Chronic Iron Overload Due to Transfusion-dependant AnemiasPhase 2NCT02125877
Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
A Phase 2 study testing Deferasirox dispersible tablet and Defearisox film-coated tablet in people with Chronic Iron Overload Due to Transfusion-dependant Anemias, run by Novartis Pharmaceuticals at 51 sites in 16 countries.
Trial summary
Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Chronic Iron Overload Due to Transfusion-dependant Anemias
- What is being tested?
- Deferasirox dispersible tablet and Defearisox film-coated tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 173 participants
- Where
- 51 sitesin 16 countries
- Study dates
- 8 Jul 2014to 24 Feb 2016
- 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female patients aged ≥ 10 years
- Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision.
- History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
- Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
Who cannot join
- Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
- Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
- ALT (SGPT) > 5xULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to screening visit 1.
- Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
- Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
- Liver disease with severity of Child-Pugh Class B or C
Trial locations and contacts
51 sites in 16 countries.
51 sites in 16 countries
Italy14 sites
Novartis Investigative Site
Brindisi, BR, 72100
Novartis Investigative Site
Cagliari, ITA, 09121
Novartis Investigative Site
Catania, CT, 95125
Novartis Investigative Site
Cona, FE, 44100
Novartis Investigative Site
Genova, GE, 16128
Novartis Investigative Site
Genova, GE, 16132
Novartis Investigative Site
Lecce, LE, 73100
Novartis Investigative Site
Milan, MI, 20122
Novartis Investigative Site
Milan, MI, 20162
Novartis Investigative Site
Naples, 80138
Novartis Investigative Site
Palermo, PA, 90127
Novartis Investigative Site
Palermo, PA, 90146
Novartis Investigative Site
Reggio Calabria, RC, 89100
Novartis Investigative Site
Verona, VR, 37126
Germany7 sites
Novartis Investigative Site
Berlin, 13353
Novartis Investigative Site
Dresden, 01307
Novartis Investigative Site
Goslar, 38642
Novartis Investigative Site
Hanover, 30170
Novartis Investigative Site
Leipzig, 04103
Novartis Investigative Site
Mannheim, Baden-Wurttemberg, 68305
Novartis Investigative Site
Potsdam, 14467
United States5 sites
Children's Hospital Boston Department of Hematology
Boston, Massachusetts, 02115
Lurie Children's Hospital of Chicago Onc Dept
Chicago, Illinois, 60611
Weill Cornell Medical College-Cornell University Onc Dept
New York, New York, 10021
Children's Hospital of Orange County Onc Dept
Orange, California, 92868-3874
Children's Hospital of Philadelphia Onc. Dept
Philadelphia, Pennsylvania, 19104-4399
Saudi Arabia4 sites
Novartis Investigative Site
Dammam, 15215
Novartis Investigative Site
Dammam, 40145
Novartis Investigative Site
Jeddah, 21589
Novartis Investigative Site
Riyadh, 11472
Greece3 sites
Novartis Investigative Site
Athens, GR, GR-115 27
Novartis Investigative Site
Patra - RIO, GR, 265 04
Novartis Investigative Site
Thessaloniki, GR, 546 42
Austria2 sites
Novartis Investigative Site
Linz, A-4010
Novartis Investigative Site
Vienna, 1140
France2 sites
Novartis Investigative Site
Lille, 59020
Novartis Investigative Site
Paris, 75010
Malaysia2 sites
Novartis Investigative Site
Kuala Lumpur, 50589
Novartis Investigative Site
Pulau Pinang, 10990
Spain2 sites
Novartis Investigative Site
Barcelona, 08041
Novartis Investigative Site
Madrid, 28033
Thailand2 sites
Novartis Investigative Site
Bangkok, 10400
Novartis Investigative Site
Bangkok, 10700
United Arab Emirates2 sites
Novartis Investigative Site
Al Ain - Abu Dhabi, United Arab Emirates
Novartis Investigative Site
Dubai, 9115
United Kingdom2 sites
Novartis Investigative Site
London, N19 5NF
Novartis Investigative Site
London, NW1 2PJ
About this study
Official title: A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance
- Being tested
- Deferasirox dispersible tablet, Defearisox film-coated tablet
- Keywords
- Iron overload, Chelation, Thalassemia, Myelodysplastic syndrome (MDS), Transfusional hemisiderosis, Deferasirox, ICL670, Dispersible tablet, Film-coated tablet
- Medical subject headings
- Iron Overload, Thalassemia, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferasirox dispersible tabletDrug
Also called ICL670, DT (dispersible tablet)
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
Defearisox film-coated tabletDrug
Also called ICL670, FCT (film-coated tablet)
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox dispersible tablet (DFX-DT) | Active comparator | Iron chelation naïve participants received DFX-DT 20 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 5 to 10 mg/kg/day, with a maximum dose of 40 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose. Receives: Drug: Deferasirox dispersible tablet |
| Deferasirox film-coated tablet (DFX-FCT) | Experimental | Participants received DFX-FCT 14 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 3.5 to 7 mg/kg/day, with a maximum dose of 28 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose Receives: Drug: Defearisox film-coated tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Safety as Measured by Frequency of Adverse Events
The percentage of participants with adverse events, serious adverse events and deaths was assessed.
