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A 12-week With Possible Extension, Prospective, Multicenter, Randomized, Open-label, 2-parallel Group, Phase IIa Study to Compare Efficacy and Safety of AB1010 at 3 or 6 mg/kg/Day in Treatment of Patients With Mastocytosis With Handicap and Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)


Phase 2
18 Years
N/A
Not Enrolling
Both
Patients With Mastocytosis With Handicap and Bearing Mutations Asp-816-Val (D816V)

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Trial Information

A 12-week With Possible Extension, Prospective, Multicenter, Randomized, Open-label, 2-parallel Group, Phase IIa Study to Compare Efficacy and Safety of AB1010 at 3 or 6 mg/kg/Day in Treatment of Patients With Mastocytosis With Handicap and Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)


Inclusion Criteria:



1. Patients with one of the following documented mastocytosis:

- Smouldering systemic mastocytosis

- Indolent systemic mastocytosis with organomegaly

- Indolent Systemic Mastocytosis having 2 infiltrated organs (skin and
bone-marrow)

- Any mastocytosis with in the last 6 months at least 3 anaphylactic shocks or
syncops requiring either use of adrenaline or medical assistance

- Cutaneous Mastocytosis (CM)

2. Skin biopsy-documented mastocytosis and evaluable disease based upon:

- Histological criteria: typical infiltrates of mast cells in a multifocal or
diffuse pattern in skin biopsy

- Clinical criteria: typical skin lesions (maculopapular, urticaria pigmentosa,
mastocytoma)

3. Missing data (c-kit molecular analysis not done) or documented presence of an
activating point mutation in the phosphotransferase domain of c-kit such as D816V
c-kit mutation in at least one infiltrated organ (bone marrow or skin)

4. Refractory to at least one of the symptomatic treatments such as:

- Anti H1

- Anti H2

- Proton pump inhibitor

- Osteoclast inhibitor

- Cromoglycate Sodium

- Antileukotriene

- Other therapies used for the symptomatic care

5. Handicap defined as at least one of the following handicaps:

- pruritus score ≥ 6

- number of flushes per week ≥ 7

- number of stools per day ≥ 4 ,

- number of mictions per day ≥ 8 ,

- QLQ-C30 score ≥ 60,

- Hamilton score ≥ 10

Exclusion Criteria:

1. Patients 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. Patient with a major surgery within 2 weeks prior to study entry

3. No vulnerable population will be included in this study

- Life expectancy < 6 months.

- Patient is < 5 years free of malignancy, except treated basal cell skin cancer
or cervical carcinoma in situ.

- Patient with grade III/IV cardiac problems as defined by the New York Heart
Association Criteria. (i.e., congestive heart failure, myocardial infarction
within 6 months of study)

- Patient has a severe and/or uncontrolled medical disease.

- Patient has a known diagnosis of human immunodeficiency virus (HIV) infection

Type of Study:

Interventional

Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

efficacy on handicaps

Outcome Description:

Pruritus score at week 12 Number of flushes per week at week 12 Hamilton score at week 12 Fatigue Impact scale at week 12

Outcome Time Frame:

week 12

Safety Issue:

No

Authority:

France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Study ID:

AB06013

NCT ID:

NCT01266369

Start Date:

February 2007

Completion Date:

May 2010

Related Keywords:

  • Patients With Mastocytosis With Handicap and Bearing Mutations Asp-816-Val (D816V)
  • mastocytosis
  • D816V
  • handicaps
  • Mastocytosis
  • Urticaria Pigmentosa
  • Mastocytoma

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