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Sequential Trial on Reduced Intensity Conditioning (RIC) Allogeneic Transplantation

Phase 1
Completed
Conditions
Hematologic Malignancies
Multiple Myeloma
Interventions
Drug: Bz (Bortezomib)
Drug: Len (lenalidomide)
Registration Number
NCT01460420
Lead Sponsor
Stichting European Myeloma Network
Brief Summary

The aim of the current study is to improve the outcome of patients with hematologic malignancies (in a phase I trial) and more specifically multiple myeloma (in a phase II trial) by 2 interventions: reduce the risk of graft-versus-host disease (GVHD) and improve the efficacy of the procedure decreasing the risk of relapses after transplant.

Currently, the standard approach used in most centers to prevent graft-versus-host disease after allogeneic transplantation is based on the combination of a calcineurin inhibitor (cyclosporine or tacrolimus) plus a short course of methotrexate. Unfortunately, this strategy is far from ideal, since the risk of acute GVHD is in the range of 30-40% among patients receiving a matched related donor transplantation and even higher among patients receiving transplantation from an unrelated donor while the incidence of chronic GVHD is 60-70% among patients receiving peripheral blood progenitor cells from either a related or unrelated donor.

As far as the patients with multiple myeloma (MM) is concerned, although the development of new drugs has markedly changed the outcome and management of these patients, allogeneic transplantation so far appears to be the only curative option, especially among those patients relapsing after first line treatment. Nevertheless, still new strategies within the allogeneic transplant setting are needed to improve its results.

Relapses may occur either extramedullary (very common in this setting) or systemic. In order to reduce the risk of systemic relapses the investigators will use maintenance therapy with Lenalidomide (Len) which, together with bortezomib (Bz) should contribute to eradicate minimal residual disease (MRD). In case the patient do not obtain complete remission or near complete remission after transplant, in addition to the maintenance therapy, the investigators will use four intensification cycles with VRD (Bz-Len-Dexamethasone).

In summary, the goal is to optimize the efficacy of allogeneic transplantation by two interventions: one focused on reducing the risk of relapse and the other on reducing the incidence of GVHD.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
49
Inclusion Criteria

Phase I: For the first 10 patients:

  • Patients with any haematological malignancy in > CR1 (first complete remission)
  • Suitable related donor human leukocyte antigen (HLA)identical
  • Age > 18 and < 70 years

For the 10 subsequent patients:

  • Patients with any haematological malignancy candidates to receive an allogeneic transplant
  • Suitable related or unrelated donor (a maximum of 1 mismatched is allowed)
  • Age > 18 and < 70 years phase II trial:
  • High-risk multiple myeloma patients at first relapse / second complete remission candidates to receive an allogeneic transplantation
  • Age:> 18 < 70 years.
  • Suitable donor, related or unrelated (a maximum of 1 mismatched is allowed)
  • Measurable disease
  • High risk first relapse is defined as:
  • First early relapse after Autologous Stem Cell Transplant (ASCT)< 24 months
  • First late relapses in case the patient does not achieve CR after second ASCT
  • First relapse in patients with poor cytogenetic features
  • All subjects must be able to comply with the Lenalidomide Pregnancy Prevention Risk Management Plan.
Exclusion Criteria

Any of the following:

  • Prior severe comorbidity such as:

    • Heart failure or previous infarction
    • Uncontrolled Hypertension
    • Arrhythmia
    • Cirrhosis
  • Peripheral neuropathy >Grade 2, 14 days prior to inclusion

  • Psychiatric disease

  • Prior history of other neoplasia except for carcinoma in situ in the last 10 years

  • Hypersensitivity to Bz, Boric acid mannitol.

