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

Mastocytosis Clinical Trial

Official title:

A 12-week With Possible Extension, Prospective, Multicenter, Randomized, Open-label, 2-parallel Group, Phase IIa Study 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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01266369
• Other study ID #: AB06013
• Status: Completed
• Phase: Phase 2
• Start date: February 2007
• Est. completion date: May 2010

Study information

• Verified date: December 2018
• Source: AB Science
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: May 2010
• Est. primary completion date: November 2007
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Mastocytosis

Intervention

Drug:
• masitinib

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
AB Science

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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