Non-operable Chronic Thromboembolic Pulmonary HypertensionPhase 3CTREPHNCT01416636

Efficacy and Tolerability of Subcutaneously Administered Treprostinil Sodium in Patients With Severe (Non-operable) Chronic Thromboembolic Pulmonary Hypertension (CTREPH)

A Phase 3 study testing Treprostinil sodium in people with Non-operable Chronic Thromboembolic Pulmonary Hypertension, run by SciPharm SàRL at 6 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The primary purpose of this study is to determine the effect on six-minute walking test (6MWT) distance after 24 weeks treatment with subcutaneous (SC) Treprostinil Sodium in patients with Severe (inoperable) Chronic Thromboembolic Pulmonary Hypertension. Read more about this study

Who is it for?: Ages 18 to 100 years
Conditions the study is for: Non-operable Chronic Thromboembolic Pulmonary Hypertension
What is being tested?: Treprostinil sodium
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 105 participants
Where: 6 sitesin 4 countries
Study dates: Mar 2009to Apr 2021
Phase: Phase 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 100 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

None listed.

Who cannot join

Subject with any form of pulmonary arterial hypertension or any disease known to cause PAH (WHO Group I)
Subjects with a total lung capacity (TLC) of < 70% predicted or a forced expiratory volume/forced vital capacity (FEV1/FVC < 50%)
Subject who received any prostanoids, within the 30 days before screening or be scheduled to receive prostanoids during the course of the study
Subject with a new type of chronic therapy (a different category of vasodilator or diuretic) for PAH added within the last month, except anticoagulants
Subject with an increased risk for hemorrhage or stroke or with a major cardiovascular event during the past 6 months.
Unstable subjects for any reason (according to the investigators discretion)

Show all 14Show fewer

Subject who received any investigational medication within 30 days prior to the screening visit of this study or be scheduled to receive another investigational drug during the course of this study
Subject with a known intolerance to any drug relevant for this trial, especially to Treprostinil sodium or prostanoids
Subject with a history or suspicion of non compliance
Subject who has any musculoskeletal disease or any other disease that would limit ambulation
Subject with other cardiovascular, liver, renal, hematologic, gastrointestinal immunologic, endocrine, metabolic, or central nervous system disease that, in the opinion of the investigator, may adversely affect the safety of the subject and /or efficacy of the study drug or limit the lifespan of the subject
Female who is considering pregnancy or who is pregnant and/or lactating
Subject who is an investigator or any other team member involved directly or indirectly in the conduct of the clinical study.
Subject who is an inmate of a psychiatric ward, prison or is suspected not to be able to give consent of his free will

Trial locations and contacts

6 sites in 4 countries.

Country

City, postcode or hospital

6 sites in 4 countries

Austria2 sites

Krankenhaus der Elisabethinen
Linz, 4020
Medical University of Vienna AKH - Division Cardiology
Vienna

Other trials in Austria

Poland2 sites

Department of Cardiac and Vascular Diseases Centre for Rare Cardiovascular Diseases John Paul II Hospital
Krakow, 31-202
NZOZ Europejskie Centrum Zdrowia Otwock
Otwock, 05-400

Other trials in Poland

Czechia1 site

II. interní klinika Všeobecná fakultní nemocnice
Prague

Other trials in Czechia

Germany1 site

Medical University Carl Gustav Carus Medizinische Klinik und Poliklinik I Medizinische Fakultät der Technischen Universität Dresden
Dresden

Other trials in Germany

About this study

Official title: A Double Blind Controlled Clinical Study to Investigate the Efficacy and Tolerability of Subcutaneous Treprostinil Sodium in Patients With Severe Non-operable Chronic Thromboembolic Pulmonary Hypertension (CTREPH)

Read the detailed description

Chronic thromboembolic pulmonary hypertension (CTEPH) is characterized by non-resolving organized thromboembolic obstructing the pulmonary vascular bed. These thrombi are resistant to thrombolytic therapy and chronic plasmatic anticoagulation. An increase in pulmonary vascular resistance (PVR), right ventricular overload, and eventually right ventricular failure ensue.

The treatment of choice for CTEPH is pulmonary endarterectomy (PEA), providing a potential cure for the disease. However, about 50 % of patients are not candidates for surgery, mainly because of distal location of thromboemboli. Despite recent advances in the treatment of pulmonary arterial hypertension (PAH), medical treatments have not been recommended for inoperable CTEPH, because of the concept that a predominantly major vessel obstructive arteriopathy would not be suitable for vasodilators. Furthermore, a major drawback of i.v. prostacyclin therapy is the need for a permanent central venous access that increases the risk of infection (0.22-0.68 per patient per year), thrombosis and new major vessel thromboembolism.

