Mucopolysaccharidosis type 3
MONDO:0018937A lysosomal disease characterized by progressive neurocognitive decline, severe intellectual deterioration, loss of functional abilities, and premature death.
Also known as: MPS3, MPSIII, Mucopoly-saccharidosis type 3, Mucopolysaccharidosis Type III, Sanfilippo disease, Sanfilippo syndrome, heparan sulphate sulfatase deficiency, mucopolysaccharidosis type III
21 clinical trials for this condition and its sub-types, 7 tagged with Mucopolysaccharidosis type 3 itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Mucopolysaccharidosis type 3
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Mucopolysaccharidosis type 3A 7 trials
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Mucopolysaccharidosis type 3B 6 trials
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Mucopolysaccharidosis type 3C 2 trials
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Mucopolysaccharidosis type 3D 0 trials
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Experimental cell shot aims to boost brain repair in kids with rare metabolic diseases
Disease control Recruiting nowThis early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. T…
Phase 1 • Sponsor: Joanne Kurtzberg, MD • Aim: Disease control
Last updated Sep 05, 2026 00:00 UTC
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One-Time gene therapy aims to halt fatal brain disease in children
Disease control Recruiting nowThis trial tests a one-time gene therapy called UX111 for children with Sanfilippo A, a rare genetic disorder that causes severe brain damage. The therapy delivers a working copy of the missing gene to cells. Researchers will measure whether it reduces harmful substances in the b…
Phase 2/3 • Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Aug 23, 2026 00:00 UTC
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CBD trial aims to ease sanfilippo syndrome symptoms
Symptom relief Recruiting nowThis study tests whether cannabidiol (CBD) can safely improve behavior, mood, sleep, and daily function in people with Sanfilippo syndrome, a rare genetic disorder. Thirty-five participants will receive either CBD or a placebo, then switch after a break. Caregivers will report on…
Phase 2/3 • Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center • Aim: Symptom relief
Last updated Sep 17, 2026 00:00 UTC
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Massive french study aims to unlock secrets of rare MPS diseases
Knowledge-focused Recruiting nowThis observational study will follow up to 1,000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers will collect medical data from patient records and ongoing checkups to map how the diseases progress and how current treatments affec…
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC