MedulloblastomaPhase 1/2NCT01125800
A Phase I Dose Finding and Safety Study of Oral LDE225 in Children and a Phase II Portion to Assess Preliminary Efficacy in Recurrent or Refractory MB
A Phase 1/2 study testing LDE225 in people with Medulloblastoma and Rhabdomyosarcoma, run by Novartis Pharmaceuticals at 14 sites in 6 countries.
Trial summary
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway. Read more about this study
- Who is it for?
- Ages 12 months to 18 years
- Conditions the study is for
- Medulloblastoma, Rhabdomyosarcoma, Neuroblastoma
- What is being tested?
- LDE225
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 76 participants
- Where
- 14 sitesin 6 countries
- Study dates
- Feb 2011to Oct 2014
- 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 12 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Phase I - Patients aged ≥12 months and <18 years, Phase II - Patients ≥12 months
- Phase I - Histologically confirmed diagnosis of medulloblastoma, rhabdomyosarcoma, neuroblastoma, hepatoblastoma, high grade glioma, or osteosarcoma, that has progressed despite treatment with standard therapies, or for which no standard treatments are available (patients with brainstem gliomas are excluded). Phase II - Histologically confirmed diagnosis of recurrent or relapsed medulloblastoma with at least one measurable lesion.
- Performance Status: Karnofsky ≥60% for patients >10 yrs, Lansky ≥50 for patients less than or equal to 10 yrs
- Protocol-defined renal , liver and bone marrow function
- Negative pregnancy test before starting study treatment. If of child bearing potential must use 'highly effective' methods of contraception.
- All patients must consent to provide a tumor sample
Who cannot join
- Systemic anti-cancer treatment within 2 weeks prior to first dose (6 weeks for nitrosourea, mitomycin and monoclonal antibodies).
- Focal radiotherapy within 4 weeks prior to first dose, or full spinal radiotherapy within 3 months of first dose.
- Unresolved toxicity greater than CTCAE grade 1 from previous anti-cancer therapy or radiotherapy (excluding neurotoxicity, alopecia, ototoxicity, lymphopenia or other specifications in the eligibility criteria for this study), or incomplete recovery from previous surgery, unless agreed by Novartis and the Principal Investigator (PI) and documented.
- Major surgery, serious illness or traumatic injury within 2 weeks of starting study therapy. Patients anticipated to require major surgery within the first 2 cycles of treatment.
- Patients requiring a nasogastric tube for drug administration (G-tubes are permitted)
- Impaired cardiac function
Show all 9Show fewer
- Pregnant or breast-feeding females
- Impairment of gastrointestinal (GI) function or GI disease
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
14 sites in 6 countries.
14 sites in 6 countries
United States4 sites
Children's Healthcare of Atlanta Childern Hosp - ATL
Atlanta, Georgia, 30342
Sidney Kimmel Comprehensive Cancer Center/Johns Hopkins Med. John Hopkins
Baltimore, Maryland, 21231
Dana Farber Cancer Institute DFCI (3)
Boston, Massachusetts, 02115
Seattle Children's Hospital CPKC412A2114
Seattle, Washington, 98105
Italy3 sites
Novartis Investigative Site
Bologna, BO, 40139
Novartis Investigative Site
Milan, MI, 20133
Novartis Investigative Site
Roma, RM, 00168
Canada2 sites
Novartis Investigative Site
Toronto, Ontario, M5G 1X8
Novartis Investigative Site
Toronto, Ontario, M5G 2M9
France2 sites
Novartis Investigative Site
Paris, 75231
Novartis Investigative Site
Villejuif, 94805
United Kingdom2 sites
Novartis Investigative Site
Newcastle upon Tyne, NE1 4LP
Novartis Investigative Site
Sutton, Surrey, SM2 5PT
About this study
Official title: A Phase I/II Study of LDE225 in Pediatric Patients With Recurrent or Refractory Medulloblastoma or Other Tumors Potentially Dependent on the Hedgehog-signaling Pathway and Adult Patients With Recurrent or Refractory Medulloblastoma
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway.
