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Study of Plitidepsin (Aplidin®) in Combination With Bortezomib and Dexamethasone in Patients With Multiple Myeloma

Phase 1
Completed
Conditions
Multiple Myeloma
Interventions
Registration Number
NCT02100657
Lead Sponsor
PharmaMar
Brief Summary

Study of Plitidepsin (Aplidin®) to determine the recommended dose (RD) of plitidepsin in Combination with Bortezomib and Dexamethasone in Patients with Relapsed and/or Refractory Multiple Myeloma.

Detailed Description

Phase I Multicenter, Open-label, Dose-escalating Clinical and Pharmacokinetic Trial of Plitidepsin (Aplidin®) to determine the recommended dose (RD) of plitidepsin in combination with bortezomib and dexamethasone in patients with relapsed and/or refractory multiple myeloma (MM), to determine the efficacy of the combination plitidepsin/bortezomib/dexamethasone, to evaluate the safety and tolerability of the combination in patients with relapsing and/or refractory MM and to study the pharmacokinetics (PK) and pharmacodynamics (PDy) of plitidepsin in combination with bortezomib and dexamethasone.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
39
Inclusion Criteria
  • Age ≥ 18 years.
  • Prior autologous transplantation (HSCT) patients are allowed.
  • Patients must have received at least one previous treatment line of induction, chemotherapy, chemotherapy and transplantation or previous treatment with bortezomib or another proteasome drug
Exclusion Criteria
  • Previous treatment with plitidepsin.
  • Active or metastatic primary malignancy other than MM.
  • Serious concomitant systemic disorders
  • History of hypersensitivity reactions to bortezomib, polyoxyl 35 castor oil or mannitol
  • Neuropathy
  • Pregnant and/or lactating women
  • HIV infection
  • Active hepatitis B or C virus infection.
  • Treatment with any Investigational Medicinal Product (IMP) in the 30 days before inclusion in the study
  • Plasma cell leukemia at the time of study entry
  • Contraindication for the use of steroids

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
plitidepsin + bortezomib + dexamethasonePlitidepsinPlitidepsin will be administered as a 3-hour (h) intravenous (i.v.) infusion on Day (D) 1 and 15, every four weeks (q4wk). Bortezomib will be administered as a subcutaneous (s.c.) injection on D1, 4, 8 and 11, q4wk, for a maximum of eight cycles. Dexamethasone will be taken orally on D1, 8, 15 and 22, q4wk
plitidepsin + bortezomib + dexamethasoneBortezomibPlitidepsin will be administered as a 3-hour (h) intravenous (i.v.) infusion on Day (D) 1 and 15, every four weeks (q4wk). Bortezomib will be administered as a subcutaneous (s.c.) injection on D1, 4, 8 and 11, q4wk, for a maximum of eight cycles. Dexamethasone will be taken orally on D1, 8, 15 and 22, q4wk
plitidepsin + bortezomib + dexamethasoneDexamethasonePlitidepsin will be administered as a 3-hour (h) intravenous (i.v.) infusion on Day (D) 1 and 15, every four weeks (q4wk). Bortezomib will be administered as a subcutaneous (s.c.) injection on D1, 4, 8 and 11, q4wk, for a maximum of eight cycles. Dexamethasone will be taken orally on D1, 8, 15 and 22, q4wk
Primary Outcome Measures
NameTimeMethod
Recommended Dose of Bortezomib in Combination With Plitidepsin and DexamethasoneAfter 28-day cycle

To determine the recommended dose (RD) of plitidepsin in combination with bortezomib and dexamethasone in patients with relapsed and/or refractory multiple myeloma (MM). To define the RD, patients will be evaluated for DLTs during the first 28-day cycle. The RD will be the highest DL at which fewer than two out of six (33%) of evaluable patients experience a DLT during the first 28-day cycle.

Number of Participants Who Experienced Dose-Limiting Toxicities (DLTs)After 28-day cycle

DLTs were defined as:

Hematological Toxicity

* Grade 3/4 neutropenia associated with fever or lasting\>7 days related to the study treatment

* Grade 3/4 thrombocytopenia accompanied by grade 3/4 hemorrhage

* Extensive bone marrow (BM) infiltration, DLT was defined as grade 4 thrombocytopenia with grade 3/4 hemorrhage or grade 4 neutropenia lasting \>7 days or with fever Non-hematological Toxicity

* Grade 3/4 nausea and vomiting refractory to antiemetic therapy

* Grade≥3 muscular Adverse events (AE) (myalgia, muscular weakness, muscle cramps, myopathy)

* Grade≥3 alanine aminotransferase (ALT)/Aspartate aminotransferase (AST) lasting more than 1 week

* Grade≥3 bilirubin increase

* Grade≥3 creatine phosphokinase (CPK) increase

* Cardiac toxicity

* Symptomatic or treatment-requiring grade ≥1 cardiac arrhythmia related to plitidepsin

* Grade≥1 left ventricular systolic dysfunction related to plitidepsin

* Neuropathic pain and peripheral sensory neuropathy related to BTZ

Recommended Dose of Plitidepsin in Combination With Bortezomib and DexamethasoneAfter 28-day cycle

To determine the recommended dose (RD) of plitidepsin in combination with bortezomib and dexamethasone in patients with relapsed and/or refractory multiple myeloma (MM). To define the RD, patients will be evaluated for DLTs during the first 28-day cycle. The RD will be the highest DL at which fewer than two out of six (33%) of evaluable patients experience a DLT during the first 28-day cycle.

Recommended Dose of Dexamethasone in Combination With Plitidepsin and BortezomibAfter 28-day cycle

To determine the recommended dose (RD) of plitidepsin in combination with bortezomib and dexamethasone in patients with relapsed and/or refractory multiple myeloma (MM). To define the RD, patients will be evaluated for DLTs during the first 28-day cycle. The RD will be the highest DL at which fewer than two out of six (33%) of evaluable patients experience a DLT during the first 28-day cycle.

Secondary Outcome Measures
NameTimeMethod
Overall Response RateResponse or disease progression was assessed on Day 1 of each cycle, assessed up to 4 years

Overall response rate (ORR), including stringent complete response (sCR), complete response (CR), very good partial response (VGPR) and partial response (PR) according to the International Myeloma Working Group (IMWG) criteria

Time to ProgressionFrom the date of the first infusion to the date of documented PD or death due to PD, up to 4 years

Time to progression (TTP) was calculated from the date of the first infusion to the date of documented PD or death due to PD.

Event-free SurvivalFrom the date of first infusion to the date of documented PD or death, up to 4 years

Event-free survival (EFS) was calculated from the date of first infusion to the date of documented PD or death, but could include additional events besides death and PD that were considered of importance

Event-free Survival Ratesfrom the date of first infusion to the date of documented PD or death, up to 4 years

Event-free survival (EFS) was calculated from the date of first infusion to the date of documented PD or death, but could include additional events besides death and PD that were considered of importance

Progression-free Survivalfrom the date of the first infusion to the date of documented PD or death, up to 4 years

Progression-free survival (PFS) was calculated from the date of the first infusion to the date of documented PD or death (regardless of the reason), whichever comes first

Progression-free Survival RatesFrom the date of the first infusion to the date of documented PD or death, up to 4 years

Progression-free survival (PFS) was calculated from the date of the first infusion to the date of documented PD or death (regardless of the reason), whichever comes first

Response According to International Myeloma Working Group CriteriaResponse or disease progression was assessed on Day 1 of each cycle, assessed up to 4 years

Stringent complete response (sCR) normal Free Light Chains (FLC) ratio. No clonal cells Complete response (CR) no serum/urine M-protein, no evidence of soft tissue plasmacytoma. \<5% clonal plasma cells 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

Duration of ResponseFrom the date of the first documentation of response to the date of PD or further therapy or death, up to 4 years

Duration of response (DR) was analyzed for all patients in whom a response had been observed and was calculated from the date of the first documentation of response to the date of PD or further therapy or death

Time to Progression RatesFrom the date of the first infusion to the date of documented PD or death due to PD, up to 4 years

Time to progression (TTP) was calculated from the date of the first infusion to the date of documented PD or death due to PD.

Trial Locations

Locations (7)

Institut Gustave Roussy

🇫🇷

Villejuif, France

Hospital Universitario Germans Trias I Pujol

🇪🇸

Badalona, Barcelona, Spain

Clínica Universidad de Navarra

🇪🇸

Pamplona, Navarra, Spain

MD Anderson Cancer Center Madrid

🇪🇸

Madrid, Spain

Hospital Universitario Virgen del Rocío

🇪🇸

Sevilla, Spain

Hospital Universitari i Politècnic la Fe

🇪🇸

Valencia, Spain

Hospital Universitario Salamanca

🇪🇸

Salamanca, Spain

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