Mevalonate Kinase DeficiencyPhase 2NCT01303380
Canakinumab in Patients With Active Hyper-IgD Syndrome
A Phase 2 study testing Canakinumab in people with Mevalonate Kinase Deficiency, run by Novartis Pharmaceuticals at 3 sites in Spain.
Trial summary
This pilot study is designed to evaluate the efficacy, the safety, and the pharmacokinetics (PK) / pharmacodynamics (PD) of canakinumab treatment in patients with HIDS. Read more about this study
- Who is it for?
- Ages 24 months and older
- Conditions the study is for
- Mevalonate Kinase Deficiency
- What is being tested?
- Canakinumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 3 sitesSpain
- Study dates
- Mar 2011to Jul 2014
- Phase
- Phase 2
- 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 24 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with a diagnosis of HIDS proven by DNA analysis and/or enzymatic studies.
- At time of start of drug treatment: active HIDS as evidenced by a physician global assessment of HIDS flare severity ≥ 2 and CRP values >10 mg/L (normal CRP < or = 10 mg/L).
- Patients who have a history of > or = 3 febrile acute HIDS flares in a 6-month period when not receiving prophylaxis treatment (e.g. anakinra daily treatment) with a duration of each flare lasting > or = 4 days and limiting the normal daily activities.
Who cannot join
- Pregnant or nursing (lactating) women.
- History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result.
- Positive Hepatitis B or Hepatitis C.
- Live vaccinations within 3 months prior to the start of the trial
- Positive tuberculosis screening test.
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
3 sites in 1 country.
Spain3 sites
Novartis Investigative Site
Esplugues de Llobregat, Barcelona, 08950
Novartis Investigative Site
Madrid, Madrid, 28046
Novartis Investigative Site
Valencia, Valencia, 46026
About this study
Official title: An Open-label, Multicenter, Efficacy and Safety Pilot Study of 6-month Canakinumab Treatment With up to 6-month Follow-up in Patients With Active Hyper-IgD Syndrome (HIDS)
This pilot study is designed to evaluate the efficacy, the safety, and the pharmacokinetics (PK) / pharmacodynamics (PD) of canakinumab treatment in patients with HIDS.
- Conditions
- Mevalonate Kinase Deficiency
- Being tested
- Canakinumab
- Keywords
- Hyper IgD syndrome, canakinumab, HIDS, mevalonate kinase deficiency, MKD
- Medical subject headings
- Mevalonate Kinase Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CanakinumabDrug
| Study group | Role | What they receive |
|---|---|---|
| Canakinumab | Experimental | Not described Receives: Drug: Canakinumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Flares Per Participant During Historical Period and Treatment Period
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a C-reactive protein (CRP) value > 10 mg/L. Flares during a historical period were defined as most recent 6-months in which the participant has not received treatment for their HIDS other than symptomatic treatment with NSAIDs and/or corticosteroids.
Measured: Historical period, Month 6 (End of treatment period)
Secondary outcomes (22)
Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value > 10 mg/L.
Measured: Month 6 (End of treatment period), Month 36 (End of Long term treatment Period 2)
Number of Participants Who Flared at Month 6, Month 24 and Month 36
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value > 10 mg/L.
Measured: Baseline, Month 6 (End of treatment period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Physician global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5- point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe.
Measured: Any flare event [Baseline up to Month 36 (End of long term treatment period 2)]
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Participant's global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5-point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe. Same investigator assessed the same participant throughout the study to ensure consistency between assessments. Investigators reviewed every participant's diary at each visit after their own clinical assessment.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Participants were assessed by participants/parent (participants aged 6-18 years) for control of signs and symptoms associated with HIDS based on 5-point scale: 0 = No control; 1 = Poor control; 2 = Somewhat control; 3 = Good control; and 4= Excellent control.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Participants were assessed by physician for control of signs and symptoms associated with HIDS based on 5-point scale: 0 = No control; 1 = Poor control; 2 = Somewhat control; 3 = Good control; and 4= Excellent control.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Fever severity was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Apthus ulcers were assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Lymphadenopathy severity was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Abdominal pain was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Time to Resolution of the Initial Flare After First Canakinumab Treatment
Time to resolution of the initial flare after first dose of canakinumab was determined.
