Duchenne Muscular DystrophyPhase 4STEADFASTNCT01999075
Stacking Exercises Aid the Decline in FVC and Sick Time
A Phase 4 study testing Lung Volume Recruitment (LVR) and Conventional Treatment in people with Duchenne Muscular Dystrophy, run by Children's Hospital of Eastern Ontario at 9 sites in Canada.
Trial summary
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths, inflate the lungs and help clear the airways of secretions by increasing the forcefulness of a… Read more about this study
- Who is it for?
- Ages 6 to 16 years
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Lung Volume Recruitment (LVR) and Conventional Treatment
- Type of treatment
- Pill or oral medicine, Injection or infusion, Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 9 sitesCanada
- Study dates
- Mar 2013to 22 Nov 2018
- 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 6 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 6-16 years - This age range was selected as there are accepted normative pulmonary function data and children 6 years of age and older are generally able to reliably perform pulmonary function tests. Children are followed in participating centres until they reach 18 years of age (allowing two years of follow-up).
- Clinical phenotypic features consistent with DMD and confirmed by either: (1) Muscle biopsy showing complete dystrophin deficiency; (2) Genetic test positive for deletion or duplication in the dystrophin gene resulting in an 'out-of-frame' mutation; or (3) Dystrophin gene sequencing showing a mutation associated with DMD.
- FVC ≥ 30% predicted - This range of pulmonary function was selected to exclude those with severe restrictive respiratory impairment, who are less likely to be able to reliably perform pulmonary function testing over a two year period.
- A caregiver willing to provide the therapy
- Fluency in English or French
Who cannot join
- Unable to perform pulmonary function tests and/or LVR manoeuvre
- Presence of an endotracheal or tracheostomy tube
- Already using LVR and/or the Respironics in-exsufflator between and during respiratory infections
- Known susceptibility to pneumothorax or pneumomediastinum
- Uncontrolled asthma or other obstructive lung disease
- Symptomatic cardiomyopathy (ejection fraction less than 50% )
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
Canada9 sites
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Stollery Children's Hospital
Edmonton, Alberta, TGG 2J3
McMaster University
Hamilton, Ontario, L8S 4K1
London Health Sciences
London, Ontario, N6A 4G5
Hôpital Ste. Justine
Montreal, Quebec, H3T 1C5
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, M4G 1R8
SickKids Hospital
Toronto, Ontario, M5G 1X8
BC Children's Hospital
Vancouver, British Columbia, V6H 3V4
About this study
Official title: Stacking Exercises Attenuate the Decline in Forced Vital Capacity and Sick Time (STEADFAST)
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths, inflate the lungs and help clear the airways of secretions by increasing the forcefulness of a cough. We believe this will slow down the steady loss of lung function, prevent lung infection, and improve quality of life. Our aim is to compare the outcome of a group of individuals with DMD treated with standard care to another group that also receives lung volume recruitment. If effective, this study will change clinical practice by including twice-daily treatment as part of the standard of care for individuals with DMD, in order to improve their lung health and quality of life.
Read the detailed description
Background: Respiratory complications are the primary cause of morbidity and mortality associated with childhood Duchenne Muscular Dystrophy (DMD). Involvement of the respiratory muscles leads to progressive hypoventilation and/or recurrent atelectasis and pneumonia secondary to decreased cough efficacy. Lung volume recruitment (LVR) is a means of stacking breaths to achieve maximal lung inflation (MIC), prevent micro-atelectasis, and improve cough efficacy. Although it has been recommended by some experts as the "standard of care" for individuals with neuromuscular disease, the strategy has not been widely implemented in DMD given the lack of clinical trials to date to support its efficacy as well as the additional burden of care required in a population already requiring multiple interventions.
Primary Objective: To determine whether LVR, in addition to conventional treatment, is successful in reducing decline from baseline in forced vital capacity (FVC) over 2 years (percent predicted, measured according to American Thoracic Society standards), compared to conventional treatment alone in children with DMD.
Secondary Objectives: To determine differences between children treated with LVR in addition to conventional treatment, compared to those treated with conventional treatment alone, in: (1) the number of courses of antibiotics, hospitalizations and intensive care admissions for respiratory exacerbations, (2) health-related quality of life, and (3) peak cough flow and other pulmonary function tests.