Conditions: Non-operable Chronic Thromboembolic Pulmonary Hypertension
Being tested: Treprostinil sodium

What participants receive

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

Treprostinil sodiumDrug

Study groups in NCT01416636
Study group	Role	What they receive
Treprostinil sodium low dose - Arm I	Active comparator	Arm I (low dose): Subject was treated with a low dose of Treprostinil sodium. Dose was escalated to an approximate target dose of 3 ng/kg/min after the first 12 weeks and was kept stable for another 12 weeks. Due to the predefined infusion rate setting schedule an interim dose of up to 6 ng/kg/min could be reached for few days at the end of the phases 1,2 and 3. This depended on the patient's exact weight and is caused by the limited infusion rate setting possibility of the infusion pump. Receives: Drug: Treprostinil sodium
Treprostinil sodium high dose - Arm II	Experimental	Subject was treated with a low dose of Treprostinil sodium. Dose was escalated to an approximate target dose of 3 ng/kg/min after the first 12 weeks and kept stable for another 12 weeks. Due to the predefined infusion rate setting schedule an interim dose of up to 6 ng/kg/min could be reached for few days at the end of the phases 1,2 and 3. This depended on the patient's exact weight and is caused by the limited infusion rate setting possibility of the infusion pump. Receives: Drug: Treprostinil sodium

What is being measured

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

Change in 6-minute Walk Test Distance After 24 Weeks
To determine the effect of subcutaneous Treprostinil sodium on 6-minute walk test distance after 24 weeks in patients with severe non-operable chronic thromboembolic pulmonary hypertension severe (inoperable) Chronic Thromboembolic Pulmonary Hypertension
Time frame of the 6-minute walk test: The 6-minute walk test was conducted at the following visits:
- baseline (day 1)
- Visit 6 (day 168)
In case of missing values, Last-Observation-Carried-Forward imputation method was used. In such cases values documented at Visit 4 (day84) were used.
Measured: Baseline and 24 weeks

Secondary outcomes (11)

