What is the mechanism of action of this treatment?

The most common treatment for Sjogren-Larsson Syndrome (SLS) being studied is ADX-629, an aldehyde trapping agent. This treatment works by eliminating harmful long-chain fatty aldehydes that accumulate due to deficient activity of fatty aldehyde dehydrogenase (FALDH) caused by mutations in the ALDH3A2 gene. By trapping and neutralizing these toxic aldehydes, ADX-629 aims to reduce biochemical abnormalities and alleviate symptoms in the skin, brain, and eyes. This mechanism is crucial for SLS patients as it directly targets the underlying cause of the disease, potentially improving their quality of life by addressing the root biochemical disruptions.

What side effects are associated with this type of intervention?

The most common treatments for Sjogren-Larsson Syndrome (SLS) include aldehyde trapping agents like ADX-629, which aim to eliminate harmful fatty aldehydes. While specific side effects of ADX-629 are not detailed, similar treatments may cause gastrointestinal disturbances, headaches, and potential allergic reactions. It is crucial to monitor patients for any adverse effects during treatment to ensure safety and efficacy.

What prior FDA approvals exist?

There are no specific FDA-approved treatments for Sjogren-Larsson Syndrome (SLS) mentioned in the provided context. ADX-629, an aldehyde trapping agent, is currently being studied for its safety and efficacy in treating SLS by eliminating fatty aldehydes and reducing aldehyde toxicity. This approach aims to address the biochemical abnormalities caused by the disease. Other treatments for similar metabolic disorders or symptoms may include enzyme replacement therapies or other biochemical modulation strategies, but these are not specified for SLS in the context provided.

What other types of research exist?

Currently, there are no specific novel alternatives to ADX-629 mentioned for the treatment of Sjogren-Larsson Syndrome in the provided context. Patients should consult with their healthcare provider to explore other potential therapies that target lipid metabolism or aldehyde toxicity.

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Aldehyde trapping agent

ADX-629 for Sjogren-Larsson Syndrome

Phase 1 & 2

Recruiting

Led By William B Rizzo, MD

Research Sponsored by William Rizzo, MD

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up tests will be done at week 1 and week 12.

Awards & highlights

No Placebo-Only Group

Summary

This trial tests if ADX-629, a medicine taken by mouth, is safe and effective for people with Sjögren-Larsson syndrome (SLS). SLS is a rare genetic disorder that causes harmful fats to build up in the body. ADX-629 helps remove these harmful fats, which may reduce symptoms in the skin, brain, and eyes.

Who is the study for?

Adults aged 18-50 with Sjogren-Larsson Syndrome (SLS), weighing at least 35 kg, able to swallow pills and follow the study plan. Participants must have ichthyosis, spasticity, a genetic confirmation of SLS, and agree to contraception if sexually active. Excluded are those in other studies, with certain health conditions or treatments that could affect results or safety.

What is being tested?

The trial is testing ADX-629's safety and effectiveness for treating SLS when taken orally for 12 weeks. It aims to reduce harmful fatty aldehydes believed to cause symptoms by trapping these molecules. The drug's impact on skin, neurological function, and eye health will also be explored.

What are the potential side effects?

While specific side effects aren't listed here, participants will be closely monitored for any adverse reactions due to ADX-629 throughout the trial period as it tests both safety and biochemical efficacy.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ monthly for 12 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~monthly for 12 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and monthly for 12 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Compliance and tolerability of ADX-629

Number of participants with abnormal drug-related safety blood tests

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Secondary study objectives

Biochemical efficacy of ADX-629 as determined by reversal of abnormal biomarkers

Other study objectives

Exploratory clinical outcomes to determine drug dependent changes in abnormal clinical measures

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: ADX-629 treatmentExperimental Treatment1 Intervention

Open label treatment with ADX-629

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ADX-629

2021

Completed Phase 2

~110

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatment for Sjogren-Larsson Syndrome (SLS) being studied is ADX-629, an aldehyde trapping agent. This treatment works by eliminating harmful long-chain fatty aldehydes that accumulate due to deficient activity of fatty aldehyde dehydrogenase (FALDH) caused by mutations in the ALDH3A2 gene. By trapping and neutralizing these toxic aldehydes, ADX-629 aims to reduce biochemical abnormalities and alleviate symptoms in the skin, brain, and eyes. This mechanism is crucial for SLS patients as it directly targets the underlying cause of the disease, potentially improving their quality of life by addressing the root biochemical disruptions.

Safety, pharmacokinetics and sialic acid production after oral administration of N-acetylmannosamine (ManNAc) to subjects with GNE myopathy.Fabry's disease presenting as syncope, angiokeratomas, and spoke-like cataracts in a young man: discussion of the differential diagnosis.Response to treatment in hereditary metabolic disease: 1993 survey and 10-year comparison.