Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
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: 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
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 email@example.com |
|Contact: Iván Alvarez-Twose, MD +34925269336 firstname.lastname@example.org |
|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
|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 May 25, 2017
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Physiological Effects of Drugs