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Indolent Systemic MastocytosisPhase 3AB06006NCT00814073

Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis

A Phase 3 study testing Masitinib in people with Indolent Systemic Mastocytosis, run by AB Science at 24 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The objective of this study is to compare the safety and efficacy of masitinib (AB1010) to placebo in patients with mastocytosis with handicap. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Indolent Systemic Mastocytosis
What is being tested?
Masitinib
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
135 participants
Where
24 sitesin 2 countries
Study dates
Dec 2008to Nov 2015
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patient with one of the following documented mastocytosis as per WHO classification: Smouldering Systemic Mastocytosis, Severe Indolent Mastocytosis
  • 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
  • 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
  • Handicapped status defined as at least two of the following handicaps, including at least one among pruritus, flushes, depression and fatigue: pruritus score ≥ 9, number of flushes per week ≥ 8, Hamilton rating scale for depression (HAMD-17) score ≥ 19, number of stools per day ≥ 4, number of mictions per day ≥ 8, Fatigue Impact Scale total score (asthenia) ≥ 75
  • Patients with OPA ≥ 2 (moderate to intolerable general handicap)
  • ECOG ≤ 2
  • Patient with adequate organ function

Who cannot join

  • Patient with one of the following mastocytosis: Cutaneous Mastocytosis, Not documented Smouldering Systemic Mastocytosis or Indolent Systemic Mastocytosis, Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD), Mast cell leukemia (MCL), Aggressive systemic mastocytosis (ASM)
  • Previous treatment with any Tyrosine Kinase Inhibitor
  • Patient with recent cardiac history of: Acute coronary syndrome, Acute heart failure, Significant ventricular arrhythmia; patient with cardiac failure class III or IV; Syncope without known aetiology within 3 months, uncontrolled severe hypertension.
  • 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
  • Change in the symptomatic treatment of mastocytosis or administration of any new treatment of mastocytosis within 4 weeks prior to baseline
  • Treatment with any investigational agent within 4 weeks prior to baseline

Trial locations and contacts

24 sites in 2 countries.

24 sites in 2 countries

France22 sites

  • CHU d'Amiens

    Amiens

  • Hôpital Avicenne

    Bobigny

  • CHU de Brest

    Brest

  • CHU de Caen

    Caen

  • CHU Clermont Ferrand

    Clermont-Ferrand, 63000

  • Hôpital Claude Huriez

    Lille

  • CHU Dupuytren

    Limoges

  • Hôpital Ambroise Paré

    Marseille

  • Hôpital Nord

    Marseille

  • Hôpital Central

    Nancy

  • CHU Hôtel Dieu

    Nantes

  • Hôpital l'Archet II

    Nice

  • Hôpital Necker

    Paris

  • Hôpital Tenon

    Paris

  • CHU Lyon Sud

    Pierre-Bénite, 69495

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite

  • CHU Milétrie

    Poitiers

  • CHU Hôpital Sud

    Rennes

  • CHU de Saint-Etienne

    Saint-Etienne

  • Hôpital Purpan

    Toulouse

  • Hôpital Bretonneau

    Tours

  • Hôpital des Hauts Clos

    Troyes

Other trials in France

United States2 sites

  • MD Anderson Cancer Centre

    Houston, Texas, 77030

  • UC Davis Health System , Department of Dermatology

    Sacramento, California, 95816

Other trials in United States

About this study

Official title: Randomized, Placebo-controlled, Phase 3 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

The objective of this study is to compare the safety and efficacy of masitinib (AB1010) to placebo in patients with mastocytosis with handicap.

Read the detailed description

This was a prospective, multicenter, randomized, placebo-controlled, parallel-group, phase 3 study, conducted in 15 countries, evaluating the efficacy and safety of masitinib (6 mg/kg/day administered orally in two daily intakes over 24-weeks with a double-blind extension period possible) for the treatment of indolent systemic mastocytosis, smoldering mastocytosis or cutaneous mastocytosis, in patients with mast cells mediator release symptoms that are refractory to conventional symptomatic treatment.

