MyelofibrosisPhase 1NCT02593760
A Study to Evaluate the Efficacy and Safety of Vismodegib in Combination With Ruxolitinib for the Treatment of Intermediate- or High-Risk Myelofibrosis (MF)
A Phase 1 study testing Ruxolitinib and Vismodegib in people with Myelofibrosis, run by Hoffmann-La Roche at 13 sites in 4 countries.
Trial summary
This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy and safety of vismodegib plus (+) ruxolitinib versus placebo + ruxolitinib in participants with intermediate- or high-risk MF. The study will be divided into 2 components. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis
- What is being tested?
- Ruxolitinib and Vismodegib
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 13 sitesin 4 countries
- Study dates
- 25 Jan 2016to 12 Jul 2017
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Pathologically confirmed diagnosis of primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF, according to the 2008 revised World Health Organization criteria
- Intermediate-1, intermediate-2, or high-risk according to the IWG-MRT Dynamic International Prognostic Scoring System
- Life expectancy >= 6 months
- Peripheral blood blast count of less than (<) 10%
- Palpable splenomegaly of greater than (>) 5 centimeters (cm) below the left costal margin
- Eastern Cooperative Oncology Group performance status of 0 to 2
- Adequate hepatic and renal function
Who cannot join
- Prior treatment with a Hedgehog or Janus kinase pathway inhibitor
- Treatment with strong cytochrome P450 3A4 inhibitors/inducers within 28 days prior to Day 1
- Prior therapy for the treatment of intermediate- or high-risk MF including chemotherapy, interferon, thalidomide, busulfan, lenalidomide, anagrelide, or androgens within 28 days prior to Day 1
- Prior splenectomy or splenic irradiation
- Inadequate bone marrow reserve
- Participants with any history of platelet counts of < 50,000/mccL or ANC of < 500/mL, except during treatment for myeloproliferative neoplasm or treatment with cytotoxic therapy for any other reason
- Planned allogeneic bone marrow transplant during the study
Trial locations and contacts
13 sites in 4 countries.
13 sites in 4 countries
United States6 sites
Oncology Hematology Care Inc
Cincinnati, Ohio, 45242
Florida Cancer Specialists-Broadway, Fort Myers
Fort Myers, Florida, 33908
Uni of Texas - Md Anderson Cancer Center; Dept of Leukemia
Houston, Texas, 77030
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
Florida Cancer Specialist, North Region
St. Petersburg, Florida, 33705
Florida Cancer Specialists
West Palm Beach, Florida, 33401
Canada3 sites
Tom Baker Cancer Centre-Calgary; Clinical Research Unit
Calgary, Alberta, T2N 4N2
Queen Elizabeth II Health Sciences Centre; Oncology
Halifax, Nova Scotia, B3H 2Y9
Centre Hospitalier De L'Universite De Montreal, Hopital Notre-Dame
Montreal, Quebec, H2L 4M1
Germany2 sites
Uniklinik RWTH Aachen; Med. Klinik IV; Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammz
Aachen, 52074
Campus Virchow-Klinikum Charité Centrum 14; Medizinische Klinik m.S. Hämatologie u. Onkologie
Berlin, 13353
Italy2 sites
Az. Osp. Di Careggi; Divisione Di Ematologia
Florence, Tuscany, 50135
A.O.U. Citta' Della Salute E Della Scienza-P.O. Molinette;S.C. Ematologia
Turin, Piedmont, 10126
About this study
Official title: A Phase IB/III, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Vismodegib in Combination With Ruxolitinib Versus Placebo and Ruxolitinib in Patients With Intermediate- or High-Risk Myelofibrosis
This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy and safety of vismodegib plus (+) ruxolitinib versus placebo + ruxolitinib in participants with intermediate- or high-risk MF. The study will be divided into 2 components. The Phase Ib portion of the study consists of participants receiving open-label vismodegib (150 milligrams [mg] orally [PO] once daily [QD]) + ruxolitinib (PO twice daily [BID]). A safety assessment will be performed after the first 10 participants have been treated for 6 weeks. An analysis for efficacy and safety is planned in the first 10 participants at Week 24. There will be a hold on participant screening and enrollment during this assessment. Another 10 participants may be enrolled, thereafter, to further assess efficacy and safety (at Week 24) before the initiation of the Phase III randomization portion of the study. Similarly, there will be another hold on participant screening and enrollment during this assessment. The participants enrolled in the Phase Ib portion of the study will continue to receive vismodegib (150 mg PO QD) + ruxolitinib (PO BID) for up to 48 weeks, if clinical benefit is observed after 24 weeks. The Phase III randomized, double-blind portion of the study will enroll approximately 84 participants. Participants will be randomly assigned in a 1:1 ratio (double blind) to receive either vismodegib (150 mg PO QD) + ruxolitinib (PO BID) or placebo (PO QD) + ruxolitinib (PO BID) for up to 48 weeks.
