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A 24-week With Possible Extension, Prospective, Multicentre, Randomized, Double Blind, Placebo-controlled, 2-parallel Group With a Randomization 1:1, Phase III Study to Compare Efficacy and Safety of Masitinib at 6 mg/kg/Day to Placebo in Treatment of Patients With Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis With Handicap


Phase 3
18 Years
N/A
Open (Enrolling)
Both
Smouldering Systemic Mastocytosis, Indolent Systemic Mastocytosis, Cutaneous Mastocytosis With Handicap, Mastocytosis

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Trial Information

A 24-week With Possible Extension, Prospective, Multicentre, Randomized, Double Blind, Placebo-controlled, 2-parallel Group With a Randomization 1:1, Phase III Study to Compare Efficacy and Safety of Masitinib at 6 mg/kg/Day to Placebo in Treatment of Patients With Smouldering Systemic, Indolent Systemic or Cutaneous Mastocytosis With Handicap


Mastocytosis is a disease characterized by mast cell invasion in various organs. Mast cells
are bone marrow derived cells which produce histamine and other substances causing allergic
and anaphylactic reactions. Accumulation of mast cells in body organs can inhibit the
functionality of the organ and eventually cause degeneration.

Mutations of the mast cell growth factor receptor (KIT protein, the product of the c-Kit
proto-oncogene) might be found in patients with mastocytosis.

Masitinib (AB1010) is a tyrosine kinase inhibitor (TKI), selectively and effectively
inhibiting c-kit. Its effect may include inhibition of cell proliferation, inhibition of
cell cycle progression and induction of apoptosis resulting in the reduction of mast cell
accumulation in body tissues. This drug is thereby specific to mastocytosis and active in
slowing or reducing the number of mast cells particularly in aggressive forms of the
disease.


Inclusion Criteria:



1. Patient with one of the following documented mastocytosis as per WHO classification:
Smouldering Systemic Mastocytosis, Indolent Systemic Mastocytosis, Cutaneous
Mastocytosis

2. Patient with documented mastocytosis and evaluable disease based upon histological
criteria: typical infiltrates of mast cells in a multifocal or diffuse pattern in
skin and/or bone marrow biopsy

3. Patient with documented treatment failure of his/her handicap(s) with at least one of
the following therapy used at optimized dose: Anti H1, Anti H2, Proton pump
inhibitor, Osteoclast inhibitor, Cromoglycate Sodium, Antileukotriene

4. Handicapped status defined as at least two of the following handicaps, including at
least one among pruritus, flushes, depression and asthenia: pruritus score ≥ 6,
number of flushes per week ≥ 7, Hamilton rating scale (depression) ≥ 10, number of
stools per day ≥ 4, number of mictions per day ≥ 8, Fatigue Impact Scale total score
(asthenia) ≥ 40

5. Patients with OPA > 2 (moderate to intolerable general handicap)

6. Male or female patient with age ≥ 18 years

Exclusion Criteria:

1. Patient with one of the following mastocytosis: Systemic Mastocytosis with an
Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD), Mast cell
leukemia (MCL), Aggressive systemic mastocytosis (ASM)

2. Previous treatment with any Tyrosine Kinase Inhibitor

3. Patient presenting with at least one of the following feature: ischemic heart
disease, cardiac failure, conduction disorders or arrythmia

4. Patient with any condition that the physician judges could be detrimental to subjects
participating in this study; including any clinically important deviations from
normal clinical laboratory values or concurrent medical events Previous treatment

5. Change in the symptomatic treatment of mastocytosis or administration of any new
treatment of mastocytosis within 4 weeks prior to baseline

6. Treatment with any investigational agent within 4 weeks prior to baseline

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Outcome Measure:

Responder rate at week 24

Outcome Time Frame:

24 weeks

Safety Issue:

No

Principal Investigator

Olivier Lortholary, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Hôpital Necker, Paris, France

Authority:

United States: Food and Drug Administration

Study ID:

AB06006

NCT ID:

NCT00814073

Start Date:

December 2008

Completion Date:

June 2013

Related Keywords:

  • Smouldering Systemic Mastocytosis
  • Indolent Systemic Mastocytosis
  • Cutaneous Mastocytosis With Handicap
  • Mastocytosis
  • Mastocytosis with handicap
  • Mastocytosis
  • Smouldering systemic mastocytosis
  • Indolent systemic mastocytosis
  • Cutaneous mastocytosis
  • Mast cell
  • Mast cell infiltration
  • Skin
  • Bone marrow
  • Pruritus
  • Flushes
  • c-kit
  • c-kit mutation
  • Wild Type
  • Mutation Asp-816-Val(D816V)
  • Mastocytosis
  • Urticaria Pigmentosa
  • Mastocytoma
  • Mastocytosis, Systemic
  • Mastocytosis, Cutaneous

Name

Location

MD Anderson Cancer CentreHouston, Texas  77030
UC Davis Health System , Department of DermatologySacramento, California  95816