Recurrent Childhood MedulloblastomaPhase 2NCT01239316
Vismodegib in Treating Younger Patients With Recurrent or Refractory Medulloblastoma
A Phase 2 study testing Laboratory Biomarker Analysis, Pharmacological Study and Vismodegib in people with Recurrent Childhood Medulloblastoma, run by National Cancer Institute (NCI) at 12 sites in United States.
Trial summary
This phase II trial studies how well vismodegib works in treating younger patients with recurrent or refractory medulloblastoma. Vismodegib may slow the growth of tumor cells. Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Recurrent Childhood Medulloblastoma
- What is being tested?
- Laboratory Biomarker Analysis, Pharmacological Study and Vismodegib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 12 sitesUnited States
- Study dates
- Nov 2010to Aug 2015
- 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 3 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients with a histologically confirmed diagnosis of medulloblastoma that is recurrent, progressive, or refractory to standard therapy and for which there is no known curative therapy
- Patient must have adequate archival formalin fixed, paraffin embedded (FFPE) primary tumor material for biology studies; specifically, adequate archival FFPE tumor material is either:
- An FFPE block, preferably in which tumor occupies an area of at least 10x10 mm if available, and is free of surgical or processing artifacts; tumor in the submitted FFPE block must not have been obtained from the CUSA trap OR
- Preferably15x5µm sections if available from an FFPE tissue block conforming to the above criteria AND
- Tissue submission must be accompanied by a copy of the pathology report
- Patients must have bi-dimensionally measureable disease in the brain or spinal cord defined as at least one lesion that can be accurately measured in at least 2 planes in order to be eligible for this study
Show all 27Show fewer
- Patients must have a body surface area (BSA) of >= 0.67m^2 and at most 2.5m^2
- Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration; this is to be documented in the database
- Karnofsky performance status of >= 50% in patients > 16 years, or Lansky performance status of >= 50% in patients >= 3 yrs and =< 16 years, assessed within two weeks prior to registration
- Must have recovered from prior treatment-related toxicity; no other myelosuppressive chemotherapy or immunotherapy within 4 weeks prior to study entry (6 weeks if prior nitrosurea)
- Decadron dose should also be stable or decreasing for at least 1 week (7days) prior to starting therapy
- Radiation therapy (XRT) >= 3 months prior to study entry for craniospinal irradiation; >= 8 weeks for local irradiation to primary tumor; >= 2 weeks prior to study entry for focal irradiation for symptomatic metastatic sites
- Off all colony stimulating factors > 1 week prior to study entry (granulocyte colony stimulating factor [GCSF], granulocyte macrophage colony stimulating factor [GM CSF], erythropoietin)
- Prior therapy will include primary therapy (including radiation therapy and chemotherapy) and a maximum of 2 additional salvage therapies; patients can enroll on the protocol after failure on primary therapy
- Absolute neutrophil count (ANC) >= 1000 µL
- Platelet count >= 50,000/uL (transfusion independent)
- Hemoglobin >= 8.0 gm/dL (may receive red blood cell [RBC] transfusions)
- Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70ml/min/1.73 m^2 or a serum creatinine =< 1.5 mg/dL
- Serum Total Bilirubin =< 1.5 x upper limit of normal (ULN) for age
- Serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 2.5 times institutional ULN for age
- Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) =< 2.5 times institutional ULN for age
- Alkaline Phosphatase =< 1.5 times institutional ULN
- Serum albumin >= 2.5 g/dL
- Patient must have recovered from the significant acute toxicities of all prior therapy before entering this study and meet all other eligibility criteria specified in the Inclusion and Exclusion Criteria
- Pregnancy should be avoided for 12 months after the last dose of GDC-0449 for females of child-bearing potential; female patients of childbearing potential must not be pregnant or breast-feeding; female patients of childbearing potential must have a negative serum or urine pregnancy test within 24 hours prior to beginning treatment
- Women of childbearing potential are required to have a negative serum pregnancy test (with a sensitivity of at least 25 mIU/mL) within 10-14 days and within 24 hours prior to the first dose of GDC-0449 (serum or urine); a pregnancy test (serum or urine) will be administered every 4 weeks if their menstrual cycles are regular or every 2 weeks if their cycles are irregular while on study within the 24-hour period prior to the administration of GDC-0449; prior to dispensing GDC-0449, the investigator must confirm and document the patient's use of two contraceptive methods, dates of negative pregnancy test, and confirm the patient's understanding of the of GDC-0449 to cause spontaneous abortion or birth defects; female patients are required to use two forms of acceptable contraception, including one barrier method during participation in the study and for the 12 months following the last dose; all patients should receive contraceptive counseling either by the investigator, or by an obstetrician (OB), gynecologist or other physician who is qualified in this area of expertise; if a woman of childbearing potential believes that her contraceptive method has failed, emergency contraception should be considered; if a patient is suspected to be pregnant, GDC-0449 should be immediately discontinued; in addition, a positive urine test must be confirmed by a serum pregnancy test; if it is confirmed that the patients is not pregnant, the patient may resume dosing with GDC-0449; if a female patient becomes pregnant during therapy or within 12 months after the last dose of GDC-0449, or if the female partner of a male patient exposed to the drug becomes pregnant while the male patient is receiving GDC-0449 or within 12 months after the last dose of GDC-0449, the investigator must be notified in order to facilitate outcome follow-up; female patients should not breastfeed a baby while on this study; female patients must NEVER donate ova while being treated with GDC-0449; all sexually active male subjects (including those who have undergone vasectomy) should utilize a barrier form of contraception during study treatment and for 12 months after the last dose as it is not known whether GDC-0449 that may be present in seminal fluid would cause serious or life-threatening birth defects in a fetus born to the female partner of a male subject; males should also not donate sperm during treatment or up to 12 months after the last dose
- Signed informed consent must be obtained including for pharmacokinetic study according to institutional guidelines
Who cannot join
- Central nervous system (CNS) embryonal tumor other than medulloblastoma; for example, patients with diagnosis of Atypical Teratoid / Rhabdoid Tumor (ATRT), primitive neuroectodermal tumor (PNET) from a non-cerebellar site within the central nervous system, ependymoblastoma, or medulloepithelioma
- Patients with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction), that would compromise the patient's ability to tolerate protocol therapy or would likely interfere with the study procedures or results
- Patients receiving any other anticancer or investigational drug therapy, or warfarin
- Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy
- Other: below given criteria are confirmed by the patient history
- Inability to swallow capsules
Show all 11Show fewer
- Malabsorption syndrome or other condition that would interfere with enteral absorption
- History of congestive heart failure
- History of ventricular arrhythmia requiring medication
- Uncontrolled hypocalcemia, hypomagnesemia, hyponatremia or hypokalemia defined as less than the lower limit of normal for the institution despite adequate electrolyte supplementation
- Clinically important history of liver disease, including viral or other hepatitis or cirrhosis
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
National Cancer Institute Pediatric Oncology Branch
Bethesda, Maryland, 20892
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Duke University Medical Center
Durham, North Carolina, 27710
Texas Children's Hospital
Houston, Texas, 77030
Children's Hospital Los Angeles
Los Angeles, California, 90027
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
Lucile Packard Children's Hospital Stanford University
Palo Alto, California, 94304
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Children With Recurrent or Refractory Medulloblastoma
This phase II trial studies how well vismodegib works in treating younger patients with recurrent or refractory medulloblastoma. Vismodegib may slow the growth of tumor cells.
Read the detailed description
PRIMARY OBJECTIVES:
- Estimate the efficacy of GDC-0449 (vismodegib) treatment for pediatric patients with recurrent or refractory medulloblastoma, as measured by the sustained objective response rates for patients without (stratum A) and with (stratum B) evidence of activation of Hedgehog (Hh) signaling pathway in their tumors.
II. Characterize the pharmacokinetics (plasma) of GDC-0449 in children/adolescents with refractory medulloblastoma.
III. To document pathologic and genomic methods to identify medulloblastomas with activation of the Hh signaling pathway.
SECONDARY OBJECTIVES:
- Document and describe toxicities associated with GDC-0449 administered on a daily schedule.
II. Estimate the duration of objective response and progression-free survival (PFS).
III. Characterize the pharmacokinetics (cerebrospinal fluid) of GDC-0449 in children/adolescents with refractory medulloblastoma.
OUTLINE: This is a multicenter study. Patients are stratified according to evidence of activation of Hedgehog signaling pathway in their tumors (without vs with vs unknown).
Patients receive vismodegib orally (PO) once daily (QD) on days 1-28. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity.
Plasma and cerebrospinal fluid samples are collected periodically for pharmacokinetic and other correlative studies.
After completion of study treatment, patients are followed up every other month for up to 12 months.
- Conditions
- Recurrent Childhood Medulloblastoma
- Being tested
- Laboratory Biomarker Analysis, Pharmacological Study, Vismodegib
- Medical subject headings
- Medulloblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Laboratory Biomarker AnalysisOther
Correlative studies
Correlative studies
VismodegibDrug
Also called Erivedge, GDC-0449, Hedgehog Antagonist GDC-0449
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (vismodegib) | Experimental | Patients receive vismodegib PO QD on days 1-28. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity. Receives: Other: Laboratory Biomarker Analysis; Other: Pharmacological Study; Drug: Vismodegib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response (CR+PR) Sustained for ≥ 8 Weeks
Objective response is either a complete response or a partial response sustained for 8 weeks in a patient. The objective response rate will be reported separately for patients of each stratum. CR is complete disappearance of all enhancing tumor. PR is >= 50% reduction in tumor size.
