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Tests treatment safety and results for Sanfilippo Syndrome
Official title An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III
ClinicalTrials.gov ID: NCT06614894
What this study is testing
What is Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day?
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for sanfilippo syndrome.
Also referred to as Ambroxol.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- A dose escalation study to evaluate the safety, tolerability, and pharmacologic properties of Ambroxol in adult participants with Sanfilippo disease(s) (MPS3).
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 99
You may be able to join if
- IRB - approved informed consent/assent signed by subject and/or parent(s) or legal guardian(s).
- Genetically confirmed diagnosis of MPS III disease.
- Genomic DNA analysis demonstrating a homozygous or compound heterozygous pathogenic variants in SGSH (type A), NAGLU (type B), HGSNAT (type C), or...
- Elevated excretion of urinary GAGs and/or serum HS (if no historical data is available, screening GAGs and serum HS values will be utilized to assess...
- Male or female; eighteen years of age and older, who is able to take Ambroxol Hydrochloride orally.
You likely can't join if
- Unwilling or unable to follow protocol requirements as per principal investigator.
- Any serious or chronic medical illness, including significant cardiac or severe debilitating pulmonary disease.
- Poorly controlled seizures, defined as more than one seizure per day for the past 6 months.
- Medications identified as a strong inducers or inhibitors of CYP3A, and changing to another alternative drug to treat the condition would place the...
- Any medical condition that, in the opinion of the PI, would make the subject unsuitable to participate in the study.
- Inability to cooperate for clinical and safety data collection.
See the full eligibility criteria
- IRB - approved informed consent/assent signed by subject and/or parent(s) or legal guardian(s).
- Genetically confirmed diagnosis of MPS III disease.
- Genomic DNA analysis demonstrating a homozygous or compound heterozygous pathogenic variants in SGSH (type A), NAGLU (type B), HGSNAT (type C), or GNS (type D) genes. Type E will not be studied.
- Elevated excretion of urinary GAGs and/or serum HS (if no historical data is available, screening GAGs and serum HS values will be utilized to assess ).
- Male or female; eighteen years of age and older, who is able to take Ambroxol Hydrochloride orally.
- Negative urine pregnancy test at screening for female people with child-bearing potential.
- The subject is willing to abstain from consumption of grapefruit, grapefruit juice, or grapefruit containing products for 72 hours prior to administration of the first dose of Ambroxol and for the duration of the...
- Unwilling or unable to follow protocol requirements as per principal investigator.
- Any serious or chronic medical illness, including significant cardiac or severe debilitating pulmonary disease.
- Poorly controlled seizures, defined as more than one seizure per day for the past 6 months.
- Medications identified as a strong inducers or inhibitors of CYP3A, and changing to another alternative drug to treat the condition would place the subject at undue risk.
- Any medical condition that, in the opinion of the PI, would make the subject unsuitable to participate in the study.
- Inability to cooperate for clinical and safety data collection.
- Known hypersensitivity to Ambroxol or any of its excipients.
- Use of genistein or Miglustat within one week of starting screening.
- Evidence of hepatitis B or hepatitis C infection upon serological testing at screening.
- Currently participating in another clinical trial or has completed an treatment trial less than 2 weeks prior to screening visit.
- The subject has received strong inducers (Note: eg, herbal supplements) or inhibitors of CYP3A within 15 days or 5 half-lives from screening, whichever is longer, prior to enrollment. This also includes the consumption...
The study team makes the final eligibility decision.
Where it's taking place
- Fairfax, Virginia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 99 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Fairfax, Virginia, United States. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.