Clinical trial · Interventional

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)

NCT00831974CI-TRIAL-00036080completedPhase 2ClinicalTrials.gov clinicaltrialsPublishedExplore in graph →

Summary

Brief summary (as posted)

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.

Conditions

Conditions (1)

Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.

Condition (as posted)	Mapped entity	Match	Confidence
Mastocytosis	Mastocytosis	ONTOLOGY_EXACT	0.98

Interventions

Interventions (1)

Intervention	Type	Mapped drug	Match
masitinib (AB1010)	Drug	Masitinib	ALIAS

Design

Arms and outcomes

Arms (2)

type
EXPERIMENTAL
label
2
description
masitinib (AB1010) 6 mg/kg/day
interventionNames
1. Drug: masitinib (AB1010)
type
EXPERIMENTAL
label
1
description
masitinib (AB1010) 3 mg/kg/day
interventionNames
1. Drug: masitinib (AB1010)

Primary outcomes (1)

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
timeFrame
12 weeks

Eligibility

Eligibility (as posted)

Sex: All
Minimum age: 18 Years

Show eligibility criteria text

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

References

Publications (1)

RESULTPaul 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 21108325