MedPath

Aplidin - Dexamethasone in Relapsed/Refractory Myeloma

Phase 3
Completed
Conditions
Relapsed/Refractory Multiple Myeloma
Interventions
Registration Number
NCT01102426
Lead Sponsor
PharmaMar
Brief Summary

Study of Plitidepsin in combination with dexamethasone versus dexamethasone alone in patients with relapsed/refractory multiple myeloma.

Detailed Description

Phase III Study in Patients with Relapsed/Refractory Multiple Myeloma to compare the efficacy of plitidepsin in combination with dexamethasone vs. dexamethasone alone measured by progression-free survival (PFS) and to evaluate tumor response, duration of response (DR), overall survival (OS) and to rule out any effect of plitidepsin on the duration of the QT/QTc interval (time corresponding to the beginning of depolarization to re-polarization of the ventricles).

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
255
Inclusion Criteria
  • Age ≥ 18 years.
  • Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) ≤ 2
  • Life expectancy ≥ 3 months.
  • Patients previously diagnosed with multiple myeloma
  • Patients must have relapsed or relapsed and refractory multiple myeloma (MM) after at least three but not more than six prior therapeutic regimens for MM, including induction therapy and stem cell transplant in candidate patients, which will be considered as only one regimen.
  • Patients must have received previous bortezomib-containing and lenalidomide-containing regimens (or thalidomide where lenalidomide is not available)
  • Women must have a negative serum pregnancy test
  • Voluntarily signed and dated written informed consent
Exclusion Criteria
  • Concomitant diseases/conditions
  • Women who are pregnant or breast feeding.
  • Concomitant medications that include corticosteroids, chemotherapy, or other therapy that is or may be active against MM
  • Known hypersensitivity to any involved study drug or any of its formulation components

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
DexamethasoneDexamethasonedexamethasone single agent
Plitidepsin+DexamethasoneDexamethasoneplitidepsin + dexamethasone combination
Plitidepsin+DexamethasonePlitidepsinplitidepsin + dexamethasone combination
Primary Outcome Measures
NameTimeMethod
Progression Free Survival (PFS) as Per Intention-to-treat (ITT)From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 5 years

To compare the efficacy of plitidepsin in combination with dexamethasone vs. dexamethasone alone as measured by progression-free survival (PFS) in patients with relapsed/refractory multiple myeloma (MM).

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Percentage of Participants With Progression Free Survival (PFS) as Per Intention-to-treat (ITT) at 6 MonthsFrom date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 6 months

To compare the efficacy of plitidepsin in combination with dexamethasone vs. dexamethasone alone as measured by progression-free survival (PFS) in patients with relapsed/refractory multiple myeloma (MM).

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Secondary Outcome Measures
NameTimeMethod
Progression-free Survival (Investigator Assessment)From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 5 years

The secondary study analysis was based on Investigator's assessment PFS data in the ITT efficacy population, defined as all patients randomized to either treatment arm. PFS was calculated from randomization to the first evidence of PD or death due to any cause. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions If the patient received further antitumor therapy before PD and within the timeframe expected for first follow-up, PFS was censored on the date of the last disease assessment prior to the administration of this antitumor therapy.

Percentage of Participants With Progression-free Survival (Investigator Assessment) at 6 MonthsFrom date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 6 months

The secondary study analysis was based on Investigator's assessment PFS data in the ITT efficacy population, defined as all patients randomized to either treatment arm. PFS was calculated from randomization to the first evidence of PD or death due to any cause. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions If the patient received further antitumor therapy before PD and within the timeframe expected for first follow-up, PFS was censored on the date of the last disease assessment prior to the administration of this antitumor therapy.

Overall SurvivalFrom randomization to the death due to any cause,assessed up to 5 years

Overall Survival (OS) is defined as the time from the date of randomization to the date of death or last contact

Percentage of Participants With Overall Survival at 12 MonthsFrom randomization to the death due to any cause,assessed up to 12 months

Overall Survival (OS) is defined as the time from the date of randomization to the date of death or last contact

Percentage of Participants With Overall Survival at 24 MonthsFrom randomization to the death due to any cause,assessed up to 24 months

Overall Survival (OS) is defined as the time from the date of randomization to the date of death or last contact

Duration of Response (Independent Review Committee)From the date of first documentation of response to the date of disease progression or death, assessed up to 5 years

DR was calculated from the date of first documentation of response to the date of disease progression or death. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Percentage of Participants With Duration of Response (Independent Review Committee) at 6 MonthsFrom the date of first documentation of response to the date of disease progression or death, assessed up to 6 months

