Clinical trial NCT03353545: Retrospective and Prospective Study of POmalidomide Plus LoW Dose Dexa Efficacy in RRMM Patients Under Real-Life Conditions
Official title: A Retrospective Chart Review and a Prospective Study of the POmalidomide Plus LoW Dose Dexamethasone Efficacy in Relapsed/ ReFractory Multiple Myeloma Patients Under Real-Life Conditions,
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Relapse/Refractory Multiple Myeloma
Lead sponsor: Genesis Pharma S.A.
Sponsor class: INDUSTRY
First posted: 2017-11-27
Enrollment: 110
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: A total of 100 adult patients with RRMM who have been initiated on Pom/LoDex treatment as indicated in the product's Summary of Product Characteristics (SmPC) (i.e., after having received at least two prior therapies including both lenalidomide and bortezomib and whose disease progressed after the last treatment) between 01 January 2016 and 28 February 2019 and who are eligible for participation in the study according to the inclusion and exclusion criteria -as outlined in Section 10- are planned to be recruited in the present clinical study. For the justification of the sample size please refer to Section 13.1.

Brief summary: The importance of real-world evidence studies stems from the following considerations. The study population of a specific clinical trial needs to meet strict inclusion and exclusion criteria, which result in a population of participants that is not necessarily representative of the study population of interest treated in routine care. Furthermore, the outcomes of a clinical trial occur under controlled conditions that do not necessarily reflect the routine healthcare practice. This is especially true among patient populations with challenging to treat disease such as in MM, where personalized therapeutic approaches are commonly considered taking into consideration the patients' age and associated comorbidities, among other factors. In addition, observational studies, due to their non-interventional nature, often show increased degree of heterogeneity across the enrolled patient populations compared to clinical studies, thus aiding generalizability of the results.

In light of the above and due to the scarcity of evidence regarding the outcomes for patients with RRMM receiving Pom/LoDex in routine clinical practice, this retrospective chart review and prospective observational study aims to assess the PFS and response to treatment as well as to obtain real-world evidence on the utilization patterns and management strategy of Pom/LoDex in routine clinical care settings in Greece.

This is a non-interventional, multicenter, single-country, retrospective chart review and prospective cohort study which will include a representative sample of patients with RRMM who have been initiated on Pom/LoDex between 01 January 2016 and 28 February 2019 in the third line and beyond treatment setting under routine care conditions in Greece.

The study will be carried out by hospital-based hematology specialists practicing in geographically diverse locations throughout Greece and will be conducted under real-world conditions of daily clinical practice.


Eligibility criteria: * Patients eligible for inclusion in this study have to meet all of the following criteria:

  * Adult subjects (aged ≥18 years) of either gender.
  * Subjects with a documented RRMM diagnosis according to IMWG or EBMT criteria prior to Pom/LoDex treatment.
  * Subjects who have been initiated on Pom/LoDex treatment as per the product's Summary of Product Characteristics (SmPC) between 01 January 2016 and 28 February 2019, after having received at least two prior therapies including both lenalidomide and bortezomib and whose disease progressed after the last treatment.\*
  * Note: Retrospective patients who at the time of their enrollment in the study have already completed 3 or more cycles with Pom/LoDex must have available at least one post-baseline response assessment
  * Subjects with available medical files/records and detailed historical data on their disease course and clinical management.
  * Provision of signed informed consent form (ICF) for collecting and analyzing medical data pertinent to the objectives of this study.\*\*

    * Note: For retrospective subjects who are deceased at the date of enrollment into the study a waiver of consent will be requested by the Hospital Scientific Committee and/or Administrative Board of each participating site to either implement a hospital ICF already in place or provide written approval of this study- specific waiver. In the occasion that waiver of consent is not granted by the Scientific Committee and/or Administrative Board of the study site, deceased subjects will not be enrolled in the study.

Exclusion Criteria:

A patient who meets any of the following criteria will be excluded from participation in this study:

* Prior malignancy (within the 3 years preceding initial diagnosis of MM).
* Concurrent administration of anti-cancer regimens for malignancies other than MM.
* Subjects currently participating or who have participated, during the treatment phase, in any investigational program with interventions outside of routine clinical practice.

Primary outcomes:
measure: Effectiveness of Pom/LoDex in terms of median PFS
timeFrame: median time from start of Pom/LoDex treatment to disease progression or death, through study completion and maximum 50 months
Secondary outcomes:
measure: Response to Pom/LoDex treatment in terms of ORR
timeFrame: from start of Pom/LoDex until achievement of PR or better, through study completion and maximum 50 months
measure: Response to Pom/LoDex treatment in terms of CBR
timeFrame: from start of Pom/LoDex until achievement of MR or better, through study completion and maximum 50 months
measure: Response to Pom/LoDex treatment in terms of DCR
timeFrame: from start of Pom/LoDex until achievement of SD or better, through study completion and maximum 50 months
measure: TTR among the RRMM study population who achieved at least partial response (PR)
timeFrame: median from start of Pom/LoDex to first documented response (TTR), through study completion and maximum 50 months
measure: DoR among the RRMM study population who achieved at least partial response (PR)
timeFrame: median from start of Pom/LoDex to first disease progrestion or death (DoR), through study completion and maximum 50 months
measure: 12-month PFS rate
timeFrame: time from start of Pom/LoDex until 12 months
measure: Time to progression
timeFrame: median time from start of Pom/LoDex until disease progression or death, through study completion and maximum 50 months
measure: Real-world utilization patterns of Pom/LoDex in the RRMM study population
timeFrame: no time frame - only proportion of patients in 12 month follow-up
Other outcomes:
measure: Evaluate the effectiveness and response to Pom/LoDex treatment in the subpopulations of RRMM patients who have been initiated on Pom/LoDex in the third line versus later line setting
timeFrame: from start of Pom/LoDex until 12 month follow-up
measure: To evaluate the improvement of the QoR with Pom/LoDex continued treatment
timeFrame: from start of Pom/LoDex until 12 month follow-up
Locations:
city: Patra
country: Greece
facility: Ag.Andreas General Hospital
Study officials:
affiliation: Genesis Pharma S.A.
name: Kiki Karvounis
role: STUDY_DIRECTOR
References:
citation: Terpos E, Repousis P, Lalayanni C, Hatjiharissi E, Assimakopoulou T, Vassilopoulos G, Pouli A, Spanoudakis E, Michalis E, Pangalis G, Ntanasis-Stathopoulos I, Poziopoulos C, Kyrtsonis MC, Pappa V, Symeonidis A, Georgopoulos C, Zikos PM, Gavriatopoulou M, Papadaki HA, Dadakaridou M, Karvounis-Marolachakis K, Katodritou E. Pomalidomide Plus Low-Dose Dexamethasone in Relapsed/Refractory Multiple Myeloma Patients: Results of the Real-World "POWERFUL" Study. J Clin Med. 2021 Apr 5;10(7):1509. doi: 10.3390/jcm10071509.