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

NCT01266369 | Completed Phase 2

• 1 other identifier: AB06013
• Study Type: interventional
• Target: 21
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (1)

• efficacy on handicaps

  Pruritus score at week 12 Number of flushes per week at week 12 Hamilton score at week 12 Fatigue Impact scale at week 12

  week 12

Study Arms (2)

masitinib 3 mg/kg/day

EXPERIMENTAL

masitinib 3 mg/kg/day

Drug: masitinib

masitinib 6 mg/kg/day

EXPERIMENTAL

masitinib 6 mg/kg/day

Drug: masitinib

Interventions

masitinib DRUG

masitinib 3 mg/kg/day; masitinib 6 mg/kg/day

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)
• 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)
• 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)
• Refractory to at least one of the symptomatic treatments such as:
• Anti H1
• Anti H2
• Proton pump inhibitor
• Osteoclast inhibitor

You may not qualify if:

• 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)
• Patient with a major surgery within 2 weeks prior to study entry
• 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

Sponsors & Collaborators

• AB Science: lead

MeSH Terms

Conditions

Mastocytosis

Interventions

masitinib

Condition Hierarchy (Ancestors)

Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Mast Cell Activation Disorders; Immune System Diseases

Study Officials

• Olivier Lortholary, MD, PhD

  Necker Hospital, Paris, France

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 23, 2010
• First Posted: December 24, 2010
• Study Start: February 1, 2007
• Primary Completion: November 1, 2007
• Study Completion: May 1, 2010
• Last Updated: December 10, 2018

Record last verified: 2018-12