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Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

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ClinicalTrials.gov Identifier: NCT00831974
Recruitment Status : Completed
First Posted : January 29, 2009
Last Update Posted : August 14, 2013
Sponsor:
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment Phase
Mastocytosis Drug: masitinib (AB1010) Phase 2

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


Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : October 2004
Primary Completion Date : February 2007

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: 2
masitinib (AB1010) 6 mg/kg/day
Drug: masitinib (AB1010)
6 mg/kg/day
Experimental: 1
masitinib (AB1010) 3 mg/kg/day
Drug: masitinib (AB1010)
3 mg/kg/day


Outcome Measures

Primary Outcome Measures :
  1. 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 Measures :
  1. 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 ]

Eligibility Criteria

Information from the National Library of Medicine

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

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
Contacts and Locations

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 ClinicalTrials.gov identifier (NCT number): NCT00831974


Sponsors and Collaborators
AB Science
Investigators
Principal Investigator: Olivier Lortholary, MD, PhD Hôpital Necker, Paris, France
More Information

Responsible Party: AB Science
ClinicalTrials.gov Identifier: NCT00831974     History of Changes
Other Study ID Numbers: AB04010
First Posted: January 29, 2009    Key Record Dates
Last Update Posted: August 14, 2013
Last Verified: January 2009

Keywords provided by AB Science:
mastocytosis
indolent
handicap
wild-type
systemic
not D816V mutated

Additional relevant MeSH terms:
Mastocytosis
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Skin Diseases