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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the safety and tolerability of vosoritide (also known as Voxzogo), a daily injection for children with MPS IVA or VI, rare genetic conditions affecting growth and bone development. Researchers seek to determine if vosoritide can improve growth rates and bone health. The trial is recruiting children aged 5 to 10 who have been diagnosed with MPS IVA or VI, are currently on enzyme replacement therapy, and face specific growth challenges. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this potentially beneficial treatment.

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.

Is there any evidence suggesting that vosoritide is likely to be safe for humans?

Research has shown that vosoritide is usually safe for patients. In earlier studies, only 3% of patients experienced a temporary drop in blood pressure, sometimes causing symptoms like vomiting or dizziness. Vosoritide already has approval for some conditions, indicating its general safety. However, ongoing trials are crucial to confirm its safety for new uses. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for mucopolysaccharidosis focus on enzyme replacement therapies to manage symptoms. But vosoritide works differently, targeting the underlying causes of growth issues related to the condition. Researchers are excited because vosoritide, a C-type natriuretic peptide analog, promotes bone growth by directly stimulating the growth plates in bones, potentially addressing a critical aspect of the disease more effectively. Unlike enzyme treatments that address only certain symptoms, vosoritide offers a novel approach by aiming to improve overall bone growth and development.

What evidence suggests that vosoritide might be an effective treatment for MPS?

Research shows that vosoritide, the treatment under study in this trial for mucopolysaccharidosis (MPS), has the potential to aid growth in patients with similar conditions. In studies with children who have achondroplasia, vosoritide significantly increased growth rates. Specifically, children aged 5 to 15 demonstrated noticeable annual growth improvements. Additionally, the FDA has already approved vosoritide for promoting growth in children with achondroplasia, suggesting it might also be effective for MPS. This treatment mimics a natural protein that promotes bone growth, which could benefit those with MPS. ^{2 4 5 6 7}

Who Is on the Research Team?

Paul Harmatz, MD

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05845749

Safety and Efficacy of Voxzogo for Growth Deficits in MPS ... The primary study endpoint is the determination of safety and tolerability of daily vosoritide treatment in MPS. Exploratory endpoints include changes in linear ...

2. biomarin.com biomarin.com/en-au/voxzogo-efficacy-data/

VOXZOGO® - Efficacy Data - BioMarin Australia Statistically significant improvements in annualised growth velocity (AGV) were seen in patients aged 5 to 15 years.

3. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT05845749

Voxzogo for Growth Deficits in MPS IVA and VI Vosoritide, a CNP analog and recently approved FDA drug, has been shown to improve linear growth in patients with achondroplasia. This proposal ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10796712/

Vosoritide Therapy in Children with Achondroplasia Data from the same study support the efficacy observed in the vosoritide trial program, with 22 patients treated for 12 months obtaining an ...

5. hcp.biomarin.com hcp.biomarin.com/en-us/voxzogo/efficacy-data/

Efficacy | VOXZOGO® (vosoritide) HCP VOXZOGO is FDA approved to increase linear growth in children of all ages with achondroplasia and open growth plates.

6. hcp.biomarin.com hcp.biomarin.com/en-us/voxzogo/safety-data/

Safety | VOXZOGO® (vosoritide) HCP VOXZOGO safety data. 2 of 60 VOXZOGO-treated patients (3%) each had one symptomatic episode of blood-pressure decrease with vomiting and/or dizziness ...

7. voxzogo.com voxzogo.com/en-us/voxzogo-safety/

VOXZOGO® (vosoritide) | Safety & Side Effects VOXZOGO may cause serious side effects, including a temporary decrease in blood pressure in some children · You can reduce the risk of decreased blood pressure ...