Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis

The recruitment status of this study is unknown because the information has not been verified recently.
Verified May 2012 by Hospital Virgen de la Salud.
Recruitment status was  Recruiting
Sponsor:
Information provided by (Responsible Party):
LUIS ESCRIBANO, Hospital Virgen de la Salud
ClinicalTrials.gov Identifier:
NCT01602939
First received: May 16, 2012
Last updated: May 19, 2012
Last verified: May 2012
  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
Advanced Systemic Mastocytosis
Drug: Cladribine and pegylated interpheron alpha-2a
Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 Hospital Virgen de la Salud:

Primary Outcome Measures:
  • To evaluate the effect of therapy on bone marrow mast cell infiltration. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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: May 2013
Estimated Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Intervention Details:
    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

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
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

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

No publications provided

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
Study First Received: May 16, 2012
Last Updated: May 19, 2012
Health Authority: Spain: Ministry of Health

Keywords provided by Hospital Virgen de la Salud:
Mast cell
Mastocytosis
Mast cell disease

Additional relevant MeSH terms:
Pigmentation Disorders
Mastocytosis
Mastocytosis, Systemic
Urticaria Pigmentosa
Mastocytosis, Cutaneous
Neoplasms
Neoplasms by Histologic Type
Neoplasms, Connective and Soft Tissue
Neoplasms, Connective Tissue
Skin Diseases
Cladribine
Antineoplastic Agents
Immunologic Factors
Immunosuppressive Agents
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on October 29, 2014