Measured: 28 weeks
Overall Safety as Measured by Changes in Laboratory Values From Baseline
The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary.
Measured: baseline (BL), 30 weeks
Secondary outcomes (10)
Frequency of Selected Gastro-intestinal (GI) Adverse Events
The percentage of participants with any GI adverse event, diarrhea, constipation, nausea, vomiting, abdominal pain was assessed.
Measured: 28 weeks
Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
The modified SICT consisted of 13 items that represent 3 domains: adherence, satisfaction and concerns. The adherence domain consisted of 7 items, 6 which were measured using a 5-point response scale and was calculated by summing the 6 items. The score range from 6 to 30 and higher scores indicated worse adherence. The satisfaction domain consisted of 3 items, 2 which were measured using a 5-point response scale and was calculated by summing the 2 items. The score range from 2 to 10 and higher scores indicated worse satisfaction. The concerns domain consisted of 3 items to address any concerns or worries with his/her medication. All 3 items were measured on a 5-point response scale and were calculated by summing the 3 items. The score range from 3 to 15 and higher scores indicated fewer concerns. For all three domains, the meaningful difference between two treatment arms was determined to be 1 point.
Measured: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Palatability Questionnaire Score
The palatability questionnaire consisted of 4 items. The first item measured the taste and aftertaste of the medication and were scored a on a 5-point response scale. The second item offered an additional response option of "no aftertaste". The last 2 items referred to whether the medication was taken, i.e. swallowed or vomited, and how the participant perceived the amount of medication to be taken. The palatability summary score was calculated using a scoring matrix from items 1, 3 and 4 scores and the score ranges from 0 - 11. Higher scores indicated the best palatability. A meaningful difference between two treatment arms was determined to be 1 point.
Measured: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary
The GI symptom diary consisted of 6 items, five which were scored using a 0 - 10 rating scale with item appropriate anchors to rate the symptom, for example, Pain in your belly: 0 = no pain and 10 = worst pain. The GI diary summary score was created using the 10 point response scale for the 5 items. The GI symptom daily diary had a minimum score of 0 and a maximum score of 50. The weekly average score for the 7 days was calculated for each individual item and the GI summary score was created from these weekly averages. Higher scores indicated worse symptoms. A meaningful difference between two treatment arms was determined to be 0.3 point.
Measured: weeks -1, 4, 8, 12, 16, 20, 24
Number of Participants With Weekly Average Compliance of Medication Consumption
A compliance questionnaire assessed whether the medication was taken. Weekly average compliance was calculated when there were at least four non-missing daily responses.
Measured: Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
Weekly Dose Violation Rate
The dose violation is defined as a dose either missed completely or not taken in accordance with the timing instruction (no later than 12:00 pm. The rate was calculated as [number of dose violations/drug exposure (days)] x 100.
Measured: weeks 1, 4, 8, 12, 16, 20, 24
Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)
Blood samples were collected to assess AUClast.
Measured: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
Blood samples were collected to assess Cmax.
Measured: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)
Blood samples were collected to assess Tmax.
Measured: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Dererasirox Plasma Concentration
Blood samples were collected to assess deferasirox concentration. Dose-adjusted calculations are presented: (concentration/actual dose)*20 for participants on DFX-DT and (concentration/actual dose)*14 for participants on DFX-FCT.
