline within 56 days prior to Visit 1 or any Pulmozyme in the 28 days before Visit 1
Involvement in a clinical intervention trial within the 4 weeks prior to Visit 1
Use of an investigational drug or device within 28 days prior to Visit 1
Any other condition that might increase the risk of participation to the patient in the judgement of the investigator

About this study

Official title: A Phase IV, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme® in 3- to 5-Year-Old Patients With Cystic Fibrosis

This was a Phase IV, multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate the effect of Pulmozyme on pulmonary function, health-related quality of life (HRQOL), and respiratory symptoms in 3- to 5-year-old children with cystic fibrosis (CF). Approximately 40 patients were planned to be enrolled in this study. However, only 3 patients were eligible for random allocation and received treatment: 1 patient in the Pulmozyme group and 2 patients in the placebo group. All 3 patients completed the study assessments but did not have usable pulmonary function test (PFT) data.

Conditions: Cystic Fibrosis
Being tested: Dornase alfa, Placebo
Keywords: Pulmozyme, CF
Medical subject headings: Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Dornase alfaDrug
Also called Pulmozyme
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
PlaceboDrug
2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days

Study groups in NCT00680316
Study group	Role	What they receive
Dornase alfa	Experimental	Not described Receives: Drug: Dornase alfa
Placebo	Placebo comparator	Not described Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment).
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.
Measured: from Visit 2 to Visit 3 (16 +/- 2 days)

Secondary outcomes (3)

Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.
Measured: from Visit 2 to Visit 3 (16 +/- 2 days)
Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10)
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Resistance is complex measure that incorporates the lack of changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (10Hz was used for the secondary endpoint).
Measured: from Visit 2 to Visit 3 (16 +/- 2 days)
Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers
The CFQ-R for Preschoolers and the CFQ-R for Parents of Preschoolers was designed specifically to measure the impact of CF for patients with a diagnosis of CF. Each question is answered using a 4-point Likert scale.
In order to calculate the domain/symptom scale scores, the following algorithm is followed
- Re-number items which have been reverse coded
- Calculate the mean of the items to be included. If more than half of the items are missing, then the score is considered missing
- Re-scale to result in a scaled score which ranges from 0 to 100, with higher scores indicating better health
Measured: from Visit 2 to Visit 3 (16 +/- 2 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jun 2008May 2009

First posted20 May 2008 (estimated)
Study startJun 2008
Primary completionMay 2009
Study completionMay 2009

Why it stopped: Most patients were unable to perform the test being used to measure lung function, thus it became clear the study would not yield clinically meaningful data

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Cystic FibrosisStudies of the same condition, open and under way.
More trials run by Genentech, Inc.The 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 4
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 3 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00680316
Sponsor's protocol ID: Z4240g
Status last verified: Nov 2016
First submitted: 16 May 2008
Last updated: 12 Jan 2017
Results posted: Yes, on ClinicalTrials.gov

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

RecruitingPhase 2
A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis
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All Cystic Fibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 12 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase IV, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme® in 3- to 5-Year-Old Patients With Cystic Fibrosis
ClinicalTrials.gov ID: NCT00680316
Acronym / sponsor ID: Z4240g
Conditions: Cystic Fibrosis
Keywords: Pulmozyme, CF
Phase: Phase 4
Study type: Interventional
Recruitment status: TerminatedReason stopped: Most patients were unable to perform the test being used to measure lung function, thus it became clear the study would not yield clinically meaningful data
Enrolment: 3 participants (actual)
Lead sponsor: Genentech, Inc.Industry (pharma, biotech, devices)
Study start: Jun 2008
Primary completion: May 2009 (actual)
Study completion: May 2009 (actual)
First posted: 20 May 2008 (estimated)
Results: Posted 17 Jun 2011

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
Dornase alfa	Experimental	Drug: Dornase alfa
Placebo	Placebo comparator	Drug: Placebo

Who may qualify

Ages: 3 to 5 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (3 listed)

Signed Informed Consent Form
Aged 3-5 years
Diagnosis of cystic fibrosis

Key exclusion criteria (4 listed)

Children taking scheduled inhaled Pulmozyme or hypertonic saline within 56 days prior to Visit 1 or any Pulmozyme in the 28 days before Visit 1
Involvement in a clinical intervention trial within the 4 weeks prior to Visit 1
Use of an investigational drug or device within 28 days prior to Visit 1
Any other condition that might increase the risk of participation to the patient in the judgement of the investigator

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
Dornase alfaAlso: Pulmozyme2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days	Drug	Dornase alfa
Placebo2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days	Drug	Placebo

Primary outcome

Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment).
Time frame: from Visit 2 to Visit 3 (16 +/- 2 days)
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.

3 secondary outcomes

Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)
Time frame: from Visit 2 to Visit 3 (16 +/- 2 days)
Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10)
Time frame: from Visit 2 to Visit 3 (16 +/- 2 days)
Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers
Time frame: from Visit 2 to Visit 3 (16 +/- 2 days)

Results and safety

Results posted to the registry on 17 Jun 2011, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Dornase Alfa	1	1
Placebo	2	2

Primary outcome: Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment).

Time frame: from Visit 2 to Visit 3 (16 +/- 2 days)

Population analysed: Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.

3 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Dornase Alfa	0 of 1 (0%)	1 of 1 (100%)	Not reported
Placebo	0 of 2 (0%)	2 of 2 (100%)	Not reported

Limitations and caveats (from the sponsor): Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Cystic Fibrosis

RecruitingPhase 2
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Active, not recruitingObservational study
CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
Cystic Fibrosis, Bone Loss
RecruitingObservational study
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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.

Michelle Freemer, M.D. study director

Genentech, Inc.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Dornase alfa in people with Cystic Fibrosis, run by Genentech, Inc..

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 3 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 12 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.

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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 17 Jun 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment). (time frame: from Visit 2 to Visit 3 (16 +/- 2 days)). 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 NCT00680316.

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 12 Jan 2017.