Aggressive Systemic MastocytosisPhase 2NCT02415608
Ibrutinib in Treating Patients With Advanced Systemic Mastocytosis
A Phase 2 study testing Ibrutinib in people with Aggressive Systemic Mastocytosis and Mast Cell Leukemia, run by Jason Robert Gotlib at 1 site in United States.
Trial summary
This phase 2 trial studies ibrutinib to see how well it works in treating patients with systemic (affecting the entire body) mastocytosis that has spread to other parts of the body and usually cannot be cured or controlled with treatment (advanced). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aggressive Systemic Mastocytosis, Mast Cell Leukemia, Systemic Mastocytosis
- What is being tested?
- Ibrutinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2015to 14 Jun 2017
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of systemic mastocytosis per 2008 World Health Organization (WHO) criteria. Those with advanced systemic mastocytosis (ASM); mast cell leukemia (MCL); or systemic mastocytosis-associated hematological clonal non-mast cell lineage disease (SM-AHNMD) required to have at least 1 organ damage finding
- Serum aspartate transaminase (AST) or alanine transaminase (ALT) ≤ 3.0 x upper limit of normal (ULN); if considered related to ASM/MCL ≤ 5 x ULN
- Estimated creatinine clearance ≥ 30 mL/min (Cockcroft-Gault)
- Total bilirubin ≤ 1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin); if considered related to ASM/MCL ≤ 3 x ULN
- Female subjects must be of non-reproductive potential, or if of childbearing potential must have a negative serum pregnancy test upon study entry
- Must agree to use highly effective methods of birth control
Show all 9Show fewer
- Written informed consent
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 3
- Life expectancy > 12 weeks
Who cannot join
- Received any investigational agent, chemotherapy, interferon-alpha, or 2-chlorodeoxyadenosine (2-CdA, cladribine) within 30 days prior to day 1; or monoclonal antibody ≤ 6 weeks prior to first administration of study treatment (patients with an AHNMD with progressive leukocytosis who require control of their counts are permitted to receive hydroxyurea)
- Diagnosis of AHNMD requiring immediate cytoreductive therapy or targeted drugs (eg, acute myeloid leukemia [AML])
- History of other malignancies, except:
- Malignancy treated with curative intent and with no known active disease present for ≥ 3 years before the first dose of study drug, and at low risk for recurrence
- Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease
- Adequately treated carcinoma in situ without evidence of disease
Show all 32Show fewer
- Concurrent systemic immunosuppressant therapy (eg, cyclosporine A, tacrolimus, etc., or chronic administration [> 14 days] of > 10 mg/day of prednisone) within 28 days of the first dose of study drug
- Vaccinated with live, attenuated vaccines within 4 weeks of first dose of study drug
- Systemic treatment for infection completed ≤ 14 days before the first dose of study drug
- Unresolved toxicities from prior anti-cancer therapy, defined as having not resolved to Common Terminology Criteria for Adverse Event (CTCAE, version 4), grade 0 or 1, or to the levels dictated in the inclusion/exclusion criteria with the exception of alopecia
- Known bleeding disorders (eg, severe von Willebrand's disease) or severe hemophilia
- History of stroke or intracranial hemorrhage within 6 months prior to enrollment
- Known history of human immunodeficiency virus (HIV) or
- Active infection with hepatitis C virus (HCV) or hepatitis B virus (HBV)
- Major surgery within 4 weeks of first dose of study drug
- Any life-threatening illness, medical condition, or organ system dysfunction that could compromise the subject's safety or put the study outcomes at undue risk
- Currently active, clinically significant cardiovascular disease, such as uncontrolled arrhythmia or class 3 or 4 congestive heart failure as defined by the New York Heart Association Functional Classification; or a history of myocardial infarction, unstable angina, or acute coronary syndrome within 6 months prior to randomization
- Unable to swallow capsules or malabsorption syndrome
- Disease significantly affecting gastrointestinal function
- Resection of the stomach or small bowel
- Symptomatic inflammatory bowel disease
- Ulcerative colitis
- Partial or complete bowel obstruction
- Requires treatment with a strong cytochrome P450 (CYP) 3A4/5 inhibitor
- Lactating or pregnant
- Unwilling or unable to participate in all required study evaluations and procedures