Measured: Up to 12 months
Pharmacokinetics (Plasma) of GDC-0449
plasma GDC-0449 concentration of day 21 in first course
Measured: up to 12 month
Secondary outcomes (3)
Progression-free Survival
Progression-free survival (PFS) is measured from the date of initial treatment with GDC-0449 until the earliest of progression or death on study. PFS is censored at the last tumor assessment date for patients without disease progression who have not died within 30 days of last exposure to study treatment. Kaplan-Meier method is used to estimate the progression-free survival.
Measured: From start of treatment up to 2 years
Duration of Objective Response
The duration of objective response is measured from the initial scan documenting complete or partial response that was subsequently confirmed until the earlier of documented progression or death on study. Duration of objective response is censored at the last tumor assessment date for patients without disease progression who have not died within 30 days of last exposure to study treatment.
Measured: From start of treatment up to 2 years
Pharmacokinetic Parameters of Vismodegib, CSF Penetration
The estimated median of cerebrospinal fluid (CSF) drug penetration is reported when expressed as an AUC ratio of CSF vismodegib to that of unbound drug in plasma.
Measured: up to 12 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2010 (estimated)
- Study startNov 2010
- Primary completionJun 2014
- Study completionAug 2015
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 Recurrent Childhood MedulloblastomaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Amar Gajjar, Principal investigator, Pediatric Brain Tumor Consortium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01239316
- Sponsor's protocol ID
- NCI-2011-02546
- Other IDs
- NCI-2011-02546, CDR0000688451, PBTC-032, PBTC-032, PBTC-032, U01CA081457
- Status last verified
- Apr 2015
- First submitted
- 10 Nov 2010
- Last updated
- 14 Jan 2016
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Robinson GW, Orr BA, Wu G, Gururangan S, Lin T, Qaddoumi I, Packer RJ, Goldman S, Prados MD, Desjardins A, Chintagumpala M, Takebe N, Kaste SC, Rusch M, Allen SJ, Onar-Thomas A, Stewart CF, Fouladi M, Boyett JM, Gilbertson RJ, Curran T, Ellison DW, Gajjar A. Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032. J Clin Oncol. 2015 Aug 20;33(24):2646-54. doi: 10.1200/JCO.2014.60.1591. Epub 2015 Jul 13.
PubMed 26169613
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Clinical Trial Evaluating the Efficacy and Safety of GDC-0449 in Children With Recurrent or Refractory Medulloblastoma
- ClinicalTrials.gov ID
- NCT01239316
- Acronym / sponsor ID
- NCI-2011-02546
- Conditions
- Recurrent Childhood Medulloblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- Nov 2010
- Primary completion
- Jun 2014 (actual)
- Study completion
- Aug 2015 (actual)
- First posted
- 11 Nov 2010 (estimated)
- Results
- Posted 14 Jan 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies how well vismodegib works in treating younger patients with recurrent or refractory medulloblastoma. Vismodegib may slow the growth of tumor cells.
Detailed description
PRIMARY OBJECTIVES:
- Estimate the efficacy of GDC-0449 (vismodegib) treatment for pediatric patients with recurrent or refractory medulloblastoma, as measured by the sustained objective response rates for patients without (stratum A) and with (stratum B) evidence of activation of Hedgehog (Hh) signaling pathway in their tumors.
II. Characterize the pharmacokinetics (plasma) of GDC-0449 in children/adolescents with refractory medulloblastoma.
III. To document pathologic and genomic methods to identify medulloblastomas with activation of the Hh signaling pathway.
SECONDARY OBJECTIVES:
- Document and describe toxicities associated with GDC-0449 administered on a daily schedule.
II. Estimate the duration of objective response and progression-free survival (PFS).
III. Characterize the pharmacokinetics (cerebrospinal fluid) of GDC-0449 in children/adolescents with refractory medulloblastoma.
OUTLINE: This is a multicenter study. Patients are stratified according to evidence of activation of Hedgehog signaling pathway in their tumors (without vs with vs unknown).
Patients receive vismodegib orally (PO) once daily (QD) on days 1-28. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity.
Plasma and cerebrospinal fluid samples are collected periodically for pharmacokinetic and other correlative studies.