Methods: We propose a 3-year multi-centre randomized controlled trial involving fifteen tertiary care pediatric hospitals across Canada. The study population consists of boys aged 6-16 years with DMD and FVC ≥ 30% of predicted. A sample size of 110 participants will be enrolled. This has been informed by chart review and survey of participating centres to be feasible, and will be re-assessed with an ongoing internal pilot study. Intervention: Participants will be allocated with a minimization procedure to receive conventional treatment (non-invasive ventilation, nutritional supplementation, physiotherapy and/or antibiotics, as decided by the treating physician) or conventional treatment plus twice daily LVR exercises performed with an inexpensive, portable self-inflating resuscitation bag containing a one-way valve and a mouthpiece. Data Analysis: The primary outcome (change in percent predicted FVC over 2 years) will be compared between the two study groups using an analysis of co-variance (ANCOVA) that takes into account baseline FVC and minimization factors.
Importance: Decline in pulmonary function among children with DMD negatively affects quality of life and predicts mortality. The relatively simple strategy of LVR has the potential to optimize pulmonary function and reduce respiratory exacerbations, thereby improving quality of life for individuals with DMD. This study is novel in that it is the first randomized controlled trial of LVR. A major strength is that the results will give support or refute recommendations regarding inclusion of LVR in the standard of care for individuals with DMD worldwide.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Lung Volume Recruitment (LVR), Conventional Treatment
- Keywords
- Duchenne Muscular Dystrophy, Pulmonary complications, Lung volume recruitment,, Breath-stacking,, Cough efficacy,, Maximal insufflation capacity
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lung Volume Recruitment (LVR)Device
LVR will be used twice per day
Conventional TreatmentOther
This may include: a. Physiotherapy, consisting of percussion, active cycle of breathing and/or postural drainage; b. Nutritional support, consisting of oral or tube-fed dietary supplements; c. Antibiotics (oral or intravenous), if there is evidence of respiratory infection; d. Non-invasive positive pressure ventilation, if there is evidence of nocturnal hypoventilation or sleep-disordered breathing; e. Systemic steroids
| Study group | Role | What they receive |
|---|---|---|
| Conventional Treatment | Placebo comparator | Conventional Treatment Receives: Other: Conventional Treatment |
| Lung Volume Recruitment | Active comparator | Conventional treatment plus the use of Lung Volume Recruitment (LVR) twice per day Receives: Device: Lung Volume Recruitment (LVR); Other: Conventional Treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in FVC (%-Predicted) From Baseline to 2 Years.
Change in FVC (%-predicted) was chosen as the primary outcome as it is a strong predictor of subsequent respiratory failure and mortality. Although survival is not a realistic endpoint for this trial, given expected mortality is less than 5% for the pediatric age group, FVC change is an appropriate clinical laboratory measure and valid surrogate endpoint to use for this trial.
Measured: 2 years
Secondary outcomes (7)
FVC Decline of 10% of Predicted
The time to reach an FVC decline of 10% of predicted will be used to calculate a hazard ratio.
Measured: 2 years
Number of Participants Prescribed Outpatient Oral Antibiotic Courses Between Baseline and 2 Years
Total number of participants who received any antibiotic courses between baseline and two years
Measured: 2 years
Health-related Quality of Life From Baseline to 2 Years
Measured biannually using the Pediatric Quality of Life Inventory (PedsQL 4.0). The PedsQL includes four subscales: Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. Each subscale is scored from 0 to 100, with higher scores indicating better health-related quality of life. A total score is computed by averaging all subscale scores.
Measured: 2 years
Change in Difference Between Assisted and Unassisted Peak Cough Flow (PCF) From Baseline to 2 Years
Change in the difference between assisted and unassisted peak cough flow (in liters per minute) from baseline to 2 years.