  • Patients unable to use appropriate contraceptive methods

  • Patients who have received an investigational drug 30 days prior to inclusion

  • Positive human immunodeficiency virus (HIV) or active viral hepatitis

  • Patients with pericardial disease

  • Patients with acute diffuse infiltrative pulmonary disease

  • Patients not willing to comply with the Lenalidomide Pregnancy Prevention Risk Management Plan

  • Patients not willing to receive thromboprophylaxis during the consolidation phase will not be eligible.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Bortezomib + LenalidomideBz (Bortezomib)After conditioning treatment and graft versus host disease prophylaxis with Bz 1.3 mg/m2 on days +1, +4 and +7 plus sirolimus/rapamycin at a dose of 6 mg po on day -5 and then 4 mg per day in order to maintain serum levels in the range of 6-12 ng /mL, a maintenance therapy with Bz 1.3 mg/m2 on days 1, 8 and 15 in cycles of 56 days up to 6 cycles post-transplant and on day +180 Len will be started at a dose of 5 mg and will be maintained until relapse.
Bortezomib + LenalidomideLen (lenalidomide)After conditioning treatment and graft versus host disease prophylaxis with Bz 1.3 mg/m2 on days +1, +4 and +7 plus sirolimus/rapamycin at a dose of 6 mg po on day -5 and then 4 mg per day in order to maintain serum levels in the range of 6-12 ng /mL, a maintenance therapy with Bz 1.3 mg/m2 on days 1, 8 and 15 in cycles of 56 days up to 6 cycles post-transplant and on day +180 Len will be started at a dose of 5 mg and will be maintained until relapse.
Primary Outcome Measures
NameTimeMethod
Phase I trial: Safety of Len + Bz in patients with hematologic malignancies Phase II trial: Safety and efficacy of an optimized strategy of allogeneic transplantation in multiple myeloma undergoing allogeneic transplantation.Up to one year after transplant

For phase I trial: safety of Len + Bz. The phase I trial safety criteria will be evaluated in terms of (1) engraftment defined as \> 500 granulocytes / microL and \> 20.000 platelets / microL x 3 consecutive days will be required for 9/10 patients, (2) incidence of neuropathy grades 3-4 attributed to Bz \> 20% (3) incidence of gastrointestinal toxicity attributed to Bz \> 20%.

For phase II trial: safety evaluated through adverse events and toxicity and efficacy evaluated as reduction of relapse rate as defined by the EBMT criteria.

Secondary Outcome Measures
NameTimeMethod
Incidence of GVHD with this combination (phase I and II)Up to one year after transplant

Evaluation of a novel combination of Bz plus Len to prevent GVHD after allogeneic transplantation in patients with haematologic malignancies/MM

Phase II: response and relapse rate of this approachUp to one year after transplant

Reduction of relapse rate as defined by the EBMT (European Group for Blood, and Marrow Transplant)criteria.

Phase II: safety of the procedureUp to one year after transplant

For all patients safety will be assessed by the reporting of adverse events starting with the first study-related procedure and up to 30 days after the treatment period. The severity of adverse events will be assessed using National Cancer Institute (NCI) common toxicity criteria (CTC).

Evaluate the efficacy on survivalUp to one year after transplant

Evaluate the efficacy of the procedure in terms of event free and overall survival

Efficacy of positron emission tomography (PET scan)and local radiotherapyUp to one year after transplant

Analyze the prognostic value and efficacy of imaging studies using PET scan and local radiotherapy in involved fields prior to or after (\> 100 days) conditioning

Trial Locations

Locations (10)

Azienda Ospedaliera Universitaria di Udine

🇮🇹

Udine, Italy

S Giovanni Battista Hospital

🇮🇹

Torino, Italy

Medizinische Klinik and Poliklinik II, University Hospital

🇩🇪

Würzburg,, Germany

Hospital Clinic i Provincial,

🇪🇸

Barcelona,, Spain

Hospital Clinico Universitario Salamanca,

🇪🇸

Salamanca,, Spain

Hospital Universitario Ramón y Cajal

🇪🇸

Madrid, Spain

Hospital Santa Creu I Sant Pau,

🇪🇸

Barcelona,, Spain

Karolinska University Hospital, Huddinge

🇸🇪

Stockholm, Sweden

Hospital Universitario Virgen del Rocío,

🇪🇸

Sevilla, Spain

Hospital Gregorio Marañón,

🇪🇸

Madrid, Spain

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