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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 between 18 and 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up tests will be done at week 1 and week 12.
Awards & highlights

Study Summary

This trial will study whether a drug is safe and effective in people with a rare disease called Sjögren-Larsson syndrome. The disease is caused by mutations in a gene and leads to the build-up of harmful fats in the body. The drug, ADX-629, is an experimental aldehyde trapping agent that is expected to eliminate these harmful fats. The primary objective of the trial is to determine whether ADX-629 is safe and tolerable for use in SLS subjects. The secondary objective is to determine the efficacy of ADX-629 in reversing the biochemical abnormalities in SLS.

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.Check my eligibility
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.See study design
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 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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
Biochemical efficacy of ADX-629 as determined by reversal of abnormal biomarkers
Other outcome measures
Exploratory clinical outcomes to determine drug dependent changes in abnormal clinical measures

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

Find a Location

Who is running the clinical trial?

William Rizzo, MDLead Sponsor
University of NebraskaLead Sponsor
539 Previous Clinical Trials
1,144,724 Total Patients Enrolled
1 Trials studying Sjogren-Larsson Syndrome
20 Patients Enrolled for Sjogren-Larsson Syndrome
William B Rizzo, MDPrincipal InvestigatorUniversity of Nebraska
2 Previous Clinical Trials
32 Total Patients Enrolled
1 Trials studying Sjogren-Larsson Syndrome
20 Patients Enrolled for Sjogren-Larsson Syndrome

Media Library

ADX-629 (Aldehyde trapping agent) Clinical Trial Eligibility Overview. Trial Name: NCT05443685 — Phase 1 & 2
Sjogren-Larsson Syndrome Research Study Groups: ADX-629 treatment
Sjogren-Larsson Syndrome Clinical Trial 2023: ADX-629 Highlights & Side Effects. Trial Name: NCT05443685 — Phase 1 & 2
ADX-629 (Aldehyde trapping agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05443685 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is it possible to partake in this clinical experiment?

"This clinical trial is hoping to enroll 10 people aged between 18 and 50 that have been diagnosed with sjogren-larsson syndrome. To be eligible, potential participants must provide written informed consent before any study procedures are initiated, weigh at least 35 kgs, demonstrate active ichthyosis and neurological symptoms of spasticity, abstain from all topical creams 7 days prior to initial baseline evaluation or the 12 week centre visit (if applicable), pass a negative pregnancy test if female of childbearing potential and adhere to medically approved contraceptive methods during the study duration or document being surgically sterile/postmenopausal; additionally they need to"

Answered by AI

How many participants are currently enrolled in this research experiment?

"Absolutely. According to clinicaltrials.gov, this medical trial began on the 2nd of January 2023 and was last updated on the 7th of November 2022. At present, only 10 participants are needed from a single site for enrollment."

Answered by AI

Is the enrollment for this trial still open to new participants?

"Clinicaltrials.gov reveals that this medical trial is open for recruitment, beginning from the initial post date of January 2nd 2023 and updated as recently as November 7th 2022."

Answered by AI

Does this research involve participants aged more than twenty?

"This research is targeting individuals aged 18 and under 50 who are willing to participate."

Answered by AI
~5 spots leftby Jun 2025