Cladribine Plus Pegylated Interpheron Alfa-2a in Systemic Mastocytosis

This study is currently recruiting participants. (see Contacts and Locations)

Verified August 2016 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: August 26, 2016

Last verified: August 2016

History of Changes

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:	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:

Drug Information available for: Cladribine

Genetic and Rare Diseases Information Center resources: Mastocytosis Systemic Mastocytosis

U.S. FDA Resources

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:	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


Ages Eligible for Study:	18 Years and older (Adult, Senior)
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


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:	August 26, 2016
Health Authority:	Spain: Ministry of Health
Individual Participant Data
Plan to Share IPD:	Undecided

Keywords provided by 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

ClinicalTrials.gov processed this record on September 23, 2016

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