Measured: Day 1 (Baseline), Day 28
Change From Baseline in Inflammation Markers Over Time up to Month 24
The C-reactive Protein (CRP) and/or Serum amyloid A protein (SAA) were used as inflammatory markers. The normal range of CRP was 0-10 mg/L.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time
Participants were assessed for health-related quality of life (HRQoL) based on Health Assessment Questionnaire (HAQ). HAQ was an eight 8 categories questionnaire representing all activities related to physical function. Each category has various sub-categories, which were rated by the participants on a 4- point difficulty scale: 0 = any difficulty; 1 = some difficulty; 2 = much difficulty; 3 = unable to do. The total score was the mean of the 8 scores, and ranged from 0 (no disability) to 3 (completely disabled).
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time
Participants or their parents (participants aged 6 to 17 years) were assessed for HRQoL based on Childhood Health Assessment Questionnaire (CHAQ). CHAQ was an eight domain questionnaire representing functional capacity and independence, evaluated for previous week. Each domain was rated on a 4-point difficulty scale: 0 = any difficulty; 1 = some difficulty; 2 = much difficulty; 3 = unable to do.The total score is the mean from the 8 scores, and ranges from 0 (no disability) to 3 (completely disabled).
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Who Received Dose Up-titration During 6-month Treatment Period
Participants who experienced a new HIDS flare between baseline and Week 4 and received an escalated dose of 450 mg of canakinumab every 6 weeks thereafter starting at Week 6 were determined.
Measured: Day 1 up to Month 6 (End of follow up)
Duration of Flares Experienced During the Study
Flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value > 10 mg/L. The change in post canakinumab treatment flare duration during the study were assessed as compared to historical period.
Measured: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Time to Flare After the Last Dose of Canakinumab During the Follow-up Period
The median time to flare by the participants after administration of the last dose of canakinumab during the follow-up period was analysed using Kaplan-Meier method.
Measured: Last dose of canakinumab treatment in follow-up period to end of follow-up period (Day 337)
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalisation, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards.
Measured: Day 1 (Start of study treatment) up to Month 36 (End of study)
Participants Who Received Rescue Treatment
Participants who experienced flares were treated with corticosteroids and NSAIDs as rescue medication.
Measured: Baseline up to Month 36 (End of study)
Serum Concentration-time Profile of Canakinumab
Canakinumab concentrations in serum were assessed for evaluating pharmacokinetics (PK) of the drug.
Measured: Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose)
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Pharmacodynamics of canakinumab was assessed by total IL-1β (sum of free and bound canakinumab) concentration, determined in serum by means of sandwich ELISA assay with limit of detection at 0.1 picogram/millilitre.
Measured: Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose)
Number of Participants Exhibiting Anti-canakinumab Antibodies at Any Visit
Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using bridging ECLIA assay.
Measured: Baseline up to Month 36 (End of study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2011 (estimated)
- Study startMar 2011
- Primary completionJul 2014
- Study completionJul 2014
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 (1)Papers the sponsor has linked to this study.
- Other trials for Mevalonate Kinase DeficiencyStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01303380
- Sponsor's protocol ID
- CACZ885D2402
- Other IDs
- 2010-020904-31
- Status last verified
- Oct 2015
- First submitted
- 23 Feb 2011
- Last updated
- 5 Nov 2015
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Arostegui JI, Anton J, Calvo I, Robles A, Iglesias E, Lopez-Montesinos B, Banchereau R, Hong S, Joubert Y, Junge G, Pascual V, Yague J. Open-Label, Phase II Study to Assess the Efficacy and Safety of Canakinumab Treatment in Active Hyperimmunoglobulinemia D With Periodic Fever Syndrome. Arthritis Rheumatol. 2017 Aug;69(8):1679-1688. doi: 10.1002/art.40146. Epub 2017 Jul 5.