Number of Participants With Clinical Worsening
Clinical worsening defined as a decrease of 6-minute walk test distance of more than 20% from baseline due to Chronic Thromboembolic Pulmonary Hypertension, decrease of New York Heart Association functional class, hospitalization with the requirement for additional Pulmonary Hypertension specific treatment and/or death due to worsening Chronic Thromboembolic Pulmonary Hypertension.
Clinical Worsening was assessed after 12 weeks and 24 weeks, participants experiencing clinical worsening at any time-point are reported.
Measured: 12 weeks and 24 weeks
Effect on Maximal Borg Score During 6-minutes Walk Test
The Borg scale was used for rating of dyspnea during 6-minutes walk test. The scale is defined from 0 to > 10 (upper bound) (0 = NOTHING AT ALL; 0.5 = VERY VERY SLIGHT (just noticeable); 1 = VERY SLIGHT; 2 = SLIGHT; 3 = MODERATE; 4 = SOMEWHAT SEVERE; 5 = SEVERE; 6-9 = VERY SEVERE; 10 = VERY VERY SEVERE (almost maximum); >10 MAXIMUM).
As can be seen with the scale, the higher scale values represent a worse outcome.
As no imputation rule applied only full-data sets were evaluated. Complete data sets were available for 48 patients randomized to high dose group and 48 patients in low dose group.
Measured: Baseline and 24 weeks
Change in WHO/NYHA (World Health Organization - New York Heart Association) Functional Class
Class I - Patients with pulmonary hypertension but without resulting limitation of physical activity. Ordinary physical activity does not cause undue dyspnea or fatigue, chest pain or near syncope.
Class II - Patients with pulmonary hypertension resulting in slight limitation of physical activity. They are comfortable at rest. Ordinary physical activity causes undue dyspnea or fatigue, chest pain or near syncope.
Class III - Patients with pulmonary hypertension resulting in marked limitation of physical activity. They are comfortable at rest. Less than ordinary activity causes undue dyspnea or fatigue, chest pain or near syncope
Class IV - Patients with pulmonary hypertension in the inability to carry out any physical activity without symptoms. These patients manifest signs of right heart failure. Dyspnea and/or fatigue may even be present at rest. Discomfort is increased by any physical activity.
Measured: Baseline and 24 weeks
Effect on Quality of Life by the MINNESOTA Questionnaire
This questionnaire is composed of 21 questions relating to limitations in lifestyle associated with Heart Failure. Respondents use a 5-point scale that ranges from 0 (none) to 5 (too much), with a score of 0 representing no limitation and a score of 5 representing maximum limitation. The change in individual score sum was evaluated and is displayed in the results, with a possible range of 0-105. Higher values indicate more limitations in Quality of Life.
As no imputation rule applied only full-data sets were evaluated. Complete data sets were available for 50 patients randomized to high dose group and 46 patients in low dose group.
Measured: Baseline and 24 weeks
Effect on N-terminal Pro-BNP Levels
baseline values, assessment after 12 and 24 weeks
As no imputation rule applied only full-data sets were evaluated. Complete data sets were available for 46 patients randomized to high dose group and 46 patients in low dose group.
Measured: Baseline and 24 weeks
Effect on Hemodynamic Parameter (PVR - Pulmonary Vascular Resistance)
baseline values, assessment after 24 weeks
As no imputation rule applied only full-data sets were evaluated. Complete data sets were available for 48 patients randomized to high dose group and 47 patients in low dose group.
Measured: Baseline and 24 weeks
Effect on Hemodynamic Parameter (CI - Cardiac Index)
baseline values, assessment after 24 weeks
As no imputation rule applied only full-data sets were evaluated. Complete data sets were available for 48 patients randomized to high dose group and 47 patients in low dose group.
Measured: Baseline and 24 weeks
Effect on Hemodynamic Parameter (CO - Cardiac Output)
baseline values, assessment after 24 weeks
As no imputation rule applied only full-data sets were evaluated. Complete data sets were available for 48 patients randomized to high dose group and 47 patients in low dose group.
Measured: Baseline and 24 weeks
Effect on Hemodynamic Parameter (mPAP - Mean Pulmonary Arterial Pressure)
baseline values, assessment after 24 weeks
As no imputation rule applied only full-data sets were evaluated. Complete data sets were available for 47 patients randomized to high dose group and 47 patients in low dose group.
Measured: Baseline and 24 weeks
Effect on Hemodynamic Parameter (mRap - Mean Right Atrial Pressure)
baseline values, assessment after 24 weeks
As no imputation rule applied only full-data sets were evaluated. Complete data sets were available for 48 patients randomized to high dose group and 47 patients in low dose group.
Measured: Baseline and 24 weeks
Effect on Signs & Symptoms of the CTEPH
baseline values, assessment after 24 weeks
Measured: Baseline and 24 weeks

Timeline

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

Mar 2009Apr 2021

First posted15 Aug 2011 (estimated)
Study startMar 2009
Primary completionNov 2016
Study completionApr 2021

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 (5)Papers the sponsor has linked to this study.
Other trials for Non-operable Chronic Thromboembolic Pulmonary HypertensionStudies 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 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 105 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01416636
Sponsor's protocol ID: 116-02
Acronym: CTREPH
Status last verified: May 2022
First submitted: 12 Aug 2011
Last updated: 7 Jun 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Lang IM, Marsh JJ, Olman MA, Moser KM, Loskutoff DJ, Schleef RR. Expression of type 1 plasminogen activator inhibitor in chronic pulmonary thromboemboli. Circulation. 1994 Jun;89(6):2715-21. doi: 10.1161/01.cir.89.6.2715.
PubMed 8205686
Klepetko W, Mayer E, Sandoval J, Trulock EP, Vachiery JL, Dartevelle P, Pepke-Zaba J, Jamieson SW, Lang I, Corris P. Interventional and surgical modalities of treatment for pulmonary arterial hypertension. J Am Coll Cardiol. 2004 Jun 16;43(12 Suppl S):73S-80S. doi: 10.1016/j.jacc.2004.02.039.
PubMed 15194182
McLaughlin VV, Presberg KW, Doyle RL, Abman SH, McCrory DC, Fortin T, Ahearn G; American College of Chest Physicians. Prognosis of pulmonary arterial hypertension: ACCP evidence-based clinical practice guidelines. Chest. 2004 Jul;126(1 Suppl):78S-92S. doi: 10.1378/chest.126.1_suppl.78S.
PubMed 15249497
Kuhn KP, Byrne DW, Arbogast PG, Doyle TP, Loyd JE, Robbins IM. Outcome in 91 consecutive patients with pulmonary arterial hypertension receiving epoprostenol. Am J Respir Crit Care Med. 2003 Feb 15;167(4):580-6. doi: 10.1164/rccm.200204-333OC. Epub 2002 Nov 21.
PubMed 12446266
Sadushi-Kolici R, Jansa P, Kopec G, Torbicki A, Skoro-Sajer N, Campean IA, Halank M, Simkova I, Karlocai K, Steringer-Mascherbauer R, Samarzija M, Salobir B, Klepetko W, Lindner J, Lang IM. Subcutaneous treprostinil for the treatment of severe non-operable chronic thromboembolic pulmonary hypertension (CTREPH): a double-blind, phase 3, randomised controlled trial. Lancet Respir Med. 2019 Mar;7(3):239-248. doi: 10.1016/S2213-2600(18)30367-9. Epub 2018 Nov 23.
PubMed 30477763

