Treatment for FRAXA Syndrome

Phase-Based Estimates
1
Effectiveness
2
Safety
Emory University Hospital, Atlanta, GA
+1 More
Eligibility
< 65
Male
Eligible conditions
FRAXA Syndrome

Study Summary

This study is evaluating whether Sulindac is safe and effective for treating males with Fragile X Syndrome.

See full description

Eligible Conditions

  • FRAXA Syndrome
  • Syndrome
  • Fragile X Syndrome

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Treatment will improve 5 primary outcomes and 6 secondary outcomes in patients with FRAXA Syndrome. Measurement will happen over the course of Day 70.

Day 70
Aberrant Behavior Checklist
Anxiety, Depression, and Mood Scale
CGI-S
Clinical Global Impression - I
EEG
Emotional Faces Tobii Eye Tracking
FXS Domain Specific Concerns
Kiddie Test of Attentional Performance (KiTAP)
NIH Cognitive Toolbox
To assess the safety and tolerability of each dose
To assess the safety and tolerability of each sulindac dose

Trial Safety

Safety Estimate

2 of 3
This is better than 68% of similar trials

Trial Design

7 Treatment Groups

Sulindac (HLX-0201), dose strength 2
Placebo group

This trial requires 60 total participants across 7 different treatment groups

This trial involves 7 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 2 and have already been tested with other people.

Sulindac (HLX-0201), dose strength 2
Drug
One capsule, twice a day
Gaboxadol (HLX-0206)
Drug
One capsule, twice a day
Sulindac (HLX-0201), dose strength 1
Drug
One capsule, twice a day
HLX-0206
Drug
One capsule, twice a day
Sulindac, dose strength 2
Drug
One capsule, twice a day
Placebo
Drug
One capsule, twice a day
Sulindac, dose strength 1
Drug
One capsule, twice a day

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: day 70
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly day 70 for reporting.

Closest Location

Emory University Hospital - Atlanta, GA

Eligibility Criteria

This trial is for male patients aged 65 and younger. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Subject consents to participate, or if the subject are not the subjects own legal guardian, offers assent supported by legally authorized representative consent show original
You are willing and able to comply with the study procedures as specified in the protocol and to comply with the study drug administration. show original
You are able to understand the spoken national language clearly and are able to read and write to complete study assessments. show original
Males aged 13 to 40 years (inclusive)
You have FXS with molecular genetic confirmation of the full FMR1 mutation (>200 CGG repeats). show original
Weight ≥45 kg
CGI-S score ≥4
You are in general good health as deemed by the Investigator, determined by physical examination, medical history and laboratory tests. show original
You are on a stable, well-tolerated dose for the previous 3 months with no further changes anticipated. show original
You agree not to discuss treatment outcomes on social media until the subject has completed their End of Therapy visit. 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 fraxa syndrome be cured?

Add answer

This is the first report of an cured case of FraxA syndrome following surgical resection. This has implications for all surgeons who see these patients and their families.

Unverified Answer

What is fraxa syndrome?

Add answer

The majority of patients suffer from fraxia syndrome at the first signs of disease, and some have it even at the time of diagnosis. It is suggested that patients, both male and female, should have regular consultations with physicians in order to manage their health to avoid complications and to improve their quality of life.

Unverified Answer

What are the signs of fraxa syndrome?

Add answer

These may include muscle weakness, joint contractures, poor muscle tone, and atrophy in some of the muscles. Other common presenting signs of fraxa syndrome may involve skin changes, including xerosis, erythema, and dry skin.\n

Unverified Answer

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

Add answer

Every year approximately 45,000 children and adults are diagnosed with Fraxa syndrome, making it the most common of the rare disorders. Fraxa syndrome can be a result of a failure of the thalidomide embryopathy model in patients with immunodeficiency. It commonly occurs in an unrelated form in children of thalidomide-exposed mothers. Fraxas are relatively less common with an overall prevalence of around 0.1% (0.2-1.2% depending on the criteria) across all geographic areas. It may become more common with increasing world population, as in other countries where thalidomide was used to help increase the numbers of children or pregnant women.

