Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

Phase 2

Completed

• Conditions: Mastocytosis
• Interventions: Drug: masitinib (AB1010)

• Registration Number: NCT00831974
• Lead Sponsor: AB Science

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.

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 Timeline

• Study Start: 2004-10
• Primary Completion: 2007-02
• Study First Submitted: 2009-01-28
• Study First Submitted QC: 2009-01-28
• Study First Posted: 2009-01-29
• Status Verified: 2018-12
• Last Update Submitted: 2018-12-08
• Last Update Posted: 2018-12-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 23

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1	masitinib (AB1010)	masitinib (AB1010) 3 mg/kg/day
2	masitinib (AB1010)	masitinib (AB1010) 6 mg/kg/day

Primary Outcome Measures

Name	Time	Method
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	12 weeks

Secondary Outcome Measures

Name	Time	Method
AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010	12 weeks