#1 Clinical Trials Search in Europe

Masitinib

Clinical trials investigating Masitinib are studying whether it can help people with serious conditions such as ALS, mast cell activation syndrome, mastocytosis, and multiple sclerosis. These studies mainly look at safety and how well the treatment works in specific patient groups. Most trials are randomized, placebo-controlled, and in phase 2 or phase 3.

Table of Contents

Overview of the Masitinib trials

These clinical trials are testing Masitinib in several different diseases, not as a general medicine article, but as a study treatment in specific research settings.[1][2][3][4][5]

All five studies listed are interventional, which means the researchers give a treatment and then measure results.[1][2][3][4][5]

The studies are mainly randomized, double-blind, and placebo-controlled, which helps compare groups more fairly and reduces bias.[1][3][4][5]

Most of the trials are in phase 3, and one trial is in phase 2.[1][2][3][4][5]

Trials in amyotrophic lateral sclerosis

Two phase 3 trials study amyotrophic lateral sclerosis (ALS), a disease that affects nerve cells and leads to muscle weakness over time.[2][5]

One trial compares Masitinib plus standard of care with placebo plus standard of care in patients with ALS, and it plans to include 327 participants.[2]

The other ALS trial compares Masitinib in combination with Riluzole versus placebo in combination with Riluzole, and it plans to include 155 participants.[5]

Both ALS studies are trying to see whether adding Masitinib can improve outcomes in people already receiving treatment for ALS.[2][5]

Trials in mast cell activation syndrome and mastocytosis

One phase 2 study is testing Masitinib in people with severe mast cell activation syndrome (MCAS) whose handicap did not improve with optimal symptomatic treatment.[1]

This study is designed as a 24-week trial and plans to compare two titration schemes of oral Masitinib with placebo.[1]

The trial enrolled 5 participants and focuses on symptoms that affect daily life, such as itching, flushing, depression, and fatigue.[1]

Another phase 3 study is testing Masitinib in patients with smouldering or indolent severe systemic mastocytosis with handicap that is unresponsive to optimal symptomatic treatment.[3]

This mastocytosis trial plans to compare oral Masitinib with placebo over 24 weeks, with a possible extension period afterward.[3]

The study enrolled 70 participants and looks at whether treatment can improve patient handicap, meaning how much the condition affects daily function.[3]

Trial in multiple sclerosis

One phase 3 study is testing Masitinib in patients with primary progressive or secondary progressive multiple sclerosis without relapse.[4]

This means the trial focuses on people whose disease is steadily getting worse rather than having relapse episodes.[4]

The study plans to follow participants for 96 weeks and compare Masitinib dose titration to placebo.[4]

It enrolled 145 participants and is designed to see whether treatment can slow disability progression in this group.[4]

How the studies are designed and who may join

These trials use different dose plans, including dose titration, which means the dose is adjusted step by step during the study.[1][3][4]

Some studies compare Masitinib with placebo alone, while others test it as an add-on to standard treatment such as standard of care or Riluzole.[2][5]

People may be eligible only if they have the exact condition named in the study and, in some trials, if their symptoms remain severe despite optimal symptomatic treatment.[1][3]

Because these are controlled trials, participants are assigned to treatment groups by chance, and the comparison group may receive placebo.[1][3][4][5]

Main endpoints measured in the trials

The phase 2 MCAS study measures a confirmed response at week 24 using four handicap scores: pruritus, flush, depression, and fatigue.[1]

The ALS trial with standard of care uses the ALSFRS-R, which is a score that measures function in people with ALS, and it looks at the change from baseline at week 48.[2]

The second ALS trial also uses the ALSFRS-R change from baseline at week 48 as its main outcome.[5]

The mastocytosis study measures response on three handicaps from week 8 to week 24 in the main analysis.[3]

The multiple sclerosis study measures time to confirmed disability progression using the EDSS, a scale that tracks worsening disability in MS.[4]

Across the trials, the main goal is to see whether Masitinib can improve symptoms, slow worsening, or increase overall response compared with placebo.[1][2][3][4][5]

Trial ID Phase Condition studied Status Enrollment
2024-511244-12-01 Phase 3 Amyotrophic Lateral Sclerosis Suspended 327
2024-516671-33-00 Phase 3 Amyotrophic Lateral Sclerosis Suspended 155
2024-516672-16-00 Phase 3 Multiple Sclerosis Suspended 145
2024-514538-19-00 Phase 3 Mastocytosis Suspended 70
2024-515193-27-00 Phase 2 Mast cell activation syndrome Suspended 5

FAQ

  • What conditions are being studied in Masitinib trials? The trials in this set study Masitinib in amyotrophic lateral sclerosis (ALS), mast cell activation syndrome (MCAS), mastocytosis, and multiple sclerosis. Each trial focuses on a different patient group and disease stage.
  • Who can take part in these trials? People may be eligible if they have the condition being studied, such as severe MCAS, ALS, severe systemic mastocytosis, or progressive multiple sclerosis without relapse. Some trials also require symptoms that do not improve with optimal standard treatment.
  • What phases are included in the Masitinib trials? The trials shown here are in phase 2 and phase 3. Phase 2 studies usually look at early signs of benefit and safety, while phase 3 studies compare Masitinib with placebo in larger groups.
  • What do these trials measure? The trials measure outcomes such as symptom improvement, disease progression, and overall response to treatment. Examples include handicap scores, ALSFRS-R score, and EDSS progression.
  • Are the trials placebo-controlled? Yes, all trials listed here compare Masitinib with placebo, often in a double-blind design. Double-blind means neither the patient nor the study team knows who gets the active treatment or placebo during the trial.

Ongoing Clinical Trials on Masitinib

  • Study on the Effectiveness and Safety of Masitinib and Riluzole for Patients with Amyotrophic Lateral Sclerosis (ALS)

    Not yet recruiting

    3 1 1
    Investigated drugs:
    France Germany Greece Latvia Slovenia Spain +1

  • Study on the Effectiveness and Safety of Masitinib with Riluzole for Patients with Amyotrophic Lateral Sclerosis (ALS)

    Not yet recruiting

    3 1
    Investigated drugs:
    Denmark France Greece Norway Sweden

  • Study on the Effectiveness and Safety of Masitinib for Patients with Severe Systemic Mastocytosis Unresponsive to Treatment

    Not yet recruiting

    3 1
    Investigated drugs:
    France Poland Spain

  • Study on Masitinib for Treating Primary or Secondary Progressive Multiple Sclerosis in Patients Without Relapse

    Not yet recruiting

    3 1
    Investigated drugs:
    France Greece Poland Spain

  • Study on Masitinib for Patients with Severe Mast Cell Activation Syndrome Unresponsive to Treatment

    Not yet recruiting

    2 1
    Investigated drugs:
    France

Glossary

  • Amyotrophic Lateral Sclerosis (ALS): A serious disease that affects nerve cells and causes muscle weakness over time.
  • Mast Cell Activation Syndrome (MCAS): A condition where mast cells release signals too often, which can cause symptoms such as itching, flushing, tiredness, and low mood.
  • Mastocytosis: A condition where there are too many mast cells in the body, which can cause different symptoms and disability.
  • Multiple Sclerosis (MS): A disease of the brain and spinal cord that can affect movement, balance, and daily function.
  • Phase 2: An earlier stage of clinical research that looks at whether a treatment may work and checks safety in a smaller group.
  • Phase 3: A later stage of research that compares a treatment with placebo or standard care in a larger group.
  • Placebo: An inactive treatment used for comparison in a trial.
  • Randomized: Assigned by chance to different study groups, so the groups are more similar.
  • Double-blind: A study design where the patient and the study team do not know which treatment is being given.
  • Primary endpoint: The main result the researchers plan to measure to see if the treatment works.
  • ITT population: Intention-to-treat population, meaning the main analysis includes all participants as originally assigned.
  • EDSS: Expanded Disability Status Scale, a score used to measure disability in multiple sclerosis.

References

  1. https://clinicaltrials.gov/study/2024-515193-27-00
  2. https://clinicaltrials.gov/study/2024-511244-12-01
  3. https://clinicaltrials.gov/study/2024-514538-19-00
  4. https://clinicaltrials.gov/study/2024-516672-16-00
  5. https://clinicaltrials.gov/study/2024-516671-33-00