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)
1 other identifier
interventional
23
0 countries
N/A
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 1, 2004
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2007
CompletedFirst Submitted
Initial submission to the registry
January 28, 2009
CompletedFirst Posted
Study publicly available on registry
January 29, 2009
CompletedDecember 11, 2018
December 1, 2018
2.3 years
January 28, 2009
December 8, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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 Outcomes (1)
AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010
12 weeks
Study Arms (2)
2
EXPERIMENTALmasitinib (AB1010) 6 mg/kg/day
1
EXPERIMENTALmasitinib (AB1010) 3 mg/kg/day
Interventions
Eligibility Criteria
You may qualify if:
- Patients with documented Indolent systemic mastocytosis with handicap (ISMwh) having at least 2 infiltrated\* organs (skin and /or bone-marrow and/or internal organ).
- Bone-marrow, or skin or internal biopsy-documented mastocytosis and evaluable disease.
- 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.
- 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
You may not qualify if:
- Performance status \> 2 (ECOG).
- Inadequate organ function, except if the abnormalities are due to involvement by mast cells
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AB Sciencelead
Related Publications (1)
Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmerini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol. 2010 Dec;85(12):921-5. doi: 10.1002/ajh.21894.
PMID: 21108325RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Olivier Lortholary, MD, PhD
Hôpital Necker, Paris, France
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
January 28, 2009
First Posted
January 29, 2009
Study Start
October 1, 2004
Primary Completion
February 1, 2007
Last Updated
December 11, 2018
Record last verified: 2018-12