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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
Sponsor:
ClinicalTrials.gov Identifier:
NCT01602939
First Posted: May 21, 2012
Last Update Posted: August 29, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
LUIS ESCRIBANO, Hospital Virgen de la Salud
  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.

Condition Intervention Phase
Systemic Mastocytosis Drug: Cladribine and pegylated interpheron alpha-2a Phase 2 Phase 3

Study Type: Interventional
Study Design: 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.

Resource links provided by NLM:


Further study details as provided by LUIS ESCRIBANO, Hospital Virgen de la Salud:

Primary Outcome Measures:
  • 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.


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: 10
Study Start Date: May 2012
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: February 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.


  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Information from the National Library of Medicine

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


Contacts
Contact: Luis Escribano, MD, PhD +34925269335 lescribanom@sescam.jccm.es
Contact: Iván Alvarez-Twose, MD +34925269336 ivana@sescam.jccm.es

Locations
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         
Sponsors and Collaborators
Hospital Virgen de la Salud
Investigators
Principal Investigator: Luis Escribano, MD, PhD Instituto de Estudios de Mastocitosis de Castilla La Mancha
  More Information

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 Submitted: May 16, 2012
First Posted: May 21, 2012
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


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