Ergoloid Mesylate for FRAXA Syndrome

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Rush University Medical Center, Chicago, IL
FRAXA Syndrome+1 More
Ergoloid Mesylate - Drug
Eligibility
18 - 65
Male
What conditions do you have?
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Study Summary

Single Blind Study of Ergoloid Mesylates, 5-HTP and the Combination in Adult Males With Fragile X Syndrome

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Eligible Conditions

  • FRAXA Syndrome

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for FRAXA Syndrome

Study Objectives

This trial is evaluating whether Ergoloid Mesylate will improve 1 primary outcome and 13 secondary outcomes in patients with FRAXA Syndrome. Measurement will happen over the course of Up to 21 weeks.

Up to 21 weeks
Aberrant Behavior Checklist (ABC)
Anxiety, Depression, and Mood Scale (ADAMS)
Clinical Global Impression Improvement
Clinical Global Impression Severity
Event Related Potentials (ERP)
Eye Tracking
Eye Tracking - Gaze Aversion
Eye Tracking - Pupilometry
KiTAP Executive Battery
NIH Toolbox Cognitive Battery Modified for Intellectual Disabilities (NIH-TCB)
Safety and tolerability
Vineland-3 Adaptive Behavior Scale
Visual Analog Scale (VAS)
iBehavior ratings

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for FRAXA Syndrome

Trial Design

4 Treatment Groups

5-hydroxytryptophan (5-HTP) and placebo
1 of 4
Ergoloid mesylates (EM) and placebo
1 of 4
Ergoloid mesylates (EM) and 5-hydroxytryptophan (5-HTP)
1 of 4
Placebo
1 of 4
Experimental Treatment
Non-Treatment Group

This trial requires 15 total participants across 4 different treatment groups

This trial involves 4 different treatments. Ergoloid Mesylate is the primary treatment being studied. Participants will be divided into 3 treatment groups. Some patients will receive a placebo treatment. The treatments being tested are in Phase 2 and have already been tested with other people.

5-hydroxytryptophan (5-HTP) and placebo5-hydroxytryptophan (5-HTP) 100 mg three times daily and 1 placebo capsule three times daily for 4 weeks.
Ergoloid mesylates (EM) and placeboErgoloid mesylates (EM) 1 mg three times daily and 1 placebo capsule three times daily for 4 weeks.
Ergoloid mesylates (EM) and 5-hydroxytryptophan (5-HTP)Ergoloid mesylates (EM) 1 mg three times daily and 5-hydroxytryptophan (5-HTP) 100 mg three times daily for 4 weeks
Placebo
Other
2 placebo capsules three times daily for 4 weeks

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 21 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 21 weeks for reporting.

Closest Location

Rush University Medical Center - Chicago, IL

Eligibility Criteria

This trial is for male patients between 18 and 65 years old. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Male aged 18 to 45 years, inclusive.
Participant has Fragile X Syndrome with a molecular genetic confirmation of the full Fragile X Mental Retardation (FMR1) mutation (≥200 CGG repeats).
Current treatment with no more than 3 prescribed psychotropic medications. Anti-epileptic medications are permitted and are not counted as psychotropic medications if they are used for treatment of seizures. Anti-epileptics for other indications, such as the treatment of mood disorders, count towards the limit of permitted medications.
Permitted concomitant psychotropic medications must be at a stable dose and dosing regimen for at least 2 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication.
Anti-epileptic medications must be at a stable dose and dosing regimen for 12 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication.
Participants with a history of seizure disorder who are currently receiving treatment with anti-epileptics must have been seizure-free for 3 months preceding screening, or must be seizure-free for 3 years if not currently receiving anti-epileptics.
Behavioral and therapy treatments/interventions must be stable for 4 weeks prior to Screening and must remain stable during the period between Screening and the commencement of study medication, and throughout the study. Minor changes in hours or times of therapy that are not considered clinically significant will not be exclusionary. Changes in therapies provided through a school program, due to school vacations, are allowed.
Participant must be willing to practice barrier methods of contraception while on study, if sexually active. Abstinence is also considered a reasonable form of birth control in this study population.
Participant has a parent, legal authorized guardian or consistent caregiver.
Participant and caregiver are able to attend the clinic regularly and reliably.

Patient Q&A Section

What is fraxa syndrome?

"Fraxa syndrome is an autosomal recessive disorder that is caused by specific gene mutations. It starts out as early on in life and continues to produce symptoms until a person is an adult. It is a rare disorder with a prevalence of only 1 in 50.000 births. Symptoms can include facial features, short stature, low bone density, hypoglycemia, or muscle weakness. In some people, it can also be associated with other neurological problems including seizures and intellectual disability." - Anonymous Online Contributor

Unverified Answer

What causes fraxa syndrome?

"In this series of patients with features of fraxa syndrome, it is suggested that the syndrome is largely a clinical diagnosis. Its features should guide treatment and research into its aetiology. The syndrome appears to be a new clinical entity, and fraxa may have specific causes." - Anonymous Online Contributor

Unverified Answer

What are common treatments for fraxa syndrome?

"Frequent co-occurrence of multiple symptoms and abnormalities in these areas suggests the possibility that common treatments (e.g., neuroleptic-type medication, physical therapy, and chiropractic) may be effective for all of these manifestations." - Anonymous Online Contributor

Unverified Answer

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

"In the U.S., fraxa syndrome is rare, and its exact incidence is currently unknown. Results from a recent clinical trial have major implications for healthcare-related decisions. However, if one takes into consideration the possible sequelae, including the expense, for not taking such measures, this may not be a valid argument for not taking these measures." - Anonymous Online Contributor

Unverified Answer

Can fraxa syndrome be cured?

"Fraxa syndrome cannot be cured. In selected patients with mild symptoms, the syndrome can be treated quite well. However, in other cases, the syndrome is likely to worsen because of its own progression." - Anonymous Online Contributor

Unverified Answer

What are the signs of fraxa syndrome?

"In a recent study, findings suggests that fraxa syndrome is a neurological disorder and is not a skin disease. There is no clear and conclusive explanation for fraxa syndrome, yet based on available studies we speculate that it is a combination of disorders for a variety of syndromes. The exact diagnosis should be established by a specialist to give a proper and more effective treatment. The combination of systemic disease with neurological disease warrants a clinical investigation to understand the exact association. At present no treatment is available, so there is an urgent need for further research." - Anonymous Online Contributor

Unverified Answer

Is ergoloid mesylate safe for people?

"This clinical trial shows that the safety of using ergoloid mesylate for 12 weeks is similar to the 4-week dosing regimen. However it is recommended that the 4-week dosing regimen be the dosing regimen to ensure safety for individuals with fragile X syndrome." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets fraxa syndrome?

"In addition to the typical presentation of fraxa syndrome in the adult, we found an increased incidence of familial onset of the disease in the pediatric subset. Children with fraxa syndromes may be detected by routine screening examination or because of a close familial history." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in ergoloid mesylate for therapeutic use?

"A recent series of studies demonstrated a promising clinical response to ergoloid mesylate. The safety profile associated with this drug and the positive clinical results indicate its feasibility for routine use." - Anonymous Online Contributor

Unverified Answer

Does fraxa syndrome run in families?

"In summary, familial cases of FD occur in approximately 4% of patients (7/177) with FD, with a statistically significant difference between familial and sporadic FD. FD is associated with an increased prevalence (12%) of FD-like features in some patients without the known FD gene mutations. This data supports the hypothesis of an interactive or inherited gene-environmental process in FD aetiology." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving ergoloid mesylate?

"Very few studies have been published investigating the effect of this therapy and our study is the only one to date evaluating such therapy in ALS patients, thus offering further clues to this medical question." - Anonymous Online Contributor

Unverified Answer

How serious can fraxa syndrome be?

"The cause of fraxa syndrome remains unknown and, therefore, this condition has a mortality rate of less than 3%. However, studies have shown that the mortality rate of untreated victims can be as high as 65%. There is no way of curing the condition and hence it is of utmost importance that individuals suffering from fraxa syndrome receive appropriate care.\n" - Anonymous Online Contributor

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