Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Part of paid clinical trials in Columbus, Ohio.

Sponsor
Ultragenyx Pharmaceutical Inc
Study ID
NCT02716246
Phase
PHASE2/PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • MPS IIIA
  • Mucopolysaccharidosis III
  • Sanfilippo A
  • Sanfilippo Syndrome

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • UX111 — BIOLOGICAL
    Self-complementary adeno-associated virus serotype 9 carrying the human SGSH gene under the control of a U1a promoter (scAAV9.U1a.hSGSH) will be delivered one time through a venous catheter inserted into a peripheral limb vein.
  • Prophylactic Immunomodulatory (IM) Therapy — DRUG
  • Optimized Prophylactic IM Therapy — DRUG
  • Adjuvant IM Therapy — DRUG
    The Principal Investigator and/or caregiver, in consultation with the medical monitor, will determine whether to initiate adjuvant IM therapy. Not all participants may receive adjuvant IM therapy.

Study Details

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

Key Dates

Start date
Apr 25, 2016
Status verified
Jun 2026
Primary completion
Mar 31, 2029
Completion
Mar 31, 2029

Study Design

Enrollment
36 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1 Low Dose
    Dose of 0.5 X 10\^13 vg/kg
  • Experimental: Cohort 2 Mid Dose
    Dose of 1 X 10\^13 vg/kg
  • Experimental: Cohort 3 High Dose
    Dose of 3 X 10\^13 vg/kg
  • Experimental: Cohort 4 High Dose (Spain Only)
    Dose of 3 X 10\^13 vg/kg

Primary Outcome Measure

Cerebrospinal Fluid (CSF) Heparan Sulfate (HS) (Disaccharide) Exposure [ Time Frame: Up to Month 24 Visit ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Nationwide Children's HospitalColumbusOhio43205-
Children's Hospital of PittsburghPittsburghPennsylvania15224-

Find similar trials in Columbus, OH

By research site
Nationwide Children's Hospital· Columbus, OHChildren's Hospital of Pittsburgh· Pittsburgh, PA

Related Studies