- Unable to understand the purpose and risks of the study and to provide a signed and dated informed consent form (ICF) and authorization to use protected health information (in accordance with national and local subject privacy regulations)
- Known hypersensitivity to any excipient contained in the drug
- Received hematopoietic growth factor support within 14 days of day 1 of ibrutinib (Jehovah's witnesses may be given an erythropoiesis-stimulating agent before and during the trial in lieu of red blood cell transfusions but anemia and/or red blood cell (RBC) transfusion dependence cannot be used for response assessment in these patients)
- Presence of the factor interacting with poly(A) polymerase alpha (PAPOLA) and cleavage and polyadenylation specific factor 1 (CPSF1) (FIP1L1)-platelet-derived growth factor receptor, alpha polypeptide (PDGFRalpha) fusion even with resistance to imatinib (such patients are no longer defined as systemic mastocytosis by the WHO)
- Received any treatment with ibrutinib prior to study entry
- The concomitant use of warfarin or other vitamin K antagonists unless felt to be of significant clinical need; low molecular weight heparin or other anticoagulants may be used instead if anticoagulation is required
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University Hospitals and Clinics
Stanford, California, 94305
About this study
Official title: A Phase 2 Study of Ibrutinib in Advanced Systemic Mastocytosis
This phase 2 trial studies ibrutinib to see how well it works in treating patients with systemic (affecting the entire body) mastocytosis that has spread to other parts of the body and usually cannot be cured or controlled with treatment (advanced). Systemic mastocytosis is a disease in which too many mast cells (a type of immune system cell) are found throughout the body. Mast cells give off chemicals such as histamine that can cause flushing (a hot, red face), itching, abdominal cramps, muscle pain, nausea, vomiting, diarrhea, low blood pressure, and shock. Ibrutinib may stop the growth of mast cells by blocking some of the enzymes needed for cell growth.
Read the detailed description
PRIMARY OBJECTIVE:
Evaluate the response rate to ibrutinib in patients with advanced systemic mastocytosis (SM) (aggressive systemic mastocytosis [ASM] or mast cell leukemia [MCL], or SM-associated hematologic non-mast cell disorder [AHNMD]) by the end of 6 cycles (6 months).
SECONDARY OBJECTIVES:
- Evaluate the tolerability and safety profile of ibrutinib in patients with advanced SM.
- Evaluate the pharmacokinetic (PK) profile of ibrutinib in a subset of patients with advanced SM.
- Evaluate changes in histopathology (blood and bone marrow) of patients with advanced SM in response to ibrutinib therapy.
- Evaluate changes in mastocytosis related symptom scores and quality-of-life (QOL) using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF).
- Evaluate the duration of response (DoR) and time to response (TTR).
- Evaluate progression-free survival (PFS) and overall survival.
OUTLINE:
Patients receive ibrutinib orally (PO) once daily (QD) on days 1 to 28. Treatment repeats every 28 days for up to 6 months in the absence of disease progression or unacceptable toxicity. Patients achieving an unconfirmed or confirmed clinical improvement (CI), partial response (PR), or complete response (CR) by the end of course 6 will be permitted to continue maintenance courses of ibrutinib on an ongoing basis until loss of response/progressive disease, or unacceptable toxicity.
After completion of study treatment, patients are followed up for 30 days and then every 6 months thereafter.
- Being tested
- Ibrutinib
- Medical subject headings
- Mastocytosis, Systemic, Leukemia, Mast-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IbrutinibDrug
Also called Imbruvica, PCI-32765, BTK Inhibitor PCI-32765, CRA-032765
Given orally in 28-day cycles
| Study group | Role | What they receive |
|---|---|---|
| Ibrutinib 420 mg/day | Experimental | Participants receive ibrutinib daily on days 1 to 28, at 420 mg/day in 28-day cycles Receives: Drug: Ibrutinib |
| Ibrutinib 560 mg/day | Experimental | Participants receive ibrutinib daily on days 1 to 28, at 560 mg/day in 28-day cycles Receives: Drug: Ibrutinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
Overall response rate (ORR) is reported as the sum of the rates of participants achieving complete remission (CR), partial remission (PR), \& clinical improvement (CI). A clinical response is a response with duration of ≥ 12 weeks.
