BRAF Mutant Metastatic MelanomaPhase 1/2NCT01512251
BKM120 Combined With Vemurafenib (PLX4032) in BRAFV600E/K Mutant Advanced Melanoma
A Phase 1/2 study testing BKM120 Combined with Vemurafenib (PLX4032) in people with BRAF Mutant Metastatic Melanoma, run by University of California, San Francisco at 1 site in United States.
Trial summary
This is a phase 1/2 clinical trial with the goal of determining whether the addition of the investigational agent BKM120 to vemurafenib will lead to improved 6-month progression-free survival in patients with BRAFV600E/K mutant melanoma. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- BRAF Mutant Metastatic Melanoma
- What is being tested?
- BKM120 Combined with Vemurafenib (PLX4032)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Jun 2012to 21 Mar 2017
- Phase
- Phase 1/2
- 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
- Histologically or cytologically confirmed diagnosis of unresectable stage III and stage IV melanoma
- BRAFV600E or BRAFV600K mutation-positive
- Age >= 18 years
- Eastern Cooperative Oncology Group (ECOG) performance status <= 2
- Patients must have at least one site of measurable disease (per RECIST for solid tumors)
- Life expectancy of ≥ 12 weeks
Show all 18Show fewer
- Adequate bone marrow function as shown by: Absolute Neutrophil Count (ANC) >= 1.5 x 109/L, Platelets >= 100 x 109/L, Hb >9 g/dL
- Total calcium (corrected for serum albumin) within normal limits (biphosphonate use for malignant hypercalcemia control is not allowed)
- Magnesium ≥ the lower limit of normal
- Potassium within normal limits for the institution
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) within normal range (or <= 3.0 x upper limit of normal (ULN) if liver metastases are present)
- Serum bilirubin within normal range (or <= 1.5 x ULN if liver metastases are present; or total bilirubin <= 3.0 x ULN with direct bilirubin within normal range in patients with well documented Gilbert Syndrome)
- Serum creatinine <= 1.5 x ULN or 24-hour clearance >= 50 mL/min
- Serum amylase <= ULN
- Serum lipase <= ULN
- International Normalized Ratio (INR) <= 2
- Fasting plasma glucose <= 120 mg/dL (6.7 mmol/L)
- Negative serum pregnancy test within 48 hours before starting study treatment
Who cannot join
- Patients who have received prior treatment with a PI3K inhibitor or a BRAF inhibitor, prior treatment with sorafenib is permitted.
- Patients with a known hypersensitivity to BKM120 or to its excipients
- Patients with untreated brain metastases are excluded; however, patients with metastatic central nervous system (CNS) tumors may participate in this trial, if the patient is > 4 weeks from therapy completion (incl. radiation and/or surgery) and clinically stable at the time of study entry
- Patients with acute or chronic liver, renal disease or pancreatitis
- Patients with the following mood disorders as judged by the Investigator or a psychiatrist, or as a result of patient's mood assessment questionnaire:
- Medically documented history of or active major depressive episode, bipolar disorder (I or II), obsessive-compulsive disorder, schizophrenia, history of suicidal attempt or ideation, or homicidal ideation
Show all 42Show fewer
- >= CTCAE grade 3 anxiety
- Meets the cut-off score of >= 10 in the Patient Health Questionnaire-9 (PHQ-9) or a cut-off of >= 15 in the General Anxiety Disorder-7 (GAD-7) mood scale, respectively, or selects a positive response of "1, 2, or 3" to question number 9 regarding potential for suicidal thoughts in the PHQ-9 (independent of the total score of the PHQ-9) will be excluded from the study unless overruled by the psychiatric assessment
- Patients with diarrhea >= CTCAE grade 2
- Patient has active cardiac disease including any of the following:
- Left ventricular ejection fraction (LVEF) < 50% as determined by multigated acquisition (MUGA) or echocardiogram (ECHO)
- QTc > 480 msec on screening ECG (using the QTcF formula)
- Personal or family history of prolonged QT syndrome
- Angina pectoris that requires the use of anti-anginal medication
- Ventricular arrhythmias except for benign premature ventricular contractions
- Supraventricular and nodal arrythmias requiring a pacemaker or not controlled with medication
- Conduction abnormality requiring a pacemaker
