Masitinib in Patients With Mastocytosis With Handicap and Bearing the D816V Mutation

This study has been completed.
Sponsor:
Information provided by:
AB Science
ClinicalTrials.gov Identifier:
NCT01266369
First received: December 23, 2010
Last updated: NA
Last verified: December 2010
History: No changes posted
  Purpose

The objective is to compare efficacy and safety of AB1010 at 3 or 6 mg/kg/day in treatment of patients with mastocytosis with handicap and bearing activating point mutations in the phosphotransferase domain of c-Kit such as the main mutation Asp-816-Val (D816V).


Condition Intervention Phase
Patients With Mastocytosis With Handicap and Bearing Mutations Asp-816-Val (D816V)
Drug: masitinib
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A 12-week With Possible Extension, Prospective, Multicenter, Randomized, Open-label, 2-parallel Group, Phase IIa Study to Compare Efficacy and Safety of AB1010 at 3 or 6 mg/kg/Day in Treatment of Patients With Mastocytosis With Handicap and Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)

Resource links provided by NLM:


Further study details as provided by AB Science:

Primary Outcome Measures:
  • efficacy on handicaps [ Time Frame: week 12 ] [ Designated as safety issue: No ]
    Pruritus score at week 12 Number of flushes per week at week 12 Hamilton score at week 12 Fatigue Impact scale at week 12


Enrollment: 21
Study Start Date: February 2007
Study Completion Date: May 2010
Primary Completion Date: November 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: masitinib 3 mg/kg/day
masitinib 3 mg/kg/day
Drug: masitinib
Experimental: masitinib 6 mg/kg/day
masitinib 6 mg/kg/day
Drug: masitinib

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with one of the following documented mastocytosis:

    • Smouldering systemic mastocytosis
    • Indolent systemic mastocytosis with organomegaly
    • Indolent Systemic Mastocytosis having 2 infiltrated organs (skin and bone-marrow)
    • Any mastocytosis with in the last 6 months at least 3 anaphylactic shocks or syncops requiring either use of adrenaline or medical assistance
    • Cutaneous Mastocytosis (CM)
  2. Skin biopsy-documented mastocytosis and evaluable disease based upon:

    • Histological criteria: typical infiltrates of mast cells in a multifocal or diffuse pattern in skin biopsy
    • Clinical criteria: typical skin lesions (maculopapular, urticaria pigmentosa, mastocytoma)
  3. Missing data (c-kit molecular analysis not done) or documented presence of an activating point mutation in the phosphotransferase domain of c-kit such as D816V c-kit mutation in at least one infiltrated organ (bone marrow or skin)
  4. Refractory to at least one of the symptomatic treatments such as:

    • Anti H1
    • Anti H2
    • Proton pump inhibitor
    • Osteoclast inhibitor
    • Cromoglycate Sodium
    • Antileukotriene
    • Other therapies used for the symptomatic care
  5. Handicap defined as at least one of the following handicaps:

    • pruritus score ≥ 6
    • number of flushes per week ≥ 7
    • number of stools per day ≥ 4 ,
    • number of mictions per day ≥ 8 ,
    • QLQ-C30 score ≥ 60,
    • Hamilton score ≥ 10

Exclusion Criteria:

  1. Patients with one of the following mastocytosis:

    • Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD)
    • Mast cell leukemia (MCL)
    • Aggressive systemic mastocytosis (ASM)
  2. Patient with a major surgery within 2 weeks prior to study entry
  3. No vulnerable population will be included in this study

    • Life expectancy < 6 months.
    • Patient is < 5 years free of malignancy, except treated basal cell skin cancer or cervical carcinoma in situ.
    • Patient with grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study)
    • Patient has a severe and/or uncontrolled medical disease.
    • Patient has a known diagnosis of human immunodeficiency virus (HIV) infection
  Contacts and Locations
No Contacts or Locations Provided
  More Information

No publications provided

Responsible Party: Alain Moussy, CEO, AB Science
ClinicalTrials.gov Identifier: NCT01266369     History of Changes
Other Study ID Numbers: AB06013
Study First Received: December 23, 2010
Last Updated: December 23, 2010
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by AB Science:
mastocytosis
D816V
handicaps

Additional relevant MeSH terms:
Mastocytosis
Urticaria Pigmentosa
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Skin Diseases
Mastocytosis, Cutaneous
Pigmentation Disorders

ClinicalTrials.gov processed this record on April 23, 2014