Description:
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.
Title
- Brief Title: Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
- Official Title: Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.
Clinical Trial IDs
- ORG STUDY ID:
EC11-187
- NCT ID:
NCT01602939
Conditions
Interventions
| Drug | Synonyms | Arms |
|---|
| Cladribine and pegylated interpheron alpha-2a | | 2CDA+IFN |
Purpose
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.
Trial Arms
| Name | Type | Description | Interventions |
|---|
| 2CDA+IFN | Experimental | | - Cladribine and pegylated interpheron alpha-2a
|
Eligibility Criteria
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.
| Maximum Eligible Age: | N/A |
| Minimum Eligible Age: | 18 Years |
| Eligible Gender: | All |
| Healthy Volunteers: | No |
Primary Outcome Measures
| Measure: | To evaluate the effect of therapy on bone marrow mast cell infiltration. |
| Time Frame: | 6 months |
| Safety Issue: | |
| Description: | Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples. |
Secondary Outcome Measures
| Measure: | To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis. |
| Time Frame: | 6 months |
| Safety Issue: | |
| Description: | Serum tryptase and any other mastocytosis-related altered biochemical parameter at diagnosis will be measured monthly until the end of therapy. |
| Measure: | To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis). |
| Time Frame: | 6 months |
| Safety Issue: | |
| Description: | Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy. |
| Measure: | To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a. |
| Time Frame: | 6 months |
| Safety Issue: | |
| Description: | Potentially drugs-related adverse events will be recorded in each case following accepted criteria (NIH CTCAE). |
| Measure: | To evaluate the effect of therapy on mastocytosis skin lesions. |
| Time Frame: | 6 moths |
| Safety Issue: | |
| Description: | Evaluation of cutaneous response will be assessed by macroscopic inspection including photographs and by skin immunohistochemestry. |
| Measure: | To evaluate the effect of therapy on mastocytosis-related organomegalies. |
| Time Frame: | 6 months |
| Safety Issue: | |
| Description: | Evaluation of organomegalies response will be assessed by abdominal ultrasound and/or computerized tomography. |
| Measure: | To evaluate the effect of therapy on mastocytosis-related bone alterations. |
| Time Frame: | 6 months |
| Safety Issue: | |
| Description: | Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography. |
Details
| Phase: | Phase 2/Phase 3 |
| Primary Purpose: | Interventional |
| Overall Status: | Unknown status |
| Lead Sponsor: | Hospital Virgen de la Salud |
Trial Keywords
- Mast cell
- Mastocytosis
- Mast cell disease
Last Updated
August 29, 2016