- Conditions
- Myelofibrosis
- Being tested
- Placebo, Ruxolitinib, Vismodegib
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PlaceboOther
Placebo will be administered PO QD for up to 48 weeks.
RuxolitinibDrug
Ruxolitinib will be administered PO BID at a starting dose depending on the participants's baseline platelet count for up to 48 weeks.
VismodegibDrug
Also called Erivedge
Vismodegib will be administered at a dose of 150 mg PO QD for up to 48 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Placebo + Ruxolitinib | Active comparator | Participants will receive placebo (PO QD) in combination with ruxolitinib (dose will depend on the participant's baseline platelet count) for up to 48 weeks. Receives: Other: Placebo; Drug: Ruxolitinib |
| Vismodegib + Ruxolitinib | Experimental | Participants will receive vismodegib (150 mg PO QD) in combination with ruxolitinib (dose will depend on the participant's baseline platelet count) for up to 48 weeks. Receives: Drug: Ruxolitinib; Drug: Vismodegib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants who Achieve a Greater Than or Equal to (>=) 35% Reduction in Spleen Volume from Baseline at Week 24
Determined by an Independent Review Committee (IRC) Using International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Revised Response Criteria
Measured: Week 24
Percentage of Participants with Complete Remission (CR) and Partial Remission (PR) at Week 24, as Determined by an IRC Using IWG-MRT Revised Response Criteria
Measured: Week 24
Secondary outcomes (20)
Plasma Vismodegib Concentration at Steady State
Measured: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Unbound Vismodegib Concentration at Steady State
Measured: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Alpha 1-Acid Glycoprotein Concentration at Steady State
Measured: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Percentage of Participants who Achieve a >= 35% Reduction in Spleen Volume from Baseline, as Determined by an IRC Using IWG-MRT Revised Response Criteria at Week 48
Measured: Baseline, Week 48
Percentage of Participants who Achieve a >= 35% Reduction in Spleen Volume from Baseline, as Determined by an Investigator at Weeks 24 and 48
Measured: Baseline, Weeks 24 and 48
Percentage of Participants with CR and PR, as Determined by an IRC Using IWG-MRT Revised Response Criteria at Week 48
Measured: Week 48
Percentage of Participants with CR and PR, as Determined by an Investigator at Weeks 24 and 48
Measured: Weeks 24 and 48
Percentage of Participants with Overall Response Rate (CR, PR, and Clinical Improvement) at Weeks 24 and 48, as Determined by an IRC Using IWG-MRT Revised Response Criteria
Measured: Weeks 24 and 48
Percentage of Participants with Overall Response Rate (CR, PR, and Clinical Improvement) at Weeks 24 and 48, as Determined by the Investigator Using IWG-MRT Revised Response Criteria
Measured: Weeks 24 and 48
Percentage of Participants who Achieve Anemia Response at Weeks 24 and 48, as Determined by the Investigator Using IWG-MRT Revised Response Criteria
Measured: Weeks 24 and 48
Percentage of Participants with Symptom Response (Participants who Achieve a >= 50% Reduction from Baseline in the Myeloproliferative Neoplasm Symptom Assessment Form [MPN-SAF] Total Symptom Score [TSS])
