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A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors
This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.
Study details:
This study includes 2 parts: phase 1 (dose escalation) and phase 2 (dose expansion). In phase 1, participants will be enrolled using a rolling 6 dose escalation scheme. The starting dose of LOXO-292 is equivalent to the adult recommended phase 2 dose of 160 milligrams (mg) twice a day (BID).
Once the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) is identified, participants will be enrolled to one of four phase 2 dose expansion cohorts depending on tumor histology and tumor genotype. Cycle length will be 28 days.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 6 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2019-06-13
Primary completion: 2024-12-15
Study completion finish: 2029-05-31
Study type
TREATMENT
Phase
PHASE1
PHASE2
Trial ID
NCT03899792
Intervention or treatment
DRUG: LOXO-292
Conditions
- • Medullary Thyroid Cancer
- • Infantile Myofibromatosis
- • Infantile Fibrosarcoma
- • Papillary Thyroid Cancer
- • Soft Tissue Sarcoma
Find a site
Closest Location:
The Children's Hospital at Westmead
Research sites nearby
Select from list below to view details:
The Children's Hospital at Westmead
Westmead, New South Wales, Australia
Royal Children's Hospital
Parkville, Victoria, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: LOXO-292
| DRUG: LOXO-292
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
To Determine the Safety of Oral LOXO-292 in Pediatric Participants with Advanced Solid Tumors: Dose Limiting Toxicities (DLTs) | For Phase 1 | During the first 28-day cycle of LOXO-292 treatment |
To Determine the Safety of Oral LOXO-292 in Pediatric Participants with Primary CNS Tumors: DLTs | For Phase 1 | During the first 28-day cycle of LOXO-292 treatment |
Overall Response Rate (ORR) Based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 per Independent Review Committee (IRC) | For Phase 2 | Baseline to Progressive Disease or Death due to any cause (Estimated up to 12 months) |
ORR Based on Response Assessment in Neuro-Oncology (RANO) per IRC | For Phase 2 | Baseline to Progressive Disease or Death due to any cause (Estimated up to 12 months) |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Plasma Concentrations of LOXO-292 | Phase 1 | Days 1 and 8 of Cycle 1, Day 1 of Cycle 3 and Day 8 after Intra-participant Dose Escalation (each cycle is 28 days) |
Area Under the Concentration-Time Curve from 0 to 24 hours (AUC0-24) of LOXO-292 | Phase 1 and Phase 2 | Days 1 and 8 of Cycle 1, Day 1 of Cycle 3 and Day 8 after Intra-participant Dose Escalation (each cycle is 28 days) |
Maximum Concentration (Cmax) of LOXO-292 | Phase 1 and Phase 2 | Days 1 and 8 of Cycle 1, Day 1 of Cycle 3 and Day 8 after Intra-participant Dose Escalation (each cycle is 28 days) |
Time to Maximum Concentration (Tmax) of LOXO-292 | Phase 1 and Phase 2 | Days 1 and 8 of Cycle 1, Day 1 of Cycle 3 and Day 8 after Intra-participant Dose Escalation (each cycle is 28 days) |
Recommended LOXO-292 Dose for Phase 2 (MTD) | For Phase 1 | Cycle 1 (28 days) |
To Assess the Preliminary Anti-Tumor Activity of LOXO-292 in Pediatric Participants with Tumors Harboring an Activating RET Alteration as Determined by ORR Based on RECIST v1.1 | For Phase 1 | Baseline to Progressive Disease or Death due to any cause (Estimated up to 12 months) |
Changes from Baseline in Pain Measures as Measured by Wong Baker Faces scales. Wong-Baker Faces Pain Scale includes pictures of facial expressions with correlating scores of 0 being 'no hurt' and 10 being 'hurts worst'. | For Phase 1 | Up to 24 months |
Changes from Baseline in Health Related Quality of Life Measures as Measured by Pediatric Quality of Life (PedsQoL) Inventory Core. PedsQoL includes a list of problems with scores of 0 being 'never a problem' and 4 being 'almost always a problem'. | For Phase 1 | Up to 24 months |
Objective Response Rate as Assessed by RECIST v1.1, as Assessed by Investigator | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, and 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
Objective Response Rate as Assessed by RANO, as Assessed by Investigator | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, and 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
Duration of Response (DOR) as Assessed by Investigator | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, and 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
Duration of Response (DOR) as Assessed by the IRC | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, and 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
Progression Free Survival (PFS) as Assessed by Investigator | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, and 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
PFS as Assessed by IRC | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, and 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
Overall survival (OS) | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, and 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
Clinical Benefit Rate (by Investigator) | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
Clinical Benefit Rate (by IRC) | For Phase 2 | Approximately every 8 weeks for one year, then every 12 weeks, 7 days after the last dose (for up to 2 years) in participants who have not progressed. |
Frequency of Adverse Events (AEs) | For Phase 2 | From the time of informed consent, for approximately 24 months (or earlier if the participants discontinues from the study), and through Safety Follow-up (28 days after the last dose) |
To Evaluate the Concordance of Prior Molecular that Detected a RET Alteration within the Participant's Tumor with Diagnostic Tests Being Evaluated by Sponsor | For Phase 2 | 6 months |
Phase 2: Post-Operative Stage on Participants Treated with LOXO-292 | Tumor stage is described according to the Tumor, Node, Metastasis (TNM)Classification of malignant tumors of the Union for International Cancer Control (UICC) | Up to 3 years |
Phase 2: Surgical Margin Status in Participants Treated with LOXO-292 | Tumor margins after surgery are classified into four groups using the International Cancer Control (UICC)-R classification and the Intergroup Rhabdomyosarcoma Staging (IRS) systems: 1) Complete tumor resection with histologically free margins, 2) Macroscopic resection but invaded margins on histology, 3)Macroscopic residual tumor and 4) Distant metastatic tumor. | Up to 3 years |
Descriptive Analysis of Pretreatment Surgical Plan | For Phase 2 | Up to 3 years |
Descriptive Analysis of Post-Treatment Plans | For Phase 2 | Up to 3 years |
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