Phase II study is to assess preliminary efficacy in both adult and pediatric patients with recurrent or refractory MB.
- Conditions
- Medulloblastoma, Rhabdomyosarcoma, Neuroblastoma, Hepatoblastoma, Glioma, Astrocytoma
- Being tested
- LDE225
- Keywords
- Recurrent,, refractory,, medulloblastoma,, rhabdomyosarcoma,, neuroblastoma,, hepatoblastoma,, astrocytoma,, children,, pediatric,, hedgehog pathway inhibitor, adult
- Medical subject headings
- Medulloblastoma, Rhabdomyosarcoma, Neuroblastoma, Hepatoblastoma, Glioma, Astrocytoma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LDE225Drug
Also called sonidegib
LDE225/sonidegib capsules were supplied to the Investigators at dose strengths of 50 mg, 100 mg, 200 mg, and 250 mg.
| Study group | Role | What they receive |
|---|---|---|
| LDE225 233mg/m2 daily dose | Experimental | Pediatric dose. Receives: Drug: LDE225 |
| LDE225 372mg/m2 daily dose | Experimental | Pediatric dose. Receives: Drug: LDE225 |
| LDE225 425 mg/m2 daily dose | Experimental | Pediatric dose. Receives: Drug: LDE225 |
| LDE225 680 mg/m2 daily dose | Experimental | Pediatric dose. Receives: Drug: LDE225 |
| LDE225 800 mg/m2 daily dose | Experimental | Adult dose Receives: Drug: LDE225 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Dose-limiting Toxicities (DLT) in Phase I
DLT was defined as an adverse event (AE) or abnormal laboratory value assessed as unrelated to disease, disease progression, intercurrent illness, or concomitant medications. DLT included any grade 3 or 4 clinically-evident toxicity, Hematology: ≥ CTCAE grade 3 neutropenia (ANC <1.0x10^9/L); ≥ CTCAE grade 3 thrombocytopenia (platelets <50x10^9/L); ≥ CTCAE grade 3 anemia (Hgb <80 g/L); Febrile neutropenia (ANC <1x10^9/L, fever ‡ 38.5°C), Renal: ≥ CTCAE grade 3 serum creatinine (>3xULN), Hepatic: ≥ CTCAE grade 3 total bilirubin (>3xULN); ≥ 10xULN ALT elevation; grade 2 total bilirubin (>1.5ULN) together with ≥ grade 3 ALT elevation (>5xULN), Cardiac: ≥ CTCAE grade 3, Other AEs: ≥ CTCAE grade 3 vomiting or nausea despite optimal antiemetic therapy, diarrhea despite optimal antidiarrheal treatment.
Measured: Baseline, End of dose escalation part (Day 42)
Maximum Tolerated Dose (MTD) of Sonidegib for Prolonged Use
MTD was defined as highest dose level for which no more than 1 participant in a dose cohort experienced dose-limiting toxicity (DLT), based on Bayesian logistic regression model (BLRM) employing the escalation with overdose control (EWOC) principle. DLT was defined as an adverse event (AE) or abnormal laboratory value assessed as unrelated to disease, disease progression, intercurrent illness, or concomitant medications.k
Measured: Baseline, End of dose escalation part (Day 42)
Percentage of Participants With Objective Response Rate (ORR) by Treatment
The tumor response to the sonidegib treatment was measured by ORR. The ORR was defined as the percentage of participants with partial response or complete response as their best overall response. Participants with stable disease, progressive disease tumor assessment were considered as non-responders. Response evaluation criteria was gadolinium chelate-enhanced brain tumor magnetic resonance imaging (Gd-MRI) for Medulloblastoma and central nervous system (CNS) tumors and response evaluation criteria in solid tumors (RECIST) version 1.0 for non-CNS tumors assessed by MRI. Complete Response (CR), Progressive Disease (PD) and Incomplete Response/Stable Disease (SD) were defined as disappearance of all non-target lesions, unequivocal progression of existing non-target lesions and Neither CR nor PD, respectively.
