Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

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)

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00831974

Enrollment

Registered

2009-01-29

Start date

2004-10-31

Completion date

Unknown

Last updated

2018-12-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Mastocytosis

Keywords

mastocytosis, indolent, handicap, wild-type, systemic, not D816V mutated

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

Interventions

DRUGmasitinib (AB1010)

3 mg/kg/day

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame
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

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

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026