CLINICAL TRIAL

4D-310 for Fabry Disease

Recruiting · 18+ · Male · Fairfax, VA

This study is evaluating whether a drug may help treat Fabry disease.

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About the trial for Fabry Disease

Treatment Groups

This trial involves 5 different treatments. 4D-310 is the primary treatment being studied. Participants will be divided into 5 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Experimental Group 1
4D-310
BIOLOGICAL
Experimental Group 2
4D-310
BIOLOGICAL
Experimental Group 3
4D-310
BIOLOGICAL
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Eligibility

This trial is for male patients aged 18 and older. There are 4 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
In medically confirmed cases of Fabry disease, the individual will often exhibit symptoms such as acroparesthesia, hypohidrosis, angiokeratoma or cornea verticillata, and/or left ventricular hypertrophy. show original
You must have been taking the same dose of ERT for at least six months (and a total of at least 12 months) before you can enroll in the study. show original
A man who is at least 18 years old. show original
The text is discussing a mutation that is associated with Fabry disease show original
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Odds of Eligibility
Unknown<50%
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: 1 year
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 year
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: 1 year.
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 4D-310 will improve 1 primary outcome and 3 secondary outcomes in patients with Fabry Disease. Measurement will happen over the course of 1 year.

Change from baseline in serum AGA activity
1 YEAR
Change from baseline in serum AGA activity
1 YEAR
Incidence and severity of adverse events
1 YEAR
Safety and tolerability of 4D-310 following a single IV dose, as assessed by incidence and severity of adverse events, serious adverse events and dose limiting toxicities, including clinically significant changes from baseline to scheduled time points in safety parameters
1 YEAR
Change from baseline in blood leukocyte AGA activity
1 YEAR
Change from baseline in blood leukocyte AGA activity
1 YEAR
Change from baseline serum globotriaosylsphingosine (lysoGb3)
1 YEAR
Change from baseline serum globotriaosylsphingosine (lysoGb3)
1 YEAR

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 fabry disease?

Fabry disease is most reliably diagnosed in the early stages by the cardiothoracic surgeons and internists in Fabry disease clinics. It is therefore important that people with unexplained unexplained symptoms are referred for diagnosis. The treatment of Fabry disease is with a lifelong regimen of angiotensin converting enzyme (ACE) inhibitor for proteinuria and treatment of heart disease in addition to the other aspects of management.

Anonymous Patient Answer

Can fabry disease be cured?

In those people who have not previously suffered from any type of atherosclerotic vascular disease, fabry disease-induced renal failure can be successfully reversed. However, in those with atherosclerotic vascular disease, kidney failure is irreversible and dialysis must be initiated. However, the treatment of individuals suffering from Fabry disease with drugs that inhibit the activity of aldosterone synthase can reduce the severity of affected people's symptoms and reduce their risk of arterial attacks.

Anonymous Patient Answer

What is fabry disease?

The most important predictor for Fabry disease in women is late-onset of disease, and in men, the most important indicators are elevated levels of LDL. Fabry disease results in accumulation of lipids in the affected tissues, particularly in the kidney, heart and skin.\n

Anonymous Patient Answer

What are common treatments for fabry disease?

The treatment of Fabry disease can be a complex decision-making process. Early management can be challenging. Physicians often lack clinical experience with the disease and should remain involved in patient counseling as well as monitoring to avoid complications.

Anonymous Patient Answer

How many people get fabry disease a year in the United States?

There are approximately 30 Fabry disease patients in the United States. There is a low incidence of individuals with Fabry disease at the same time in different countries. The clinical manifestations are similar, although the renal presentations vary.

Anonymous Patient Answer

What causes fabry disease?

This may have implications for the management of the condition, as well as for general awareness of a rare genetic condition that is often mistaken between the populations from developing and developed countries.\n

Anonymous Patient Answer

How does 4d-310 work?

For patients with Fabry disease who have no effective treatment, or who cannot tolerate the medications on the market, 4d-310 could be a promising therapy.

Anonymous Patient Answer

What are the latest developments in 4d-310 for therapeutic use?

The 4D-310 platform enables drug development with high level of fidelity to pharmaceutical regulatory regulatory standards, while remaining technically feasible to generate in quantities and quality sufficient to meet a therapeutic candidate's needs. A 4D-310-derived compound, 4D-310L, is a potent and selective nonpeptide antagonist of the κ-opioid receptor (KOR), which could potentially serve as a novel drug therapy for multiple indications.

Anonymous Patient Answer

What are the common side effects of 4d-310?

In the clinical trials investigating the systemic pharmacokinetics of 4D310, no serious adverse events were reported. However, 4D310 has a potential immunogenicity, which may lead to the development of other autoimmune syndromes such as erythema nodosum, systemic lupus erythema, arthritis, and autoimmune hemolytic anemia. Therefore, 4D310 should be used with caution in patients with autoimmune disorders. 4D310 may also induce high-normal liver enzymes and elevated bilirubin. These side effects should be monitored during 4D310 therapy.

Anonymous Patient Answer

Who should consider clinical trials for fabry disease?

In the clinical trials performed in this study, the disease activity of patients with Fabry disease differed according to sex and disease type. These differences may have contributed to the lack of efficacy of some drugs and support that clinical trials should be performed using a pooled analysis, which is recommended in clinical practice. The clinical trials of beta blocker therapy can help to evaluate the efficacy of this treatment, particularly, as a secondary outcome, in female Fabry patients with cardiac involvement or in those who also have neurological manifestations.

Anonymous Patient Answer

Have there been any new discoveries for treating fabry disease?

The first treatment of this disease was published in 1985. The latest discovery of this disease is a drug which is given to patients after a kidney transplant from a person with this illness. Although the drugs to treat fabry disease have already been discovered, there is still no real cure for this condition. There are no treatments that will keep these patients from having to rely on hemodialysis when and if they have to have a kidney transplant. Newer drugs and recombinant proteins [containing the Fabry protein] that should be effective for this disease are under development. Although Fabry disease could result in a life-threatening kidney transplant, it is still considered a severe disease because the life expectancy for patients who have this disease is only a few years.

Anonymous Patient Answer

Have there been other clinical trials involving 4d-310?

There are currently no studies in which the 4D-310 compound was given as part of a clinical trial. To date all studies of 4D-310 and related compounds have been performed on animals. The ultimate aim of clinical trials is to identify the most effective compound with desired therapeutic benefit and minimal side effects. For clinical trials, the aim is not to test a new drug, but to identify which drug is the most effective. There is no evidence of safety or efficacy in humans, so 4D-310 is not currently available for use in humans beyond testing as of December 2016.

Anonymous Patient Answer
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