Measured: Baseline, Weeks 24 and 48
Duration of Response, as Determined by the Investigator and an IRC Using IWG-MRT Revised Response Criteria or Death from Any Cause During the Study
Measured: Baseline up to 28 days after the last dose of study drug (52 weeks)
Percentage of Participants with Improvement from Baseline in Bone Marrow Fibrosis at Weeks 24 and 48, as Determined by the Investigator Using the European Consensus Grading System
Measured: Baseline, Weeks 24 and 48
Percentage of Participants with Improvement from Baseline in Bone Marrow Fibrosis at Weeks 24 and 48, as Determined by Independent Pathology Review Using the European Consensus Grading System
Measured: Baseline, Weeks 24 and 48
Progression-Free Survival
Measured: Baseline up to the end of the study (up to 1 year after completing 48 weeks of treatment by the last participant)
Percentage of Participants who Achieve a >= 50% Reduction in Fatigue from Baseline to Weeks 24 and 48 as Measured by MPN-SAF TSS
Measured: Baseline, Weeks 24 and 48
Percentage of Participants who Achieve a >= 50% Reduction in Other Symptom and Impact Item Scores from Baseline to Weeks 24 and 48, as Measured by the MPN-SAF
Measured: Baseline, Weeks 24 and 48
Percentage of Participants who Achieve a Meaningful Improvement on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 Scale Scores from Baseline to Weeks 24 and 48
Meaningful improvement is defined as a 10-point change.
Measured: Baseline, Weeks 24 and 48
Overall Survival
Measured: Baseline up to the end of the study (up to 1 year after completing 48 weeks treatment by the last participant)
Percentage of Participants with Adverse Events (AEs)
Measured: Baseline up to Month 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2015 (estimated)
- Study start25 Jan 2016
- Primary completion29 Mar 2017
- Study completion12 Jul 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for MyelofibrosisStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02593760
- Sponsor's protocol ID
- WO29806
- Other IDs
- 2015-001620-33, 2015
- Status last verified
- May 2018
- First submitted
- 29 Oct 2015
- Last updated
- 11 May 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Couban S, Benevolo G, Donnellan W, Cultrera J, Koschmieder S, Verstovsek S, Hooper G, Hertig C, Tandon M, Dimier N, Malhi V, Passamonti F. A phase Ib study to assess the efficacy and safety of vismodegib in combination with ruxolitinib in patients with intermediate- or high-risk myelofibrosis. J Hematol Oncol. 2018 Sep 24;11(1):122. doi: 10.1186/s13045-018-0661-x.
PubMed 30249277
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IB/III, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Vismodegib in Combination With Ruxolitinib Versus Placebo and Ruxolitinib in Patients With Intermediate- or High-Risk Myelofibrosis
- ClinicalTrials.gov ID
- NCT02593760
- Acronym / sponsor ID
- WO29806
- Conditions
- Myelofibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 25 Jan 2016 (actual)
- Primary completion
- 29 Mar 2017 (actual)
- Study completion
- 12 Jul 2017 (actual)
- First posted
- 2 Nov 2015 (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.
