Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
This study is currently recruiting participants.
Verified August 2016 by LUIS ESCRIBANO, Hospital Virgen de la Salud
Information provided by (Responsible Party):
LUIS ESCRIBANO, Hospital Virgen de la Salud
First received: May 16, 2012
Last updated: August 26, 2016
Last verified: August 2016
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.
Drug: Cladribine and pegylated interpheron alpha-2a
||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
||Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.
Primary Outcome Measures:
Secondary Outcome Measures:
- 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.
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||February 2017 (Final data collection date for primary outcome measure)
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.
|Ages Eligible for Study:
||18 Years and older (Adult, Senior)
|Sexes Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01602939
|Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle
|Toledo, Spain, 45071 |
|Contact: Luis Escribano, MD, PhD +34925269335 firstname.lastname@example.org |
|Contact: Iván Alvarez-Twose, MD +34925269336 email@example.com |
|Principal Investigator: Luis Escribano, MD, PhD |
|Sub-Investigator: Iván Alvarez-Twose, MD |
Hospital Virgen de la Salud
||Luis Escribano, MD, PhD
||Instituto de Estudios de Mastocitosis de Castilla La Mancha
||LUIS ESCRIBANO, Director of the Instituto de Estudios de Mastocitosis de Castilla La Mancha, Hospital Virgen de la Salud
History of Changes
|Other Study ID Numbers:
|Study First Received:
||May 16, 2012
||August 26, 2016
|Individual Participant Data (IPD) Sharing Statement:
|Plan to Share IPD:
Keywords provided by LUIS ESCRIBANO, Hospital Virgen de la Salud:
Mast cell disease
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on September 21, 2017
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Physiological Effects of Drugs