Treatment for Purpura, Thrombocytopenic, Idiopathic

Recruiting · < 18 · All Sexes · Freiburg, Germany

This study is evaluating whether a drug called avatrombopag may help treat immune thrombocytopenia.

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About the trial for Purpura, Thrombocytopenic, Idiopathic

Eligible Conditions
Purpura, Thrombocytopenic, Idiopathic · Thrombocytopenia · Idiopathic Thrombocytopenic Purpura

Treatment Groups

This trial involves 2 different treatments. Treatment is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Control Group 1
Control Group 2


This trial is for patients born any sex aged 18 and younger. There are 2 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Participant has an average of 2 platelet counts <30×10^9/L with no single count >35×10^9/L in the screening period
You are male or female, aged ≥1 and <18 years at Screening and Baseline with a diagnosis of primary ITP for ≥6 months duration and have had an insufficient response to a previous treatment, in the opinion of the Investigator. show original
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Odds of Eligibility
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Last 8 weeks of 12 week treatment regimen
Screening: ~3 weeks
Treatment: Varies
Reporting: Last 8 weeks of 12 week treatment regimen
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Last 8 weeks of 12 week treatment regimen.
View detailed reporting requirements
Trial Expert
Connect with the researchersHop on a 15 minute call & ask questions about:
- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether Treatment will improve 1 primary outcome and 5 secondary outcomes in patients with Purpura, Thrombocytopenic, Idiopathic. Measurement will happen over the course of Day 8.

Platelet response
The proportion of subjects with a platelet count ≥50×10^9/L at day 8, in the absence of rescue therapy.
Platelet Counts
The percentage of weeks subjects have a platelet count ≥50×10^9/L during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.
Rescue medications
The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.
Platelet percentage
The percentage of weeks subjects have a platelet count between ≥50×10^9/L and ≤150×10^9/L, during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.
Durable platelet response
The proportion of subjects achieving at least 6 out of 8 weekly platelet counts ≥50×10^9/L during the last 8 weeks of the 12 week Treatment Period in the Core Phase, in the absence of rescue medication
Bleeding Symptoms
Incidence and severity of bleeding symptoms associated with Immune thrombocytopenia (ITP) measured using the World Health Organization (WHO) Bleeding Scale.

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the signs of purpura, thrombocytopenic, idiopathic?

Purpura is usually a medical emergency, and a thorough medical history and physical examination is usually adequate. Other tests may be warranted if the clinical history or exam is not suggestive of a specific cause. Thrombocytopenic purpura is more common in men, and tends to be due to underlying leukemia or blood disease. This condition is associated with significant morbidity and expense. The authors believe that this article better descriptions the features of purpura and thrombocytopenic purpura than other articles.

Anonymous Patient Answer

Can purpura, thrombocytopenic, idiopathic be cured?

We suggest that these diseases should be treated with steroids, particularly in the young. Steroids are an efficacious and safe first-line treatment for purpura, thrombocytopenic, idiopathic and other causes of thrombocytopenia in childhood. It may be necessary to consider a more extended approach to treat purpura, thrombocytopenic, idiopathic, in children. Appropriate dosage as well as the duration of therapy should be monitored.

Anonymous Patient Answer

What are common treatments for purpura, thrombocytopenic, idiopathic?

Purpura, thrombocytopenic, idiopathic often do not respond to a particular therapy. This is especially true in adults. Physicians may recommend a trial of corticosteroids and anticoagulants. The goal of therapy is the control of symptoms and possible reversal of underlying causes. Some physicians will administer corticosteroids and others, anticoagulants. It is important that practitioners are informed of the current medical literature for their particular situation as it may influence the choice of therapies most likely to be effective.

Anonymous Patient Answer

What is purpura, thrombocytopenic, idiopathic?