- Symptomatic pericarditis
- Patient has a history of cardiac dysfunction including any of the following:
- Myocardial infarction within the last 6 months, documented by persistent elevated cardiac enzymes or persistent regional wall abnormalities on assessment of left ventricular ejection fraction (LVEF) function
- History of documented congestive heart failure (New York Heart Association functional classification III-IV)
- Documented cardiomyopathy
- Poorly controlled diabetes mellitus (HbA1c > 8 %)
- Other concurrent severe and/or uncontrolled concomitant medical conditions that could cause unacceptable safety risks or compromise compliance with the protocol
- Significant symptomatic deterioration of lung function; if clinically indicated, pulmonary function tests including measures of predicted lung volumes, Diffusing capacity of the lungs for carbon monoxide (DLCO), oxygen (O2) saturation at rest on room air should be considered to exclude pneumonitis or pulmonary infiltrates
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of BKM120 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection)
- Patients who have been treated with any hematopoietic colony-stimulating growth factors (e.g., Granulocyte colony-stimulating factor (G-CSF), Granulocyte-macrophage colony-stimulating factor (GM-CSF)) <= 2 weeks prior to starting study drug; erythropoietin or darbepoetin therapy, if initiated at least 2 weeks prior to enrollment, may be continued
- Patients who are currently receiving treatment with medication with a known risk to prolong the QT interval or inducing Torsades de Pointes and the treatment cannot either be discontinued or switched to a different medication prior to starting study drug
- Patients receiving chronic treatment with steroids or another immunosuppressive agent; topical applications, inhaled sprays, eye drops or local injections are allowed; patients with previously treated brain metastases, who are on stable low dose corticosteroids treatment for at least 14 days before start of study treatment are eligible
- Patients who have taken herbal medications and certain fruits within 7 days prior to starting study drug - herbal medications include, but are not limited to St. John's Wort, Kava, ephedra (ma huang), gingko biloba, dehydroepiandrosterone (DHEA), yohimbe, saw palmetto, and ginseng; fruits include CYP3A inhibitors: Seville oranges, grapefruit, pomelos, or exotic citrus fruits
- Patients who are currently treated with drugs known to be moderate and strong inhibitors or inducers of isoenzyme CYP3A, and the treatment cannot be discontinued or switched to a different medication prior to starting study drug; note that co-treatment with weak inhibitors of CYP3A is allowed).
- Patients who have received chemotherapy or targeted anticancer therapy <= 4 weeks (6 weeks for nitrosourea, antibodies or mitomycin-C) prior to starting study drug must have resolution of treatment related adverse events to baseline or grade 1 before starting the trial
- Patients who have received wide field radiotherapy <= 4 weeks or limited field radiation for palliation ≤ 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy
- Patients who have undergone major surgery <= 2 weeks prior to starting study drug or who have not recovered from side effects of such therapy
- Patients who are currently taking therapeutic doses of warfarin sodium or any other coumadin-derivative anticoagulant
- Women who are pregnant or breast feeding or adults of reproductive potential not employing an effective method of birth control; women of child-bearing potential must have a negative serum pregnancy test <= 72 hours prior to initiating treatment; double barrier contraceptives must be used through the trial by both sexes; oral, implantable, or injectable contraceptives are therefore not considered effective for this study
- Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or six months of spontaneous amenorrhea with serum follicle stimulating hormone (FSH) levels > 40 milli-international units per millilitre (mIU/mL) [for US only: and estradiol < 20 pg/mL] or have had surgical bilateral oophorectomy (with or without hysterectomy) at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, must use highly effective contraception during treatment for 3 months in total after study drug discontinuation. Highly effective contraception is defined as either: True abstinence-when this is in line with the preferred and usual lifestyle of the subject, periodic abstinence and withdrawal are not acceptable methods of contraception; Sterilization-have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago; Male partner sterilization-for female subjects, the vasectomised male partner should be the sole partner for that patient; Use of a combination of any two of the following barrier methods of contraception-condom or Occlusive cap with spermicidal foam/gel/film/cream/vaginal suppository
- Fertile males, defined as all males physiologically capable of conceiving offspring must use condom during treatment, for 3 months in total after study drug discontinuation and should not father a child in this period
- Known diagnosis of human immunodeficiency virus (HIV) infection
- History of another malignancy within 3 years, except cured or curable basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix; patients with lesions curable by excision must have these lesions excised prior to the initiation of treatment on study
- Patient is unable or unwilling to abide by the study protocol or cooperate fully with the investigator
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, San Francisco
San Francisco, California, 94115
About this study
Official title: A Phase 1/2 Trial of BKM120 Combined With Vemurafenib (PLX4032) in BRAFV600E/K Mutant Advanced Melanoma (Novartis Study Number CBKM120ZUS21T)
This is a phase 1/2 clinical trial with the goal of determining whether the addition of the investigational agent BKM120 to vemurafenib will lead to improved 6-month progression-free survival in patients with BRAFV600E/K mutant melanoma.
Read the detailed description
The phase 1 portion of this trial is a dose escalation study; the phase 2 portion is a single-stage, single arm prospective clinical trial. All patients will receive continuous doses of vemurafenib twice a day and BKM120 once a day.
In the phase 1 portion of the study, there will be a 7 day lead-in period to allow for single dose pharmacokinetic analysis of BKM120 alone. Cycle 1 (28 days) is the dose-limiting toxicity (DLT) period. During phase 1, vemurafenib and BKM120 doses will be escalated using a standard 3+3 dose escalation scheme with the goal of identifying the recommended phase 2 dose.
In the phase 2 portion of the study, patients will receive continuous doses of vemurafenib and BKM120 starting on day 1 of the first cycle. In the phase 2 portion of the study, patients will receive vemurafenib and BKM120 at the recommended phase 2 dose.
- Conditions
- BRAF Mutant Metastatic Melanoma
- Being tested
- BKM120 Combined with Vemurafenib (PLX4032)
- Keywords
- BKM120, BRAF, Melanoma, PI3K
- Medical subject headings
- Melanoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BKM120 Combined with Vemurafenib (PLX4032)Drug
Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D)
Dose Level -1: BKM120 60 mg daily, Vemurafenib 480 mg bid
Dose Level 1: BKM120 60 mg daily, Vemurafenib 720 mg bid
Dose Level 2: BKM120 80 mg daiy, Vemurafenib 720 mg bid
Dose Level 3: BKM120 100 mg daiy, Vemurafenib 720 mg bid
Dose Level 4: BKM120 100 mg daiy, Vemurafenib 960 mg bid
Phase II is a single-stage, single arm prospective trial: patients will receive BKM120 and vemurafenib at the RP2D
| Study group | Role | What they receive |
|---|---|---|
| No Previous Treatment | Other | 150 mg oral dabrafenib twice a day until disease progression, death, or unacceptable adverse events. Receives: Drug: BKM120 Combined with Vemurafenib (PLX4032) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1 - Safety & Recommended Phase 2 Dose (RP2D)
RP2D determined by maximum tolerated dose (MTD), post-dose-limiting toxicity (DLT) period toxicity, and pharmacokinetic data
Measured: 28 days
Phase 2 - Progression-free Survival Rate
6 month progression-free survival rate (PFS6) determined by tumor assessments, clinical tests and laboratory tests
Measured: 6 months
Secondary outcomes (6)
Secondary Outcome 1 Phase 2 - Objective Response Rate
Objective response rate determined by tumor assessments, clinical tests and laboratory tests.
Measured: Day 28 (+/- 3) of even-numbered treatment cycles until progression
Secondary Outcome 2 Phase 2 - Safety and Tolerability
Determined by clinical and laboratory tests, and adverse events (AE) assessments
Measured: During study treatment, up to 2 years
Secondary Outcome 3 Phase 2 - Phosphatase and TENsin (PTEN) Expression
PTEN expression associated with better PFS determined by laboratory tests.
Measured: No time limit
Secondary Outcome 4 Phase 2 - Phosphatidylinositol 3-kinase (PI3K)-Pathway Signaling Reduction Levels
Greater reduction in PI3K-pathway signaling associated with better PFS determined by laboratory tests and tumor assessments.
Measured: No time limit
Secondary Outcome 5 Phase 2 - PI3K Pathway Gene Expression Levels
Responding tumors lack gene expression signatures of PI3K pathway activation, and progressing tumors demonstrate gene expression signatures of PI3K pathway activation - determined by laboratory tests and tumor assessments.
Measured: No time limit
Secondary Outcome 6 Phase 2 - MAPK Pathway Gene Expression Levels
Responding tumors lack gene expression signatures of MAPK pathway activation, and progressing tumors demonstrate gene expression signatures of MAPK pathway activation - determined by laboratory tests and tumor assessments.
Measured: No time limit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2012 (estimated)
- Study start9 Jun 2012
- Primary completion13 Dec 2013
- Study completion21 Mar 2017
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 BRAF Mutant Metastatic MelanomaStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Principal investigator, Alain Algazi, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01512251
- Sponsor's protocol ID
- 11952
- Other IDs
- CBKM120ZUS21T
- Status last verified
- Aug 2020
- First submitted
- 20 Dec 2011
- Last updated
- 17 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Algazi AP, Rotow J, Posch C, Ortiz-Urda S, Pelayo A, Munster PN, Daud A. A dual pathway inhibition strategy using BKM120 combined with vemurafenib is poorly tolerated in BRAF V600E/K mutant advanced melanoma. Pigment Cell Melanoma Res. 2019 Jul;32(4):603-606. doi: 10.1111/pcmr.12777. Epub 2019 Apr 13. No abstract available.
PubMed 30801911
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Trial of BKM120 Combined With Vemurafenib (PLX4032) in BRAFV600E/K Mutant Advanced Melanoma (Novartis Study Number CBKM120ZUS21T)
- ClinicalTrials.gov ID
- NCT01512251
- Acronym / sponsor ID
- 11952
- Conditions
- BRAF Mutant Metastatic Melanoma
- Keywords
- BKM120, BRAF, Melanoma, PI3K
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- 9 Jun 2012 (actual)
- Primary completion
- 13 Dec 2013 (actual)
- Study completion
- 21 Mar 2017 (actual)
- First posted
- 19 Jan 2012 (estimated)
- Results
- Posted 16 May 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 1/2 clinical trial with the goal of determining whether the addition of the investigational agent BKM120 to vemurafenib will lead to improved 6-month progression-free survival in patients with BRAFV600E/K mutant melanoma.
Detailed description
The phase 1 portion of this trial is a dose escalation study; the phase 2 portion is a single-stage, single arm prospective clinical trial. All patients will receive continuous doses of vemurafenib twice a day and BKM120 once a day.
In the phase 1 portion of the study, there will be a 7 day lead-in period to allow for single dose pharmacokinetic analysis of BKM120 alone. Cycle 1 (28 days) is the dose-limiting toxicity (DLT) period. During phase 1, vemurafenib and BKM120 doses will be escalated using a standard 3+3 dose escalation scheme with the goal of identifying the recommended phase 2 dose.
In the phase 2 portion of the study, patients will receive continuous doses of vemurafenib and BKM120 starting on day 1 of the first cycle. In the phase 2 portion of the study, patients will receive vemurafenib and BKM120 at the recommended phase 2 dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| No Previous Treatment | Other | Drug: BKM120 Combined with Vemurafenib (PLX4032)150 mg oral dabrafenib twice a day until disease progression, death, or unacceptable adverse events. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Histologically or cytologically confirmed diagnosis of unresectable stage III and stage IV melanoma
- BRAFV600E or BRAFV600K mutation-positive
- Age >= 18 years
- Eastern Cooperative Oncology Group (ECOG) performance status <= 2
- Patients must have at least one site of measurable disease (per RECIST for solid tumors)
Key exclusion criteria (42 listed)
- Patients who have received prior treatment with a PI3K inhibitor or a BRAF inhibitor, prior treatment with sorafenib is permitted.
- Patients with a known hypersensitivity to BKM120 or to its excipients
- Patients with untreated brain metastases are excluded; however, patients with metastatic central nervous system (CNS) tumors may participate in this trial, if the patient is > 4 weeks from therapy completion (incl. radiation and/or surgery) and clinically stable at the time of study entry
- Patients with acute or chronic liver, renal disease or pancreatitis
- Patients with the following mood disorders as judged by the Investigator or a psychiatrist, or as a result of patient's mood assessment questionnaire:
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 |
|---|---|---|
| BKM120 Combined with Vemurafenib (PLX4032)Phase I is 3+3 dose escalation study to identify the recommended phase 2 dose (RP2D) Dose Level -1: BKM120 60 mg daily, Vemurafenib 480 mg bid Dose Level 1: BKM120 60 mg daily, Vemurafenib 720 mg bid Dose Level 2: BKM120 80 mg daiy, Vemurafenib 720 mg bid Dose Level 3: BKM120 100 mg daiy, Vemurafenib 720 mg bid Dose Level 4: BKM120 100 mg daiy, Vemurafenib 960 mg bid Phase II is a single-stage, single arm prospective trial: patients will receive BKM120 and vemurafenib at the RP2D | Drug | No Previous Treatment |
Primary outcome
Phase 1 - Safety & Recommended Phase 2 Dose (RP2D)
Time frame: 28 days
RP2D determined by maximum tolerated dose (MTD), post-dose-limiting toxicity (DLT) period toxicity, and pharmacokinetic data
Phase 2 - Progression-free Survival Rate
Time frame: 6 months
6 month progression-free survival rate (PFS6) determined by tumor assessments, clinical tests and laboratory tests
6 secondary outcomes
Secondary Outcome 1 Phase 2 - Objective Response Rate
Time frame: Day 28 (+/- 3) of even-numbered treatment cycles until progression
Secondary Outcome 2 Phase 2 - Safety and Tolerability
Time frame: During study treatment, up to 2 years
Secondary Outcome 3 Phase 2 - Phosphatase and TENsin (PTEN) Expression
Time frame: No time limit
Secondary Outcome 4 Phase 2 - Phosphatidylinositol 3-kinase (PI3K)-Pathway Signaling Reduction Levels
Time frame: No time limit
Secondary Outcome 5 Phase 2 - PI3K Pathway Gene Expression Levels
Time frame: No time limit
Secondary Outcome 6 Phase 2 - MAPK Pathway Gene Expression Levels
Time frame: No time limit
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 30801911Algazi AP, Rotow J, Posch C, Ortiz-Urda S, Pelayo A, Munster PN, Daud A. A dual pathway inhibition strategy using BKM120 combined with vemurafenib is poorly tolerated in BRAF V600E/K mutant advanced melanoma. Pigment Cell Melanoma Res. 2019 Jul;32(4):603-606. doi: 10.1111/pcmr.12777. Epub 2019 Apr 13. No abstract available.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BKM120 Combined with Vemurafenib (PLX4032) in people with BRAF Mutant Metastatic Melanoma, run by University of California, San Francisco 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 18 inclusion and 42 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 17 Aug 2020. 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 combined 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.
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 16 May 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?
Phase 1 - Safety & Recommended Phase 2 Dose (RP2D) (time frame: 28 days). 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 NCT01512251, 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 17 Aug 2020.