This multicenter, randomized, double-blind, placebo-controlled study will evaluate the efficacy and safety of vismodegib plus (+) ruxolitinib versus placebo + ruxolitinib in participants with intermediate- or high-risk MF. The study will be divided into 2 components. The Phase Ib portion of the study consists of participants receiving open-label vismodegib (150 milligrams [mg] orally [PO] once daily [QD]) + ruxolitinib (PO twice daily [BID]). A safety assessment will be performed after the first 10 participants have been treated for 6 weeks. An analysis for efficacy and safety is planned in the first 10 participants at Week 24. There will be a hold on participant screening and enrollment during this assessment. Another 10 participants may be enrolled, thereafter, to further assess efficacy and safety (at Week 24) before the initiation of the Phase III randomization portion of the study. Similarly, there will be another hold on participant screening and enrollment during this assessment. The participants enrolled in the Phase Ib portion of the study will continue to receive vismodegib (150 mg PO QD) + ruxolitinib (PO BID) for up to 48 weeks, if clinical benefit is observed after 24 weeks. The Phase III randomized, double-blind portion of the study will enroll approximately 84 participants. Participants will be randomly assigned in a 1:1 ratio (double blind) to receive either vismodegib (150 mg PO QD) + ruxolitinib (PO BID) or placebo (PO QD) + ruxolitinib (PO BID) for up to 48 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo + Ruxolitinib | Active comparator | Other: Placebo, Drug: RuxolitinibParticipants will receive placebo (PO QD) in combination with ruxolitinib (dose will depend on the participant's baseline platelet count) for up to 48 weeks. |
| Vismodegib + Ruxolitinib | Experimental | Drug: Ruxolitinib, Drug: VismodegibParticipants will receive vismodegib (150 mg PO QD) in combination with ruxolitinib (dose will depend on the participant's baseline platelet count) for up to 48 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Pathologically confirmed diagnosis of primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF, according to the 2008 revised World Health Organization criteria
- Intermediate-1, intermediate-2, or high-risk according to the IWG-MRT Dynamic International Prognostic Scoring System
- Life expectancy >= 6 months
- Peripheral blood blast count of less than (<) 10%
- Palpable splenomegaly of greater than (>) 5 centimeters (cm) below the left costal margin
Key exclusion criteria (7 listed)
- Prior treatment with a Hedgehog or Janus kinase pathway inhibitor
- Treatment with strong cytochrome P450 3A4 inhibitors/inducers within 28 days prior to Day 1
- Prior therapy for the treatment of intermediate- or high-risk MF including chemotherapy, interferon, thalidomide, busulfan, lenalidomide, anagrelide, or androgens within 28 days prior to Day 1
- Prior splenectomy or splenic irradiation
- Inadequate bone marrow reserve
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 |
|---|---|---|
| PlaceboPlacebo will be administered PO QD for up to 48 weeks. | Other | Placebo + Ruxolitinib |
| RuxolitinibRuxolitinib will be administered PO BID at a starting dose depending on the participants's baseline platelet count for up to 48 weeks. | Drug | Placebo + Ruxolitinib, Vismodegib + Ruxolitinib |
| VismodegibAlso: ErivedgeVismodegib will be administered at a dose of 150 mg PO QD for up to 48 weeks. | Drug | Vismodegib + Ruxolitinib |
Primary outcome
Percentage of Participants who Achieve a Greater Than or Equal to (>=) 35% Reduction in Spleen Volume from Baseline at Week 24
Time frame: Week 24
Determined by an Independent Review Committee (IRC) Using International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Revised Response Criteria
Percentage of Participants with Complete Remission (CR) and Partial Remission (PR) at Week 24, as Determined by an IRC Using IWG-MRT Revised Response Criteria
Time frame: Week 24
20 secondary outcomes
Plasma Vismodegib Concentration at Steady State
Time frame: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Unbound Vismodegib Concentration at Steady State
Time frame: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Alpha 1-Acid Glycoprotein Concentration at Steady State
Time frame: Predose (0 hour) on Weeks 6, 12, 24, 36, and 48