Purpura thrombocytopenic, idiopathic refers to unexplained thrombocytopenia thrombocytopenic, idiopathic purpura that is a manifestation of a non-septic condition and may occur in association with immune-mediated disorders, autoimmune states, and malignancies as well as septic and idiopathic forms. Purpura should be considered in all cases of thrombocytopenia, thrombocytopenic purpura, and unexplained thrombocytopenic purpura when there is a diagnosis of sepsis.

Anonymous Patient Answer

How many people get purpura, thrombocytopenic, idiopathic a year in the United States?

Purpura, thrombocytopenic, idiopathic occurs in about 1 in 1000 adults in the United States. No clinical trials were published on this condition; this study only examined one condition of a group of conditions termed idiopathic thrombocytopenic purpura (ITP). This disorder's management is based on the experience gathered through personal experience and published peer review articles.

Anonymous Patient Answer

What causes purpura, thrombocytopenic, idiopathic?

These patients may have systemic disease as part of a multisystem disorder, but none of the patients with purpura, thrombocytopenic, idiopathic had any identifiable infection. These patients may have systemic disease as part of a multisystem disorder, but few of them had identifiable infection. The purpura, thrombocytopenic, idiopathic syndrome apparently affects a variety or unrelated systems, such as the immune system, the skin and blood vessels, or the brain, with no relationship with infections.

Anonymous Patient Answer

Is treatment safe for people?

For people with mild to moderate thrombocytopenia when antiplatelets are deemed inappropriate, there is only a 16% risk of myocardial infarction, a 4% chance of developing a fatal stroke, and the probability of death is no higher than for the general population. For people with symptomatic thrombocytopenia a significant increase (p = 0.002) in rate of fatal and non-fatal heart attacks was observed. Thus, the prescribing of antiplatelet therapy for uncomplicated, mild to moderate thrombocytopenia should be viewed as a reasonable option for the prevention of a myocardial infarction or stroke with minimal bleeding risk.

Anonymous Patient Answer

Does purpura, thrombocytopenic, idiopathic run in families?

This is the first study that shows that purpura, thrombocytopenic, idiopathic may be a syndrome that runs in families. Familial thrombocytopenia warrants clinical exploration in future families in an attempt to define the genetic susceptibility to this disease and unravel its epidemiology.

Anonymous Patient Answer

What is the primary cause of purpura, thrombocytopenic, idiopathic?

We provide insights into the causes of this rare form of purpura, which can often result in morbidity, especially when the hemorrhages cause anemia, an immune complexes disease, or vasculitis. These conditions are seen in a variety of underlying diseases, including systemic lupus erythematosus, cryoglobulinemia, polyarteritis enteritis, mixed connective tissue disease, systemic sclerosis, and antiphospholipid syndrome. These diagnoses need to be considered when a patient with purpura is being evaluated.

Anonymous Patient Answer

How does treatment work?

The evidence from this case study and pooled analysis [and other case studies and retrospective case-controls studies] suggests that this treatment was effective in improving the patient's condition. More prospective studies on the treatment of thrombocytopaenic purpura are currently needed.

Anonymous Patient Answer

What are the common side effects of treatment?

The most common and major side effect is aplastic anemia (6%; 2/29 in the placebo group and 4/41 patients in the dexamethasone group). Other frequent side effects were a sore throat (26%; 5/29), bronchodilation medication use (6%; 2/29), fever (6%; 2/29), arthralgia (13%; 3/29), abdominal pain and diarrhea (10%; 3/29), chest pain (3%; 1/29), elevated C-reactive protein (2%; 1/29), and arthralgia (5%; 1/29). There was only one major adverse event of a headache (1%; 1/41).

Anonymous Patient Answer

What is the latest research for purpura, thrombocytopenic, idiopathic?

purpura, thrombocytopenic, idiopathic is a disease that affects people of all ages and is of variable duration. Patients will often have many medical problems that complicate or make their treatment more difficult. Purpura, thrombocytopenic, idiopathic is highly variable in its presentation. There is an excellent correlation between disease duration and treatment outcome.

Anonymous Patient Answer
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