DR was calculated from the date of first documentation of response to the date of disease progression or death. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Duration of Response (Investigator Assessment)From the date of first documentation of response to the date of disease progression or death, assessed up to 5 years

DR was calculated from the date of first documentation of response to the date of disease progression or death. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Percentage of Participants With Duration of Response (Investigator Assessment) at 6 MonthsFrom the date of first documentation of response to the date of disease progression or death, assessed up to 6 months

DR was calculated from the date of first documentation of response to the date of disease progression or death. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Best Overall Response (Independent Review Committee)From the date of first documentation of response to the date of disease progression or death, assessed up to 5 years

Very good partial response (VGPR) serum and urine M-protein detectable but not electrophoresis or \>90% reduction in serum M-protein and urine M-protein \<100 mg/24h Partial response (PR) ≥50% reduction in serum M-protein and 90% reduction in 24h urine M-protein or to \<200 mg/24h. 50% reduction in size of any soft tissue plasmacytomas Minor response (MR) 25%-49% reduction of serum M-protein and 50-89% reduction of 24h urine M-protein. 25-49% reduction in size of soft tissue plasmacytomas. No increase in size or number of bone lesions Stable disease (SD) No sCR, CR, VGPR, PR, MR or PD Progressive disease (PD) 25% increase from the lowest value: serum M-protein, urine M-protein, BM plasma cell. Increase in size, new bone lesions, soft tissue plasmacytomas or serum calcium \>11.5 mg/dL; NE, not evaluable

Overall Response Rate (Independent Review Committee)From the date of first documentation of response to the date of disease progression or death, assessed up to 5 years

Overall response rate including sCR, CR, VGPR, PR and MR. Very good partial response (VGPR) serum and urine M-protein detectable but not electrophoresis or \>90% reduction in serum M-protein and urine M-protein \<100 mg/24h Partial response (PR) ≥50% reduction in serum M-protein and 90% reduction in 24h urine M-protein or to \<200 mg/24h. 50% reduction in size of any soft tissue plasmacytomas Minor response (MR) 25%-49% reduction of serum M-protein and 50-89% reduction of 24h urine M-protein. 25-49% reduction in size of soft tissue plasmacytomas. No increase in size or number of bone lesions

Overall Response Rate (Independent Review Committee) Excluding MRFrom the date of first documentation of response to the date of disease progression or death, assessed up to 5 years

Includes sCR, CR, VGPR and PR (excludes MR). Very good partial response (VGPR) serum and urine M-protein detectable but not electrophoresis or \>90% reduction in serum M-protein and urine M-protein \<100 mg/24h Partial response (PR) ≥50% reduction in serum M-protein and 90% reduction in 24h urine M-protein or to \<200 mg/24h. 50% reduction in size of any soft tissue plasmacytomas

Best Overall Response (Investigator Assessment)From the date of first documentation of response to the date of disease progression or death, assessed up to 5 years

Very good partial response (VGPR) serum and urine M-protein detectable but not electrophoresis or \>90% reduction in serum M-protein and urine M-protein \<100 mg/24h Partial response (PR) ≥50% reduction in serum M-protein and 90% reduction in 24h urine M-protein or to \<200 mg/24h. 50% reduction in size of any soft tissue plasmacytomas Minor response (MR) 25%-49% reduction of serum M-protein and 50-89% reduction of 24h urine M-protein. 25-49% reduction in size of soft tissue plasmacytomas. No increase in size or number of bone lesions Stable disease (SD) No sCR, CR, VGPR, PR, MR or PD Progressive disease (PD) 25% increase from the lowest value: serum M-protein, urine M-protein, BM plasma cell. Increase in size, new bone lesions, soft tissue plasmacytomas or serum calcium \>11.5 mg/dL; NE, not evaluable

Overall Response Rate (Investigator Assessment)From the date of first documentation of response to the date of disease progression or death, assessed up to 5 years

Includes sCR, CR, VGPR, PR and MR. Very good partial response (VGPR) serum and urine M-protein detectable but not electrophoresis or \>90% reduction in serum M-protein and urine M-protein \<100 mg/24h Partial response (PR) ≥50% reduction in serum M-protein and 90% reduction in 24h urine M-protein or to \<200 mg/24h. 50% reduction in size of any soft tissue plasmacytomas Minor response (MR) 25%-49% reduction of serum M-protein and 50-89% reduction of 24h urine M-protein. 25-49% reduction in size of soft tissue plasmacytomas. No increase in size or number of bone lesions

Overall Response Rate (Investigator Assessment) Excluding MRFrom the date of first documentation of response to the date of disease progression or death, assessed up to 5 years

