Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

Mastocytosis Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00831974
• Other study ID #: AB04010
• Status: Completed
• Phase: Phase 2
• Start date: October 2004

Study information

• Verified date: December 2018
• Source: AB Science
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 23
• Est. primary completion date: February 2007
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Mastocytosis

Intervention

Drug:
• masitinib (AB1010)
3 mg/kg/day
• masitinib (AB1010)
6 mg/kg/day

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
AB Science

References & Publications (1)

Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmérini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phas — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFa, eosinophils, histamine levels), pharmacokinetic profile of AB1010		12 weeks

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