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Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

Primary Purpose

Mastocytosis

Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
masitinib (AB1010)
masitinib (AB1010)
Sponsored by
AB Science
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mastocytosis focused on measuring mastocytosis, indolent, handicap, wild-type, systemic, not D816V mutated

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Patients with documented Indolent systemic mastocytosis with handicap (ISMwh) having at least 2 infiltrated* organs (skin and /or bone-marrow and/or internal organ).
  2. Bone-marrow, or skin or internal biopsy-documented mastocytosis and evaluable disease.
  3. The absence of an activating point mutation in the phosphotransferase domain of c-Kit such as D816V c-Kit mutation in at least one of the two infiltrated organs: bone marrow and/or skin and/or other tissue.
  4. Handicap defined as at least one of the following handicaps:

    • a number of flush per day ≥ 1 ,
    • a pruritus score ≥ 9 ,
    • a number of stools per day ≥ 4 ,
    • a Pollakyuria (on a per day basis) ≥ 8 ,
    • a QLQ-C30 score ≥ 83 ,
    • a Hamilton rating scale for depression ≥ 12

Exclusion Criteria:

  1. Performance status > 2 (ECOG).
  2. Inadequate organ function, except if the abnormalities are due to involvement by mast cells

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Experimental

    Experimental

    Arm Label

    2

    1

    Arm Description

    masitinib (AB1010) 6 mg/kg/day

    masitinib (AB1010) 3 mg/kg/day

    Outcomes

    Primary Outcome Measures

    Response on: Pruritus score, Number of flush per day, Pollakyuria (on a daily basis), Number of stools per day, QLQ-C30 score, Hamilton Rating Scale for depression

    Secondary Outcome Measures

    AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010

    Full Information

    First Posted
    January 28, 2009
    Last Updated
    December 8, 2018
    Sponsor
    AB Science
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00831974
    Brief Title
    Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
    Official Title
    Phase IIa, Open-label, Randomized Study of Oral AB1010 in Patients With Systemic Indolent Mastocytosis With Handicap and Not Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    December 2018
    Overall Recruitment Status
    Completed
    Study Start Date
    October 2004 (undefined)
    Primary Completion Date
    February 2007 (Actual)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    AB Science

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    This is a 12 weeks study aimed at assessing the safety and efficacy of 2 doses of AB1010 in patients suffering from indolent systemic mastocytosis with handicap.
    Detailed Description
    Efficacy will be assessed based on: Pruritus score Number of flush per day Pollakyuria (on a daily basis) Number of stools per day QLQ-C30 score Hamilton Rating Scale for depression

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Mastocytosis
    Keywords
    mastocytosis, indolent, handicap, wild-type, systemic, not D816V mutated

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Randomized
    Enrollment
    23 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    2
    Arm Type
    Experimental
    Arm Description
    masitinib (AB1010) 6 mg/kg/day
    Arm Title
    1
    Arm Type
    Experimental
    Arm Description
    masitinib (AB1010) 3 mg/kg/day
    Intervention Type
    Drug
    Intervention Name(s)
    masitinib (AB1010)
    Intervention Description
    3 mg/kg/day
    Intervention Type
    Drug
    Intervention Name(s)
    masitinib (AB1010)
    Intervention Description
    6 mg/kg/day
    Primary Outcome Measure Information:
    Title
    Response on: Pruritus score, Number of flush per day, Pollakyuria (on a daily basis), Number of stools per day, QLQ-C30 score, Hamilton Rating Scale for depression
    Time Frame
    12 weeks
    Secondary Outcome Measure Information:
    Title
    AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010
    Time Frame
    12 weeks

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Patients with documented Indolent systemic mastocytosis with handicap (ISMwh) having at least 2 infiltrated* organs (skin and /or bone-marrow and/or internal organ). Bone-marrow, or skin or internal biopsy-documented mastocytosis and evaluable disease. The absence of an activating point mutation in the phosphotransferase domain of c-Kit such as D816V c-Kit mutation in at least one of the two infiltrated organs: bone marrow and/or skin and/or other tissue. Handicap defined as at least one of the following handicaps: a number of flush per day ≥ 1 , a pruritus score ≥ 9 , a number of stools per day ≥ 4 , a Pollakyuria (on a per day basis) ≥ 8 , a QLQ-C30 score ≥ 83 , a Hamilton rating scale for depression ≥ 12 Exclusion Criteria: Performance status > 2 (ECOG). Inadequate organ function, except if the abnormalities are due to involvement by mast cells
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Olivier Lortholary, MD, PhD
    Organizational Affiliation
    Hôpital Necker, Paris, France
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    21108325
    Citation
    Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmerini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol. 2010 Dec;85(12):921-5. doi: 10.1002/ajh.21894.
    Results Reference
    result

    Learn more about this trial

    Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

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