Measured: Baseline, Day 28 of Cycle 2, End of treatment (Within 14 days of last dose)
Secondary outcomes (6)
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Treatment Related AEs and Death During the Study
An AE was defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. A SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes. Treatment related AEs were defined as AEs that were suspected to be related to study treatment as per investigator. On-treatment deaths were deaths which occurred up to 30 days after last date of study treatment.
Measured: Baseline (start of study treatment) up to End of treatment (Within 14 days of last dose)
Area Under the Drug Concentration Time Curve From Time Zero to 24 Hours After Dosing (AUC0-24h) of Sonidegib in Phase I
AUC(0-24h) was defined as the area under the drug concentration time curve calculated using linear trapezoidal summation from time zero to 24 hours after dosing.
Measured: Pre-dose, 0.5, 1, 2, 4, 7 hours (± 15 min) post-dose at Day 1 and Day 22 of Cycle 1
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Sonidegib in Phase I
Tmax was defined as the time required to reach maximum observed plasma concentration. Tmax was directly determined from the raw plasma concentration time data.
Measured: Pre-dose, 0.5, 1, 2, 4, 7 hours (± 15 min) post-dose at Day 1 and Day 22 of Cycle 1
Maximum Observed Plasma Concentration (Cmax) of Sonidegib in Phase I
Maximum observed plasma concentration following drug administration was calculated from the raw plasma concentration time data.
Measured: Pre-dose, 0.5, 1, 2, 4, 7 hours (± 15 min) post-dose at Day 1 and Day 22 of Cycle 1
Percentage of Pediatric Participants With Objective Response Rate (ORR) by Hedgehog (Hh) Signaling Pathway Status
ORR was determined in the participants with mutations on Hh gene (Hh positive) and the participants without mutations on Hh gene (Hh negative).
Measured: Baseline, Day 28 of Cycle 2, End of treatment (Within 14 days of last dose)
Duration of Response by Treatment
Duration of overall response (complete response (CR) or partial response (PR)) was calculated for those participants whose best overall response was CR or PR. The start date was the date of the first documented tumor response (CR or PR) and the end date was the date of the event defined as the first documented progression or death due to underlying cancer or after the same treatment line. If a participant did not have a progression or death, the duration of response was censored at the date of last adequate tumor assessment in that treatment line.
Measured: Baseline, Day 28 of Cycle 2, End of treatment (Within 14 days of last dose)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2010 (estimated)
- Study startFeb 2011
- Primary completionOct 2014
- Study completionOct 2014
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 MedulloblastomaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01125800
- Sponsor's protocol ID
- CLDE225X2104
- Other IDs
- 2010-019348-37
- Status last verified
- Feb 2016
- First submitted
- 12 May 2010
- Last updated
- 20 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of LDE225 in Pediatric Patients With Recurrent or Refractory Medulloblastoma or Other Tumors Potentially Dependent on the Hedgehog-signaling Pathway and Adult Patients With Recurrent or Refractory Medulloblastoma
- ClinicalTrials.gov ID
- NCT01125800
- Acronym / sponsor ID
- CLDE225X2104
- Conditions
- Medulloblastoma, Rhabdomyosarcoma, Neuroblastoma, Hepatoblastoma, Glioma, Astrocytoma
- Keywords
- Recurrent,, refractory,, medulloblastoma,, rhabdomyosarcoma,, neuroblastoma,, hepatoblastoma,, astrocytoma,, children,
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 76 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Feb 2011
- Primary completion
- Oct 2014 (actual)
- Study completion
- Oct 2014 (actual)
- First posted
- 18 May 2010 (estimated)
- Results
- Posted 2 Mar 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other tumors potentially dependent on Hedgehog signaling pathway.
