Pulmonary SarcoidosisPhase 3HySSASNCT02200146
Hydroxychloroquine as Steroid-Sparing Agent in Pulmonary Sarcoidosis (HySSAS).
A Phase 3 study testing Prednisone and Hydroxychloroquine + Prednisone in people with Pulmonary Sarcoidosis, run by University of Milano Bicocca at 1 site in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Pulmonary Sarcoidosis
- What is being tested?
- Prednisone and Hydroxychloroquine + Prednisone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 94 participants
- Where
- 1 siteItaly
- Study dates
- Mar 2009to Sep 2013
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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%
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Hydroxychloroquine as Steroid-Sparing Agent in Pulmonary Sarcoidosis (HySSAS). A Multicenter, Prospective, Controlled, Randomized Trial.
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.
- Conditions
- Pulmonary Sarcoidosis
- Being tested
- Prednisone, Hydroxychloroquine + Prednisone
- Keywords
- sarcoidosis, treatment, lung, glucocorticoids, hydroxychloroquine
- Medical subject headings
- Sarcoidosis, Pulmonary, Sarcoidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PrednisoneDrug
Prednisone per os 0,5 mg/kg/die once/day for 3 months. After 3 months, between responders, prednisone was slowly tapered (5 mg/week maintaining the new reduced dose for one week) to 0,2 mg/kg/die for further 6 months.
Hydroxychloroquine + PrednisoneDrug
Hydroxychloroquine per os 200 mg/die (or adjusted for body weight if less than 61 kg), twice/day + prednisone 0,15 mg/kg per os daily, once/day for 3 months, than for further 6 months between responders.
| Study group | Role | What they receive |
|---|---|---|
| Prednisone | Active comparator | Prednisone per os 0,5 mg/kg/die once/day for 3 months. After 3 months, between responders, prednisone was slowly tapered (5 mg/week maintaining the new reduced dose for one week) to 0,2 mg/kg/die for further 6 months. Receives: Drug: Prednisone |
| Hydroxychloroquine + Prednisone | Experimental | Hydroxychloroquine per os 200 mg/die (or adjusted for body weight if less than 61 kg), twice/day + prednisone 0,15 mg/kg per os daily, once/day for 3 months, than for further 6 months between responders. Receives: Drug: Hydroxychloroquine + Prednisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Subjects is considered clinically cured at the 3 month visit if they will have radiographic success (determined if Chest X-Ray is resolved or improved compared to the baseline; improvement was assessed if there were reduction in hilar adenopathies, less pulmonary involvement, changing in radiographic stage) PLUS a change in at least one of the followings: symptoms (determined by dyspnea or cough index score decrease compared to the baseline), and/or functional improvement (determined by an increase in % of predicted Forced Vital Capacity and/or increase in % of predicted Single-Breath Diffusion capacity of Lung for Carbon monoxide DLCO-SB compared to the baseline), and/or increase in resting Partial pressure of Oxygen in the artery blood (PaO2), and/or worst oxygen saturation increase during 6 Minute Walk Test (6MWT) and/or increase in distance walked at 6MWT, compared to the baseline
Measured: Baseline- After 3 months of treatment
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)
Measured: Baseline - After 9 months of treatment
Secondary outcomes (6)
Secondary EFFICACY endpoint was the change from baseline in radiographic success rate after 9 month of therapy (measured by High Resolution Chest Tomography HRCT)
The radiographic success is determined if HRCT is resolved or improved compared to the baseline after 9 months
Measured: Baseline- After 9 months of therapy
Secondary SAFETY endpoint was the change from baseline in Body Mass Index
Measured: Baseline - After 3, 6 and 9 months
Secondary SAFETY endpoint was the change from baseline in HbA1c
Measured: At 3, 6 and 9 months of therapy
Secondary SAFETY endpoint was the change from baseline in clinical laboratory tests (including inflammatory markers)
Measured: At 3, 6 and 9 months of therapy
Secondary SAFETY endpoint was the change from baseline in bone turnover markers and mineral metabolism
Measured: At months 3 and 9 from the start of therapy
Secondary safety endpoint was the number of participants with Serious and Non-Serious Adverse Events
Measured: Within the 9 months of therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2014 (estimated)
- Study startMar 2009
- Primary completionJul 2013
- Study completionSep 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary SarcoidosisStudies 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
- None (open label)
- Enrolment
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Milano Bicocca
- Sponsor type
- University, hospital or other
- Collaborators
- Agenzia Italiana del Farmaco
- Responsible party
- Sponsor
- Investigators
- Alberto Pesci, Principal investigator, Università Milano Bicocca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02200146
- Sponsor's protocol ID
- HySSAS-FARM639KLZ
- Acronym
- HySSAS
- Status last verified
- Jul 2014
- First submitted
- 10 Jul 2014
- Last updated
- 25 Jul 2014
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hydroxychloroquine as Steroid-Sparing Agent in Pulmonary Sarcoidosis (HySSAS). A Multicenter, Prospective, Controlled, Randomized Trial.
