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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01266369
Recruitment Status : Completed
First Posted : December 24, 2010
Last Update Posted : December 10, 2018
Information provided by (Responsible Party):
AB Science

Brief Summary:
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 or disease Intervention/treatment Phase
Mastocytosis Drug: masitinib Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 21 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (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)
Study Start Date : February 2007
Actual Primary Completion Date : November 2007
Actual Study Completion Date : May 2010

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. efficacy on handicaps [ Time Frame: week 12 ]
    Pruritus score at week 12 Number of flushes per week at week 12 Hamilton score at week 12 Fatigue Impact scale at week 12

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01266369

Sponsors and Collaborators
AB Science
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Principal Investigator: Olivier Lortholary, MD, PhD Necker Hospital, Paris, France

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Responsible Party: AB Science Identifier: NCT01266369     History of Changes
Other Study ID Numbers: AB06013
First Posted: December 24, 2010    Key Record Dates
Last Update Posted: December 10, 2018
Last Verified: December 2018
Keywords provided by AB Science:
Additional relevant MeSH terms:
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Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Skin Diseases
Immune Complex Diseases
Immune System Diseases