Study at a glance

From the registry record, last updated by the sponsor on 7 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Double Blind Controlled Clinical Study to Investigate the Efficacy and Tolerability of Subcutaneous Treprostinil Sodium in Patients With Severe Non-operable Chronic Thromboembolic Pulmonary Hypertension (CTREPH)
ClinicalTrials.gov ID: NCT01416636
Acronym / sponsor ID: CTREPH / 116-02
Conditions: Non-operable Chronic Thromboembolic Pulmonary Hypertension
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 105 participants (actual)
Lead sponsor: SciPharm SàRLIndustry (pharma, biotech, devices)
Study start: Mar 2009
Primary completion: Nov 2016 (actual)
Study completion: Apr 2021 (actual)
First posted: 15 Aug 2011 (estimated)
Results: Posted 10 Jun 2019

Clinical summary

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

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Treprostinil sodium low dose - Arm I	Active comparator	Drug: Treprostinil sodiumArm I (low dose): Subject was treated with a low dose of Treprostinil sodium. Dose was escalated to an approximate target dose of 3 ng/kg/min after the first 12 weeks and was kept stable for another 12 weeks. Due to the predefined infusion rate setting schedule an interim dose of up to 6 ng/kg/min could be reached for few days at the end of the phases 1,2 and 3. This depended on the patient's exact weight and is caused by the limited infusion rate setting possibility of the infusion pump.
Treprostinil sodium high dose - Arm II	Experimental	Drug: Treprostinil sodiumSubject was treated with a low dose of Treprostinil sodium. Dose was escalated to an approximate target dose of 3 ng/kg/min after the first 12 weeks and kept stable for another 12 weeks. Due to the predefined infusion rate setting schedule an interim dose of up to 6 ng/kg/min could be reached for few days at the end of the phases 1,2 and 3. This depended on the patient's exact weight and is caused by the limited infusion rate setting possibility of the infusion pump.

Who may qualify

Ages: 18 to 100 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (0 listed)

Key exclusion criteria (14 listed)

Subject with any form of pulmonary arterial hypertension or any disease known to cause PAH (WHO Group I)
Subjects with a total lung capacity (TLC) of < 70% predicted or a forced expiratory volume/forced vital capacity (FEV1/FVC < 50%)
Subject who received any prostanoids, within the 30 days before screening or be scheduled to receive prostanoids during the course of the study
Subject with a new type of chronic therapy (a different category of vasodilator or diuretic) for PAH added within the last month, except anticoagulants
Subject with an increased risk for hemorrhage or stroke or with a major cardiovascular event during the past 6 months.

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
Treprostinil sodium	Drug	Treprostinil sodium high dose - Arm II, Treprostinil sodium low dose - Arm I

Primary outcome

Change in 6-minute Walk Test Distance After 24 Weeks
Time frame: Baseline and 24 weeks
To determine the effect of subcutaneous Treprostinil sodium on 6-minute walk test distance after 24 weeks in patients with severe non-operable chronic thromboembolic pulmonary hypertension severe (inoperable) Chronic Thromboembolic Pulmonary Hypertension Time frame of the 6-minute walk test: The 6-minute walk test was conducted at the following visits: * baseline (day 1) * Visit 6 (day 168) In case of missing values, Last-Observation-Carried-Forward imputation method was used. In such cases values documented at Visit 4 (day84) were used.

