Systemic Mastocytosis Clinical Trial
Official title:
Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.
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.
Status | Recruiting |
Enrollment | 10 |
Est. completion date | June 2017 |
Est. primary completion date | February 2017 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 18 Years and older |
Eligibility |
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. |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Spain | Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle | Toledo |
Lead Sponsor | Collaborator |
---|---|
Hospital Virgen de la Salud |
Spain,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | To evaluate the effect of therapy on bone marrow mast cell infiltration. | Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples. | 6 months | No |
Secondary | To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis. | Serum tryptase and any other mastocytosis-related altered biochemical parameter at diagnosis will be measured monthly until the end of therapy. | 6 months | No |
Secondary | To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis). | Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy. | 6 months | No |
Secondary | To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a. | Potentially drugs-related adverse events will be recorded in each case following accepted criteria (NIH CTCAE). | 6 months | Yes |
Secondary | To evaluate the effect of therapy on mastocytosis skin lesions. | Evaluation of cutaneous response will be assessed by macroscopic inspection including photographs and by skin immunohistochemestry. | 6 moths | No |
Secondary | To evaluate the effect of therapy on mastocytosis-related organomegalies. | Evaluation of organomegalies response will be assessed by abdominal ultrasound and/or computerized tomography. | 6 months | No |
Secondary | To evaluate the effect of therapy on mastocytosis-related bone alterations. | Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography. | 6 months | No |
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