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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.
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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 2 | Experimental | masitinib (AB1010) 6 mg/kg/day |
|
| 1 | Experimental | masitinib (AB1010) 3 mg/kg/day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| masitinib (AB1010) | Drug | 3 mg/kg/day |
| |
| masitinib (AB1010) |
| Measure | Description | 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 |
| Measure | Description | 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 |
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Inclusion Criteria:
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:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Olivier Lortholary, MD, PhD | Hôpital Necker, Paris, France | Principal Investigator |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21108325 | Result | 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. |
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| ID | Term |
|---|---|
| D008415 | Mastocytosis |
| ID | Term |
|---|---|
| D009372 | Neoplasms, Connective Tissue |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| C526575 | masitinib |
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| Drug |
6 mg/kg/day |
|
| D000090362 | Mast Cell Activation Disorders |
| D007154 | Immune System Diseases |