Measured: Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2014 (estimated)
- Study start8 Jul 2014
- Primary completion24 Feb 2016
- Study completion24 Feb 2016
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 Chronic Iron Overload Due to Transfusion-dependant AnemiasStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 173 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
- NCT02125877
- Sponsor's protocol ID
- CICL670F2201
- Status last verified
- Jul 2017
- First submitted
- 26 Apr 2014
- Last updated
- 25 Jul 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Taher AT, Origa R, Perrotta S, Kouraklis A, Ruffo GB, Kattamis A, Goh AS, Huang V, Zia A, Herranz RM, Porter JB. Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias. Health Qual Life Outcomes. 2018 Nov 19;16(1):216. doi: 10.1186/s12955-018-1041-5.
PubMed 30453981
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
- ClinicalTrials.gov ID
- NCT02125877
- Acronym / sponsor ID
- CICL670F2201
- Keywords
- Iron overload, Chelation, Thalassemia, Myelodysplastic syndrome (MDS), Transfusional hemisiderosis, Deferasirox, ICL670, Dispersible tablet
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 173 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2014 (actual)
- Primary completion
- 24 Feb 2016 (actual)
- Study completion
- 24 Feb 2016 (actual)
- First posted
- 29 Apr 2014 (estimated)
- Results
- Posted 18 Oct 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Deferasirox dispersible tablet (DFX-DT) | Active comparator | Drug: Deferasirox dispersible tabletIron chelation naïve participants received DFX-DT 20 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 5 to 10 mg/kg/day, with a maximum dose of 40 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose. |
| Deferasirox film-coated tablet (DFX-FCT) | Experimental | Drug: Defearisox film-coated tabletParticipants received DFX-FCT 14 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 3.5 to 7 mg/kg/day, with a maximum dose of 28 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male and female patients aged ≥ 10 years
- Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision.
- History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
- Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
Key exclusion criteria (6 listed)
- Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
- Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
- ALT (SGPT) > 5xULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to screening visit 1.
- Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
- Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
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 |
|---|---|---|
| Deferasirox dispersible tabletAlso: ICL670, DT (dispersible tablet)Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use. | Drug | Deferasirox dispersible tablet (DFX-DT) |
| Defearisox film-coated tabletAlso: ICL670, FCT (film-coated tablet)Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use. | Drug | Deferasirox film-coated tablet (DFX-FCT) |
Primary outcome
Overall Safety as Measured by Frequency of Adverse Events
Time frame: 28 weeks
The percentage of participants with adverse events, serious adverse events and deaths was assessed.
Overall Safety as Measured by Changes in Laboratory Values From Baseline
Time frame: baseline (BL), 30 weeks
The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary.
10 secondary outcomes
Frequency of Selected Gastro-intestinal (GI) Adverse Events
Time frame: 28 weeks
Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
Time frame: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Palatability Questionnaire Score
Time frame: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary
Time frame: weeks -1, 4, 8, 12, 16, 20, 24
Number of Participants With Weekly Average Compliance of Medication Consumption
Time frame: Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
Weekly Dose Violation Rate
Time frame: weeks 1, 4, 8, 12, 16, 20, 24
Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)
Time frame: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
Time frame: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)
Time frame: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Dererasirox Plasma Concentration
Time frame: Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30453981Taher AT, Origa R, Perrotta S, Kouraklis A, Ruffo GB, Kattamis A, Goh AS, Huang V, Zia A, Herranz RM, Porter JB. Patient-reported outcomes from a randomized phase II study of the deferasirox film-coated tablet in patients with transfusion-dependent anemias. Health Qual Life Outcomes. 2018 Nov 19;16(1):216. doi: 10.1186/s12955-018-1041-5.
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
51 sites in 16 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Deferasirox dispersible tablet and Defearisox film-coated tablet in people with Chronic Iron Overload Due to Transfusion-dependant Anemias, run by Novartis Pharmaceuticals at 51 sites in 16 countries.
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 4 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 25 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 51 sites in Italy, Germany, United States, Saudi Arabia, Greece and 11 more countries. 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 18 Oct 2016). 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?
Overall Safety as Measured by Frequency of Adverse Events (time frame: 28 weeks). 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?
On the ClinicalTrials.gov record for NCT02125877, 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 25 Jul 2017.