Phase II study is to assess preliminary efficacy in both adult and pediatric patients with recurrent or refractory MB.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| LDE225 233mg/m2 daily dose | Experimental | Drug: LDE225Pediatric dose. |
| LDE225 372mg/m2 daily dose | Experimental | Drug: LDE225Pediatric dose. |
| LDE225 425 mg/m2 daily dose | Experimental | Drug: LDE225Pediatric dose. |
| LDE225 680 mg/m2 daily dose | Experimental | Drug: LDE225Pediatric dose. |
| LDE225 800 mg/m2 daily dose | Experimental | Drug: LDE225Adult dose |
Who may qualify
- Ages
- 12 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Phase I - Patients aged ≥12 months and <18 years, Phase II - Patients ≥12 months
- Phase I - Histologically confirmed diagnosis of medulloblastoma, rhabdomyosarcoma, neuroblastoma, hepatoblastoma, high grade glioma, or osteosarcoma, that has progressed despite treatment with standard therapies, or for which no standard treatments are available (patients with brainstem gliomas are excluded). Phase II - Histologically confirmed diagnosis of recurrent or relapsed medulloblastoma with at least one measurable lesion.
- Performance Status: Karnofsky ≥60% for patients >10 yrs, Lansky ≥50 for patients less than or equal to 10 yrs
- Protocol-defined renal , liver and bone marrow function
- Negative pregnancy test before starting study treatment. If of child bearing potential must use 'highly effective' methods of contraception.
Key exclusion criteria (9 listed)
- Systemic anti-cancer treatment within 2 weeks prior to first dose (6 weeks for nitrosourea, mitomycin and monoclonal antibodies).
- Focal radiotherapy within 4 weeks prior to first dose, or full spinal radiotherapy within 3 months of first dose.
- Unresolved toxicity greater than CTCAE grade 1 from previous anti-cancer therapy or radiotherapy (excluding neurotoxicity, alopecia, ototoxicity, lymphopenia or other specifications in the eligibility criteria for this study), or incomplete recovery from previous surgery, unless agreed by Novartis and the Principal Investigator (PI) and documented.
- Major surgery, serious illness or traumatic injury within 2 weeks of starting study therapy. Patients anticipated to require major surgery within the first 2 cycles of treatment.
- Patients requiring a nasogastric tube for drug administration (G-tubes are permitted)
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 |
|---|---|---|
| LDE225Also: sonidegibLDE225/sonidegib capsules were supplied to the Investigators at dose strengths of 50 mg, 100 mg, 200 mg, and 250 mg. | Drug | LDE225 233mg/m2 daily dose, LDE225 372mg/m2 daily dose, LDE225 425 mg/m2 daily dose, LDE225 680 mg/m2 daily dose, LDE225 800 mg/m2 daily dose |
Primary outcome
Number of Participants With Dose-limiting Toxicities (DLT) in Phase I
Time frame: Baseline, End of dose escalation part (Day 42)
DLT was defined as an adverse event (AE) or abnormal laboratory value assessed as unrelated to disease, disease progression, intercurrent illness, or concomitant medications. DLT included any grade 3 or 4 clinically-evident toxicity, Hematology: ≥ CTCAE grade 3 neutropenia (ANC <1.0x10^9/L); ≥ CTCAE grade 3 thrombocytopenia (platelets <50x10^9/L); ≥ CTCAE grade 3 anemia (Hgb <80 g/L); Febrile neutropenia (ANC <1x10^9/L, fever ‡ 38.5°C), Renal: ≥ CTCAE grade 3 serum creatinine (>3xULN), Hepatic: ≥ CTCAE grade 3 total bilirubin (>3xULN); ≥ 10xULN ALT elevation; grade 2 total bilirubin (>1.5ULN) together with ≥ grade 3 ALT elevation (>5xULN), Cardiac: ≥ CTCAE grade 3, Other AEs: ≥ CTCAE grade 3 vomiting or nausea despite optimal antiemetic therapy, diarrhea despite optimal antidiarrheal treatment.
