Know Cancer

or
forgot password

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)


Phase 2
18 Years
N/A
Not Enrolling
Both
Mastocytosis

Thank you

Trial Information

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)


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


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

Type of Study:

Interventional

Study Design:

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

Outcome Measure:

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

Outcome Time Frame:

12 weeks

Safety Issue:

No

Principal Investigator

Olivier Lortholary, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Hôpital Necker, Paris, France

Authority:

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

Study ID:

AB04010

NCT ID:

NCT00831974

Start Date:

October 2004

Completion Date:

Related Keywords:

  • Mastocytosis
  • mastocytosis
  • indolent
  • handicap
  • wild-type
  • systemic
  • not D816V mutated
  • Mastocytosis
  • Urticaria Pigmentosa
  • Mastocytoma

Name

Location