Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis

Phase 2

• Conditions: Systemic Mastocytosis
• Interventions: Drug: Cladribine and pegylated interpheron alpha-2a

• Registration Number: NCT01602939
• Lead Sponsor: Hospital Virgen de la Salud

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 Description

Not available

Study Timeline

• Study Start: 2012-05
• Study First Submitted: 2012-05-16
• Study First Submitted QC: 2012-05-17
• Study First Posted: 2012-05-21
• Status Verified: 2016-08
• Last Update Submitted: 2016-08-26
• Last Update Posted: 2016-08-29
• Primary Completion: 2017-02
• Study Completion: 2017-06

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 10

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
2CDA+IFN	Cladribine and pegylated interpheron alpha-2a	-

Primary Outcome Measures

Name	Time	Method
To evaluate the effect of therapy on bone marrow mast cell infiltration.	6 months	Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.

Secondary Outcome Measures

Name	Time	Method
To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis.	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).	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.	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.	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.	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.	6 months	Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography.

Trial Locations

Locations (1)

Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle; 🇪🇸 Toledo, Spain