- ClinicalTrials.gov ID
- NCT02200146
- Acronym / sponsor ID
- HySSAS / HySSAS-FARM639KLZ
- Conditions
- Pulmonary Sarcoidosis
- Keywords
- sarcoidosis, treatment, lung, glucocorticoids, hydroxychloroquine
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- University of Milano BicoccaUniversity, hospital or other
- Collaborators
- Agenzia Italiana del Farmaco
- Study start
- Mar 2009
- Primary completion
- Jul 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 25 Jul 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Prednisone | Active comparator | Drug: PrednisonePrednisone per os 0,5 mg/kg/die once/day for 3 months. After 3 months, between responders, prednisone was slowly tapered (5 mg/week maintaining the new reduced dose for one week) to 0,2 mg/kg/die for further 6 months. |
| Hydroxychloroquine + Prednisone | Experimental | Drug: Hydroxychloroquine + PrednisoneHydroxychloroquine per os 200 mg/die (or adjusted for body weight if less than 61 kg), twice/day + prednisone 0,15 mg/kg per os daily, once/day for 3 months, than for further 6 months between responders. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (15 listed)
- 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
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 |
|---|---|---|
| PrednisonePrednisone per os 0,5 mg/kg/die once/day for 3 months. After 3 months, between responders, prednisone was slowly tapered (5 mg/week maintaining the new reduced dose for one week) to 0,2 mg/kg/die for further 6 months. | Drug | Prednisone |
| Hydroxychloroquine + PrednisoneHydroxychloroquine per os 200 mg/die (or adjusted for body weight if less than 61 kg), twice/day + prednisone 0,15 mg/kg per os daily, once/day for 3 months, than for further 6 months between responders. | Drug | Hydroxychloroquine + Prednisone |
Primary outcome
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.
Time frame: Baseline- After 3 months of treatment
Subjects is considered clinically cured at the 3 month visit if they will have radiographic success (determined if Chest X-Ray is resolved or improved compared to the baseline; improvement was assessed if there were reduction in hilar adenopathies, less pulmonary involvement, changing in radiographic stage) PLUS a change in at least one of the followings: symptoms (determined by dyspnea or cough index score decrease compared to the baseline), and/or functional improvement (determined by an increase in % of predicted Forced Vital Capacity and/or increase in % of predicted Single-Breath Diffusion capacity of Lung for Carbon monoxide DLCO-SB compared to the baseline), and/or increase in resting Partial pressure of Oxygen in the artery blood (PaO2), and/or worst oxygen saturation increase during 6 Minute Walk Test (6MWT) and/or increase in distance walked at 6MWT, compared to the baseline
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)
Time frame: Baseline - After 9 months of treatment
6 secondary outcomes
Secondary EFFICACY endpoint was the change from baseline in radiographic success rate after 9 month of therapy (measured by High Resolution Chest Tomography HRCT)
Time frame: Baseline- After 9 months of therapy
Secondary SAFETY endpoint was the change from baseline in Body Mass Index
Time frame: Baseline - After 3, 6 and 9 months
Secondary SAFETY endpoint was the change from baseline in HbA1c
Time frame: At 3, 6 and 9 months of therapy
Secondary SAFETY endpoint was the change from baseline in clinical laboratory tests (including inflammatory markers)
Time frame: At 3, 6 and 9 months of therapy
Secondary SAFETY endpoint was the change from baseline in bone turnover markers and mineral metabolism
Time frame: At months 3 and 9 from the start of therapy
Secondary safety endpoint was the number of participants with Serious and Non-Serious Adverse Events
Time frame: Within the 9 months of therapy
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2014.
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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.
Alberto Pesci principal investigator
Università Milano Bicocca
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Prednisone and Hydroxychloroquine + Prednisone in people with Pulmonary Sarcoidosis, run by University of Milano Bicocca at 1 site in Italy.
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 2 inclusion and 15 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 25 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
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. (time frame: Baseline- After 3 months of treatment). The study lists 2 primary outcomes in all. 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 25 Jul 2014.