PubMed 28482144
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multicenter, Efficacy and Safety Pilot Study of 6-month Canakinumab Treatment With up to 6-month Follow-up in Patients With Active Hyper-IgD Syndrome (HIDS)
- ClinicalTrials.gov ID
- NCT01303380
- Acronym / sponsor ID
- CACZ885D2402
- Conditions
- Mevalonate Kinase Deficiency
- Keywords
- Hyper IgD syndrome, canakinumab, HIDS, mevalonate kinase deficiency, MKD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Mar 2011
- Primary completion
- Jul 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 24 Feb 2011 (estimated)
- Results
- Posted 19 Aug 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This pilot study is designed to evaluate the efficacy, the safety, and the pharmacokinetics (PK) / pharmacodynamics (PD) of canakinumab treatment in patients with HIDS.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Canakinumab | Experimental | Drug: Canakinumab |
Who may qualify
- Ages
- 24 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patients with a diagnosis of HIDS proven by DNA analysis and/or enzymatic studies.
- At time of start of drug treatment: active HIDS as evidenced by a physician global assessment of HIDS flare severity ≥ 2 and CRP values >10 mg/L (normal CRP < or = 10 mg/L).
- Patients who have a history of > or = 3 febrile acute HIDS flares in a 6-month period when not receiving prophylaxis treatment (e.g. anakinra daily treatment) with a duration of each flare lasting > or = 4 days and limiting the normal daily activities.
Key exclusion criteria (6 listed)
- Pregnant or nursing (lactating) women.
- History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result.
- Positive Hepatitis B or Hepatitis C.
- Live vaccinations within 3 months prior to the start of the trial
- Positive tuberculosis screening test.
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 |
|---|---|---|
| Canakinumab | Drug | Canakinumab |
Primary outcome
Number of Flares Per Participant During Historical Period and Treatment Period
Time frame: Historical period, Month 6 (End of treatment period)
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a C-reactive protein (CRP) value > 10 mg/L. Flares during a historical period were defined as most recent 6-months in which the participant has not received treatment for their HIDS other than symptomatic treatment with NSAIDs and/or corticosteroids.
22 secondary outcomes
Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period
Time frame: Month 6 (End of treatment period), Month 36 (End of Long term treatment Period 2)
Number of Participants Who Flared at Month 6, Month 24 and Month 36
Time frame: Baseline, Month 6 (End of treatment period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Time frame: Any flare event [Baseline up to Month 36 (End of long term treatment period 2)]
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Time to Resolution of the Initial Flare After First Canakinumab Treatment
Time frame: Day 1 (Baseline), Day 28
Change From Baseline in Inflammation Markers Over Time up to Month 24
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Percentage of Participants Who Received Dose Up-titration During 6-month Treatment Period
Time frame: Day 1 up to Month 6 (End of follow up)
Duration of Flares Experienced During the Study
Time frame: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)
Time to Flare After the Last Dose of Canakinumab During the Follow-up Period
Time frame: Last dose of canakinumab treatment in follow-up period to end of follow-up period (Day 337)
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 (Start of study treatment) up to Month 36 (End of study)
Participants Who Received Rescue Treatment
Time frame: Baseline up to Month 36 (End of study)
Serum Concentration-time Profile of Canakinumab
Time frame: Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose)
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Time frame: Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose)
Number of Participants Exhibiting Anti-canakinumab Antibodies at Any Visit
Time frame: Baseline up to Month 36 (End of study)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 28482144Arostegui JI, Anton J, Calvo I, Robles A, Iglesias E, Lopez-Montesinos B, Banchereau R, Hong S, Joubert Y, Junge G, Pascual V, Yague J. Open-Label, Phase II Study to Assess the Efficacy and Safety of Canakinumab Treatment in Active Hyperimmunoglobulinemia D With Periodic Fever Syndrome. Arthritis Rheumatol. 2017 Aug;69(8):1679-1688. doi: 10.1002/art.40146. Epub 2017 Jul 5.
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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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Canakinumab in people with Mevalonate Kinase Deficiency, run by Novartis Pharmaceuticals at 3 sites in Spain.
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 3 inclusion and 6 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 5 Nov 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Spain. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 19 Aug 2015). 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?
Number of Flares Per Participant During Historical Period and Treatment Period (time frame: Historical period, Month 6 (End of treatment period)). 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 NCT01303380, which also lists 1 linked publication.
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 5 Nov 2015.