Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
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ClinicalTrials.gov Identifier: NCT01602939 |
Recruitment Status
:
Recruiting
First Posted
: May 21, 2012
Last Update Posted
: August 29, 2016
|
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Systemic Mastocytosis | Drug: Cladribine and pegylated interpheron alpha-2a | Phase 2 Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 10 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations. |
Study Start Date : | May 2012 |
Estimated Primary Completion Date : | February 2017 |
Estimated Study Completion Date : | June 2017 |

Arm | Intervention/treatment |
---|---|
Experimental: 2CDA+IFN |
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. |
- To evaluate the effect of therapy on bone marrow mast cell infiltration. [ Time Frame: 6 months ]Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.
- 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.

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Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01602939
Contact: Luis Escribano, MD, PhD | +34925269335 | lescribanom@sescam.jccm.es | |
Contact: Iván Alvarez-Twose, MD | +34925269336 | ivana@sescam.jccm.es |
Spain | |
Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle | Recruiting |
Toledo, Spain, 45071 | |
Contact: Luis Escribano, MD, PhD +34925269335 lescribanom@sescam.jccm.es | |
Contact: Iván Alvarez-Twose, MD +34925269336 ivana@sescam.jccm.es | |
Principal Investigator: Luis Escribano, MD, PhD | |
Sub-Investigator: Iván Alvarez-Twose, MD |
Principal Investigator: | Luis Escribano, MD, PhD | Instituto de Estudios de Mastocitosis de Castilla La Mancha |
Responsible Party: | LUIS ESCRIBANO, Director of the Instituto de Estudios de Mastocitosis de Castilla La Mancha, Hospital Virgen de la Salud |
ClinicalTrials.gov Identifier: | NCT01602939 History of Changes |
Other Study ID Numbers: |
EC11-187 |
First Posted: | May 21, 2012 Key Record Dates |
Last Update Posted: | August 29, 2016 |
Last Verified: | August 2016 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Keywords provided by LUIS ESCRIBANO, Hospital Virgen de la Salud:
Mast cell Mastocytosis Mast cell disease |
Additional relevant MeSH terms:
Mastocytosis Mastocytosis, Systemic Neoplasms, Connective Tissue Neoplasms, Connective and Soft Tissue Neoplasms by Histologic Type Neoplasms |
Skin Diseases Cladribine Antineoplastic Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |