Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis

Hospital Virgen de la Salud

Status and phase: Unknown; Phase 3; Phase 2

Conditions: Systemic Mastocytosis

Treatments: Drug: Cladribine and pegylated interpheron alpha-2a

Study type: Interventional

Funder types: Other

Identifiers

NCT01602939

EC11-187

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

2CDA+IFN

Experimental group

Treatment:

Drug: Cladribine and pegylated interpheron alpha-2a