After completion of study treatment, patients are followed up every other month for up to 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (vismodegib) | Experimental | Other: Laboratory Biomarker Analysis, Other: Pharmacological Study, Drug: VismodegibPatients receive vismodegib PO QD on days 1-28. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 3 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (27 listed)
- Patients with a histologically confirmed diagnosis of medulloblastoma that is recurrent, progressive, or refractory to standard therapy and for which there is no known curative therapy
- Patient must have adequate archival formalin fixed, paraffin embedded (FFPE) primary tumor material for biology studies; specifically, adequate archival FFPE tumor material is either:
- Patients must have bi-dimensionally measureable disease in the brain or spinal cord defined as at least one lesion that can be accurately measured in at least 2 planes in order to be eligible for this study
- Patients must have a body surface area (BSA) of >= 0.67m^2 and at most 2.5m^2
- Patients with neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration; this is to be documented in the database
Key exclusion criteria (11 listed)
- Central nervous system (CNS) embryonal tumor other than medulloblastoma; for example, patients with diagnosis of Atypical Teratoid / Rhabdoid Tumor (ATRT), primitive neuroectodermal tumor (PNET) from a non-cerebellar site within the central nervous system, ependymoblastoma, or medulloepithelioma
- Patients with any clinically significant unrelated systemic illness (serious infections or significant cardiac, pulmonary, hepatic or other organ dysfunction), that would compromise the patient's ability to tolerate protocol therapy or would likely interfere with the study procedures or results
- Patients receiving any other anticancer or investigational drug therapy, or warfarin
- Patients with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy
- Other: below given criteria are confirmed by the patient history
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 |
|---|---|---|
| Laboratory Biomarker AnalysisCorrelative studies | Other | Treatment (vismodegib) |
| Pharmacological StudyCorrelative studies | Other | Treatment (vismodegib) |
| VismodegibAlso: Erivedge, GDC-0449, Hedgehog Antagonist GDC-0449Given PO | Drug | Treatment (vismodegib) |
Primary outcome
Objective Response (CR+PR) Sustained for ≥ 8 Weeks
Time frame: Up to 12 months
Objective response is either a complete response or a partial response sustained for 8 weeks in a patient. The objective response rate will be reported separately for patients of each stratum. CR is complete disappearance of all enhancing tumor. PR is >= 50% reduction in tumor size.
Pharmacokinetics (Plasma) of GDC-0449
Time frame: up to 12 month
plasma GDC-0449 concentration of day 21 in first course
3 secondary outcomes
Progression-free Survival
Time frame: From start of treatment up to 2 years
Duration of Objective Response
Time frame: From start of treatment up to 2 years
Pharmacokinetic Parameters of Vismodegib, CSF Penetration
Time frame: up to 12 month
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 26169613Robinson GW, Orr BA, Wu G, Gururangan S, Lin T, Qaddoumi I, Packer RJ, Goldman S, Prados MD, Desjardins A, Chintagumpala M, Takebe N, Kaste SC, Rusch M, Allen SJ, Onar-Thomas A, Stewart CF, Fouladi M, Boyett JM, Gilbertson RJ, Curran T, Ellison DW, Gajjar A. Vismodegib Exerts Targeted Efficacy Against Recurrent Sonic Hedgehog-Subgroup Medulloblastoma: Results From Phase II Pediatric Brain Tumor Consortium Studies PBTC-025B and PBTC-032. J Clin Oncol. 2015 Aug 20;33(24):2646-54. doi: 10.1200/JCO.2014.60.1591. Epub 2015 Jul 13.
Related trials for Recurrent Childhood Medulloblastoma
Active, not recruitingPhase 2
Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma
RecruitingPhase 1
Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)
Medulloblastoma, Childhood, Atypical Teratoid/Rhabdoid Tumor of CNS
RecruitingPhase 1
GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults
Medulloblastoma, Central Nervous System Embryonal Tumor
RecruitingPhase 1/2
Liothyronine in Combination With BIT Regimen for Medulloblastoma With or Without Minimal Residual Disease
Medulloblastoma, Medulloblastoma, Childhood
RecruitingPhase 1
HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors
Neoplasms, Brain, Glioblastoma Multiforme
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.
Amar Gajjar principal investigator
Pediatric Brain Tumor Consortium
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Laboratory Biomarker Analysis, Pharmacological Study and Vismodegib in people with Recurrent Childhood Medulloblastoma, run by National Cancer Institute (NCI) at 12 sites 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 27 inclusion and 11 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 14 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites 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 14 Jan 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?
Objective Response (CR+PR) Sustained for ≥ 8 Weeks (time frame: Up to 12 months). 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 NCT01239316, 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 14 Jan 2016.