Percentage of Participants who Achieve a >= 35% Reduction in Spleen Volume from Baseline, as Determined by an IRC Using IWG-MRT Revised Response Criteria at Week 48
Time frame: Baseline, Week 48
Percentage of Participants who Achieve a >= 35% Reduction in Spleen Volume from Baseline, as Determined by an Investigator at Weeks 24 and 48
Time frame: Baseline, Weeks 24 and 48
Percentage of Participants with CR and PR, as Determined by an IRC Using IWG-MRT Revised Response Criteria at Week 48
Time frame: Week 48
Percentage of Participants with CR and PR, as Determined by an Investigator at Weeks 24 and 48
Time frame: Weeks 24 and 48
Percentage of Participants with Overall Response Rate (CR, PR, and Clinical Improvement) at Weeks 24 and 48, as Determined by an IRC Using IWG-MRT Revised Response Criteria
Time frame: Weeks 24 and 48
Percentage of Participants with Overall Response Rate (CR, PR, and Clinical Improvement) at Weeks 24 and 48, as Determined by the Investigator Using IWG-MRT Revised Response Criteria
Time frame: Weeks 24 and 48
Percentage of Participants who Achieve Anemia Response at Weeks 24 and 48, as Determined by the Investigator Using IWG-MRT Revised Response Criteria
Time frame: Weeks 24 and 48
Percentage of Participants with Symptom Response (Participants who Achieve a >= 50% Reduction from Baseline in the Myeloproliferative Neoplasm Symptom Assessment Form [MPN-SAF] Total Symptom Score [TSS])
Time frame: Baseline, Weeks 24 and 48
Duration of Response, as Determined by the Investigator and an IRC Using IWG-MRT Revised Response Criteria or Death from Any Cause During the Study
Time frame: Baseline up to 28 days after the last dose of study drug (52 weeks)
Percentage of Participants with Improvement from Baseline in Bone Marrow Fibrosis at Weeks 24 and 48, as Determined by the Investigator Using the European Consensus Grading System
Time frame: Baseline, Weeks 24 and 48
Percentage of Participants with Improvement from Baseline in Bone Marrow Fibrosis at Weeks 24 and 48, as Determined by Independent Pathology Review Using the European Consensus Grading System
Time frame: Baseline, Weeks 24 and 48
Progression-Free Survival
Time frame: Baseline up to the end of the study (up to 1 year after completing 48 weeks of treatment by the last participant)
Percentage of Participants who Achieve a >= 50% Reduction in Fatigue from Baseline to Weeks 24 and 48 as Measured by MPN-SAF TSS
Time frame: Baseline, Weeks 24 and 48
Percentage of Participants who Achieve a >= 50% Reduction in Other Symptom and Impact Item Scores from Baseline to Weeks 24 and 48, as Measured by the MPN-SAF
Time frame: Baseline, Weeks 24 and 48
Percentage of Participants who Achieve a Meaningful Improvement on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 Scale Scores from Baseline to Weeks 24 and 48
Time frame: Baseline, Weeks 24 and 48
Overall Survival
Time frame: Baseline up to the end of the study (up to 1 year after completing 48 weeks treatment by the last participant)
Percentage of Participants with Adverse Events (AEs)
Time frame: Baseline up to Month 48
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 11 May 2018.
- Derived from this study: PubMed 30249277Couban S, Benevolo G, Donnellan W, Cultrera J, Koschmieder S, Verstovsek S, Hooper G, Hertig C, Tandon M, Dimier N, Malhi V, Passamonti F. A phase Ib study to assess the efficacy and safety of vismodegib in combination with ruxolitinib in patients with intermediate- or high-risk myelofibrosis. J Hematol Oncol. 2018 Sep 24;11(1):122. doi: 10.1186/s13045-018-0661-x.
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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.
Clinical Trials study director
Hoffmann-La Roche
13 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ruxolitinib and Vismodegib in people with Myelofibrosis, run by Hoffmann-La Roche at 13 sites in 4 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 7 inclusion and 7 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 11 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Canada, Germany, 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Percentage of Participants who Achieve a Greater Than or Equal to (>=) 35% Reduction in Spleen Volume from Baseline at Week 24 (time frame: Week 24). 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 NCT02593760, 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 11 May 2018.