What side effects are associated with this type of intervention?

Common treatments for Mucopolysaccharidosis (MPS) include enzyme replacement therapies (ERTs) such as laronidase, idursulfase, and galsulfase. These treatments often aim to reduce the accumulation of glycosaminoglycans and improve physical function. Known side effects of ERTs can include infusion-related reactions such as fever, rash, and headache, as well as more severe allergic reactions like anaphylaxis. For growth-promoting treatments similar to Vosoritide, potential side effects may include injection site reactions, hypotension, and increased risk of bone fractures. It is important to monitor patients closely for these adverse effects during treatment.

What prior FDA approvals exist?

The FDA has approved several treatments for Mucopolysaccharidosis (MPS), primarily focusing on enzyme replacement therapies (ERTs) to address the underlying enzyme deficiencies. These include laronidase (Aldurazyme) for MPS I, idursulfase (Elaprase) for MPS II, and galsulfase (Naglazyme) for MPS VI. While these treatments help manage the systemic symptoms of MPS, they do not specifically target bone growth or skeletal abnormalities. Vosoritide, a CNP analog promoting endochondral ossification and increasing bone growth, represents a novel approach in this context, although it is still under investigation for its efficacy and safety in MPS types IVA and VI.

What other types of research exist?

Promising alternatives to Vosoritide for MPS patients include enzyme replacement therapies (ERT) such as Elosulfase alfa for MPS IVA and Galsulfase for MPS VI, which address the underlying enzyme deficiencies. Gene therapies are also emerging as potential treatments, with several in clinical trials aiming to correct the genetic defects causing MPS. Additionally, substrate reduction therapies and small molecule chaperones are being investigated to enhance the efficacy of existing treatments.

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Growth Hormone Therapy

Voxzogo for Mucopolysaccharidosis

Phase 1 & 2

Waitlist Available

Led By Paul Harmatz, MD

Research Sponsored by University of California, San Francisco

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinical Diagnosis of MPS IVA or VI

MPS Diagnosis Confirmed by either: Demonstration of 2 pathogenic or likely pathogen mutations (or homozygous for single mutation) and elevated GAG (either before or during ERT treatment), OR Demonstration of diagnostic enzyme deficiency, elevated GAG (either before or during ERT treatment), and a normal second sulfatase

Must not have

Treated with medications known to affect QC/QTc

Untreated severe sleep apnea

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 72 weeks

Awards & highlights

No Placebo-Only Group

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While the trial primarily focuses on safety and tolerability, potential side effects aren't detailed here but may include reactions related to injection site, general body responses to new medications such as fatigue or nausea, and specific concerns related to hormone therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with MPS IVA or VI.

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

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My guardian can give me the study drug every day.

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I can stand by myself for at least one minute without help.

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I am between 5 and 9 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not on medications that affect heart rhythm.

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

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

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I have taken my enzyme replacement therapy less than 75% of the time in the last 6 months.

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I have undergone gene therapy in the past.

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I have had surgery to lengthen my bones or fix my spine.

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I have never received enzyme replacement therapy.

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I have been diagnosed with growth hormone deficiency.

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I am currently taking or have taken hormone therapy for cancer.

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I have a history of chronic kidney problems with low filtration rates.

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I have been on oral steroids for the past 6 months.

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I have previously been treated with or am currently on vosoritide.

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I am not allergic to vosoritide.

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I have had cancer before.

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I have a long-term inflammation condition not linked to MPS.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 72 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~72 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 72 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Safety and tolerability: Incidence of adverse events while treated with vosoritide

Secondary study objectives

Change in height velocity while treated with vosoritide

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: VosoritideExperimental Treatment1 Intervention

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

0 / 20Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Vosoritide, a C-type natriuretic peptide (CNP) analog, promotes endochondral ossification and increases bone growth, which is crucial for Mucopolysaccharidosis (MPS) patients. MPS patients often experience skeletal abnormalities and growth deficiencies due to the accumulation of glycosaminoglycans (GAGs) that interfere with normal bone development. By enhancing the signaling pathways that promote bone growth, treatments like Vosoritide can help mitigate skeletal complications, thereby improving the quality of life and physical function for MPS patients.