Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
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|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01602939
|Contact: Luis Escribano, MD, PhDemail@example.com|
|Contact: Iván Alvarez-Twose, MDfirstname.lastname@example.org|
|Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle||Recruiting|
|Toledo, Spain, 45071|
|Contact: Luis Escribano, MD, PhD +34925269335 email@example.com|
|Contact: Iván Alvarez-Twose, MD +34925269336 firstname.lastname@example.org|
|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|