Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis - Full Text View

Purpose

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.

Condition	Intervention	Phase
Mastocytosis	Drug: masitinib (AB1010)	Phase 2

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
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)

Resource links provided by NLM:

Genetics Home Reference related topics: Help Me Understand Genetics

U.S. FDA Resources

Further study details as provided by AB Science:

Primary Outcome Measures:

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 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Enrollment:	23
Study Start Date:	October 2004
Primary Completion Date:	February 2007 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: 2 masitinib (AB1010) 6 mg/kg/day	Drug: masitinib (AB1010) 6 mg/kg/day
Experimental: 1 masitinib (AB1010) 3 mg/kg/day	Drug: masitinib (AB1010) 3 mg/kg/day

Detailed 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

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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:
- 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:

Performance status > 2 (ECOG).
Inadequate organ function, except if the abnormalities are due to involvement by mast cells

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00831974

Sponsors and Collaborators

AB Science

Investigators

Principal Investigator:

Olivier Lortholary, MD, PhD

Hôpital Necker, Paris, France

More Information

No publications provided

Responsible Party:	Alain Moussy, AB Science
ClinicalTrials.gov Identifier:	NCT00831974 History of Changes
Other Study ID Numbers:	AB04010
Study First Received:	January 28, 2009
Last Updated:	January 28, 2009
Health Authority:	France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by AB Science:

mastocytosis
indolent
handicap

wild-type
systemic
not D816V mutated

Additional relevant MeSH terms:

Mastocytosis
Urticaria Pigmentosa
Mastocytoma
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type
Neoplasms
Skin Diseases
Mastocytosis, Cutaneous
Pigmentation Disorders

ClinicalTrials.gov processed this record on October 18, 2012