Unverified Answer

What causes fraxa syndrome?

Add answer

There is a growing body of evidence that fraxia syndrome has both genetic and environmental factors. The disease, which is also known as fraxa, occurs in men of the elderly class. Fraxia syndrome is characterized by various symptoms similar to those of schizophrenia. Because patients are usually treated with a variety of standard antipsychotics, such as quetiapine, clozapine, olanzapine, risperidone, and aripiprazole, a diagnosis of these symptoms must be clarified by a psychiatric evaluation.

Unverified Answer

What are common treatments for fraxa syndrome?

Add answer

More than 40% of the patients with Fraxa syndrome will suffer from some form of cancer, which can present as bone pain and/or pathologic fracture. For patients who have both cancer and Fraxa syndrome, it is important to discuss different treatment options for the specific disease and to use a multidisciplinary approach.

Unverified Answer

What is the latest research for fraxa syndrome?

Add answer

The term “Fraxa” was brought into existence in 1960.\nFraxa is a genetic disorder resulting in the inability to produce the olfactory receptors. The main symptoms include anosmia and dysosmia. Many studies focusing on anosmia and dysosmia were made in patients suffering from Fraxas. Though the exact reason regarding why anosmia and dysosmia are the main symptoms for aFraxas are not determined, multiple theories have contributed to the current state of knowledge. Furthermore, the exact etiology of the disorder remains unknown.

Unverified Answer

Who should consider clinical trials for fraxa syndrome?

Add answer

The use of a simple algorithm to identify eligible participants (1 patient with Friedreich's ataxia disease or FRDA; 1 patient with sporadic juvenile-onset Ataxia Disease or JOPAD) should be considered to help to identify patients with FRDA or JOPAD.

Unverified Answer

Is treatment typically used in combination with any other treatments?

Add answer

The present study suggests that the use of the treatment is combination drug therapy (prednisone with azathioprine plus methotrexate). These drugs can produce an efficacious effect in the treatment of active dermatologic manifestations, or active disease flares; however, this could not be demonstrated in most of the patients in this study.

Unverified Answer

Have there been any new discoveries for treating fraxa syndrome?

Add answer

[About a fifth of patients with fraxa syndrome complain about their disease worsening over the last 2 years, with new complaints developing most often in the period after having been diagnosed] (Power) (https://www.withpower.com/d/fraxa-syndrome-and-causes). However, there is only one type of treatment for fraxia syndrome. Fraxa Syndrome patients can search for a clinical trial at Power (www.withpower.com/d/fraxa-syndrome) to find the clinical trials tailored to your condition, treatment, or location. [http://www.withpower.

Unverified Answer

What is the average age someone gets fraxa syndrome?

Add answer

The average age for fraxa syndrome onset is 61 with a range 38-79. Age is not the only cause for the syndrome, other causes include physical/neurological diseases, endocrine malfunction, or genetics. Fraxa syndrome onset is [not likely to precede birth (mean = 59 years) by a decade or 2] except for those [with an earlier onset age because of a family history, with or without known genetic link (mean = 42)], however, a family history may be a significant factor. Fraxa syndrome also typically starts during the years of adolescence.

Unverified Answer

How does treatment work?

Add answer

Data from a recent study has shown that the majority of patients have persistent positive responses to the treatment regimen. Patient satisfaction with treatment is also high, highlighting the fact that treatment works. Data from a recent study of this study also indicate that a positive response in terms of treatment outcome depends heavily on the baseline patient expectations. The ability to identify the appropriate patients for therapy and tailor the therapy may allow many patients with FRAXA syndrome to achieve a low level of symptoms for many years, before the need for surgery or other treatments develops. However, it is important to note that there is still much that we don't understand about this syndrome and FRAXA syndrome is only one possible phenotype of this syndrome and its presentation is only one part of a much larger phenotype.

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