Mucopolysaccharidosis Type IIIA study: DNL126
Official title Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
ClinicalTrials.gov ID: NCT06181136
What this study is testing
What is DNL126?
DNL126 is an investigational medicine, given as an IV infusion (through a vein), being studied as a potential treatment for mucopolysaccharidosis type iiia.
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
- This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). In Part 1, the core study period is 25 weeks (approximately 6 months); followed by an open-label extension (OLE), which extends through Week 97 (approximately 18 months); and a long-term extension (LTE), which extends through Week 241 (Year 5).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 18
You may be able to join if
- Confirmed diagnosis of MPS IIIA
- For Cohorts A2 and C1: No more than 1 participant may have predictors of a slow-progressing phenotype
- For Cohort A3: Approximately 2 participants will have predictors of the slow-progressing phenotype
- An older sibling with the same genotype and severe MPS IIIA, in the opinion of the investigator
- A definitive genotype indicative of severe MPS IIIA, in the opinion of the investigator
You likely can't join if
- Have unstable or poorly controlled medical condition(s) or significant medical or psychological comorbidity or comorbidities that, in the opinion of...
- Have lost the ability to walk independently, in the opinion of the investigator
- Are unable to take the majority of nutrition via mouth, in the opinion of the investigator
- For Cohort B only: Are homozygous or compound heterozygous for the N-sulfoglucosamine sulfohydrolase (SGSH) S298P mutation or any other mutation...
- Have used any CNS-targeted MPS IIIA enzyme replacement therapy (ERT) (eg, intrathecal SGSH or TfR-mediated SGSH delivery to CNS) within 3 months...
- Have a prior history of hematopoietic stem cell transplantation
See the full eligibility criteria
- Confirmed diagnosis of MPS IIIA
- For Cohorts A2 and C1: No more than 1 participant may have predictors of a slow-progressing phenotype
- For Cohort A3: Approximately 2 participants will have predictors of the slow-progressing phenotype
- An older sibling with the same genotype and severe MPS IIIA, in the opinion of the investigator
- A definitive genotype indicative of severe MPS IIIA, in the opinion of the investigator
- Clinical symptoms of MPS IIIA prior to 28 months of age that, in the opinion of the investigator, are indicative of severe MPS IIIA
- For Cohort B2: Are an older sibling of a participant in Cohort B1 (who has already been confirmed to be eligible for dosing) with MPS IIIA, the same causative genotype, and who has severe MPS IIIA in the opinion of the...
- For Cohort C1: Have up-to-date age-specific vaccination status, including SARS-CoV-2 (COVID-19) vaccines, according to country-specific guidelines at the time of the screening visit. Key
- Have unstable or poorly controlled medical condition(s) or significant medical or psychological comorbidity or comorbidities that, in the opinion of the investigator, would interfere with safe participation in the trial...
- Have lost the ability to walk independently, in the opinion of the investigator
- Are unable to take the majority of nutrition via mouth, in the opinion of the investigator
- For Cohort B only: Are homozygous or compound heterozygous for the N-sulfoglucosamine sulfohydrolase (SGSH) S298P mutation or any other mutation known to be associated with slow-progressing phenotype
- Have used any CNS-targeted MPS IIIA enzyme replacement therapy (ERT) (eg, intrathecal SGSH or TfR-mediated SGSH delivery to CNS) within 3 months before Day 1
- Have a prior history of hematopoietic stem cell transplantation
- Have a prior history of gene therapy
- Have used genistein or anakinra within 7 days of screening or intended use of genistein or anakinra during the study
- Have a documented likely pathogenic mutation sufficient to cause disease (eg, taking into account zygosity) of other genes that are known to be associated with developmental delay, seizures, or other significant CNS...
- Have clinically significant thrombocytopenia, other clinically significant coagulation abnormality, significant active bleeding, or require treatment with an anticoagulant or more than two antiplatelet agents
- Contraindication for lumbar punctures
- Contraindication for MRI scan
- Have a clinically significant history of stroke, status epilepticus, head trauma with loss of consciousness, or any clinically significant CNS disease that is not MPS IIIA-related within 3 months of screening
- Have had a ventriculoperitoneal (VP) shunt placed or a clinically significant VP shunt malfunction within 30 days of screening
- Have any clinically significant CNS trauma or disorder, including severe untreated intracranial hypertension or brain surgery, that, in the opinion of the investigator, may interfere with assessment of study endpoints...
The study team makes the final eligibility decision.
Where it is enrolling
- Oakland, California, United States
- Iowa City, Iowa, United States
- Chapel Hill, North Carolina, United States
- Houston, Texas, 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, but 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, 0 years to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Oakland, California, United States; Iowa City, Iowa, United States; Chapel Hill, North Carolina, United States; Houston, Texas, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.