search  
 


Home

Who are we?

Why
register?


Signup for
registration


Online registration

Log in to register
your trial


Search a trial

NRT en CCMO

Contact

NEDERLANDS





MetaRegister
van CCT (UK)


ISRCTN-Register
van CCT (UK)


STEPS study


- candidate number15910
- NTR NumberNTR4328
- ISRCTNISRCTN wordt niet meer aangevraagd.
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR19-dec-2013
- Secondary IDsNL44805.078.13 
- Public TitleSTEPS study
- Scientific TitleSteroid Treatment Effect in Pulmonary Sarcoidosis
- ACRONYMSTEPS study
- hypothesisSarcoidosis is a granulomatous disorder of unknown cause. Current therapy is immunosuppressive, not curative and often ineffective, as we do not thoroughly understand the underlying pathogenesis. Oral corticosteroids are the first line of therapy in pulmonary sarcoidosis patients with significant pulmonary symptoms and/or progressive disease as determined by radiology or lung function. In the latter case, treatment is primarily aimed at preventing organ damage, although previous studies did not conclusively demonstrate a beneficial effect in preventing disease progression or pulmonary fibrosis. Nevertheless, current treatment guidelines advocate relatively long and high initial treatment regimes, without clear evidence for the optimal dose and duration of treatment. Importantly, prolonged treatment with prednisone is known to induce considerable side-effects/co-morbidity.

Several expert-opinions suggest a lower initial dose and shorter initial phase, based on retrospective case series. However, there is considerable variety in the response to treatment between sarcoidosis individuals and immunological mechanisms underlying this phenomenon have not been elucidated yet. These data suggest that treatment of sarcoidosis patients should be individualized, pursuing the lowest prednisone dose for the shortest period possible. However, prospective data on the early response towards prednisone treatment and tapering is lacking in a well-characterized cohort of pulmonary sarcoidosis patients.