11 secondary outcomes

Number of Participants With Clinical Worsening
Time frame: 12 weeks and 24 weeks
Effect on Maximal Borg Score During 6-minutes Walk Test
Time frame: Baseline and 24 weeks
Change in WHO/NYHA (World Health Organization - New York Heart Association) Functional Class
Time frame: Baseline and 24 weeks
Effect on Quality of Life by the MINNESOTA Questionnaire
Time frame: Baseline and 24 weeks
Effect on N-terminal Pro-BNP Levels
Time frame: Baseline and 24 weeks
Effect on Hemodynamic Parameter (PVR - Pulmonary Vascular Resistance)
Time frame: Baseline and 24 weeks
Effect on Hemodynamic Parameter (CI - Cardiac Index)
Time frame: Baseline and 24 weeks
Effect on Hemodynamic Parameter (CO - Cardiac Output)
Time frame: Baseline and 24 weeks
Effect on Hemodynamic Parameter (mPAP - Mean Pulmonary Arterial Pressure)
Time frame: Baseline and 24 weeks
Effect on Hemodynamic Parameter (mRap - Mean Right Atrial Pressure)
Time frame: Baseline and 24 weeks
Effect on Signs & Symptoms of the CTEPH
Time frame: Baseline and 24 weeks

Results and safety

Results posted to the registry on 10 Jun 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Treprostinil Sodium High Dose (~30ng/kg/Min)	53	45
Treprostinil Sodium Low Dose (~3ng/kg/Min)	52	46

Primary outcome: Change in 6-minute Walk Test Distance After 24 Weeks

Time frame: Baseline and 24 weeks. Mean (Standard Deviation). Unit: m

Measure	High Dose n = 53	Low Dose n = 52
Value	45.43 (71.29)	3.83 (56.21)

High Dose vs Low Dose: p = 0.002, ANCOVA
High Dose vs Low Dose: p = 0.0003, Wilcoxon (Mann-Whitney)

Population analysed: All randomized subjects who received at least one dose of study medication.

11 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
High Dose	9 of 53 (17%)	53 of 53 (100%)	2 of 53
Low Dose	10 of 52 (19.2%)	51 of 52 (98.1%)	1 of 52

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 Jun 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 8205686Lang IM, Marsh JJ, Olman MA, Moser KM, Loskutoff DJ, Schleef RR. Expression of type 1 plasminogen activator inhibitor in chronic pulmonary thromboemboli. Circulation. 1994 Jun;89(6):2715-21. doi: 10.1161/01.cir.89.6.2715.
Background: PubMed 15194182Klepetko W, Mayer E, Sandoval J, Trulock EP, Vachiery JL, Dartevelle P, Pepke-Zaba J, Jamieson SW, Lang I, Corris P. Interventional and surgical modalities of treatment for pulmonary arterial hypertension. J Am Coll Cardiol. 2004 Jun 16;43(12 Suppl S):73S-80S. doi: 10.1016/j.jacc.2004.02.039.
Background: PubMed 15249497McLaughlin VV, Presberg KW, Doyle RL, Abman SH, McCrory DC, Fortin T, Ahearn G; American College of Chest Physicians. Prognosis of pulmonary arterial hypertension: ACCP evidence-based clinical practice guidelines. Chest. 2004 Jul;126(1 Suppl):78S-92S. doi: 10.1378/chest.126.1_suppl.78S.
Background: PubMed 12446266Kuhn KP, Byrne DW, Arbogast PG, Doyle TP, Loyd JE, Robbins IM. Outcome in 91 consecutive patients with pulmonary arterial hypertension receiving epoprostenol. Am J Respir Crit Care Med. 2003 Feb 15;167(4):580-6. doi: 10.1164/rccm.200204-333OC. Epub 2002 Nov 21.
Derived from this study: PubMed 30477763Sadushi-Kolici R, Jansa P, Kopec G, Torbicki A, Skoro-Sajer N, Campean IA, Halank M, Simkova I, Karlocai K, Steringer-Mascherbauer R, Samarzija M, Salobir B, Klepetko W, Lindner J, Lang IM. Subcutaneous treprostinil for the treatment of severe non-operable chronic thromboembolic pulmonary hypertension (CTREPH): a double-blind, phase 3, randomised controlled trial. Lancet Respir Med. 2019 Mar;7(3):239-248. doi: 10.1016/S2213-2600(18)30367-9. Epub 2018 Nov 23.

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.

Irene Lang, MD principal investigator

Medical University of Vienna

6 sites in 4 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Treprostinil sodium in people with Non-operable Chronic Thromboembolic Pulmonary Hypertension, run by SciPharm SàRL at 6 sites in 4 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 0 inclusion and 14 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 7 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in Austria, Poland, Czechia, Germany. 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 phase mean?

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 10 Jun 2019). 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 Test Distance After 24 Weeks (time frame: Baseline and 24 weeks). 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 NCT01416636, which also lists 5 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 7 Jun 2022.