Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis

This study is currently recruiting participants.

Verified May 2012 by Hospital Virgen de la Salud

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

History of Changes

Tracking Information

First Received Date ^ICMJE

May 16, 2012

Last Updated Date

May 19, 2012

Start Date ^ICMJE

May 2012

Estimated Primary Completion Date

March 2013 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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.

Original Primary Outcome Measures ^ICMJE

To evaluate the effect of therapy on the grade of bone marrow and cutaneous mast cell infiltration, as well as on organomegalies or bone alterations. [ 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. Evaluation of cutaneous response will be assessed by macroscopic inspection including photographs and by skin immunohistochemestry. Evaluation of organomegalies response will be assessed by abdominal ultrasound and/or computerized tomography. Evaluation of bone response will be assessed by dual X-ray absorptiometry and/or X-ray survey and/or computerized tomography.

Change History

Complete list of historical versions of study NCT01602939 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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.

Original Secondary Outcome Measures ^ICMJE

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).

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis

Official Title ^ICMJE

Subcutaneous Cladribine Plus Pegylated Interpheron Alfa-2a in Advanced Systemic Mastocytosis With D816V and Other Exon 17 KIT Mutations.

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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2
Phase 3

Study Design ^ICMJE

Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Advanced Systemic Mastocytosis

Intervention ^ICMJE

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.

Study Arm (s)

Not Provided

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

May 2013

Estimated Primary Completion Date

March 2013 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.

Gender

Both

Ages

18 Years and older

Accepts Healthy Volunteers

Contacts ^ICMJE

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

Location Countries ^ICMJE

Spain

Administrative Information

NCT Number ^ICMJE

NCT01602939

Other Study ID Numbers ^ICMJE

EC11-187

Has Data Monitoring Committee

Responsible Party

LUIS ESCRIBANO, Hospital Virgen de la Salud

Study Sponsor ^ICMJE

Hospital Virgen de la Salud

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

Luis Escribano, MD, PhD

Instituto de Estudios de Mastocitosis de Castilla La Mancha

Information Provided By

Hospital Virgen de la Salud

Verification Date

May 2012

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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