Measured: 2 years
Change in Maximal Insufflation Capacity (MIC)-Vital Capacity (VC) From Baseline to 2 Years
Change in maximal insufflation capacity (MIC)-vital capacity (VC) in liters from Baseline to 2 Years
Measured: 2 years
Change in Maximum Inspiratory Pressures (MIP), From Baseline to 2 Years
Change in maximum inspiratory pressures (MIP, in centimeters of water), From Baseline to 2 Years
Measured: 2 years
Change in Maximal Expiratory Pressures (MEP), From Baseline to 2 Years
Change in maximal expiratory pressures (MEP, in liters), From Baseline to 2 Years
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2013 (estimated)
- Study startMar 2013
- Primary completion22 Nov 2018
- Study completion22 Nov 2018
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.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- 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
- Single blind
- Who is masked
- Investigator
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Eastern Ontario
- Sponsor type
- University, hospital or other
- Collaborators
- Jesse's Journey
- Responsible party
- Principal investigator, Sherri Katz, Children's Hospital of Eastern Ontario
- Investigators
- Sherri Katz, MD, Principal investigator, Children's Hospital of Eastern OntarioIan MacLusky, MD, Study director, Children's Hospital of Eastern OntarioNicholas Barrowman, PhD, Study director, Children's Hospital of Eastern Ontario
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01999075
- Sponsor's protocol ID
- 12/26E
- Acronym
- STEADFAST
- Status last verified
- Nov 2024
- First submitted
- 14 Nov 2013
- Last updated
- 9 Jan 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Dussah N, McKim DA, Mah JK, McMillan HJ, Campbell C, Bijelic V, Barrowman N, Momoli F, Blinder H, Aaron S, McAdam LC, Nguyen TTD, Tarnopolsky M, Wensley D, Zielinski D, Wolfe L, Katz SL. Patterns of Adherence to Lung Volume Recruitment Therapy Amongst Individuals With Duchenne Muscular Dystrophy: A Secondary Analysis of the STEADFAST Randomized Controlled Trial. Pediatr Pulmonol. 2025 Oct;60(10):e71357. doi: 10.1002/ppul.71357.
PubMed 41147267Katz SL, Mah JK, McMillan HJ, Campbell C, Bijelic V, Barrowman N, Momoli F, Blinder H, Aaron SD, McAdam LC, Nguyen TTD, Tarnopolsky M, Wensley DF, Zielinski D, Rose L, Sheers N, Berlowitz DJ, Wolfe L, McKim D. Routine lung volume recruitment in boys with Duchenne muscular dystrophy: a randomised clinical trial. Thorax. 2022 Aug;77(8):805-811. doi: 10.1136/thoraxjnl-2021-218196. Epub 2022 Mar 2.
PubMed 35236763Morrow B, Argent A, Zampoli M, Human A, Corten L, Toussaint M. Cough augmentation techniques for people with chronic neuromuscular disorders. Cochrane Database Syst Rev. 2021 Apr 22;4(4):CD013170. doi: 10.1002/14651858.CD013170.pub2.
PubMed 33887060
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Stacking Exercises Attenuate the Decline in Forced Vital Capacity and Sick Time (STEADFAST)
- ClinicalTrials.gov ID
- NCT01999075
- Acronym / sponsor ID
- STEADFAST / 12/26E
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Pulmonary complications, Lung volume recruitment,, Breath-stacking,, Cough efficacy,, Maximal insufflation capacity
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Children's Hospital of Eastern OntarioUniversity, hospital or other
- Collaborators
- Jesse's Journey
- Study start
- Mar 2013
- Primary completion
- 22 Nov 2018 (actual)
- Study completion
- 22 Nov 2018 (actual)
- First posted
- 3 Dec 2013 (estimated)
- Results
- Posted 9 Jan 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Duchenne Muscular Dystrophy is complicated by weak breathing muscles and lung infections. "Lung volume recruitment" is a technique performed using a face mask or mouthpiece and a hand-held resuscitation bag to stack breaths, inflate the lungs and help clear the airways of secretions by increasing the forcefulness of a cough. We believe this will slow down the steady loss of lung function, prevent lung infection, and improve quality of life. Our aim is to compare the outcome of a group of individuals with DMD treated with standard care to another group that also receives lung volume recruitment. If effective, this study will change clinical practice by including twice-daily treatment as part of the standard of care for individuals with DMD, in order to improve their lung health and quality of life.
Detailed description
Background: Respiratory complications are the primary cause of morbidity and mortality associated with childhood Duchenne Muscular Dystrophy (DMD). Involvement of the respiratory muscles leads to progressive hypoventilation and/or recurrent atelectasis and pneumonia secondary to decreased cough efficacy. Lung volume recruitment (LVR) is a means of stacking breaths to achieve maximal lung inflation (MIC), prevent micro-atelectasis, and improve cough efficacy. Although it has been recommended by some experts as the "standard of care" for individuals with neuromuscular disease, the strategy has not been widely implemented in DMD given the lack of clinical trials to date to support its efficacy as well as the additional burden of care required in a population already requiring multiple interventions.
Primary Objective: To determine whether LVR, in addition to conventional treatment, is successful in reducing decline from baseline in forced vital capacity (FVC) over 2 years (percent predicted, measured according to American Thoracic Society standards), compared to conventional treatment alone in children with DMD.
Secondary Objectives: To determine differences between children treated with LVR in addition to conventional treatment, compared to those treated with conventional treatment alone, in: (1) the number of courses of antibiotics, hospitalizations and intensive care admissions for respiratory exacerbations, (2) health-related quality of life, and (3) peak cough flow and other pulmonary function tests.
Methods: We propose a 3-year multi-centre randomized controlled trial involving fifteen tertiary care pediatric hospitals across Canada. The study population consists of boys aged 6-16 years with DMD and FVC ≥ 30% of predicted. A sample size of 110 participants will be enrolled. This has been informed by chart review and survey of participating centres to be feasible, and will be re-assessed with an ongoing internal pilot study. Intervention: Participants will be allocated with a minimization procedure to receive conventional treatment (non-invasive ventilation, nutritional supplementation, physiotherapy and/or antibiotics, as decided by the treating physician) or conventional treatment plus twice daily LVR exercises performed with an inexpensive, portable self-inflating resuscitation bag containing a one-way valve and a mouthpiece. Data Analysis: The primary outcome (change in percent predicted FVC over 2 years) will be compared between the two study groups using an analysis of co-variance (ANCOVA) that takes into account baseline FVC and minimization factors.
Importance: Decline in pulmonary function among children with DMD negatively affects quality of life and predicts mortality. The relatively simple strategy of LVR has the potential to optimize pulmonary function and reduce respiratory exacerbations, thereby improving quality of life for individuals with DMD. This study is novel in that it is the first randomized controlled trial of LVR. A major strength is that the results will give support or refute recommendations regarding inclusion of LVR in the standard of care for individuals with DMD worldwide.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Conventional Treatment | Placebo comparator | Other: Conventional TreatmentConventional Treatment |
| Lung Volume Recruitment | Active comparator | Device: Lung Volume Recruitment (LVR), Other: Conventional TreatmentConventional treatment plus the use of Lung Volume Recruitment (LVR) twice per day |
Who may qualify
- Ages
- 6 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Age 6-16 years - This age range was selected as there are accepted normative pulmonary function data and children 6 years of age and older are generally able to reliably perform pulmonary function tests. Children are followed in participating centres until they reach 18 years of age (allowing two years of follow-up).
- Clinical phenotypic features consistent with DMD and confirmed by either: (1) Muscle biopsy showing complete dystrophin deficiency; (2) Genetic test positive for deletion or duplication in the dystrophin gene resulting in an 'out-of-frame' mutation; or (3) Dystrophin gene sequencing showing a mutation associated with DMD.
- FVC ≥ 30% predicted - This range of pulmonary function was selected to exclude those with severe restrictive respiratory impairment, who are less likely to be able to reliably perform pulmonary function testing over a two year period.
- A caregiver willing to provide the therapy
- Fluency in English or French
Key exclusion criteria (6 listed)
- Unable to perform pulmonary function tests and/or LVR manoeuvre
- Presence of an endotracheal or tracheostomy tube
- Already using LVR and/or the Respironics in-exsufflator between and during respiratory infections
- Known susceptibility to pneumothorax or pneumomediastinum
- Uncontrolled asthma or other obstructive lung disease
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 |
|---|---|---|
| Lung Volume Recruitment (LVR)LVR will be used twice per day | Device | Lung Volume Recruitment |
| Conventional TreatmentThis may include: a. Physiotherapy, consisting of percussion, active cycle of breathing and/or postural drainage; b. Nutritional support, consisting of oral or tube-fed dietary supplements; c. Antibiotics (oral or intravenous), if there is evidence of respiratory infection; d. Non-invasive positive pressure ventilation, if there is evidence of nocturnal hypoventilation or sleep-disordered breathing; e. Systemic steroids | Other | Conventional Treatment, Lung Volume Recruitment |
Primary outcome
Change in FVC (%-Predicted) From Baseline to 2 Years.