CR is defined as all 4 criteria:
- No presence of compact neoplastic mast cell aggregates
- Serum tryptase level < 20 ng/mL
- Peripheral blood count remission defined as absolute neutrophil count (ANC) ≥1 x 10e9/L + normal differential, Hb ≥11 g/dL, \& platelet count ≥100x10e9/L
- Complete resolution of palpable hepatosplenomegaly \& all biopsy-proven or suspected SM-related organ damage
PR is defined as all 3 criteria with response duration ≥12 weeks, that is not CR or progressive disease:
- ≥ 50% reduction in neoplastic mast cells
- Serum tryptase level reduced ≥50%
- Resolution of 1+ biopsy-proven or suspected systemic mastocytosis (SM)-related organ damage findings
CI is defined as any improvement in any of the above measures.
Measured: Up to 6 months
Secondary outcomes (10)
Number of Participants With Adverse Events
Adverse events will be graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03, and reported as the number and percentage of participants having any adverse event; by each grade of adverse event; and by affected body systems.
Measured: 30 days
Ibrutinib Pharmacokinetics (PK)
Plasma concentration-time profiles for each subject and mean plasma concentration-time profiles for each dose level will be plotted, plasma concentration data for ibrutinib at each time point will be summarized by descriptive statistics, and PK parameters such as maximum concentration (Cmax), minimum concentration, time at which the Cmax is reached, and area under the curve will be summarized with mean, geometric mean, medium, minimum, maximum, standard deviation, and coefficient of variation.
Measured: 28 days
Change of Mast Cell Burden
The change in the number of neoplastic mast cells in tissues (blood and/or bone marrow), ie, a measure of mast cell burden, will be assessed by immunophenotyping and/or immunohistochemistry (depending on patient and disease specifics) using mast cell markers, eg, CD25, CD30, CD117, tryptase, reticulin, Wright-Giemsa staining, and/or hematoxylin-eosin staining, in peripheral blood smears or bone marrow samples. For each participant, the data are used to collectively determine a single assessment for the number of mast cells present at baseline and after treatment. The outcome is reported as the median change in that level of mast cells, with full range, from baseline up to 2 years.
Measured: 2 years
Serum Tryptase Levels
Serum tryptase level is a surrogate marker for the desired histopathologic response, ie, reduction in mast cell burden. Serum tryptase levels are reported as the median of the percent reduction, with full range, from baseline up to 2 years.
Measured: 2 years
Total Symptom Score (TSS)
The totality of systemic mastocytosis was assessed by the total symptom score as measured by a Myeloproliferative Neoplasm Symptom Assessment Form modified for mast cell disorders [MPN-SAF (MCD)], and reported as the change in median score with standard deviation at baseline and 30 days. The MPN-SAF is a single, 27-question questionnaire that scores the following general measures on a scale of 0 (best) to 10 (worst): fatigue levels, effects of fatigue, satiety, pain, activity, concentration, dizziness, sleep, mood, anxiety, sexual function, itching, flushing, fever, weight loss, respiratory functions, diarrhea, lesions, and allergic reactions (some of these general terms may describe more than 1 assessment). The score on the MPN-SAF is the sum total of all 27 scores, and the range of scores is from a minimum of 0 (best; symptoms for all assessment absent) to a maximum of 270 (worst; score of 10 on all assessments).
Measured: 30 days
Change in Quality of Life (QoL)
The quality of life (QoL) component of the Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF) modified for mast cell symptoms, a scale of life quality ranking from 0 (best) to 10 (worst), was assessed at baseline and after 1 cycle of ibrutinib treatment (30 days), and reported as the median change in score with standard deviation.
Measured: 30 days
Duration of Response (DoR)
Duration of response (DoR) was assessed through 2 years of treatment, and reported as the median with standard deviation, with response duration censored at last response assessment in the event of death or progression not documented.
