Spinal Muscular AtrophyPhase 2NCT02644668
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
A Phase 2 study testing Reldesemtiv 150 mg and Reldesemtiv 450 mg in people with Spinal Muscular Atrophy, run by Cytokinetics at 19 sites in 2 countries.
Trial summary
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal muscular atrophy (SMA). Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- Reldesemtiv 150 mg and Reldesemtiv 450 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 19 sitesin 2 countries
- Study dates
- 14 Jan 2016to 31 May 2018
- Phase
- Phase 2
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Able to comprehend and willing to sign an Informed Consent Form (ICF) for patients 18 years of age and older. For patients less than 18 years of age, parent(s)/legal guardian(s) of patients must provide written informed consent prior to participation in the study and informed assent will be obtained from minors at least 12 years of age when required by regulation.
- Males or females with genetically confirmed diagnosis of SMA who are Type II, III or IV and at least 12 years of age
- Ambulatory patients, once having achieved a standing position independently, must be able to complete at least one lap in the 6-minute walk test (at least 50 meters) within 6 minutes without assistance.
- Non-ambulatory patients (defined as individuals who are effectively requiring a wheelchair for all mobility needs; they may be able to stand or walk short distances, but unable to walk 50 meters without assistance in 6 minutes). Non-ambulatory patients must be able to tolerate an upright sitting position, with support, continuously for 3 hours
- Hammersmith (HFMS-E) score ≥ 10 and ≤ 54
- Contracture of the elbow flexion and knee flexion ≤ 90 degrees
Show all 20Show fewer
- Pre-study clinical laboratory findings within the normal range or, if outside the normal range, deemed not clinically significant by the Investigator
- Able to swallow an oral suspension and in the opinion of the Investigator, is expected to continue to be able to do so for the duration of the trial. Administration via a feeding tube is not allowed.
- Forced vital capacity (FVC) > 20% predicted
- Male patients who have reached puberty must agree to do either of the following from Screening until 10 weeks after the last dose of the investigational product unless they have had a vasectomy and confirmed sperm count is zero:
- Abstain from sexual intercourse, OR
- If having heterosexual intercourse, must use a condom and their female partners who are of childbearing potential must use a highly effective contraception method*
- Female patients who have had their first period will be considered of childbearing potential unless they are anatomically and physiologically incapable of becoming pregnant. If of childbearing potential, the female patients must:
- Have a negative urine/serum pregnancy test at Screening AND
- Abstain from heterosexual intercourse from Screening until 10 weeks after the last dose of investigational product OR
- If having heterosexual intercourse, must use a highly effective contraception method* and require the male partners to use a condom from Screening until 10 weeks after the last dose of investigational product
- Highly effective contraception methods include:
- Established use of oral, injected or implanted hormonal methods of contraception
- Placement of an intrauterine device (IUD) or intrauterine system (IUS)
- Male patients must agree to refrain from sperm donation from Screening until 10 weeks after the final study drug administration
Who cannot join
- History of significant hypersensitivity, intolerance, or allergy to any drug compound, food, or other substance, unless approved by the Investigator
- Hospitalization within 2 months of Screening
- History of stomach or intestinal surgery or resection that would potentially alter absorption and/or excretion of orally administered drugs (appendectomy, hernia repair, and/or cholecystectomy will be allowed)
- A clinically significant illness within 4 weeks of Screening
- History of alcoholism or drug addiction within 2 years prior to Screening
- History of smoking more than 10 cigarettes (or equivalent amount of tobacco) per day within 3 months prior to Screening
Show all 12Show fewer
- Patient has used a strong CYP3A4 inhibitor within 7 days prior to first dose of study drug or a strong CYP3A4 inducer within 14 days prior to first dose of study drug
- Any other medical condition that would interfere with performance of testing including (but not limited to) significant joint pain or arthritis limiting mobility, and chronic neuromuscular pain sufficient to require ongoing analgesic medication
- Participation by two people at the same time that are living in the same household
- Participation in any other investigational study drug trial in which receipt of an investigational study drug occurred within 30 days or five half-lives of the other investigational study drug, whichever is greater, prior to Screening
- An ALT or AST greater than 2-fold the upper limit of normal (ULN) or has total bilirubin greater than the ULN at screening. These assessments may be repeated once at the investigator's discretion (within the screening window)
- Currently taking nusinersen, or has taken it in the past, or plans to take it during the course the study
Trial locations and contacts
19 sites in 2 countries.
19 sites in 2 countries
United States15 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins Hospital Institute for Clinical and Translational Research Pediatric Clinical Research Unit
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
Duke University Medical Center
Durham, North Carolina, 27705
University of Kansas Medical Center
Kansas City, Kansas, 66160
UCLA
Los Angeles, California, 90095
Hospital for Special Care
New Britain, Connecticut, 06053
University of California Irvine
Orange, California, 92868
Nemours Childrens Hospital
Orlando, Florida, 32827
Pediatric Neuromuscular Clinic Stanford University
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
The University of Utah, Clinical Neurosciences Center
Salt Lake City, Utah, 84132
Washington University School of Medicine
St Louis, Missouri, 63110
Canada4 sites
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Children's Hospital - LHSC
London, Ontario, N6A 4G5
Montreal Neurological Institute and Hospital
Montreal, Quebec, H3A 2B4
Children's and Women's Health Centre of British Columbia
Vancouver, British Columbia, V6H 3V4
About this study
Official title: A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular Atrophy
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal muscular atrophy (SMA).
Read the detailed description
CY 5021 is a Phase 2, double-blind, randomized, placebo-controlled, multiple dose study of reldesemtiv in 2 sequential ascending dose cohorts of patients with SMA. Patients will be randomized 2:1 to receive reldesemtiv or placebo twice daily for 8 weeks. Patients randomized to reldesemtiv in Cohort 1 will receive a dose of 150 mg twice daily and patients randomized to reldesemtiv in Cohort 2 will receive 450 mg twice daily. Within each cohort, randomization will be stratified by ambulatory status (ambulatory versus non ambulatory).
The primary objective of the study is to determine the PD effects of reldesemtiv on measures of pulmonary function, respiratory function, muscle strength, and motor function. Other PD measures include changes in the timed up and go (TUG) test, a 6-minute walk test (6MWT), and patient and investigator global assessments. Secondary objectives include the safety of multiple doses of reldesemtiv and an evaluation of the pharmacokinetics of reldesemtiv.
- Conditions
- Spinal Muscular Atrophy
- Being tested
- Placebo, Reldesemtiv 150 mg, Reldesemtiv 450 mg
- Keywords
- Reldesemtiv
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboDrug
Granules for oral suspension (placebo)
Reldesemtiv 150 mgDrug
Also called CK-2127107
Granules for oral suspension, 18.7% reldesemtiv
Reldesemtiv 450 mgDrug
Also called CK-2127107
Granules for oral suspension, 56.0% reldesemtiv
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Patients randomized to this treatment arm will receive a placebo suspension twice daily for 8 weeks. Receives: Drug: Placebo |
| Reldesemtiv 150 mg twice daily | Experimental | Patient randomized to this treatment arm will receive reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks. Receives: Drug: Reldesemtiv 150 mg |
| Reldesemtiv 450 mg twice daily | Experimental | Patients randomized to this treatment arm will receive reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks. Receives: Drug: Reldesemtiv 450 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Week 8 in Forced Vital Capacity (FVC)
FVC was measured using a calibrated spirometer (in units of liters). Patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs).
Measured: baseline and 8 weeks
Change From Baseline to Week 8 in Maximum Inspiratory Pressure (MIP)
MIP was measured (in units of cm H20) using a calibrated spirometer with an inspiratory pressure valve attached. For the test, patients were asked to inhale as forcefully as possible, to their maximum pressure.
Measured: baseline and 8 weeks
Change From Baseline to Week 8 in Maximum Expiratory Pressure (MEP)
MEP was measured (in units of cm H20) using a calibrated spirometer with an exspiratory pressure valve attached. For the test, patients were asked to maximally inhale then perform a forced exhalation with as forcefully as possible.
Measured: baseline and 8 weeks
Muscle Strength Mega-Score at Week 8
Muscle strength of 3 muscle groups (elbow flexion, knee extension, and shoulder abduction) were measured bilaterally using a hand-held dynamometer. Muscle strength was measured twice for each body location; if the variability between the 2 measures was > 15%, a third measure was obtained.
The maximum muscle strength of the 2 measurements was identified and transformed as a percent change from baseline using the equation: ([postbaseline value - baseline value] / baseline value) × 100.
The mega-score was a composite score that averaged strength across the 3 muscle groups. It was calculated as the mean of the non-missing transformed muscle strength scores among the 3 muscle groups each measure bilaterally (totaling 6 body locations).
Measured: baseline and 8 weeks
Change From Baseline to Week 8 in the Hammersmith Functional Motor Scale-Expanded (HFMS-E)
The HFMS-E evaluated the level of independent mobility and motor skills through assessment of 33 test-items, each scored from 0 (worse) to 2 (better). The total score was calculated as the sum of the scores among the 33 test items, and has a range from 0 to 66.
Measured: baseline and 8 weeks
Change From Baseline to Week 8 in Revised Upper Limb Module (RULM)
The RULM assessed motor function in the upper limbs (specifically shoulder, elbow, wrist, and hand function) that related to activities of everyday life. The RULM consisted of 20 items, 1 of which was scored on a 7-point scale (from 0 to 6), 18 were scored on a 3-point scale (from 0 to 2), and 1 was scored on a 2-point scale (0 or 1). The total score was the sum of each response and could range from a minimum of 0 to a maximum of 43 points. Higher scores reflected better motor function.
Measured: baseline and 8 weeks
Change From Baseline to Week 8 in the TUG Test
The TUG test measured the time (in seconds) it took for a patient to rise from a chair, walk 3 meters, turn around, walk back to the chair and sit down.
Measured: baseline and 8 weeks
Change From Baseline to Week 8 in the 6MWT
The 6MWT measured the distance (in meters) a patient walked in 6 minutes.
Measured: baseline and 8 weeks
Patient Global Assessment at the End of Week 8
Patients assessed whether they felt the same, better, or worse than prior to dosing on Day 1.
Measured: 8 weeks
Investigator Global Assessment at the End of Week 8
The Investigator assessed whether patient appeared the same, better, or worse than prior to dosing on Day 1.
Measured: 8 weeks
Secondary outcomes (2)
Reldesemtiv Maximum Observed Plasma Concentration (Cmax)
Determined by evaluation of reldesemtiv plasma concentrations from blood samples collected prior to dosing and at 1, 3, and 6 hours following dosing
Measured: End of Week 8
Reldesemtiv Area Under the Plasma Concentration-time Curve From 0 to 12 Hours (AUC0-12)
Determined by evaluation of reldesemtiv plasma concentrations from blood samples collected prior to dosing and at 1, 3, and 6 hours following dosing
Measured: End of Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jan 2016 (estimated)
- Study start14 Jan 2016
- Primary completion31 May 2018
- Study completion31 May 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.
- Other trials for Spinal Muscular AtrophyStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cytokinetics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Astellas Pharma Global Development, Inc.
- Responsible party
- Sponsor
- Investigators
- MD, Cytokinetics, Study director, Cytokinetics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02644668
- Sponsor's protocol ID
- CY 5021
- Status last verified
- Aug 2020
- First submitted
- 23 Dec 2015
- Last updated
- 31 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT02644668
- Acronym / sponsor ID
- CY 5021
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Reldesemtiv
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- CytokineticsIndustry (pharma, biotech, devices)
- Collaborators
- Astellas Pharma Global Development, Inc.
- Study start
- 14 Jan 2016 (actual)
- Primary completion
- 31 May 2018 (actual)
- Study completion
- 31 May 2018 (actual)
- First posted
- 1 Jan 2016 (estimated)
- Results
- Posted 31 Aug 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal muscular atrophy (SMA).
Detailed description
CY 5021 is a Phase 2, double-blind, randomized, placebo-controlled, multiple dose study of reldesemtiv in 2 sequential ascending dose cohorts of patients with SMA. Patients will be randomized 2:1 to receive reldesemtiv or placebo twice daily for 8 weeks. Patients randomized to reldesemtiv in Cohort 1 will receive a dose of 150 mg twice daily and patients randomized to reldesemtiv in Cohort 2 will receive 450 mg twice daily. Within each cohort, randomization will be stratified by ambulatory status (ambulatory versus non ambulatory).
The primary objective of the study is to determine the PD effects of reldesemtiv on measures of pulmonary function, respiratory function, muscle strength, and motor function. Other PD measures include changes in the timed up and go (TUG) test, a 6-minute walk test (6MWT), and patient and investigator global assessments. Secondary objectives include the safety of multiple doses of reldesemtiv and an evaluation of the pharmacokinetics of reldesemtiv.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPatients randomized to this treatment arm will receive a placebo suspension twice daily for 8 weeks. |
| Reldesemtiv 150 mg twice daily | Experimental | Drug: Reldesemtiv 150 mgPatient randomized to this treatment arm will receive reldesemtiv suspension at a dose of 150 mg, twice daily for 8 weeks. |
| Reldesemtiv 450 mg twice daily | Experimental | Drug: Reldesemtiv 450 mgPatients randomized to this treatment arm will receive reldesemtiv suspension at a dose of 450 mg, twice daily for 8 weeks. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (20 listed)
- Able to comprehend and willing to sign an Informed Consent Form (ICF) for patients 18 years of age and older. For patients less than 18 years of age, parent(s)/legal guardian(s) of patients must provide written informed consent prior to participation in the study and informed assent will be obtained from minors at least 12 years of age when required by regulation.
- Males or females with genetically confirmed diagnosis of SMA who are Type II, III or IV and at least 12 years of age
- Ambulatory patients, once having achieved a standing position independently, must be able to complete at least one lap in the 6-minute walk test (at least 50 meters) within 6 minutes without assistance.
- Non-ambulatory patients (defined as individuals who are effectively requiring a wheelchair for all mobility needs; they may be able to stand or walk short distances, but unable to walk 50 meters without assistance in 6 minutes). Non-ambulatory patients must be able to tolerate an upright sitting position, with support, continuously for 3 hours
- Hammersmith (HFMS-E) score ≥ 10 and ≤ 54
Key exclusion criteria (12 listed)
- History of significant hypersensitivity, intolerance, or allergy to any drug compound, food, or other substance, unless approved by the Investigator
- Hospitalization within 2 months of Screening
- History of stomach or intestinal surgery or resection that would potentially alter absorption and/or excretion of orally administered drugs (appendectomy, hernia repair, and/or cholecystectomy will be allowed)
- A clinically significant illness within 4 weeks of Screening
- History of alcoholism or drug addiction within 2 years prior to Screening
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 |
|---|---|---|
| PlaceboGranules for oral suspension (placebo) | Drug | Placebo |
| Reldesemtiv 150 mgAlso: CK-2127107Granules for oral suspension, 18.7% reldesemtiv | Drug | Reldesemtiv 150 mg twice daily |
| Reldesemtiv 450 mgAlso: CK-2127107Granules for oral suspension, 56.0% reldesemtiv | Drug | Reldesemtiv 450 mg twice daily |
Primary outcome
Change From Baseline to Week 8 in Forced Vital Capacity (FVC)
Time frame: baseline and 8 weeks
FVC was measured using a calibrated spirometer (in units of liters). Patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs).
