International Observational, Study to Evaluate the Benefit/Risk of Vandetanib (Caprelsa™) 300 mg in RET Mutation Negative and RET Mutation Positive Patients with Symptomatic, Aggressive, Sporadic, Unresectable, Locally Advanced/Metastatic Medullary Thyroid Cancer (Caprelsa 104)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation
• Data collection methods:
  Combined primary and secondary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Multinational, multicenter, Non-Interventional (observational), prospective (for patients with RET mutation positive or negative status) and retrospective (for patients with RET mutation negative status) study.

Study drug and medical condition

• Name of medicine:
  Caprelsa
• Study drug International non-proprietary name (INN) or common name:
  VANDETANIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L01XE) Protein kinase inhibitors
• Medical condition to be studied:
  Medullary thyroid cancer

Population studied

• Short description of the study population:
  Patients with symptomatic, aggressive, sporadic, unresectable, locally advanced/metastatic MTC, treated with vandetanib 300 mg/once daily and with a RET mutation positive or negative status, prospectively. In addition, patients with symptomatic, aggressive, sporadic, unresectable, locally advanced/metastatic MTC, treated with vandetanib 300 mg/once daily, at any time, and with a RET mutation negative status, will be allowed to enter the study retrospectively. Also, patients with symptomatic, aggressive, sporadic, unresectable, locally advanced/metastatic MTC not prescribed vandetanib 300 mg but who are RET mutation negative will be allowed to enter the study both retrospectively and prospectively.
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Medullary thyroid cancer patients
• Estimated number of subjects:
  50

Study design details

• Main study objective:
  - To determine the Objective Response Rate (ORR), Disease Control Rate (DCR), the duration of response and time to response; - To compare PFS for patients treated with vandetanib RET mutation positive (RET+) and RET mutation negative (RET-); - To explore the clinical outcomes among RET- patients not treated with vandetanib; ; - To evaluate the incidence of QTc prolongation and associated risks, SAEs and AEs
• Outcomes:
  Assessment of ; -Objective Response Rate ; -Disease control rate ; -Duration of Response ; -Progression Free Survival ; ; Evaluation of Safety ; -QTc prolongation ; -Adverse Events ; -Vital signs; -Laboratory data
• Data analysis plan:
  1.Efficacy analyses on the evaluable population: Estimate ORR and DCR for RET+ and RET- patients summarized as qualitative variable with corresponding 95% CI by RET mutation status, by study and overall. Time to Response and Duration of Response. Kaplan Meier survival curves. Median PFS in RET+ and RET- patients. Other outcome evaluations (including CTN and CEA) are descriptive. ; ; 2. Safety analysis on the safety population: Extent of exposure as number of days of exposure to drug. Duration of exposure summarized descriptively by RET mutation/study and overall. Number and percentage of patients with TEAEs by SOC order and decreasing frequency of PT within each SOC. Same presentation for pre-treatments AEs, SAEs, TEAEs, TEAEs leading to drug and study discontinuation, TEAEs by grade, TEAE leading to death. AE incidence table by RET mutation status, study and overall, for all types of TEAEs. Other safety evaluations including vital signs, ECG and laboratory data (descriptive).

Documents

Study results

rdct-obs14778-synopsis (addendum)-PDFA

rdct-obs14778-synopsis-PDFA