Measured: 2 years
Time-to-Response (TTR)
Time-to-response (TTR) was assessed through 2 years of treatment, and reported as the median with standard deviation, censored at last response assessment in the event of death or progression not documented.
Measured: 2 years
Progression-free Survival (PFS)
Participants were assessed for progression-free survival (PFS) from the start of treatment through 2 years of treatment. The outcome is reported as the number of participants who were alive without disease progression after 2 years of treatment.
Measured: 2 years
Overall Survival (OS)
Overall survival (OS) was assessed through 2 years of treatment, and recorded as the time from the start of treatment to either progression or death, with values censored at the last response assessment if the participant did not progress or die during that period. OS is reported as reported as the median with standard deviation.
Measured: 26 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2015 (estimated)
- Study startMar 2015
- Primary completion4 Nov 2016
- Study completion14 Jun 2017
Why it stopped: Slow accrual
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.
- Other trials for Aggressive Systemic MastocytosisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jason Robert Gotlib
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor-investigator, Jason Robert Gotlib, Stanford University
- Investigators
- Jason Gotlib, Principal investigator, Stanford University Hospitals and Clinics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02415608
- Sponsor's protocol ID
- IRB-31815
- Other IDs
- NCI-2014-02341, HEMMPD0021, P30CA124435
- Status last verified
- Aug 2018
- First submitted
- 24 Mar 2015
- Last updated
- 20 Sep 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of Ibrutinib in Advanced Systemic Mastocytosis
- ClinicalTrials.gov ID
- NCT02415608
- Acronym / sponsor ID
- IRB-31815
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow accrual
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Jason Robert GotlibUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- Mar 2015
- Primary completion
- 4 Nov 2016 (actual)
- Study completion
- 14 Jun 2017 (actual)
- First posted
- 14 Apr 2015 (estimated)
- Results
- Posted 20 Sep 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase 2 trial studies ibrutinib to see how well it works in treating patients with systemic (affecting the entire body) mastocytosis that has spread to other parts of the body and usually cannot be cured or controlled with treatment (advanced). Systemic mastocytosis is a disease in which too many mast cells (a type of immune system cell) are found throughout the body. Mast cells give off chemicals such as histamine that can cause flushing (a hot, red face), itching, abdominal cramps, muscle pain, nausea, vomiting, diarrhea, low blood pressure, and shock. Ibrutinib may stop the growth of mast cells by blocking some of the enzymes needed for cell growth.
Detailed description
PRIMARY OBJECTIVE:
Evaluate the response rate to ibrutinib in patients with advanced systemic mastocytosis (SM) (aggressive systemic mastocytosis [ASM] or mast cell leukemia [MCL], or SM-associated hematologic non-mast cell disorder [AHNMD]) by the end of 6 cycles (6 months).
SECONDARY OBJECTIVES:
- Evaluate the tolerability and safety profile of ibrutinib in patients with advanced SM.
- Evaluate the pharmacokinetic (PK) profile of ibrutinib in a subset of patients with advanced SM.
- Evaluate changes in histopathology (blood and bone marrow) of patients with advanced SM in response to ibrutinib therapy.
- Evaluate changes in mastocytosis related symptom scores and quality-of-life (QOL) using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF).
- Evaluate the duration of response (DoR) and time to response (TTR).
- Evaluate progression-free survival (PFS) and overall survival.
OUTLINE:
Patients receive ibrutinib orally (PO) once daily (QD) on days 1 to 28. Treatment repeats every 28 days for up to 6 months in the absence of disease progression or unacceptable toxicity. Patients achieving an unconfirmed or confirmed clinical improvement (CI), partial response (PR), or complete response (CR) by the end of course 6 will be permitted to continue maintenance courses of ibrutinib on an ongoing basis until loss of response/progressive disease, or unacceptable toxicity.
