Mucopolysaccharidosis type 3A
MONDO:0009655A rare autosomal recessive lysosomal storage disease caused by deficiency of the enzyme heparan sulfate sulfatase. It is characterized by behavioral changes, sleep disturbances, mental developmental delays and seizures.
Also known as: MPS III A, MPS3A, MPSIIIA, Sanfilippo A, Sanfilippo syndrome a, Sanfilippo syndrome type A, heparan sulfamidase deficiency, mucopolysaccharidosis type 3A
10 clinical trials for this condition and its sub-types, 7 tagged with Mucopolysaccharidosis type 3A itself.
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Can a direct brain infusion slow sanfilippo syndrome?
Disease control OngoingThis trial tests an experimental enzyme replacement called GC1130A in children with Sanfilippo syndrome type A, a rare genetic disease that damages the brain. The enzyme is given through a small device placed under the scalp that delivers it directly into the fluid around the bra…
Phase 1 • Sponsor: GC Biopharma Corp • Aim: Disease control
Last updated Sep 10, 2026 00:00 UTC
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Can a weekly infusion reach the brain to fight a rare childhood disease?
Disease control OngoingThis trial tests an experimental drug called JR-441 in children with mucopolysaccharidosis type IIIA (MPS IIIA), a rare genetic condition that harms the brain and body. Researchers give the drug as a weekly intravenous infusion to see if it is safe and tolerable. They also measur…
Phase 1/2 • Sponsor: JCR Pharmaceuticals Co., Ltd. • Aim: Disease control
Last updated Sep 06, 2026 00:00 UTC
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Gene therapy for sanfilippo a: does it last?
Disease control By invitation onlyThis study follows 41 children with Sanfilippo A (MPS IIIA) who previously received UX111 gene therapy in earlier trials. Researchers will monitor safety and how well the therapy controls the disease over time, using tests like the Bayley cognitive scale. No new gene therapy is g…
Phase 3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jul 19, 2026 00:00 UTC
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New hope for kids with rare brain disease: drug targets toxic buildup
Disease control OngoingThis study tests a drug called DNL126 in 20 children with Sanfilippo syndrome type A, a rare genetic disorder that causes brain damage. The drug is given through a vein and aims to reduce harmful substances in the brain and body. The trial lasts about 6 months, with options to co…
Phase 1/2 • Sponsor: Denali Therapeutics Inc. • Aim: Disease control
Last updated Jul 15, 2026 00:00 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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New study monitors heart risks in rare genetic disease
Knowledge-focused OngoingThis study follows 30 people with mucopolysaccharidoses (MPS) over three years to see how their heart and arteries change. Researchers use neck ultrasounds and blood tests to measure artery thickness, stiffness, and signs of inflammation. The goal is to better understand cardiova…
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC