Hypertension,PulmonaryPhase 2/3SAPPHIRENCT03001414
Study of Angiogenic Cell Therapy for Progressive Pulmonary Hypertension
A Phase 2/3 study testing Autologous EPCs transfected with human eNOS in people with Hypertension,Pulmonary, run by Northern Therapeutics at 4 sites in Canada.
Trial summary
The SAPPHIRE clinical trial seeks to establish the efficacy and safety of repeated monthly dosing of autologous EPCs transfected with human eNOS (heNOS) in patients with symptomatic severe PAH on available PAH-targeted medical therapy. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Hypertension,Pulmonary
- What is being tested?
- Autologous EPCs transfected with human eNOS
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- 4 sitesCanada
- Study dates
- 28 Sep 2017to 1 Nov 2023
- Phase
- Phase 2/3
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years, ≤ 80 years
- Established diagnosis of PAH due to the following:
- Idiopathic or heritable PAH;
- Scleroderma associated PAH (limited or diffuse);
- Drugs (anorexigens) or toxins;
- Congenital heart defects (atrial septal defects, ventricular septal defects, and patent ductus arteriosus) repaired ≥ 1 years
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- World Health Organization (WHO) functional class II, III, or IV on appropriate stable therapy for PAH for at least 3 months prior to the screening period and up until randomization, apart from modification of anticoagulant or diuretic dosages, or small adjustments in prostaglandin dose that are considered by the Investigator to be consistent with stable parenteral therapy.
- Able to walk unassisted (oxygen use allowed). Aids for carrying oxygen (such as a wheel chair or walker) are permitted provided they are not also required as mobility aids.
- An average 6-Minute Walk Distance (6MWD) of ≥ 125 meters and ≤ 440 meters on two consecutive tests during the Screening period
- Previous diagnostic right heart cardiac catheterization (RHC) at the time of PAH diagnosis with findings consistent with PAH: specifically, mean pulmonary arterial pressure (mPAP) ≥ 25 mmHg (at rest); pulmonary vascular resistance (PVR) ≥ 3 WU; pulmonary capillary wedge pressure (PCWP) (or left ventricular end diastolic pressure) ≤12 mmHg if PVR ≥ 3 to < 5 Woods Units (WU), or pulmonary capillary wedge pressure (PCWP) (or left ventricular end diastolic pressure) ≤ 15 mmHg if PVR ≥ 5 WU. If repeat testing has occurred since initial diagnosis, the most recent results should be used.
- Echocardiography performed within 12 months prior to the Screening Period confirming a left atrial volume index (LAVI) of ≤ 34 ml/m2 and the absence of any clinically significant left heart disease including evidence of more than mild left-sided valvular heart disease, systolic or diastolic left ventricular dysfunction
- Ventilation and perfusion (VQ) nuclear scan performed as part of the initial workup to establish the diagnosis of PAH showing absence (i.e. low probability) of pulmonary embolism. If repeat testing has occurred since initial diagnosis, the most recent results should be used. In the absence of a VQ scan to establish eligibility, a CT angiogram that has been reviewed by a radiologist with expertise in the work up for pulmonary endarterectomy and deemed negative for chronic thromboembolic disease may be used instead.
- Pulmonary function tests conducted within 2 years prior to the Screening Period to confirm: total lung capacity (TLC) ≥ 65% the predicted value; and forced expiratory volume at one second (FEV1) of ≥ 65% the predicted value
- Must have a resting arterial oxygen saturation (SaO2) ≥88% with or without supplemental oxygen as measured by pulse oximetry at the Screening Visit
- Must not be enrolled in an exercise training program for pulmonary rehabilitation within 3 months prior to the Screening Visit and must agree not to enroll in an exercise training program for pulmonary rehabilitation during the Screening Period and the first 6 months of the study. Participants enrolled in an exercise program for pulmonary rehabilitation 3 months prior to screening may enter the study if they agree to maintain their current level of rehabilitation for the first 6 months of the study
- Women of child-bearing potential (defined as less than 1 year post-menopausal and not surgically sterile) must be practicing abstinence or using two highly effective methods of contraception (defined as a method of birth control that results in a low failure rate, i.e., less than 1% per year, such as approved hormonal contraceptives, barrier methods [such as a condom or diaphragm] used with a spermicide, or an intrauterine device). Subjects must have a negative ß-human chorionic gonadotropin (hCG) pregnancy test during the Screening period and negative urine pregnancy test results at all other study visits
- Must be willing and able to comply with study requirements and restrictions.
Who cannot join
- Pregnant or lactating
- PAH related to any condition not covered under inclusion criteria, including but not limited to pulmonary venous hypertension, pulmonary veno-occlusive disease, pulmonary capillary hemangiomatosis, or chronic thromboembolic pulmonary hypertension
- Evidence of more than mild interstitial lung disease on Chest CT within the last 5 years (last 3 years for patients with scleroderma associated PAH)
- Treatment with an investigational drug, device or therapy within 3 months prior to the screening period or is scheduled to receive an investigational drug, device or therapy during the course of the study
- Any musculoskeletal disease or any other disease that would significantly limit ambulation
- Unrepaired or recently repaired (< 1 year) congenital systemic-to-pulmonary shunt other than patent foramen ovale
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- Patients having three or more of the following four AMBITION study heart failure preserved ejection fractio (HFpEF) risk factors will be excluded:
- BMI ≥ 30 kg/m2,
- History of essential hypertension,
- Diabetes mellitus (any type)
- Historical evidence of significant coronary artery disease (CAD) by any one of the following:
- History of myocardial infarction (MI)
- History of percutaneous coronary intervention (PCI)
- Prior coronary angiography evidence of CAD (>50% stenosis in ≥1 vessel)
- Previous positive Stress Test
- Previousoronary artery bypass grafting (CABG)
- Stable angina
- Creatinine clearance <30 ml/min (using the Cockroft-Gault formula) or requires hemodialysis
- Inability to undergo the apheresis procedure due to poor venous access or laboratory tests that are not within acceptable ranges (not including international normalized ratio (INR) for patients on Coumadin)
- Childs-Pugh class C liver cirrhosis
- Previous atrial septostomy
- Any other clinically significant illness or abnormal laboratory values (measured during the screening period) that, in the opinion of the Investigator, might put the subject at risk of harm during the study or might adversely affect the interpretation of the study data
- Anticipated survival less than 1 year due to concomitant disease
- History of cancer in the past 5 years (except for low grade and fully resolved non-melanoma skin cancer)
- Results during screening consistent with current infection with HIV, Hepatitis B (HBV) or C (HCV), human T-cell lymphotropic virus (HTLV- I/II) or syphilis
- Systemic arterial systolic blood pressure < 85 mm Hg
- Known allergy to gentamicin or amphotericin
- Patients who have participated in any gene therapy study or an angiogenic growth factor protein study
- Patients unable to provide informed consent and comply with the visit schedule.
Trial locations and contacts
4 sites in 1 country.
Canada4 sites
Peter Lougheed Center, University of Calgary
Calgary, Alberta, T1Y 6J4
London Health Sciences Center
London, Ontario, N6A 5W9
University of Ottawa Heart Institute
Ottawa, Ontario, K1Y 4W7
Toronto General Hospital
Toronto, Ontario, M5G 2N2
About this study
Official title: Study of Angiogenic Cell Therapy for Progressive Pulmonary Hypertension: Intervention With Repeat Dosing of eNOS-enhanced EPCs
The SAPPHIRE clinical trial seeks to establish the efficacy and safety of repeated monthly dosing of autologous EPCs transfected with human eNOS (heNOS) in patients with symptomatic severe PAH on available PAH-targeted medical therapy.
Read the detailed description
SAPPHIRE will use autologous progenitor cell-based gene delivery to enhance lung microvascular repair and regeneration in patients with severe symptomatic PAH. A total of 45 patients will be enrolled in this multi-centre, late phase, randomized, double-blind, placebo-controlled, 3-arm protocol. Up to nine centres across Canada will participate.
