Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

NCT ID: NCT00831974

Last Updated: 2018-12-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-10-31

Brief Summary

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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.

Detailed Description

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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

Conditions

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Mastocytosis

Keywords

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mastocytosis indolent handicap wild-type systemic not D816V mutated

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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2

masitinib (AB1010) 6 mg/kg/day

Group Type EXPERIMENTAL

masitinib (AB1010)

Intervention Type DRUG

6 mg/kg/day

1

masitinib (AB1010) 3 mg/kg/day

Group Type EXPERIMENTAL

masitinib (AB1010)

Intervention Type DRUG

3 mg/kg/day

Interventions

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masitinib (AB1010)

3 mg/kg/day

Intervention Type DRUG

masitinib (AB1010)

6 mg/kg/day

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AB Science

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Olivier Lortholary, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Hôpital Necker, Paris, France

References

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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.

Reference Type RESULT
PMID: 21108325 (View on PubMed)

Other Identifiers

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AB04010

Identifier Type: -

Identifier Source: org_study_id