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
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.

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 October 01, 2014