NCT01602939

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started May 2012

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2012

Completed
15 days until next milestone

First Submitted

Initial submission to the registry

May 16, 2012

Completed
5 days until next milestone

First Posted

Study publicly available on registry

May 21, 2012

Completed
4.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2017

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2017

Completed
Last Updated

August 29, 2016

Status Verified

August 1, 2016

Enrollment Period

4.8 years

First QC Date

May 16, 2012

Last Update Submit

August 26, 2016

Conditions

Systemic Mastocytosis

Keywords

Mast cellMastocytosisMast cell disease

Outcome Measures

Primary Outcomes (1)

  • 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.

    6 months

Secondary Outcomes (6)

  • To determine the effect of therapy on serum tryptase levels and other altered peripheral blood parameters due to mastocytosis.

    6 months

  • To evaluate the effect of therapy on mast cell-mediator release symptoms: pruritus, flushing, gastrointestinal symptoms or anaphylaxis).

    6 months

  • To determine de safety of combined therapy with low doses of cladribine plus pegylated interpheron alpha-2a.

    6 months

  • To evaluate the effect of therapy on mastocytosis skin lesions.

    6 moths

  • To evaluate the effect of therapy on mastocytosis-related organomegalies.

    6 months

  • +1 more secondary outcomes

Study Arms (1)

2CDA+IFN

EXPERIMENTAL
Drug: Cladribine and pegylated interpheron alpha-2a

Interventions

Cladribine and pegylated interpheron alpha-2aDRUG

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.

2CDA+IFN

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Instituto de Estudios de Mastocitosis de Castilla La Mancha; Hospital Virgen del Valle

Toledo, Toledo, 45071, Spain

RECRUITING

MeSH Terms

Conditions

Mastocytosis, SystemicMastocytosisMast Cell Activation Disorders

Interventions

Cladribine

Condition Hierarchy (Ancestors)

Neoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasmsImmune System Diseases

Intervention Hierarchy (Ancestors)

2-ChloroadenosineAdenosinePurine NucleosidesPurinesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic CompoundsDeoxyadenosinesDeoxyribonucleosidesNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Officials

  • Luis Escribano, MD, PhD

    Instituto de Estudios de Mastocitosis de Castilla La Mancha

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Luis Escribano, MD, PhD

CONTACT

+34925269335lescribanom@sescam.jccm.es

Iván Alvarez-Twose, MD

CONTACT

+34925269336ivana@sescam.jccm.es

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director of the Instituto de Estudios de Mastocitosis de Castilla La Mancha

Study Record Dates

First Submitted

May 16, 2012

First Posted

May 21, 2012

Study Start

May 1, 2012

Primary Completion

February 1, 2017

Study Completion

June 1, 2017

Last Updated

August 29, 2016

Record last verified: 2016-08

Locations

ES(1)