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| Sponsor: | AB Science |
|---|---|
| Information provided by: | AB Science |
| ClinicalTrials.gov Identifier: | NCT00814073 |
Purpose
The objective of this study is to compare the safety and efficacy of masitinib (AB1010) to placebo in patients with mastocytosis with handicap.
| Condition | Intervention | Phase |
|---|---|---|
|
Smouldering Systemic Mastocytosis Indolent Systemic Mastocytosis Cutaneous Mastocytosis With Handicap Mastocytosis |
Drug: masitinib (AB1010) Drug: placebo |
Phase 3 |
| Study Type: | 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 |
| Official Title: | 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 |
| Estimated Enrollment: | 200 |
| Study Start Date: | December 2008 |
| Estimated Study Completion Date: | June 2013 |
| Estimated Primary Completion Date: | December 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: 1
masitinib (AB1010) 6 mg/kg/day per os
|
Drug: masitinib (AB1010)
masitinib (AB1010) 6 mg/kg/day per os
Other Name: AB1010
|
|
Placebo Comparator: 2
placebo
|
Drug: placebo
matching placebo
|
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.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Olivier Lortholary, M.D., Ph.D. | +33 (0)1 44 38 15 70 | olivier.lortholary@nck.aphp.fr |
| United States, California | |
| UC Davis Health System , Department of Dermatology | Recruiting |
| Sacramento, California, United States, 95816 | |
| Contact: LIU Fu-Tong, MD | |
| United States, Texas | |
| MD Anderson Cancer Centre | Recruiting |
| Houston, Texas, United States, 77030 | |
| France | |
| CHU d'Amiens | Recruiting |
| Amiens, France | |
| Hôpital Avicenne | Recruiting |
| Bobigny, France | |
| CHU de Brest | Recruiting |
| Brest, France | |
| CHU de Caen | Recruiting |
| Caen, France | |
| CHU Clermont Ferrand | Not yet recruiting |
| Clermont Ferrand, France, 63000 | |
| Hôpital Claude Huriez | Recruiting |
| Lille, France | |
| CHU Dupuytren | Recruiting |
| Limoges, France | |
| Hôpital Nord | Recruiting |
| Marseille, France | |
| Hôpital Ambroise Paré | Recruiting |
| Marseille, France | |
| Hôpital Central | Recruiting |
| Nancy, France | |
| CHU Hôtel Dieu | Recruiting |
| Nantes, France | |
| Hôpital l'Archet II | Recruiting |
| Nice, France | |
| Hôpital Tenon | Recruiting |
| Paris, France | |
| Hôpital Necker | Recruiting |
| Paris, France | |
| CHU Lyon Sud | Not yet recruiting |
| Pierre Bénite, France, 69495 | |
| Centre Hospitalier Lyon Sud | Recruiting |
| Pierre-Bénite, France | |
| CHU Milétrie | Recruiting |
| Poitiers, France | |
| CHU Hôpital Sud | Recruiting |
| Rennes, France | |
| CHU de Saint-Etienne | Recruiting |
| Saint-Etienne, France | |
| Hôpital Purpan | Recruiting |
| Toulouse, France | |
| Hôpital Bretonneau | Recruiting |
| Tours, France | |
| Hôpital des Hauts Clos | Recruiting |
| Troyes, France | |
| Principal Investigator: | Olivier Lortholary, MD, PhD | Hôpital Necker, Paris, France |
More Information
| Responsible Party: | Alain Moussy, CEO, AB Science |
| ClinicalTrials.gov Identifier: | NCT00814073 History of Changes |
| Other Study ID Numbers: | AB06006 |
| Study First Received: | December 22, 2008 |
| Last Updated: | January 27, 2012 |
| Health Authority: | United States: Food and Drug Administration France: Afssaps - French Health Products Safety Agency |
|
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 Neoplasms, Connective Tissue |
Neoplasms, Connective and Soft Tissue Neoplasms by Histologic Type Neoplasms Skin Diseases Pigmentation Disorders |