Ignite Creation Date:
2024-05-06 @ 3:27 AM
Last Modification Date:
2024-10-26 @ 11:33 AM
Study NCT ID:
NCT02290613
Status:
COMPLETED
Last Update Posted:
2020-04-30
First Post:
2014-10-31
Brief Title:
Early Treatment of Borderline Pulmonary Arterial Hypertension Associated With Systemic Sclerosis SSc-APAH
Sponsor:
Heidelberg University
Organization:
Heidelberg University
Study Overview
Official Title:
Early Treatment of Borderline Pulmonary Arterial Hypertension Associated With Systemic Sclerosis SSc-APAH A Randomized Controlled Double-blind Parallel Group Proof-of-concept Trial EDITA
Status:
COMPLETED
Status Verified Date:
2020-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
EDITA
Brief Summary:
Trial Design Patients with borderline PAH indicated by borderline mPAP values will be included in this single centre study This clinical investigation is performed as a Proof-of-Concept PoC investigator initiated trial IIT using a prospective randomized double-blind parallel group placebo-controlled phase IIA clinical study design On their first visit their medical history will be obtained and physical examination will be conducted Moreover an electrocardiogram ECG laboratory testing NT-proBNP uric acid and other laboratory tests echocardiography at rest and right heart catheterization will be carried out If patients have been identified within the last 6 months before screening investigations by right heart catheterization the measurements are considered valid as baseline investigations and will not be repeated If patients fulfill the inclusion criteria and still suffer from borderline mPAP values they will be invited to join the study The clinical investigations will begin within 28 days The prospective study will comprise a 6 months study period 180 2 weeks plus the screening phase up to 28 days and a follow-up phase of 30 7 days
Detailed Description:
Treatment naïve patients with SSc-APAH will be included in the investigator initiated trial IIT to assess efficacy and safety of ambrisentan As patients life-expectancy after diagnosis of untreated patients is only one year we put forward a screening to identify borderline PAH patients and treat them before PH manifests Therapy with ambrisentan reached a significant improvement in SSc-IPAH patients Galiè et al 2008 In PAH mPAP improved about 15 due to ambrisentan Klinger et al 2011
Thus especially patients with SSc-APAH have a high need for an early diagnosis and therapy It is important to determine factors predictive of incident SSc-APAH and PH as well as the event rate of PAH and PH occurrence Early identification and intervention with specific modern therapies as with ambrisentan may improve hemodynamic symptoms exercise capacity quality of life and outcomes in this patient population in particular in SSc-patients of borderline-PAH It is considered reasonable that the development of manifest APAH might be preventable in this defined population with SSc and early pulmonary vascular changes A reliable trial testing this latter hypothesis cannot be performed without critical evidence which defines the response to medical PAH-targeted therapy in borderline-PAH and the associated disease progression of manifest PAH
Due to the positive results in the treatment of patients with SSc-APAH the initiation of this proof-of-concept study is justified
Previously identified patients with borderline PAH indicated by borderline mPAP values will be included in this single centre randomized controlled double-blind parallel group proof-of-concept PoC phase IIa IIT If assessments necessary for screening have already been made under the screening for PH in Systemic sclerosis trial non-drug trial Ethics committee of Heidelberg S3602009 these examinations may be used for screening for this trial as long as they have been performed within the given time frame of the screening period
On their first visit the patients medical history will be obtained and physical examination will be conducted Moreover an electrocardiogram ECG laboratory testing NT-proBNP uric acid and other laboratory tests echocardiography at rest and during exercise and right heart catheterization will be carried out If patients fulfill the inclusion criteria and still suffer from borderline mPAP values they will be invited to join the study Patients will be asked to sign the informed consent form ICF before the initial screening will be conducted Randomization will be performed after a maximum of 28 days and medication or placebo will be provided If patients have been identified within the last 6 months before baseline by right heart catheterization the measurements are considered valid for the baseline visit to spare patients a repetition of this invasive procedure Non-invasive measurements that are out of the time-frame have to be repeated for the study An 11 oral ambrisentan oral Placebo randomization will be performed
Patients will be randomized into either
A treatment arm with ambrisentan treatment 19 patients
A placebo arm 19 patients will receive placebo Safety and tolerability will be controlled at each study visit until the end of study day 180 2 weeks If necessary the dose will be adapted As to common practice of the clinic the patient will adapt the dose according to tolerability and after consultation by phone or personally with one of the investigators
Thus especially patients with SSc-APAH have a high need for an early diagnosis and therapy It is important to determine factors predictive of incident SSc-APAH and PH as well as the event rate of PAH and PH occurrence Early identification and intervention with specific modern therapies as with ambrisentan may improve hemodynamic symptoms exercise capacity quality of life and outcomes in this patient population in particular in SSc-patients of borderline-PAH It is considered reasonable that the development of manifest APAH might be preventable in this defined population with SSc and early pulmonary vascular changes A reliable trial testing this latter hypothesis cannot be performed without critical evidence which defines the response to medical PAH-targeted therapy in borderline-PAH and the associated disease progression of manifest PAH
Due to the positive results in the treatment of patients with SSc-APAH the initiation of this proof-of-concept study is justified
Previously identified patients with borderline PAH indicated by borderline mPAP values will be included in this single centre randomized controlled double-blind parallel group proof-of-concept PoC phase IIa IIT If assessments necessary for screening have already been made under the screening for PH in Systemic sclerosis trial non-drug trial Ethics committee of Heidelberg S3602009 these examinations may be used for screening for this trial as long as they have been performed within the given time frame of the screening period
On their first visit the patients medical history will be obtained and physical examination will be conducted Moreover an electrocardiogram ECG laboratory testing NT-proBNP uric acid and other laboratory tests echocardiography at rest and during exercise and right heart catheterization will be carried out If patients fulfill the inclusion criteria and still suffer from borderline mPAP values they will be invited to join the study Patients will be asked to sign the informed consent form ICF before the initial screening will be conducted Randomization will be performed after a maximum of 28 days and medication or placebo will be provided If patients have been identified within the last 6 months before baseline by right heart catheterization the measurements are considered valid for the baseline visit to spare patients a repetition of this invasive procedure Non-invasive measurements that are out of the time-frame have to be repeated for the study An 11 oral ambrisentan oral Placebo randomization will be performed
Patients will be randomized into either
A treatment arm with ambrisentan treatment 19 patients
A placebo arm 19 patients will receive placebo Safety and tolerability will be controlled at each study visit until the end of study day 180 2 weeks If necessary the dose will be adapted As to common practice of the clinic the patient will adapt the dose according to tolerability and after consultation by phone or personally with one of the investigators
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None