Clinical Trials /

Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis

NCT01602939

Description:

The aim of this study is to evaluate the efficacy in terms of clinical and biological response rates of Cladribine plus Pegylated Interpheron alpha-2a therapy in patients with advanced systemic mastocytosis carrying D816V or other exon 17 KIT mutations.

Related Conditions:
  • Systemic Mastocytosis
Recruiting Status:

Unknown status

Phase:

Phase 2/Phase 3

Trial Eligibility

Document

Title

  • Brief Title: Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
  • Official Title: Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.

Clinical Trial IDs

  • ORG STUDY ID: EC11-187
  • NCT ID: NCT01602939

Conditions

  • Systemic Mastocytosis

Interventions

DrugSynonymsArms
Cladribine and pegylated interpheron alpha-2a2CDA+IFN

Purpose

The aim of this study is to evaluate the efficacy in terms of clinical and biological response rates of Cladribine plus Pegylated Interpheron alpha-2a therapy in patients with advanced systemic mastocytosis carrying D816V or other exon 17 KIT mutations.

Trial Arms

NameTypeDescriptionInterventions
2CDA+IFNExperimental
  • Cladribine and pegylated interpheron alpha-2a

Eligibility Criteria

        Inclusion Criteria:

          -  Age older than 18 years.

          -  Diagnosis of advanced systemic mastocytosis (aggressive systemic mastocytosis or
             proggressing systemic mastocytosis) with D816V or other exon 17 KIT mutations.

          -  ECOG ≤ 3.

          -  Signed informed consent.

        Exclusion Criteria:

          -  Impaired liver function (total bilirubin ≥ 2.0 mg/dl, AST or ALT > 3 x upper limit of
             normal)not related to mastocytosis.

          -  Impaired renal function (≥ 2.0 mg/dL)not related to mastocytosis.

          -  Grade III-IV cytopenias not related to mastocytosis. Severe cardiopathy (grade III/IV
             of NYHA, or left ventricular ejection fraction < 50%).

          -  Pregnancy or breastfeeding.

          -  Female patients who do not use contraceptive methods.
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:To evaluate the effect of therapy on bone marrow mast cell infiltration.
Time Frame:6 months
Safety Issue:
Description:Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.

Secondary Outcome Measures

Measure:To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis.
Time Frame:6 months
Safety Issue:
Description:Serum tryptase and any other mastocytosis-related altered biochemical parameter at diagnosis will be measured monthly until the end of therapy.
Measure:To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis).
Time Frame:6 months
Safety Issue:
Description:Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy.
Measure:To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a.
Time Frame:6 months
Safety Issue:
Description:Potentially drugs-related adverse events will be recorded in each case following accepted criteria (NIH CTCAE).
Measure:To evaluate the effect of therapy on mastocytosis skin lesions.
Time Frame:6 moths
Safety Issue:
Description:Evaluation of cutaneous response will be assessed by macroscopic inspection including photographs and by skin immunohistochemestry.
Measure:To evaluate the effect of therapy on mastocytosis-related organomegalies.
Time Frame:6 months
Safety Issue:
Description:Evaluation of organomegalies response will be assessed by abdominal ultrasound and/or computerized tomography.
Measure:To evaluate the effect of therapy on mastocytosis-related bone alterations.
Time Frame:6 months
Safety Issue:
Description:Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography.

Details

Phase:Phase 2/Phase 3
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Hospital Virgen de la Salud

Trial Keywords

  • Mast cell
  • Mastocytosis
  • Mast cell disease

Last Updated

August 26, 2016