The following info and data is provided "as is" to help patients around the globe.
We do not endorse or review these studies in any way.
Brief Title: A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML
Official Title: A Phase II, Open-label, Single Arm Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Twice Daily Midostaurin (PKC412) Combined With Standard Chemotherapy and as a Single Agent Post-consolidation Therapy in Children With Untreated FLT3-mutated AML
Study ID: NCT03591510
Brief Summary: This study will evaluate the safety, efficacy and pharmacokinetics of midostaurin in combination with standard chemotherapy in pediatrics patients with newly diagnosed FLT3-mutated Acute Myeloid Leukemia. The study has two parts: Part 1 to define the Recommended Phase 2 Dose, and Part 2 to evaluate safety and tolerability and efficacy of midostaurin. Both parts will consist of 2 induction blocks, 3 consolidation blocks, 12 cycles of post-consolidation consisting of continuous therapy with midostaurin, and a follow-up phase.
Detailed Description: This trial is an open label, multi center single arm study to evaluate twice daily oral midostaurin with standard induction, consolidation chemotherapy with sequential midostaurin therapy for 5 treatment blocks (2 induction blocks, 3 consolidation blocks, followed by single agent midostaurin post consolidation therapy for 12 cycles). The total maximum planned duration on treatment is 17 cycles (5 blocks and 12 cycles). A block is defined as the time from start of study treatment to the time of hematopoietic recovery, at the latest at Day (D) 42, or determination of persistent disease, whichever occur first. In both Part 1 and Part 2, patients will receive the first course of induction chemotherapy according to local standard and duration is from 8 to 12 days. Upon FLT3 mutation confirmation, patients will receive midostaurin for 14 days. After determination of remission and hematopoietic recovery, patients will receive Block 2. In Part 1: * Block 2 FLADx treatment duration is D1 to D6, and midostaurin from D8 to D21. Patients who achieve documented CR (and hematopoietic recovery at the latest at D42 from the first day of Block 2) will receive Block 3. * Block 3 consolidation HAM treatment duration is D1 to D4, followed by midostaurin D8 to D21. Patients who achieve hematopoietic recovery at the latest at D42 from the first day of Block 3 will receive Block 4. Patients who relapse will discontinue further study treatment. * Block 4 HA3E treatment duration is D1 to D5 followed by midostaurin D8 to D21. Patients who achieve hematopoietic recovery at the latest at D42 from the first day of Block 4 will receive Block 5. * Block 5 HiDAC treatment duration is D1 to D3 followed by midostaurin D8 to D21. Patients who relapse will discontinue further study treatment. Patients in continuous remission with hematopoietic recovery will receive continuous post consolidation therapy of midostaurin, during 12 cycles (28 days per cycle). In Part 1 of the study, patients in cohorts of 3 will receive sequential midostaurin administered at 30mg/m2bid. If the 30mg/m2 bid is well tolerated as measured by the Dose Limited Toxicity (DLT) rate during Bock 1, additional patients in cohort of 3 will be treated with sequential midostaurin at 60mg/m2 bid. When the recommended phase 2 dose (RP2D) is confirmed, subsequent patients will be treated in Part 2 of the study at the RP2D. In Part 2: * Block 2 HAM treatment duration is D1 to D4 and midostaurin from D8 to D21. Patients who achieve documented CR (and hematopoietic recovery at the latest at D42 from the first day of Block 2) will receive Block 3. * Block 3 consolidation HA3E treatment duration is D1 to D5, followed by midostaurin D8 to D21. Patients who achieve hematopoietic recovery at the latest at D42 from the first day of Block 3 will receive Block 4. Patients who relapse will discontinue further study treatment. * Block 4 HAM treatment duration is D1 to D4 followed by midostaurin D8 to D21. Patients who achieve hematopoietic recovery at the latest at D42 from the first day of Block 4 will receive Block 5. * Block 5 HiDAC treatment duration is D1 to D3 followed by midostaurin D8 to D21. Patients who relapse will discontinue further study treatment. Patients in continuous remission with hematopoietic recovery will receive continuous post consolidation therapy of midostaurin, during 12 cycles (28 days per cycle). Patients who relapse will discontinue further study treatment.
Minimum Age: 3 Months
Eligible Ages: CHILD
Sex: ALL
Healthy Volunteers: No
Novartis Investigative Site, Aurora, Colorado, United States
Novartis Investigative Site, Miami, Florida, United States
Novartis Investigative Site, Wien, , Austria
Novartis Investigative Site, Brno, , Czechia
Novartis Investigative Site, Praha 5, , Czechia
Novartis Investigative Site, Regensburg, Bavaria, Germany
Novartis Investigative Site, Berlin, , Germany
Novartis Investigative Site, Essen, , Germany
Novartis Investigative Site, Freiburg, , Germany
Novartis Investigative Site, Halle, , Germany
Novartis Investigative Site, Athens, , Greece
Novartis Investigative Site, Bologna, BO, Italy
Novartis Investigative Site, Genova, GE, Italy
Novartis Investigative Site, Monza, MB, Italy
Novartis Investigative Site, Padova, PD, Italy
Novartis Investigative Site, Pavia, PV, Italy
Novartis Investigative Site, Roma, RM, Italy
Novartis Investigative Site, Torino, TO, Italy
Novartis Investigative Site, Napoli, , Italy
Novartis Investigative Site, Kobe-city, Hyogo, Japan
Novartis Investigative Site, Setagaya-ku, Tokyo, Japan
Novartis Investigative Site, Osaka, , Japan
Novartis Investigative Site, Saitama, , Japan
Novartis Investigative Site, Shizuoka, , Japan
Novartis Investigative Site, Amman, , Jordan
Novartis Investigative Site, Seoul, , Korea, Republic of
Novartis Investigative Site, Seoul, , Korea, Republic of
Novartis Investigative Site, Gdansk, , Poland
Novartis Investigative Site, Krakow, , Poland
Novartis Investigative Site, Ekaterinburg, , Russian Federation
Novartis Investigative Site, Moscow, , Russian Federation
Novartis Investigative Site, Ljubljana, , Slovenia
Novartis Investigative Site, Adana, , Turkey
Novartis Investigative Site, Antalya, , Turkey
Novartis Investigative Site, Istanbul, , Turkey
Name: Novartis Pharmaceuticals
Affiliation: Novartis Pharmaceuticals
Role: STUDY_DIRECTOR