Clinical trial NCT04004338: Carfilzomib in Combination for the Treatment of RR MM
Official title: Patient Related Outcomes in Real Life Prospective Follow up Study: Carfilzomib in Combination for the Treatment of RR MM
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Multiple Myeloma
Relapse Multiple Myeloma
Refractory Multiple Myeloma
Keywords: Carfilzomib
Multiple Myeloma
Lead sponsor: Black Sea Hematology Association
Sponsor class: OTHER
Collaborators: name: Amgen
First posted: 2019-07-02
Enrollment: 300
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include patients with relapsed/refractory MM who have received 1 to 3 prior lines of therapy with documented data in the medical record regarding diagnosis (month and year), the regimens used in 1st, 2nd, and 3rd line as applicable, whether stem cell transplant was part of 1st, 2nd, and 3rd line of therapy at participating clinical sites in Turkey.

Brief summary: Carfilzomib is approved in Turkey for the treatment of adult relapsed multiple myeloma patients who have received at least one prior treatment. It is approved for use in combination with lenalidomide and dexamethasone (KRd) in and with dexamethasone alone (Kd). The purpose of this study is to describe contemporary, real-world patterns of patient characteristics, clinical disease presentation, prior therapeutic regimen chosen, and clinical outcomes in participants with relapsed/refractory (R/R) multiple myeloma (MM) who receive Carfilzomib combination treatment. Real-world evidence is crucial to understand how carfilzomib-based regimens are used in practice and in relation to local prescribing information.

This is a prospective, non-interventional, observational study.

The study population will include patients with relapsed/refractory MM who have received 1 to 3 prior lines of therapy with documented data in the medical record regarding diagnosis (month and year), the regimens used in 1st, 2nd, and 3rd line as applicable, whether stem cell transplant was part of 1st, 2nd, and 3rd line of therapy at participating clinical sites in Turkey.

Detailed description: Carfilzomib is approved in Turkey for the treatment of adult relapsed multiple myeloma patients who have received at least one prior treatment. It is approved for use in combination with lenalidomide and dexamethasone (KRd) in and with dexamethasone alone (Kd). The purpose of this study is to describe contemporary, real-world patterns of patient characteristics, clinical disease presentation, prior therapeutic regimen chosen, and clinical outcomes in participants with relapsed/refractory (R/R) multiple myeloma (MM) who receive Carfilzomib combination treatment. Real-world evidence is crucial to understand how carfilzomib-based regimens are used in practice and in relation to local prescribing information.

This is a prospective, non-interventional, observational study.

The study population will include patients with relapsed/refractory MM who have received 1 to 3 prior lines of therapy with documented data in the medical record regarding diagnosis (month and year), the regimens used in 1st, 2nd, and 3rd line as applicable, whether stem cell transplant was part of 1st, 2nd, and 3rd line of therapy at participating clinical sites in Turkey.

For the primary and secondary objectives, analyses will be descriptive and include estimations; no formal hypotheses will be tested.

The study will enroll 300 participants. This multi-center trial will be conducted in Turkish Hematology clinics. The overall time to participate in this study is 12 months. Participants will be evaluated and followed-up for a period of at 12 months, until death, are lost to follow-up, or the end of the study, whichever comes first.

All hematology centers that treat MM will be eligible for participation in this study; it is likely that most participating sites will be academic institutions and/or large specialized hematology centers that treat a relatively high volume of patients with MM. It is estimated that a selection of 20 sites will be required to achieve a target of 300 patients in this study.

It is estimated that 300 RR MM patients receiving treatment will be included in the study. It is expected that this sample size will provide acceptable precision around the estimates of the primary and secondary study outcomes.

Eligibility criteria: Inclusion Criteria:

* 18 years and older
* Relapsed/refractory MM patients who have received 1 to 3 prior lines of therapy
* Is willing and able to sign informed consent (ICF) to participate
* Patients receiving carfilzomib equal or less than 2 months (≤2 cycles) according to regulatory approvals

Exclusion Criteria:

* Is reporting to a site in this study for a second opinion (consultation only) or participants whose frequency of consult and follow-up are not adequate for case report form (eCRF) completion.
* Is participating in another study (observational or interventional) that prohibits participation in this study.
* Patients receiving carfilzomib more than 2 months (\>2 cycles).

Primary outcomes:
measure: Progression free survival
timeFrame: 24 months
Secondary outcomes:
measure: Dyspnea Frequency
timeFrame: 24 months
Other outcomes:
Locations:
city: Samsun
country: Turkey
facility: 19 Mayıs University Faculty of Medicine
Study officials:
affiliation: 19 Mayıs University Faculty of Medicine
name: Mehmet Turgut, Prof
role: PRINCIPAL_INVESTIGATOR
References: