Voxzogo for Mucopolysaccharidosis

Overseen By Leslie Lynch, MS

Age: < 18

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: University of California, San Francisco

Must be taking: ERT

Must not be taking: Growth hormones, ACE inhibitors

Disqualifiers: Growth hormone deficiency, Hypothyroidism, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 3

Duration: 48 weeks

6 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you have been on enzyme replacement therapy (ERT) for at least 12 months before joining. However, you cannot participate if you have taken certain medications like growth hormone therapy, ACE inhibitors, or beta-blockers in the past 6 months. The protocol does not specify if you need to stop other current medications, but you should discuss your specific situation with the study team.

What is the purpose of this trial?

This trial tests vosoritide in children aged 5-10 with severe growth issues from MPS IVA or VI. Vosoritide works by mimicking a natural protein that helps bones grow longer. Vosoritide has been previously tested in children with achondroplasia, showing positive effects on growth.

Research Team

Paul Harmatz, MD

Principal Investigator

University of California, San Francisco

Eligibility Criteria

This trial is for children aged 5 to under 10 with MPS IVA or VI, who are shorter than average and have been on ERT treatment for at least a year. They should be able to stand without support and their guardians must be capable of giving them daily injections. Kids can't join if they've had certain health issues like cancer, heart problems, untreated thyroid issues, or severe kidney problems.

Inclusion Criteria

I have been diagnosed with MPS IVA or VI.

You are at the earliest stage of physical development.

My MPS diagnosis is confirmed by genetic tests or enzyme deficiency.

Exclusion Criteria

I am not on medications that affect heart rhythm.

I have severe sleep apnea that hasn't been treated.

My BMI is below the 5th percentile, indicating malnutrition.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

24 weeks

Pre-treatment Observation

Subjects are followed to gather information on safety profiles and determine pre-treatment annualized growth velocity

24 weeks

Treatment

Participants receive daily subcutaneous vosoritide therapy at 15 ug/kg/day

48 weeks

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

Treatment Details

Interventions

• Vosoritide

Trial Overview The study tests Voxzogo (vosoritide) given as a daily shot for nearly a year in kids with growth deficits due to MPS IVA or VI. It aims to see how safe the drug is and how well it's tolerated while also looking at its effects on growth rates and bone health markers.

Participant Groups

Group I: Vosoritide Experimental Treatment 1 Intervention

This is a single arm open label study of daily SQ dose of vosoritide

Vosoritide is already approved in United States for the following indications:

Approved in United States as Voxzogo for:

• To increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses
• to increase linear growth in pediatric patients less than 5 years of age with achondroplasia with open epiphyses

Who Is Running the Clinical Trial?

University of California, San Francisco

Lead Sponsor

Trials

2,636

Recruited

19,080,000+

UCSF Benioff Children's Hospital Oakland

Lead Sponsor

Trials

80

Recruited

19,100+