Clinical trial NCT02200146: Hydroxychloroquine as Steroid-Sparing Agent in Pulmonary Sarcoidosis (HySSAS).
Official title: Hydroxychloroquine as Steroid-Sparing Agent in Pulmonary Sarcoidosis (HySSAS). A Multicenter, Prospective, Controlled, Randomized Trial.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pulmonary Sarcoidosis
Keywords: sarcoidosis
treatment
lung
glucocorticoids
hydroxychloroquine
Lead sponsor: University of Milano Bicocca
Sponsor class: OTHER
Collaborators: name: Agenzia Italiana del Farmaco
First posted: 2014-07-25
Enrollment: 94
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The aim of the study is determining the non-inferiority in the overall success rate and the safety for a combination therapy with hydroxychloroquine plus low dose glucocorticoids compared to that for high dose glucocorticoids at 3 and 9 months in patients with pulmonary sarcoidosis.


Eligibility criteria: Inclusion Criteria:

* patients between 18 and 70 years
* parenchymal pulmonary involvement at Chest X-Ray (CXR) AND one of the follows: physiologic abnormalities on pulmonary function testing and/or respiratory symptoms, and/or exercise-induced abnormalities.

Exclusion Criteria:

* Unable to understand protocol and to sign informed consent or not suitable candidate to comply with the requirements of this study, in the opinion of the investigator
* Cardiac and neurological sarcoidosis or any other organ involvement
* End stage lung disease at high-resolution computed tomography (HRCT)
* Clinical evidence of active infection
* Documented exposure to beryllium
* Patients with Forced Expiratory Volume at one second (FEV1) changes after salbutamol inhalation ≥20%
* Comorbidity: advanced liver cirrhosis or abnormal liver function, unstable cardiac disease, moderate to severe renal insufficiency, poorly controlled diabetes
* Pregnancy or lactation
* A tuberculin skin test (5 I.U.) more than 5 mm
* Psoriasis
* Homozygous glucose-6-phosphatase deficiency
* Known hypersensitivity to hydroxychloroquine or 4-aminoquinoline derivatives
* Visual field changes attributable to 4-aminoquinolines
* Concomitant therapies: any patient enrolled in the study must be off all prohibited medications at least 4 weeks before screening. Once patients completed the washout period, they may enter the screening period that may last up to 30 days
* Previous therapies: any patient enrolled must be off all medications for sarcoidosis at least 4 weeks before screening.

Primary outcomes:
measure: The primary EFFICACY measure is the per-subject overall success rate at the 3 month visit. Overall response is defined as a combined radiographic and clinical responses.
timeFrame: Baseline- After 3 months of treatment
measure: The primary SAFETY endpoint is the percent change in lumbar spine (L1-L4) bone mineral density from baseline to month 9 as measured by Dual energy X-ray Absorptiometry (DXA)
timeFrame: Baseline - After 9 months of treatment
Secondary outcomes:
measure: Secondary EFFICACY endpoint was the change from baseline in radiographic success rate after 9 month of therapy (measured by High Resolution Chest Tomography HRCT)
timeFrame: Baseline- After 9 months of therapy
measure: Secondary SAFETY endpoint was the change from baseline in Body Mass Index
timeFrame: Baseline - After 3, 6 and 9 months
measure: Secondary SAFETY endpoint was the change from baseline in HbA1c
timeFrame: At 3, 6 and 9 months of therapy
measure: Secondary SAFETY endpoint was the change from baseline in clinical laboratory tests (including inflammatory markers)
timeFrame: At 3, 6 and 9 months of therapy
measure: Secondary SAFETY endpoint was the change from baseline in bone turnover markers and mineral metabolism
timeFrame: At months 3 and 9 from the start of therapy
measure: Secondary safety endpoint was the number of participants with Serious and Non-Serious Adverse Events
timeFrame: Within the 9 months of therapy
Other outcomes:
Locations:
city: Milano
country: Italy
facility: Università degli Studi di Milano - Bicocca
Study officials:
affiliation: Università Milano Bicocca
name: Alberto Pesci
role: PRINCIPAL_INVESTIGATOR
References: