CLINICAL TRIAL

Elaprase for Syndrome

Waitlist Available · < 65 · Male · Coyoacan, Mexico

This study is evaluating whether Elaprase treatment can be combined with idursulfase IT treatment.

See full description

About the trial for Syndrome

Eligible Conditions
Syndrome · Mucopolysaccharidosis II · Hunter Syndrome

Treatment Groups

This trial involves 2 different treatments. Elaprase 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 2 & 3 and have had some early promising results.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Elaprase
DRUG
Idursulfase-IT
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

Eligibility

This trial is for male patients aged 65 and younger. There are 3 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Participants must have completed Visit Week 52 assessments in Study HGT-HIT-094 (NCT02055118).
The participant's parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the participant's parent(s) or legally authorized guardian(s) and the participant's consent/assent, as relevant, must be obtained.
The participant has continued to receive Elaprase on a regular basis in Study HGT-HIT-094 (NCT02055118).
View All
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
Similar Trials

Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline through Month 121
Screening: ~3 weeks
Treatment: Varies
Reporting: Baseline through Month 121
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline through Month 121.
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 Elaprase will improve 8 primary outcomes and 14 secondary outcomes in patients with Syndrome. Measurement will happen over the course of From start of study drug administration up to 121 months.

Number of Participants who Report Anti-idursulfase Antibodies in Serum
FROM START OF STUDY DRUG ADMINISTRATION UP TO 121 MONTHS
The presence of idursulfase-specific antibodies will be assessed.
FROM START OF STUDY DRUG ADMINISTRATION UP TO 121 MONTHS
Number of Participants who Report Anti-idursulfase Antibodies in Cerebrospinal Fluid (CSF)
FROM START OF STUDY DRUG ADMINISTRATION UP TO 121 MONTHS
The presence of idursulfase-specific antibodies will be assessed.
FROM START OF STUDY DRUG ADMINISTRATION UP TO 121 MONTHS
Number of Participants With Adverse Events
FROM START OF STUDY DRUG ADMINISTRATION UP TO 121 MONTHS
An adverse event is any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered investigational product-related.
FROM START OF STUDY DRUG ADMINISTRATION UP TO 121 MONTHS
Number of Participants With Clinically Significant Changes in Vital Signs, Laboratory Parameters, and 12-lead Electrocardiogram (ECG) Findings
FROM START OF STUDY DRUG ADMINISTRATION UP TO 121 MONTHS
Number of participants with clinically significant changes will be reported.
FROM START OF STUDY DRUG ADMINISTRATION UP TO 121 MONTHS
Change From Baseline in Age Equivalents Score of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Sub Domains
BASELINE THROUGH MONTH 121
The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following subdomains of 5 key domains: Communication (Receptive, Expressive, Written), Daily Living Skills (Personal, Domestic, Community), Socialization (Interpersonal Relationships, Play and Leisure Time, Coping Skills), Motor Skills (Gross, Fine). The mean age equivalent score will be obtained by averaging out the age-equivalent scores for the all the sub-domains except for Gross and Fine motor skills (range: 0, unbound).
BASELINE THROUGH MONTH 121
Change From Baseline in Standard Scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II) Domains
BASELINE THROUGH MONTH 121
The VABS-II test measures adaptive behaviors, including the ability to cope with environmental changes, to learn new everyday skills, and to demonstrate independence. This test measures the following 4 key domains: communication, daily living skills, socialization, motor skills, and the adaptive behaviour composite (a composite of the other 4 domains). The standard scores represent a score (mean = 100 and standard deviation of 15) on which higher scores indicate a higher level of cognitive ability.
BASELINE THROUGH MONTH 121
See More

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 common treatments for syndrome?

Symptom management is the most common category of medical treatment in the ambulatory setting. The majority of visits (69%) are for diagnosis and/or evaluation. Physical, mental, and behavioral health issues are also common. There are several common treatments for medical conditions, including the management of diarrhea.

Anonymous Patient Answer

What is syndrome?

The syndrome model is a useful method for identifying syndromes in complex diseases. The definition of syndrome as something related to disease but not the disease itself can lead to different definitions of syndrome by different researchers. The definition and the definition of syndromes by different researchers can lead to different syndromes and to different definitions of syndromes within each syndrome. It is therefore important to consider some aspects of the definition of syndromes when designing syndrome databases such as the NCI-funded NIH-funded Syndromic Collections of Excellence (SYCE).

Anonymous Patient Answer

What are the signs of syndrome?

There are a number of causes responsible for syndrome. There is discussion as to the appropriate treatment for each syndrome and its treatment is highly dependent on each individual case.

Anonymous Patient Answer

What causes syndrome?

The authors suggest that syndromic disorders may result from a combination of genetic and lifestyle factors, a complex genetic mix, the influence of environmental factors and individualized treatment.

Anonymous Patient Answer

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

Data from a recent study indicated that syndrome is a relatively very common condition in United States, with an incidence of 2.3% per year. Although the etiology of syndrome remains unknown, the number of individuals affected with it will be expanding.

Anonymous Patient Answer

Can syndrome be cured?

The term syndrome, or group of symptoms, cannot be cured. Nevertheless, a syndromic presentation may be resolved in one patient or family. In the absence of specific information, the term syndrome may be helpful in communicating the complexity and breadth of the problems to multiple providers and the patient.

Anonymous Patient Answer

What is the latest research for syndrome?

Syndromes are the presentation of the same disease but have some notable differences. They can be identified according to the presence/absence of symptoms. The different presentation of the syndrome can have significant effects on how well a patient can be diagnosed and treated. This article presents recent advances in syndrome research.

Anonymous Patient Answer

How serious can syndrome be?

The seriousness of the disease as determined by the number of patients affected and the number of deaths should be taken into account. Results from a recent paper also indicates that syndrome is a disease in the category of severe diseases. This requires more attention and, perhaps, more specific treatments.

Anonymous Patient Answer

What are the latest developments in elaprase for therapeutic use?

ELAPRASE is effective and well tolerated in severe adult cases of lHAS. Results of this study suggest that ELAPRASE is less efficacious in neonatal cases of lHAS. ELAPRASE significantly reduces ELAPROTEN, indicating a reduction in elafin activity. Reduction of elafin in the blood coincides with a reduction in elafin activity in cultured monocytes and platelets. Further work will focus on the clinical significance of these findings and, if translocated to clinical use, whether ELAPRASE is effective at a lower dose than the normal dose.

Anonymous Patient Answer

What does elaprase usually treat?

Elaprase is an oral, sustained-release formulation of rhAGP with an increased half-life that is approved for the treatment of hemophilia A. It is manufactured by CSL Behring in the Czech Republic. A prescription medication, it is marketed and distributed by Schering-Plough, in collaboration with CSL Behring, LLC. CSL Behring offers elaprase as a self-administered product to people who require treatment at least once weekly. Because elaprase has an increased half-life relative to rhAGP, elaprase has been studied as a therapeutic option for people with hemophilia A.

Anonymous Patient Answer

What is the average age someone gets syndrome?

The average age of onset is 64.8 years of age, with a minimum age of 58 years. This mean age is somewhat variable by medical center; it tends to be higher when more patients were diagnosed in a single year. The syndrome may appear later in life and have a different etiology and course. The average interval from disease onset to diagnosis is 10.3 months.

Anonymous Patient Answer

Does syndrome run in families?

Two thirds of cases in our cohort were familial, supporting the notion that familial factors play a contributory role in the pathogenesis of LEN. Because of recent evidence of the role of microRNAs in several autoimmune diseases as well as the increasing interest in miRNA in cancer genetics, we speculate that a miRNA-induced dysregulation of the human leukocyte antigens might in part or wholly explain the familial pattern of the disease in our cases.

Anonymous Patient Answer
See if you qualify for this trial
Get access to this novel treatment for Syndrome by sharing your contact details with the study coordinator.