Against this background, we aim to describe the pulmonary response to corticosteroid therapy and tapering in a cohort of newly treated pulmonary sarcoidosis patients, using a hand-held spirometer. With results of this study, in the future, we hope to individualize the treatment and tapering strategy for sarcoidosis patients. This could lead to prednisone dose sparing, reduction in co-morbidity and an increased quality of life of sarcoidosis patients. This treatment strategy has already been performed successfully in other chronic pulmonary diseases requiring prednisone treatment, like asthma.
- Healt Condition(s) or Problem(s) studiedSarcoidosis, Prednison, Treatment, Lung
- Inclusion criteria-Patients with stage II/III sarcoidosis (based on a chest X-ray or equivalent on a CT-scan) (established using the criteria of the World Association of Sarcoidosis and Other Granulomatous Disorders (WASOG));
- A forced vital capacity (FVC) < 80% of predicted;
- A pulmonary indication for prednisolone treatment (determined by the treating physician and conform current clinical guidelines);
- Written informed consent;
- Age > 18.
- Exclusion criteria- Previous immunosuppressive treatment for sarcoidosis (e.g. prednisone, methotrexate (MTX), Infliximab);
- Use of systemic immunosuppressive therapy within the previous 3 months for another disease then sarcoidosis;
- Contra-indication for steroids;
- Other conditions which could influence pulmonary function, such as: o Concurrent chronico bstructive pulmonary disorder(COPD);
o Concurrent active asthma (according to GINA guidelines);
o Fibrotic lung disease;
o Pregnancy;
o Morbideobesitas(BMI>30);
- Pulmonary malignancies.
- mec approval receivedyes
- multicenter trialyes
- randomisedno
- groupFactorial
- Type2 or more arms, non-randomized
- Studytypeobservational
- planned startdate 19-dec-2013
- planned closingdate1-jul-2017
- Target number of participants50
- InterventionsPatients participating in this study will be treated according to current guidelines. As such, diagnostic procedures or treatment will not be postponed during participation. Standard procedures will be executed during regular visits at the outpatient clinic. In course of the research:
1. Daily home monitoring of the pulmonary function (PF) will be performed with a hand-held spirometer (MicroDiary, CareFusion) during the first three months of treatment;
2. The patient is asked to keep record of his/her symptomatic response to therapy by filling out a weekly dyspnoea score (MRC scale) and a fatigue score (FAS score) during the first three months of treatment at home;
3. During the regular visits at the outpatient clinic, patient reported response to therapy will be objectified by two standardized questionnaires (SGRQ and SF-36), in-hospital pulmonary function tests at the department of pulmonology, a chest X-ray, thorax-HRCT (only if physician’s choice), weight and laboratory data will be recorded;
4. The patient will be asked to donate an additional 60 ml peripheral blood for immunological analysis at the baseline visit, month 1,3 and 12 (i.e. amount of macrophages, NK-cells, dendritic cells, T-cells, B-cells and cytokines; apoptotic susceptibility towards FasL, IL-2 deprivation and prednisone; expression of activation, differentiation and apoptotic markers; immunosuppressive function of lymphocyte subsets and HLA-type will be examined). Blood will be collected during routine venapunction.
- Primary outcome1. Repeated measurements of the FVC and prednisone dose used
- Secondary outcome1. Demographic data and clinical status (age, gender, ethnicity, smoking status, co- morbidities, extra thoracic manifestations, date and method of sarcoidosis diagnosis);
2. Patient reported pulmonary complaints objectified in dyspnoea (Medical Research Council (MRC) scale), quality of life (St. George Respiratory Questionaire (SGRQ)), general health (SF-36) and fatigue scores (Fatigue Assessment Scale (FAS));
3. Repeated measurements of weight;
4. Extent of pulmonary disease (in-hospital pulmonary function data and radiology data);
5. Laboratory data (ACE, calcium, glucose, sIL2R, creatinine, urine analysis (calcium/creatinine/protein), CRP);
6. Exploratory immunological analysis of lymphocyte subsets.
- TimepointsBaseline visit, months 1,3,6,9,12
- Trial web siteN/A
- statusopen: patient inclusion
- CONTACT FOR PUBLIC QUERIES Caroline Broos
- CONTACT for SCIENTIFIC QUERIES Caroline Broos
- Sponsor/Initiator Erasmus Medical Center
- Funding
(Source(s) of Monetary or Material Support)
Erasmus Medical Center
- PublicationsN/a
- Brief summarySarcoidosis is a granulomatous disorder of unknown cause. Current therapy is immunosuppressive, not curative and often ineffective, as we do not thoroughly understand the underlying pathogenesis. Oral corticosteroids are the first line of therapy in pulmonary sarcoidosis patients with significant pulmonary symptoms and/or progressive disease as determined by radiology or lung function. In the latter case, treatment is primarily aimed at preventing organ damage, although previous studies did not conclusively demonstrate a beneficial effect in preventing disease progression or pulmonary fibrosis. Nevertheless, current treatment guidelines advocate relatively long and high initial treatment regimes, without clear evidence for the optimal dose and duration of treatment. Importantly, prolonged treatment with prednisone is known to induce considerable side-effects/co-morbidity. Several expert-opinions suggest a lower initial dose and shorter initial phase, based on retrospective case series. However, there is considerable variety in the response to treatment between sarcoidosis individuals and immunological mechanisms underlying this phenomenon have not been elucidated yet. These data suggest that treatment of sarcoidosis patients should be individualized, pursuing the lowest prednisone dose for the shortest period possible. However, prospective data on the early response towards prednisone treatment and tapering is lacking in a well-characterized cohort of pulmonary sarcoidosis patients. Against this background, we aim to describe the pulmonary response to corticosteroid therapy and tapering in a cohort of newly treated pulmonary sarcoidosis patients, using a hand-held spirometer. With results of this study, in the future, we hope to individualize the treatment and tapering strategy for sarcoidosis patients. This could lead to prednisone dose sparing, reduction in co-morbidity and an increased quality of life of sarcoidosis patients. This treatment strategy has already been performed successfully in other chronic pulmonary diseases requiring prednisone treatment, like asthma.
- Main changes (audit trail)20-jan-2015: Amendement:
- Inclusion criterium changed into: "A forced vital capacity (FVC) ≤ 80% of predicted AND/OR an absolute decline of ≥ 10% predicted PVC within 12 months"
- Change in interventions: "3. During the regular visits at the outpatient clinic, patient reported response to therapy will be objectified by two standardized questionnaires (SGRQ, SF-36, KSQ), in-hospital pulmonary function tests at the department of pulmonology, a chest X-ray, thorax-HRCT (only if physician’s choice), weight and laboratory data will be recorded;
5. When available, the long-term outcome of prednisone treatment will be assessed at 2, 3 and 5 years following treatment initiation via patient records described at routine follow-up visits at the outpatient clinics."
- Secondary outcome changed: "2. Patient reported pulmonary complaints objectified in dyspnoea (Medical Research Council (MRC) scale), quality of life (King’s Sarcoidosis Questionnaire (KSQ), St. George Respiratory Questionaire (SGRQ)), general health (SF-36) and fatigue scores (Fatigue Assessment Scale (FAS));"
- RECORD19-dec-2013 - 21-jan-2015


  • Indien u gegevens wilt toevoegen of veranderen, kunt u een mail sturen naar nederlands@trialregister.nl