Maximum Tolerated Dose (MTD) of Sonidegib for Prolonged Use
Time frame: Baseline, End of dose escalation part (Day 42)
MTD was defined as highest dose level for which no more than 1 participant in a dose cohort experienced dose-limiting toxicity (DLT), based on Bayesian logistic regression model (BLRM) employing the escalation with overdose control (EWOC) principle. DLT was defined as an adverse event (AE) or abnormal laboratory value assessed as unrelated to disease, disease progression, intercurrent illness, or concomitant medications.k
Percentage of Participants With Objective Response Rate (ORR) by Treatment
Time frame: Baseline, Day 28 of Cycle 2, End of treatment (Within 14 days of last dose)
The tumor response to the sonidegib treatment was measured by ORR. The ORR was defined as the percentage of participants with partial response or complete response as their best overall response. Participants with stable disease, progressive disease tumor assessment were considered as non-responders. Response evaluation criteria was gadolinium chelate-enhanced brain tumor magnetic resonance imaging (Gd-MRI) for Medulloblastoma and central nervous system (CNS) tumors and response evaluation criteria in solid tumors (RECIST) version 1.0 for non-CNS tumors assessed by MRI. Complete Response (CR), Progressive Disease (PD) and Incomplete Response/Stable Disease (SD) were defined as disappearance of all non-target lesions, unequivocal progression of existing non-target lesions and Neither CR nor PD, respectively.
6 secondary outcomes
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Treatment Related AEs and Death During the Study
Time frame: Baseline (start of study treatment) up to End of treatment (Within 14 days of last dose)
Area Under the Drug Concentration Time Curve From Time Zero to 24 Hours After Dosing (AUC0-24h) of Sonidegib in Phase I
Time frame: Pre-dose, 0.5, 1, 2, 4, 7 hours (± 15 min) post-dose at Day 1 and Day 22 of Cycle 1
Time to Reach Maximum Observed Plasma Concentration (Tmax) of Sonidegib in Phase I
Time frame: Pre-dose, 0.5, 1, 2, 4, 7 hours (± 15 min) post-dose at Day 1 and Day 22 of Cycle 1
Maximum Observed Plasma Concentration (Cmax) of Sonidegib in Phase I
Time frame: Pre-dose, 0.5, 1, 2, 4, 7 hours (± 15 min) post-dose at Day 1 and Day 22 of Cycle 1
Percentage of Pediatric Participants With Objective Response Rate (ORR) by Hedgehog (Hh) Signaling Pathway Status
Time frame: Baseline, Day 28 of Cycle 2, End of treatment (Within 14 days of last dose)
Duration of Response by Treatment
Time frame: Baseline, Day 28 of Cycle 2, End of treatment (Within 14 days of last dose)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Medulloblastoma
RecruitingPhase 1/2
PLX038 in Primary Central Nervous System Tumors Containing MYC or MYCN Amplifications
Glioma, Medulloblastoma
RecruitingPhase 2
Digoxin Medulloblastoma Study
Medulloblastoma, Medulloblastoma, Non-WNT/Non-SHH
RecruitingPhase 1
Adoptive T Cell Therapy, DC Vaccines, and Hematopoietic Stem Cells Combined With Immune checkPOINT Blockade in Patients With Medulloblastoma
Recurrent Group 3 Medulloblastoma, Recurrent Group 4 (Non-SHH/Non-WNT) Medulloblastoma
RecruitingEarly Phase 1
Pilot Study of IT Topotecan and Maintenance Chemotherapy for HR-EBTs in Children < 6 Years, Post Consolidation
CNS Embryonal Tumor, CNS, Medulloblastoma
RecruitingPhase 1
Chemo-immunotherapy Using Ibrutinib Plus Indoximod for Patients With Pediatric Brain Cancer
Ependymoma, Medulloblastoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
14 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing LDE225 in people with Medulloblastoma and Rhabdomyosarcoma, run by Novartis Pharmaceuticals at 14 sites in 6 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 6 inclusion and 9 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 20 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Italy, Canada, France, United Kingdom and 1 more country. 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 2 Mar 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?
Number of Participants With Dose-limiting Toxicities (DLT) in Phase I (time frame: Baseline, End of dose escalation part (Day 42)). The study lists 3 primary outcomes in all. 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 Mar 2017.