Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
AB Science
ClinicalTrials.gov Identifier:
NCT00831974
First received: January 28, 2009
Last updated: August 13, 2013
Last verified: January 2009
  Purpose

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 Intervention Phase
Mastocytosis
Drug: masitinib (AB1010)
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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)

Resource links provided by NLM:


Further study details as provided by AB Science:

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 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 23
Study Start Date: October 2004
Primary Completion Date: February 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00831974

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

No publications provided

Responsible Party: AB Science
ClinicalTrials.gov Identifier: NCT00831974     History of Changes
Other Study ID Numbers: AB04010
Study First Received: January 28, 2009
Last Updated: August 13, 2013
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

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

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 22, 2014