After completion of study treatment, patients are followed up for 30 days and then every 6 months thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ibrutinib 420 mg/day | Experimental | Drug: IbrutinibParticipants receive ibrutinib daily on days 1 to 28, at 420 mg/day in 28-day cycles |
| Ibrutinib 560 mg/day | Experimental | Drug: IbrutinibParticipants receive ibrutinib daily on days 1 to 28, at 560 mg/day in 28-day cycles |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis of systemic mastocytosis per 2008 World Health Organization (WHO) criteria. Those with advanced systemic mastocytosis (ASM); mast cell leukemia (MCL); or systemic mastocytosis-associated hematological clonal non-mast cell lineage disease (SM-AHNMD) required to have at least 1 organ damage finding
- Serum aspartate transaminase (AST) or alanine transaminase (ALT) ≤ 3.0 x upper limit of normal (ULN); if considered related to ASM/MCL ≤ 5 x ULN
- Estimated creatinine clearance ≥ 30 mL/min (Cockcroft-Gault)
- Total bilirubin ≤ 1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin); if considered related to ASM/MCL ≤ 3 x ULN
- Female subjects must be of non-reproductive potential, or if of childbearing potential must have a negative serum pregnancy test upon study entry
Key exclusion criteria (32 listed)
- Received any investigational agent, chemotherapy, interferon-alpha, or 2-chlorodeoxyadenosine (2-CdA, cladribine) within 30 days prior to day 1; or monoclonal antibody ≤ 6 weeks prior to first administration of study treatment (patients with an AHNMD with progressive leukocytosis who require control of their counts are permitted to receive hydroxyurea)
- Diagnosis of AHNMD requiring immediate cytoreductive therapy or targeted drugs (eg, acute myeloid leukemia [AML])
- History of other malignancies, except:
- Concurrent systemic immunosuppressant therapy (eg, cyclosporine A, tacrolimus, etc., or chronic administration [> 14 days] of > 10 mg/day of prednisone) within 28 days of the first dose of study drug
- Vaccinated with live, attenuated vaccines within 4 weeks of first dose of study drug
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 |
|---|---|---|
| IbrutinibAlso: Imbruvica, PCI-32765, BTK Inhibitor PCI-32765, CRA-032765Given orally in 28-day cycles | Drug | Ibrutinib 420 mg/day, Ibrutinib 560 mg/day |
Primary outcome
Overall Response Rate (ORR)
Time frame: Up to 6 months
Overall response rate (ORR) is reported as the sum of the rates of participants achieving complete remission (CR), partial remission (PR), \& clinical improvement (CI). A clinical response is a response with duration of ≥ 12 weeks. CR is defined as all 4 criteria: * No presence of compact neoplastic mast cell aggregates * Serum tryptase level < 20 ng/mL * Peripheral blood count remission defined as absolute neutrophil count (ANC) ≥1 x 10e9/L + normal differential, Hb ≥11 g/dL, \& platelet count ≥100x10e9/L * Complete resolution of palpable hepatosplenomegaly \& all biopsy-proven or suspected SM-related organ damage PR is defined as all 3 criteria with response duration ≥12 weeks, that is not CR or progressive disease: * ≥ 50% reduction in neoplastic mast cells * Serum tryptase level reduced ≥50% * Resolution of 1+ biopsy-proven or suspected systemic mastocytosis (SM)-related organ damage findings CI is defined as any improvement in any of the above measures.
10 secondary outcomes
Number of Participants With Adverse Events
Time frame: 30 days
Ibrutinib Pharmacokinetics (PK)
Time frame: 28 days
Change of Mast Cell Burden
Time frame: 2 years
Serum Tryptase Levels
Time frame: 2 years
Total Symptom Score (TSS)
Time frame: 30 days
Change in Quality of Life (QoL)
Time frame: 30 days
Duration of Response (DoR)
Time frame: 2 years
Time-to-Response (TTR)
Time frame: 2 years
Progression-free Survival (PFS)
Time frame: 2 years
Overall Survival (OS)
Time frame: 26 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Sep 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Jason Gotlib principal investigator
Stanford University Hospitals and Clinics
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ibrutinib in people with Aggressive Systemic Mastocytosis and Mast Cell Leukemia, run by Jason Robert Gotlib at 1 site in United States.
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 9 inclusion and 32 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: stopped early, as of the sponsor's last update on 20 Sep 2018. 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 United States. 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 20 Sep 2018). 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 Response Rate (ORR) (time frame: Up to 6 months). 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 20 Sep 2018.