Ambroxol for Sanfilippo Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores the safety and effectiveness of Ambroxol Hydrochloride for treating adults with Sanfilippo syndrome, a rare genetic disorder affecting the brain and body. It tests various doses of Ambroxol to determine the optimal balance between safety and benefits. Ideal participants have a confirmed diagnosis of Sanfilippo syndrome and can take oral medication. As a Phase 2, Phase 3 trial, this research evaluates the treatment's effectiveness in a smaller group and represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking therapy.
Will I have to stop taking my current medications?
The trial requires that you stop taking medications that are strong inducers or inhibitors of CYP3A, as well as genistein or Miglustat, before starting the trial. If you are on these medications, you may need to switch to alternatives that do not interfere with the trial.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that Ambroxol is generally safe and well-tolerated. It often treats breathing problems in both adults and children, with most side effects being mild. Studies have demonstrated that Ambroxol can be safely used at high doses, and trials have confirmed its safety. This suggests that Ambroxol is likely safe for treating Sanfilippo syndrome, although researchers are still studying the effects of higher doses for this specific condition.12345
Why do researchers think this study treatment might be promising for Sanfilippo syndrome?
Researchers are excited about using Ambroxol Hydrochloride for Sanfilippo Syndrome because it introduces a novel approach to treating this rare genetic disorder. Unlike existing treatments that primarily focus on managing symptoms, Ambroxol Hydrochloride acts on cellular pathways to enhance lysosomal function, potentially addressing the root cause of the disease. This drug is being tested at different doses, which could lead to a more personalized treatment strategy and improved outcomes for patients. The excitement stems from the possibility that this treatment could significantly alter disease progression, offering new hope for families affected by Sanfilippo Syndrome.
What evidence suggests that Ambroxol Hydrochloride might be an effective treatment for Sanfilippo Syndrome?
Research suggests that Ambroxol Hydrochloride might help people with Sanfilippo syndrome. Animal studies have shown that Ambroxol could reduce the buildup of harmful substances in cells, a major issue in Sanfilippo syndrome. These early findings indicate that Ambroxol might improve cellular function and possibly slow disease progression. Previous patients have generally tolerated Ambroxol well, which is encouraging for its use in treating this condition. Although more research is needed in humans, Ambroxol offers hope as a potential treatment for Sanfilippo syndrome. Participants in this trial will receive different dosages of Ambroxol Hydrochloride to evaluate its effectiveness and safety.14567
Who Is on the Research Team?
Ozlem Goker-Alpan, MD
Principal Investigator
Lysosomal & Rare Disorders Research & Treatment Center, Inc.
Are You a Good Fit for This Trial?
This trial is for adults with Sanfilippo disease, also known as Mucopolysaccharidosis Type III (MPS III). Participants must be at least 18 years old. Specific inclusion and exclusion criteria are not listed but typically involve health status and history.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive Ambroxol with dose escalation over 52 weeks, including regular assessments and safety monitoring
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Ambroxol Hydrochloride
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ozlem Goker-Alpan
Lead Sponsor
Team Sanfilippo
Collaborator