Clinical Trial: Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

Brief Summary: 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.

Detailed Summary:
Sponsor: Hospital Virgen de la Salud

Current Primary Outcome: To evaluate the effect of therapy on bone marrow mast cell infiltration. [ Time Frame: 6 months ]

Original Primary Outcome: To evaluate the effect of therapy on the grade of bone marrow and cutaneous mast cell infiltration, as well as on organomegalies or bone alterations. [ Time Frame: 6 months ]

Current Secondary Outcome:

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

Original Secondary Outcome:

• To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis. [ Time Frame: 6 months ]
  Serum tryptase and any other mastocytosis-related altered biochemical parameter at diagnosis will be measured monthly until the end of therapy.
• To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis). [ Time Frame: 6 months ]
  Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy.
• To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a. [ Time Frame: 6 months ]
  Potentially drugs-related adverse events will be recorded in each case following accepted criteria (NIH CTCAE).

Dates:
Date Received: May 16, 2012
Date Started: May 2012
Date Completion: June 2017
Last Updated: August 26, 2016
Last Verified: August 2016