Consented study participants who meet all eligibility criteria during the screening period will be scheduled to undergo apheresis. Following successful apheresis collection and receipt of the cell samples by the cell manufacturing facility, randomization will take place though a web-based system. Manufacturing of the cell therapy product will then be performed by the cell manufacturing facility according to the assigned treatment allocation:
Arm 1: Placebo (Plasma-Lyte A; 4 monthly IV infusions) in Course 1 (1st 6 months) followed by Autologous EPCs transfected with human eNOS in Course 2 (2nd 6 months; 4 monthly IV infusions)
Arm 2: Autologous EPCs transfected with human eNOS in Course 1 (1st 6 months; 4 monthly IV infusions) followed by Placebo (Plasma-Lyte A) in Course 2 (2nd 6 months; 4 monthly IV infusions)
Arm 3: Autologous EPCs transfected with human eNOS in Course 1 (1st 6 months; 4 monthly IV infusions) followed by a repeat dosing with Autologous EPCs transfected with human eNOS in Course 2 (2nd 6 months; 4 monthly IV infusions)
Approximately 5-9 days later, the study product will be transported to the investigative site where the initial treatment will be delivered to the study participant in an outpatient setting which is equipped for continuous monitoring of vital signs and oxygen saturation. Participants will subsequently be monitored for a minimum of 1 hour and discharged from the clinic once judged by the study investigator to be clinically stable.
Treatment and follow-up assessments will take place over a 12-month period (11 study visits in total). Once the 12-month trial data collection is completed, the trial will convert to a registry with the goal of collecting long-term safety information through annual telephone contacts for 10 years. Participants will be permitted to enroll in other clinical trials during the registry period.
- Conditions
- Hypertension,Pulmonary
- Being tested
- Placebo followed by Autologous EPCs transfected with human eNOS, Autologous EPCs transfected with human eNOS followed by Placebo, Autologous EPCs transfected with human eNOS
- Medical subject headings
- Hypertension, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placebo followed by Autologous EPCs transfected with human eNOSBiological
4 doses of placebo in first 6 months followed by 4 doses of autologous EPCs transfected with eNOS in second 6 months
Autologous EPCs transfected with human eNOS followed by PlaceboBiological
4 doses of autologous EPCs transfected with eNOS in first 6 months followed by 4 doses of placebo in second 6 months
Autologous EPCs transfected with human eNOSBiological
4 doses of autologous EPCs transfected with eNOS in first 6 months which is repeated in second 6 months
| Study group | Role | What they receive |
|---|---|---|
| Placebo followed by Autologous EPCs transfected with eNOS | Experimental | 4 monthly IV injections of Placebo (Plasma-Lyte A) during Course 1 followed by 4 monthly IV injections of Autologous EPCs transfected with human eNOS (total of 80 million cells) during Course 2 Receives: Biological: Placebo followed by Autologous EPCs transfected with human eNOS |
| Autologous EPCs transfected with eNOS followed by Placebo | Experimental | 4 monthly IV injections of Autologous EPCs transfected with human eNOS (total of 80 million cells) during Course 1 followed by 4 monthly IV injections of Placebo (Plasma-Lyte A) during Course 2 Receives: Biological: Autologous EPCs transfected with human eNOS followed by Placebo |
| Autologous EPCs transfected with eNOS | Experimental | 4 monthly IV injections of Autologous EPCs transfected with human eNOS in Course 1 followed by 4 monthly injections of Autologous EPCs transfected with human eNOS in Course 2 (total of 160 million cells) Receives: Biological: Autologous EPCs transfected with human eNOS |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in 6 Minute Walk Distance (6MWD) From Baseline
Change was calculated as the distance walked at 6 months minus the distance at baseline.
Measured: Baseline, 6 months
Secondary outcomes (8)
Change in 6 Minute Walk Distance (6MWD) From Baseline
Comparison of change from baseline after delivery of 4 versus 8 doses of study product (Arms 2 versus Arm 3). Change was calculated by the distance walked at 12 months minus the distance walked at baseline.
Measured: Baseline and 12 months
Change in 6 Minute Walk Distance (6MWD) From Baseline
Comparison of change from baseline to assess durability of any measured improvement (Arm 2 only). Durability of any improvement at 3 versus 9 months post-treatment (measured at 6 and 12 months).
Measured: Baseline, 6 months, 12 months
Change in 6MWD From Baseline
Incremental effect of 8 versus 4 doses at 6 and 12 months in arm 3 only.
Measured: Baseline, 6 months, 12 months
Change in Pulmonary Vascular Resistance From Baseline
Comparison of change from baseline after 4 doses of study product (Arms 2 and 3 versus Arm 1)
Measured: 6 months
Change in Pulmonary Vascular Resistance From Baseline
Comparison of change from baseline after delivery of 4 versus 8 doses of study product (Arm 2 versus Arm 3)
Measured: Baseline and 12 months
Incremental Effect of 8 Versus 4 Doses of Cell Therapy Product on PVR
Incremental effect of 8 versus 4 doses of cell therapy product on PVR at 6 and 12 months (Arm 3 only)
Measured: 6 and12 months
Change in Quality of Life Measures From Baseline
Comparison of change from baseline measured by the Short Form 36 (SF-36) Health Survey after delivery of 4 doses of study product (Arms 2 and 3 versus Arm 1).
