Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
Primary Purpose
Systemic Mastocytosis
Status
Unknown status
Phase
Phase 2
Locations
Spain
Study Type
Interventional
Intervention
Cladribine and pegylated interpheron alpha-2a
Sponsored by
About this trial
This is an interventional treatment trial for Systemic Mastocytosis focused on measuring Mast cell, Mastocytosis, Mast cell disease
Eligibility 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.
Sites / Locations
- Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del ValleRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
2CDA+IFN
Arm Description
Outcomes
Primary Outcome Measures
To evaluate the effect of therapy on bone marrow mast cell infiltration.
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.
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).
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.
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.
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.
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.
Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography.
Full Information
NCT ID
NCT01602939
First Posted
May 16, 2012
Last Updated
August 26, 2016
Sponsor
Hospital Virgen de la Salud
1. Study Identification
Unique Protocol Identification Number
NCT01602939
Brief Title
Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
Official Title
Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.
Study Type
Interventional
2. Study Status
Record Verification Date
August 2016
Overall Recruitment Status
Unknown status
Study Start Date
May 2012 (undefined)
Primary Completion Date
February 2017 (Anticipated)
Study Completion Date
June 2017 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Hospital Virgen de la Salud
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Systemic Mastocytosis
Keywords
Mast cell, Mastocytosis, Mast cell disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
2CDA+IFN
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Cladribine and pegylated interpheron alpha-2a
Intervention Description
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.
Primary Outcome Measure Information:
Title
To evaluate the effect of therapy on bone marrow mast cell infiltration.
Description
Evaluation of bone marrow response will be assessed by immunohistochemestry, citology, flow cytometry and molecular analyses of bone marrow samples.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis.
Description
Serum tryptase and any other mastocytosis-related altered biochemical parameter at diagnosis will be measured monthly until the end of therapy.
Time Frame
6 months
Title
To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis).
Description
Specific questionnaires regarding mast cell-mediator release symptoms will be filled monthly by each patient until the end of therapy.
Time Frame
6 months
Title
To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a.
Description
Potentially drugs-related adverse events will be recorded in each case following accepted criteria (NIH CTCAE).
Time Frame
6 months
Title
To evaluate the effect of therapy on mastocytosis skin lesions.
Description
Evaluation of cutaneous response will be assessed by macroscopic inspection including photographs and by skin immunohistochemestry.
Time Frame
6 moths
Title
To evaluate the effect of therapy on mastocytosis-related organomegalies.
Description
Evaluation of organomegalies response will be assessed by abdominal ultrasound and/or computerized tomography.
Time Frame
6 months
Title
To evaluate the effect of therapy on mastocytosis-related bone alterations.
Description
Evaluation of bone response will be assessed by X-ray survey and/or computerized tomography.
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Luis Escribano, MD, PhD
Phone
+34925269335
Email
lescribanom@sescam.jccm.es
First Name & Middle Initial & Last Name or Official Title & Degree
Iván Alvarez-Twose, MD
Phone
+34925269336
Email
ivana@sescam.jccm.es
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Luis Escribano, MD, PhD
Organizational Affiliation
Instituto de Estudios de Mastocitosis de Castilla La Mancha
Official's Role
Principal Investigator
Facility Information:
Facility Name
Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle
City
Toledo
ZIP/Postal Code
45071
Country
Spain
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Luis Escribano, MD, PhD
Phone
+34925269335
Email
lescribanom@sescam.jccm.es
First Name & Middle Initial & Last Name & Degree
Iván Alvarez-Twose, MD
Phone
+34925269336
Email
ivana@sescam.jccm.es
First Name & Middle Initial & Last Name & Degree
Luis Escribano, MD, PhD
First Name & Middle Initial & Last Name & Degree
Iván Alvarez-Twose, MD
12. IPD Sharing Statement
Plan to Share IPD
Undecided
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Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis
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