Trial Information
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)
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
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
Type of Study:
Interventional
Study Design:
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
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
Outcome Time Frame:
12 weeks
Safety Issue:
No
Principal Investigator
Olivier Lortholary, MD, PhD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Hôpital Necker, Paris, France
Authority:
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Study ID:
AB04010
NCT ID:
NCT00831974
Start Date:
October 2004
Completion Date:
Related Keywords:
- Mastocytosis
- mastocytosis
- indolent
- handicap
- wild-type
- systemic
- not D816V mutated
- Mastocytosis
- Urticaria Pigmentosa
- Mastocytoma