Odevixibat for Syndrome

2
Effectiveness
3
Safety
Instytut Pomnik-Centrum Zdrowia Dzieck, Warszawa, Poland
Syndrome+1 More
Odevixibat - Drug
Eligibility
Any Age
All Sexes
Eligible conditions
Syndrome

Study Summary

This study is evaluating whether a drug called odevixibat (A4250) is safe and effective in people with Alagille syndrome.

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Eligible Conditions

  • Syndrome
  • Alagille Syndrome

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether Odevixibat will improve 1 primary outcome and 5 secondary outcomes in patients with Syndrome. Measurement will happen over the course of Baseline to week 72.

Baseline to Weeks, 4, 12, 24, 48, and 72
Change in Global Symptom Relief
Baseline to week 72
Change in Quality of Life
Change in Sleep Parameters
Change in pruritus
Change in serum bile acids
Safety and Tolerability

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

Control
Odevixibat (A4250)

This trial requires 63 total participants across 2 different treatment groups

This trial involves 2 different treatments. Odevixibat is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.

Odevixibat (A4250)
Drug
Capsules for oral administration once daily for 72 weeks.
ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline to weeks, 4, 12, 24, 48, and 72
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly baseline to weeks, 4, 12, 24, 48, and 72 for reporting.

Closest Location

Boston Children's Hospital - Boston, MA

Eligibility Criteria

This trial is for patients born any sex of any age. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
All sexually active participants must use a reliable contraceptive method with ≤1% failure rate from signed informed consent through 90 days after last dose of study drug. show original
Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study
The study requires both caregivers and age-appropriate patients to be willing and able to use an electronic diary device. show original
The 24-week Treatment Period of Study A4250-012 has been completed show original

Patient Q&A Section

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

Can syndrome be cured?

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There is considerable difficulty in assigning a diagnosis, and the terminology of the disease has a profound impact on patients and family members. The patient is most often aware of his or her limitations and may not wish the diagnosis of his or her condition disclosed to anyone, let alone to the person to whom it may be the worst secret. A cure for a disease without definitive clinical, genetic and pathological information has proven elusive: therefore a cure for all syndrome is unlikely. For each new syndrome to be understood and addressed, it seems that a cure must be found. There are few ways of finding such a cure. The most reliable are the clinical trials of new drugs which are designed to address the shortcomings of the diagnosis and the symptoms of each syndrome.

Unverified Answer

What is syndrome?

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Syndromes can also be defined according to a specific set of symptoms or presentations, rather than by their definition. These definitions can be grouped into descriptive syndromes that represent a collection of similar conditions, or descriptive syndromes that are groups of similar syndromes. The latter definition, for example, is frequently used in the nosologic classification of childhood psychiatric disorders and can be used in this article.

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How many people get syndrome a year in the United States?

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The prevalence of SAs in one year in the United States is 6.7%. There is a trend toward increasing prevalence over the last 12 years. Most SAs (66.5%) are diagnosed in the first two years after being evaluated. The highest rate of SAs was found amongst individuals with symptoms of depression with a history of an anxiety disorder.

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What are common treatments for syndrome?

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Syndrome therapies include diet, exercise, relaxation counseling, cognitive behavior therapy, and others. \n\nIn recent years, there has been a surge of interest in the field of psychopharmacology, which deals with the study of the effects of drugs in the physiology or the mood of the organism.\n\nPsychopharmacology is concerned with the effects of chemical substances on emotional-sensory or cognitive stimuli. The most commonly studied classes for pharmacology are receptor agonists, receptor antagonists, monoamine reuptake inhibitors, and drugs affecting dopamine function (including dopaminergic stimulants, such as cocaine, methamphetamine, and nicotine). Stimulants are drugs that promote arousal.

Unverified Answer

What are the signs of syndrome?

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Signs of a syndrome may include unexplained loss of appetite, anorexia, constipation or diarrhea, and a fast heart rate. These are generally not cause for medical concern. However, they can be alarming signs and a condition which requires immediate evaluation.

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What causes syndrome?

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This article discusses how we should look at syndromes in the eye of the beholder, what they have in common and what we should pay attention to. When we are considering the concept of syndromes--or what it may mean to make a diagnosis--it is important to see them in the context of the wider system in which they are embedded. We have not yet figured out how to accurately construct system maps of syndromes and how effectively to organize the information on which they are based. We could benefit from developing clearer definitions of our concepts of syndromes, from considering the relationships between different concepts, and perhaps from examining how syndromes might fit in with other categories in systematic classification.

Unverified Answer

What are the common side effects of odevixibat?

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Common side effects with odevixibat included nausea, abdominal pain and diarrhea. There were more serious side effects such as vomiting and hypotension (low blood pressure), which were most likely attributable to the underlying medical condition associated with this medication. The side effects of odevixibat were comparable in individuals to controls.

Unverified Answer

Does odevixibat improve quality of life for those with syndrome?

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Odevixibat was well-tolerated with no clinically meaningful adverse events. Odevixibat improved overall quality of life in both the short-term and the longer term as measured by the Q-OF-36 and the EpiNHLD-2.

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Who should consider clinical trials for syndrome?

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The majority of patients who are given clinical trials do not meet the criteria for a clinical trial because of self-reported seriousness, self-reported risk-taking behavior, and reported reluctance to accept an unproven treatment. Many patient-reported outcomes of a clinical trial evaluating a clinical intervention are unimportant or not sufficiently important to change patient behavior. Most patients with rare (not serious) syndrome receive no treatment due to ethical and legal reasons.

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Have there been any new discoveries for treating syndrome?

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This is a review of research that has made very important discoveries for the treatment of syndrome. We will also discuss some of the controversies involved in the treatment of syndrome and the advances in diagnostic tools needed for the development of treatment for syndrome.

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What is the average age someone gets syndrome?

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There is no average age of presenting with syndrome. The presentation of syndrome varies widely between geographic areas and racial groups. Results from a recent clinical trial is part of a National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)/National Institutes of Health-supported initiative to explore rare genetic conditions in children, adults, and families.

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How serious can syndrome be?

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This is a rare and unexplained phenomenon known as syndrome, a clinical diagnosis of a group of disorders characterized by a variety of medical (symptoms), psychologic (traits), educational (cognitive), and social problems. Although this illness is rare, its severity depends on the number of symptoms and severity scale. Symptoms of patients with syndrome range from serious to life-threatening. It was recommended to report a diagnosis of syndrome to a reputable specialist. The treatment of most syndromes could be similar to that of the patients affected by epilepsy or bipolar disorder.

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