Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
|ClinicalTrials.gov Identifier: NCT01602939|
Recruitment Status : Recruiting
First Posted : May 21, 2012
Last Update Posted : August 29, 2016
|Condition or disease||Intervention/treatment||Phase|
|Systemic Mastocytosis||Drug: Cladribine and pegylated interpheron alpha-2a||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.|
|Study Start Date :||May 2012|
|Estimated Primary Completion Date :||February 2017|
|Estimated Study Completion Date :||June 2017|
Drug: Cladribine and pegylated interpheron alpha-2a
Cladribine (0.07 mg/Kg/day) s.c for 5 consecutive days each month for a total of 6 months.Cladribine daily doses could be increased up to 0.14 mg/Kg in the fourth, fifth and sixth cycles of therapy if no objetive response is achieved after the third cycle.
Pegylated Interpheron alpha-2a (1 mcgr/Kg) s.c weekly for a total of 6 months.
- To evaluate the effect of therapy on bone marrow mast cell infiltration. [ Time Frame: 6 months ]Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01602939
|Contact: Luis Escribano, MD, PhDfirstname.lastname@example.org|
|Contact: Iván Alvarez-Twose, MDemail@example.com|
|Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle||Recruiting|
|Toledo, Spain, 45071|
|Contact: Luis Escribano, MD, PhD +34925269335 firstname.lastname@example.org|
|Contact: Iván Alvarez-Twose, MD +34925269336 email@example.com|
|Principal Investigator: Luis Escribano, MD, PhD|
|Sub-Investigator: Iván Alvarez-Twose, MD|
|Principal Investigator:||Luis Escribano, MD, PhD||Instituto de Estudios de Mastocitosis de Castilla La Mancha|