Scale used ranging from 0-100. Lower scores indicate more significant limitations.
Measured: Baseline and 6 months
Change in Quality of Life Measures From Baseline
Comparison in change from baseline measured by SF-36 Survey after delivery of 4 versus 8 doses of study product (Arm 2 versus Arm 3).
Scale used ranging from 0-100. Lower scores indicate more significant limitations.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Dec 2016 (estimated)
- Study start28 Sep 2017
- Primary completion4 May 2023
- Study completion1 Nov 2023
Why it stopped: Lower than anticipated enrollment
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 Hypertension,PulmonaryStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northern Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ottawa Hospital Research Institute
- Responsible party
- Sponsor
- Investigators
- Duncan J Stewart, MD FRCPC, Study director, Northern Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03001414
- Sponsor's protocol ID
- CT-PAH002
- Acronym
- SAPPHIRE
- Status last verified
- Dec 2024
- First submitted
- 15 Dec 2016
- Last updated
- 31 Jan 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Angiogenic Cell Therapy for Progressive Pulmonary Hypertension: Intervention With Repeat Dosing of eNOS-enhanced EPCs
- ClinicalTrials.gov ID
- NCT03001414
- Acronym / sponsor ID
- SAPPHIRE / CT-PAH002
- Conditions
- Hypertension,Pulmonary
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lower than anticipated enrollment
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Northern TherapeuticsIndustry (pharma, biotech, devices)
- Collaborators
- Ottawa Hospital Research Institute
- Study start
- 28 Sep 2017 (actual)
- Primary completion
- 4 May 2023 (actual)
- Study completion
- 1 Nov 2023 (actual)
- First posted
- 23 Dec 2016 (estimated)
- Results
- Posted 31 Jan 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The SAPPHIRE clinical trial seeks to establish the efficacy and safety of repeated monthly dosing of autologous EPCs transfected with human eNOS (heNOS) in patients with symptomatic severe PAH on available PAH-targeted medical therapy.
Detailed description
SAPPHIRE will use autologous progenitor cell-based gene delivery to enhance lung microvascular repair and regeneration in patients with severe symptomatic PAH. A total of 45 patients will be enrolled in this multi-centre, late phase, randomized, double-blind, placebo-controlled, 3-arm protocol. Up to nine centres across Canada will participate.
Consented study participants who meet all eligibility criteria during the screening period will be scheduled to undergo apheresis. Following successful apheresis collection and receipt of the cell samples by the cell manufacturing facility, randomization will take place though a web-based system. Manufacturing of the cell therapy product will then be performed by the cell manufacturing facility according to the assigned treatment allocation:
Arm 1: Placebo (Plasma-Lyte A; 4 monthly IV infusions) in Course 1 (1st 6 months) followed by Autologous EPCs transfected with human eNOS in Course 2 (2nd 6 months; 4 monthly IV infusions)
Arm 2: Autologous EPCs transfected with human eNOS in Course 1 (1st 6 months; 4 monthly IV infusions) followed by Placebo (Plasma-Lyte A) in Course 2 (2nd 6 months; 4 monthly IV infusions)
Arm 3: Autologous EPCs transfected with human eNOS in Course 1 (1st 6 months; 4 monthly IV infusions) followed by a repeat dosing with Autologous EPCs transfected with human eNOS in Course 2 (2nd 6 months; 4 monthly IV infusions)
Approximately 5-9 days later, the study product will be transported to the investigative site where the initial treatment will be delivered to the study participant in an outpatient setting which is equipped for continuous monitoring of vital signs and oxygen saturation. Participants will subsequently be monitored for a minimum of 1 hour and discharged from the clinic once judged by the study investigator to be clinically stable.
