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Avapritinib for Systemic Mastocytosis

Not currently recruiting at 50 trial locations

Overseen ByBlueprint Medicines

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Blueprint Medicines Corporation

Must not be taking: Cytoreductive, Antibody, Interferon, others

Disqualifiers: Cutaneous mastocytosis, Aggressive SM, others

Prior Safety DataThis treatment has passed at least one previous human trial

Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests avapritinib for individuals with indolent systemic mastocytosis (ISM), a condition where certain immune cells accumulate in the body, causing symptoms like skin rashes, stomach pain, and fatigue. The researchers aim to determine if avapritinib can better control these symptoms compared to a placebo (a non-active treatment). Participants will receive either the medication or a placebo, but all will eventually have access to avapritinib. This trial suits those with ISM who experience moderate-to-severe symptoms not well-managed by their current care. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you must stop all current medications, but it does require that you have not taken certain treatments like cytoreductive therapy or investigational agents for a specific period before starting the trial. If you are on corticosteroids, your dose must be stable for at least 14 days.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that avapritinib is generally safe for people with indolent systemic mastocytosis. In earlier studies, patients taking avapritinib experienced ongoing improvements in symptoms and quality of life. These studies found no significant long-term harmful effects from the treatment.

The evidence suggests that patients can safely take avapritinib for extended periods. While some individuals might experience side effects, they are usually manageable. It is important to consult a healthcare provider about any concerns or questions before joining a clinical trial.¹ ² ³ ⁴ ⁵

Why do researchers think this study treatment might be promising for systemic mastocytosis?

Researchers are excited about avapritinib for systemic mastocytosis because it targets a specific mutation in the KIT gene, which is often the root cause of the disease. Unlike other treatments that may only address symptoms or rely on broader mechanisms, avapritinib directly inhibits this mutation, potentially leading to more effective disease control. Additionally, it is administered orally, offering convenience compared to some existing therapies that require injections or infusions. This new approach could offer patients a more targeted and easier-to-take option.

What evidence suggests that avapritinib might be an effective treatment for indolent systemic mastocytosis?

Research has shown that avapritinib, which participants in this trial may receive, can help treat symptoms of systemic mastocytosis. In earlier studies, patients taking avapritinib experienced significant symptom improvement over time. Specifically, after 24 weeks of treatment, 43% of patients reported only mild symptoms, compared to just 10% at the start. Additionally, avapritinib has been linked to a better quality of life and lasting symptom relief. These findings suggest that avapritinib could be a good option for those whose symptoms are not well-controlled by other treatments.¹ ⁴ ⁵ ⁶ ⁷

Are You a Good Fit for This Trial?

This trial is for patients with indolent systemic mastocytosis (ISM) who haven't found relief from their symptoms through standard care. They must have a specific symptom score, confirmed ISM by pathology review, and be able to perform daily activities with minimal assistance. People can't join if they've had certain treatments recently, other SM subtypes, prior avapritinib use, or specific heart rhythm issues.

Inclusion Criteria

My current treatment hasn't relieved my symptoms.

I am on a stable dose of corticosteroids, not exceeding 20 mg/d prednisone or its equivalent, for at least 14 days.

My systemic mastocytosis was confirmed by a specialized lab review.

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Exclusion Criteria

I haven't had radiotherapy or PUVA therapy in the last 14 days.

I have never been treated with avapritinib.

I haven't had specific cancer treatments in the required time before the trial starts.

See 3 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part 1 Treatment

Participants receive avapritinib or placebo in continuous 28-day cycles to determine optimal dosing

6 months

Monthly visits (in-person)

Part 2 Treatment

Participants receive avapritinib or placebo in continuous 28-day cycles to assess efficacy and safety

6 months

Monthly visits (in-person)

Part 3 Treatment

All participants receive avapritinib in continuous 28-day cycles

6 months

Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 5 years

What Are the Treatments Tested in This Trial?

Interventions

Avapritinib
Placebo

Trial Overview The study tests Avapritinib's effectiveness and safety against a placebo in ISM patients whose symptoms persist despite best supportive care. It's randomized and double-blind: neither doctors nor participants know who gets the real drug or placebo until the study ends. Everyone eventually receives Avapritinib during part three of the trial.

How Is the Trial Designed?

7Treatment groups

Experimental Treatment

Placebo Group

Group I: (Part 3) Avapritinib RP2D + BSCExperimental Treatment1 Intervention