Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01289028 | Efficacy of Nilotinib in Adult Patients With Gastrointestinal Stromal Tumors Resistant to Imatinib and Sunitinib. | PHASE3 | COMPLETED | 125 | — | — | Nov 1, 2008 | Jul 1, 2014 | Jan 2, 2017 | 19 | Germany, Italy |
| NCT00471328 | Efficacy and Safety of Nilotinib (AMN107) Compared With Current Treatment Options in Patients With GIST Who Have Failed Both Imatinib and Sunitinib | PHASE3 | COMPLETED | 248 | — | — | Mar 1, 2007 | Jun 1, 2011 | Jun 12, 2012 | 35 | United States, Australia +11 |
| NCT01863745 | Nilotinib Roll-over Protocol for Patients in Novartis-sponsored Nilotinib Study and Benefiting From Nilotinib Treatment | PHASE2 | COMPLETED | 15 | — | — | Jun 25, 2013 | Oct 2, 2023 | Aug 19, 2024 | 11 | Japan |
| NCT00441155 | Patients Completing Core Protocol (CAMN107A2103), Exhibiting Stable Disease (SD), Partial Response (PR) or Complete Response (CR) to Nilotinib in Combination With Imatinib | PHASE1 | COMPLETED | 14 | — | — | Nov 1, 2006 | Jan 1, 2011 | May 13, 2013 | 4 | United States, France +1 |
Neither sufficient shrinkage to qualify for Partial Response nor sufficient increase to qualify for Progression Disease, taking as reference the smallest sum of the longest diameter since the treatment started.
The primary efficacy variable was defined as the proportion of patients with a best overall response of CR by Week 16/Month 4 based on local assessment according to RECIST (Version 1.0). This is an at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum longest diameter.
Complete response (CR) is the Disappearance of all target lesions.
Progression-free survival (PFS) is the time from date of randomization to the date of first documented progression or death due to any cause. If a participant has not had an event, progression-free survival is censored at the time of last adequate tumor assessment. Progression is defined according to Modified Response Evaluation Criteria in Solid Tumors (modified RECIST) criteria as at least a 20% increase in the sum of the longest diameter of target lesions, worsening of the non-target lesions or the appearance of one or more new lesions.
PFS is defined as the time from the first date of cross-over to nilotinib therapy from the control arm to the date of the first observation of documented disease progression. Tumor assessment was based on the local investigator's measurement using Modified Response Evaluation Criteria in Solid Tumors (modified RECIST) criteria. Progression is defined according to modified RECIST criteria as at least a 20% increase in the sum of the longest diameter of target lesions, worsening of the non-target lesions or the appearance of one or more new lesions.
Number of participants with adverse events (AEs) (any AE regardless of seriousness), serious adverse events (SAEs), Fatal SAEs and treatment related AEs.
Assessed by AE evaluation, including safety laboratory and cardiac safety (ECG, cardiac enzymes, ECHO) measures.
| Arm | Type | Description |
|---|---|---|
| Nilotinib | EXPERIMENTAL | nilotinib 400 mg twice daily (bid). |
| Control/cross-over to Nilotinib | ACTIVE_COMPARATOR | In core study phase, patients in this arm received Best Supportive Care (BSC) with or without imatinib or sunitinib at the last tolerated dose or at the investigator's choice until documented disease progression followed by cross-over to nilotinib arm. Patients entering the extension study on this control arm were permitted to cross over to nilotinib arm only upon documented disease progression. |
| Monotherapy: AMN107 | EXPERIMENTAL | initial dose of imatinib (dose level 1) was 400 mg bid was administered orally on a continuous daily schedule and was not escalated during the study |
| Combination Therapy: AMN107 + Imatinib | ACTIVE_COMPARATOR | six possible doses of Nilotinib (100 mg once daily (qd), 200 mg qd, 400 mg qd, 200 mg bid, 300 mg bid, and 400 mg bid). four possible doses of Imatinib (0 mg, 400 mg qd, 600 mg qd, and 400 mg bid).the initial dose of nilotinib (dose level 1) was 200 mg qd and could have been escalated up to 400 mg bid |
| Name | Type | Description |
|---|---|---|
| Nilotinib | DRUG | - |
| Best Supportive Care (BSC) +/- imatinib or sunitinib | OTHER | Can include pain medication, localized radiotherapy, nutritional support, and/or oxygen therapy and blood transfusions. Imatinib or sunitinib can be administered at the last tolerated dose and regimen or at the Investigator's choice. |
| Nilotinib, Imatinib | DRUG | Nilotinib 50 mg and 200 mg gelatin capsules and Imatinib 100 mg and 400 mg film-coated tablets in bottles.. Medication labels for each study drug complied with the legal requirements of each country, were printed in the local language. |
Inclusion Criteria: * Histologically confirmed diagnosis of GIST that is unresectable and/or metastatic and therefore not amenable to surgery or combined modality with curative intent. * Radiologically confirmed disease progression during imatinib therapy at a dose of at least 400 mg daily and/or r...