A study protocol amendment restricted enrolment to patients with severe indolent and smoldering systemic mastocytosis. The objective of this phase 3 study was therefore to evaluate masitinib efficacy and safety in severe systemic mastocytosis patients, with or without D816V mutation of c-Kit. The primary objective of the phase 3 study was to detect a statistically significant difference between masitinib (plus optimal concomitant symptomatic treatments) and placebo (plus optimal concomitant symptomatic treatments) in cumulative response on four severe symptoms, referred to also as handicaps.

Being tested
Masitinib, Placebo, Best Supportive Care
Keywords
Mastocytosis with handicap, Mastocytosis, Mast cell, Mast cell infiltration, Skin, Bone marrow, Pruritus, Flushes, c-kit, c-kit mutation, Wild Type, Mutation Asp-816-Val(D816V), Indolent systemic mastocytosis, smoldering systemic mastocytosis
Medical subject headings
Mastocytosis, Systemic, Mastocytosis, Pruritus, Blushing, Piebaldism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MasitinibDrug

    Also called AB1010

    Masitinib 6 mg/kg/day

  • PlaceboDrug

    Matching placebo

  • Best Supportive CareOther

    Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, proton pump inhibitors (PPI), sodium cromoglicate, antidepressants, leukotriene antagonists, interferon-alpha, 2-CdA, and corticosteroids.

Study groups in NCT00814073
Study groupRoleWhat they receive
Masitinib & BSCExperimentalMasitinib (6 mg/kg/day) administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC)
Receives: Drug: Masitinib; Other: Best Supportive Care
Placebo & BSCPlacebo comparatorMatching placebo administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC)
Receives: Drug: Placebo; Other: Best Supportive Care

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Cumulative response (4R75%)

    The prospectively declared primary endpoint (4R75%) was cumulative response in at least one of four severe baseline symptoms of mast cell mediator release (pruritus, flushes, depression, or asthenia). Response was defined as a 75% improvement from baseline for any of these four symptoms. Cumulative response was defined as the number of actual responses between weeks 8 and 24, divided by the total number of possible responses over the same treatment period (ie, with five scheduled visits, each patient had a maximum of five to 20 possible responses depending on the number of severe baseline symptoms).

    Measured: 24 weeks

Secondary outcomes (2)

  1. Cumulative response (3R75%)

    Cumulative response in at least one of three severe baseline symptoms (pruritus, flushes, or depression)

    Measured: 24 weeks

  2. Cumulative response (2R75%)

    Cumulative response in at least one of three severe baseline symptoms (pruritus or flushes)

    Measured: 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Dec 2008 (estimated)
  2. Study startDec 2008
  3. Primary completionNov 2015
  4. Study completionNov 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
135 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00814073
Sponsor's protocol ID
AB06006
Acronym
AB06006
Status last verified
Nov 2019
First submitted
22 Dec 2008
Last updated
3 Dec 2019
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Arock M. A new therapeutic advance for symptomatic systemic mastocytosis? Lancet. 2017 Feb 11;389(10069):576-578. doi: 10.1016/S0140-6736(16)31655-5. Epub 2017 Jan 7. No abstract available.

    PubMed 28069280

  • Lortholary O, Chandesris MO, Bulai Livideanu C, Paul C, Guillet G, Jassem E, Niedoszytko M, Barete S, Verstovsek S, Grattan C, Damaj G, Canioni D, Fraitag S, Lhermitte L, Georgin Lavialle S, Frenzel L, Afrin LB, Hanssens K, Agopian J, Gaillard R, Kinet JP, Auclair C, Mansfield C, Moussy A, Dubreuil P, Hermine O. Masitinib for treatment of severely symptomatic indolent systemic mastocytosis: a randomised, placebo-controlled, phase 3 study. Lancet. 2017 Feb 11;389(10069):612-620. doi: 10.1016/S0140-6736(16)31403-9. Epub 2017 Jan 7.

    PubMed 28069279

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Masitinib in people with Indolent Systemic Mastocytosis, run by AB Science at 24 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 24 sites in France, United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Cumulative response (4R75%) (time frame: 24 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00814073, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Dec 2019.

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