Very good partial response (VGPR) serum and urine M-protein detectable but not electrophoresis or \>90% reduction in serum M-protein and urine M-protein \<100 mg/24h Partial response (PR) ≥50% reduction in serum M-protein and 90% reduction in 24h urine M-protein or to \<200 mg/24h. 50% reduction in size of any soft tissue plasmacytomas

Trial Locations

Locations (82)

1003

🇬🇧

Bournemouth, United Kingdom

201

🇦🇹

Wien, Austria

1102

🇺🇸

New York, New York, United States

302

🇧🇪

Gent, Belgium

304

🇧🇪

Brugge, Belgium

706

🇩🇪

Frankfurt, Germany

708

🇩🇪

Freiburg, Germany

602

🇫🇷

Nantes, France

108

🇦🇺

Adelaide, Australia

105

🇦🇺

Parkville, Australia

301

🇧🇪

Brussels, Belgium

801

🇮🇹

Genova, Italy

1503

🇰🇷

Seoul, Korea, Republic of

702

🇩🇪

Munchen, Germany

202

🇦🇹

Graz, Austria

1507

🇰🇷

Hwasun, Korea, Republic of

204

🇦🇹

Innsbruck, Austria

205

🇦🇹

Wien, Austria

1303

🇬🇷

Patras, Greece

806

🇮🇹

Bari, Italy

501

🇨🇿

Praha, Czechia

503

🇨🇿

Hradec Kralove, Czechia

1401

🇮🇪

Dublin, Ireland

803

🇮🇹

Rozzano, Italy

1505

🇰🇷

Jeonju, Korea, Republic of

707

🇩🇪

Frankfurt, Germany

601

🇫🇷

Lille, France

606

🇫🇷

Rouen, France

703

🇩🇪

Heidelberg, Germany

1601

🇳🇿

Christchurch, New Zealand

705

🇩🇪

Essen, Germany

804

🇮🇹

San Giovanni Rotondo, Italy

1502

🇰🇷

Anyang, Korea, Republic of

1506

🇰🇷

Incheon, Korea, Republic of

1704

🇵🇱

Opole, Poland

805

🇮🇹

Reggio Emilia, Italy

604

🇫🇷

Vandœuvre-lès-Nancy, France

1504

🇰🇷

Seoul, Korea, Republic of

104

🇦🇺

South Brisbane, Australia

1103

🇺🇸

Los Angeles, California, United States

1104

🇺🇸

Canton, Ohio, United States

1107

🇺🇸

Tuscaloosa, Alabama, United States

1105

🇺🇸

Jacksonville, Florida, United States

102

🇦🇺

Canberra, Australia

101

🇦🇺

Geelong, Australia

106

🇦🇺

Perth, Australia

109

🇦🇺

Woodville, Australia

208

🇦🇹

Wien, Austria

303

🇧🇪

Brussels, Belgium

502

🇨🇿

Brno, Czechia

709

🇩🇪

Düsseldorf, Germany

1302

🇬🇷

Thessaloniki, Greece

1301

🇬🇷

Athens, Greece

704

🇩🇪

Würzburg, Germany

802

🇮🇹

Torino, Italy

1501

🇰🇷

Daejeon, Korea, Republic of

1508

🇰🇷

Seongnam, Korea, Republic of

1509

🇰🇷

Seoul, Korea, Republic of

902

🇳🇱

Rotterdam, Netherlands

901

🇳🇱

Rotterdam, Netherlands

1602

🇳🇿

Takapuna, New Zealand

1802

🇵🇹

Braga, Portugal

1201

🇪🇸

Barcelona, Spain

1801

🇵🇹

Porto, Portugal

2001

🇵🇷

San Juan, Puerto Rico

1001

🇬🇧

London, United Kingdom

1203

🇪🇸

Barcelona, Spain

1209

🇪🇸

Barcelona, Spain

1210

🇪🇸

Madrid, Spain

1207

🇪🇸

Madrid, Spain

1206

🇪🇸

Murcia, Spain

1204

🇪🇸

Palma de Mallorca, Spain

1208

🇪🇸

Salamanca, Spain

1202

🇪🇸

San Sebastián, Spain

1205

🇪🇸

Valencia, Spain

1901

🇨🇳

Taipei, Taiwan

1902

🇨🇳

Taipei, Taiwan

1903

🇨🇳

Taipei, Taiwan

1004

🇬🇧

Bradford, United Kingdom

1005

🇬🇧

Nottingham, United Kingdom

203

🇦🇹

Salzburg, Austria

1703

🇵🇱

Warszawa, Poland

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