Treatment and follow-up assessments will take place over a 12-month period (11 study visits in total). Once the 12-month trial data collection is completed, the trial will convert to a registry with the goal of collecting long-term safety information through annual telephone contacts for 10 years. Participants will be permitted to enroll in other clinical trials during the registry period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo followed by Autologous EPCs transfected with eNOS | Experimental | Biological: Placebo followed by Autologous EPCs transfected with human eNOS4 monthly IV injections of Placebo (Plasma-Lyte A) during Course 1 followed by 4 monthly IV injections of Autologous EPCs transfected with human eNOS (total of 80 million cells) during Course 2 |
| Autologous EPCs transfected with eNOS followed by Placebo | Experimental | Biological: Autologous EPCs transfected with human eNOS followed by Placebo4 monthly IV injections of Autologous EPCs transfected with human eNOS (total of 80 million cells) during Course 1 followed by 4 monthly IV injections of Placebo (Plasma-Lyte A) during Course 2 |
| Autologous EPCs transfected with eNOS | Experimental | Biological: Autologous EPCs transfected with human eNOS4 monthly IV injections of Autologous EPCs transfected with human eNOS in Course 1 followed by 4 monthly injections of Autologous EPCs transfected with human eNOS in Course 2 (total of 160 million cells) |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Age ≥ 18 years, ≤ 80 years
- Established diagnosis of PAH due to the following:
- World Health Organization (WHO) functional class II, III, or IV on appropriate stable therapy for PAH for at least 3 months prior to the screening period and up until randomization, apart from modification of anticoagulant or diuretic dosages, or small adjustments in prostaglandin dose that are considered by the Investigator to be consistent with stable parenteral therapy.
- Able to walk unassisted (oxygen use allowed). Aids for carrying oxygen (such as a wheel chair or walker) are permitted provided they are not also required as mobility aids.
- An average 6-Minute Walk Distance (6MWD) of ≥ 125 meters and ≤ 440 meters on two consecutive tests during the Screening period
Key exclusion criteria (29 listed)
- Pregnant or lactating
- PAH related to any condition not covered under inclusion criteria, including but not limited to pulmonary venous hypertension, pulmonary veno-occlusive disease, pulmonary capillary hemangiomatosis, or chronic thromboembolic pulmonary hypertension
- Evidence of more than mild interstitial lung disease on Chest CT within the last 5 years (last 3 years for patients with scleroderma associated PAH)
- Treatment with an investigational drug, device or therapy within 3 months prior to the screening period or is scheduled to receive an investigational drug, device or therapy during the course of the study
- Any musculoskeletal disease or any other disease that would significantly limit ambulation
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 |
|---|---|---|
| Placebo followed by Autologous EPCs transfected with human eNOS4 doses of placebo in first 6 months followed by 4 doses of autologous EPCs transfected with eNOS in second 6 months | Biological | Placebo followed by Autologous EPCs transfected with eNOS |
| Autologous EPCs transfected with human eNOS followed by Placebo4 doses of autologous EPCs transfected with eNOS in first 6 months followed by 4 doses of placebo in second 6 months | Biological | Autologous EPCs transfected with eNOS followed by Placebo |
| Autologous EPCs transfected with human eNOS4 doses of autologous EPCs transfected with eNOS in first 6 months which is repeated in second 6 months | Biological | Autologous EPCs transfected with eNOS |
Primary outcome
Change in 6 Minute Walk Distance (6MWD) From Baseline
Time frame: Baseline, 6 months
Change was calculated as the distance walked at 6 months minus the distance at baseline.
8 secondary outcomes
Change in 6 Minute Walk Distance (6MWD) From Baseline
Time frame: Baseline and 12 months
Change in 6 Minute Walk Distance (6MWD) From Baseline
Time frame: Baseline, 6 months, 12 months
Change in 6MWD From Baseline
Time frame: Baseline, 6 months, 12 months
Change in Pulmonary Vascular Resistance From Baseline
Time frame: 6 months
Change in Pulmonary Vascular Resistance From Baseline
Time frame: Baseline and 12 months
Incremental Effect of 8 Versus 4 Doses of Cell Therapy Product on PVR
Time frame: 6 and12 months
Change in Quality of Life Measures From Baseline
Time frame: Baseline and 6 months
Change in Quality of Life Measures From Baseline
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2025.
- 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.
Duncan J Stewart, MD FRCPC study director
Northern Therapeutics, Inc.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Autologous EPCs transfected with human eNOS in people with Hypertension,Pulmonary, run by Northern Therapeutics at 4 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 17 inclusion and 29 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 31 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 4 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 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 6 Minute Walk Distance (6MWD) From Baseline (time frame: Baseline, 6 months). 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 Jan 2025.