Time frame: 2 years
Change in FVC (%-predicted) was chosen as the primary outcome as it is a strong predictor of subsequent respiratory failure and mortality. Although survival is not a realistic endpoint for this trial, given expected mortality is less than 5% for the pediatric age group, FVC change is an appropriate clinical laboratory measure and valid surrogate endpoint to use for this trial.
7 secondary outcomes
FVC Decline of 10% of Predicted
Time frame: 2 years
Number of Participants Prescribed Outpatient Oral Antibiotic Courses Between Baseline and 2 Years
Time frame: 2 years
Health-related Quality of Life From Baseline to 2 Years
Time frame: 2 years
Change in Difference Between Assisted and Unassisted Peak Cough Flow (PCF) From Baseline to 2 Years
Time frame: 2 years
Change in Maximal Insufflation Capacity (MIC)-Vital Capacity (VC) From Baseline to 2 Years
Time frame: 2 years
Change in Maximum Inspiratory Pressures (MIP), From Baseline to 2 Years
Time frame: 2 years
Change in Maximal Expiratory Pressures (MEP), From Baseline to 2 Years
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jan 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41147267Dussah N, McKim DA, Mah JK, McMillan HJ, Campbell C, Bijelic V, Barrowman N, Momoli F, Blinder H, Aaron S, McAdam LC, Nguyen TTD, Tarnopolsky M, Wensley D, Zielinski D, Wolfe L, Katz SL. Patterns of Adherence to Lung Volume Recruitment Therapy Amongst Individuals With Duchenne Muscular Dystrophy: A Secondary Analysis of the STEADFAST Randomized Controlled Trial. Pediatr Pulmonol. 2025 Oct;60(10):e71357. doi: 10.1002/ppul.71357.
- Derived from this study: PubMed 35236763Katz SL, Mah JK, McMillan HJ, Campbell C, Bijelic V, Barrowman N, Momoli F, Blinder H, Aaron SD, McAdam LC, Nguyen TTD, Tarnopolsky M, Wensley DF, Zielinski D, Rose L, Sheers N, Berlowitz DJ, Wolfe L, McKim D. Routine lung volume recruitment in boys with Duchenne muscular dystrophy: a randomised clinical trial. Thorax. 2022 Aug;77(8):805-811. doi: 10.1136/thoraxjnl-2021-218196. Epub 2022 Mar 2.
- Derived from this study: PubMed 33887060Morrow B, Argent A, Zampoli M, Human A, Corten L, Toussaint M. Cough augmentation techniques for people with chronic neuromuscular disorders. Cochrane Database Syst Rev. 2021 Apr 22;4(4):CD013170. doi: 10.1002/14651858.CD013170.pub2.
- Individual participant dataSharing planned: No
Related trials for Duchenne Muscular Dystrophy
RecruitingObservational study
Biomarker Development for Muscular Dystrophies
Myotonic Dystrophy, Duchenne Muscular Dystrophy
Active, not recruitingObservational study
Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Recruiting
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Active, not recruitingPhase 1
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
RecruitingObservational study
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD), Chronic Pain
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.
Sherri Katz, MD principal investigator
Children's Hospital of Eastern Ontario
Ian MacLusky, MD study director
Children's Hospital of Eastern Ontario
Nicholas Barrowman, PhD study director
Children's Hospital of Eastern Ontario
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Lung Volume Recruitment (LVR) and Conventional Treatment in people with Duchenne Muscular Dystrophy, run by Children's Hospital of Eastern Ontario at 9 sites in Canada.
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 5 inclusion and 6 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: finished, as of the sponsor's last update on 9 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Canada. 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 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 9 Jan 2025). 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 FVC (%-Predicted) From Baseline to 2 Years. (time frame: 2 years). 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 NCT01999075, which also lists 3 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 9 Jan 2025.