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

NCT ID: NCT01266369

Last Updated: 2018-12-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-02-28
• Study Completion Date: 2010-05-31

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).

Conditions

Mastocytosis

Keywords

mastocytosis; D816V; handicaps

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

masitinib 3 mg/kg/day

masitinib 3 mg/kg/day

Group Type: EXPERIMENTAL

masitinib

Intervention Type: DRUG

masitinib 6 mg/kg/day

masitinib 6 mg/kg/day

Group Type: EXPERIMENTAL

masitinib

Intervention Type: DRUG

Interventions

masitinib

Intervention Type: DRUG

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

AB Science

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Olivier Lortholary, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Necker Hospital, Paris, France

Other Identifiers

AB06013

Identifier Type: -

Identifier Source: org_study_id