Change From Baseline to Week 8 in Maximum Inspiratory Pressure (MIP)
Time frame: baseline and 8 weeks
MIP was measured (in units of cm H20) using a calibrated spirometer with an inspiratory pressure valve attached. For the test, patients were asked to inhale as forcefully as possible, to their maximum pressure.
Change From Baseline to Week 8 in Maximum Expiratory Pressure (MEP)
Time frame: baseline and 8 weeks
MEP was measured (in units of cm H20) using a calibrated spirometer with an exspiratory pressure valve attached. For the test, patients were asked to maximally inhale then perform a forced exhalation with as forcefully as possible.
Muscle Strength Mega-Score at Week 8
Time frame: baseline and 8 weeks
Muscle strength of 3 muscle groups (elbow flexion, knee extension, and shoulder abduction) were measured bilaterally using a hand-held dynamometer. Muscle strength was measured twice for each body location; if the variability between the 2 measures was > 15%, a third measure was obtained. The maximum muscle strength of the 2 measurements was identified and transformed as a percent change from baseline using the equation: ([postbaseline value - baseline value] / baseline value) × 100. The mega-score was a composite score that averaged strength across the 3 muscle groups. It was calculated as the mean of the non-missing transformed muscle strength scores among the 3 muscle groups each measure bilaterally (totaling 6 body locations).
Change From Baseline to Week 8 in the Hammersmith Functional Motor Scale-Expanded (HFMS-E)
Time frame: baseline and 8 weeks
The HFMS-E evaluated the level of independent mobility and motor skills through assessment of 33 test-items, each scored from 0 (worse) to 2 (better). The total score was calculated as the sum of the scores among the 33 test items, and has a range from 0 to 66.
Change From Baseline to Week 8 in Revised Upper Limb Module (RULM)
Time frame: baseline and 8 weeks
The RULM assessed motor function in the upper limbs (specifically shoulder, elbow, wrist, and hand function) that related to activities of everyday life. The RULM consisted of 20 items, 1 of which was scored on a 7-point scale (from 0 to 6), 18 were scored on a 3-point scale (from 0 to 2), and 1 was scored on a 2-point scale (0 or 1). The total score was the sum of each response and could range from a minimum of 0 to a maximum of 43 points. Higher scores reflected better motor function.
Change From Baseline to Week 8 in the TUG Test
Time frame: baseline and 8 weeks
The TUG test measured the time (in seconds) it took for a patient to rise from a chair, walk 3 meters, turn around, walk back to the chair and sit down.
Change From Baseline to Week 8 in the 6MWT
Time frame: baseline and 8 weeks
The 6MWT measured the distance (in meters) a patient walked in 6 minutes.
Patient Global Assessment at the End of Week 8
Time frame: 8 weeks
Patients assessed whether they felt the same, better, or worse than prior to dosing on Day 1.
Investigator Global Assessment at the End of Week 8
Time frame: 8 weeks
The Investigator assessed whether patient appeared the same, better, or worse than prior to dosing on Day 1.
2 secondary outcomes
Reldesemtiv Maximum Observed Plasma Concentration (Cmax)
Time frame: End of Week 8
Reldesemtiv Area Under the Plasma Concentration-time Curve From 0 to 12 Hours (AUC0-12)
Time frame: End of Week 8
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
MD, Cytokinetics study director
Cytokinetics, Inc.
19 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Reldesemtiv 150 mg and Reldesemtiv 450 mg in people with Spinal Muscular Atrophy, run by Cytokinetics at 19 sites in 2 countries.
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 20 inclusion and 12 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 31 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, 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 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?
Yes. Results are posted in the registry (first posted 31 Aug 2020). 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 From Baseline to Week 8 in Forced Vital Capacity (FVC) (time frame: baseline and 8 weeks). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Aug 2020.