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Growth Hormone Therapy

Voxzogo for Mucopolysaccharidosis

Phase 1 & 2
Recruiting
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

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 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

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

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.

Find a Location

Who is running the clinical trial?

University of California, San FranciscoLead Sponsor
2,570 Previous Clinical Trials
15,175,704 Total Patients Enrolled
UCSF Benioff Children's Hospital OaklandLead Sponsor
79 Previous Clinical Trials
19,045 Total Patients Enrolled
Paul Harmatz, MDPrincipal InvestigatorUniversity of California, San Francisco
5 Previous Clinical Trials
54 Total Patients Enrolled

Media Library

Vosoritide (Growth Hormone Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05845749 — Phase 1 & 2
Mucopolysaccharidosis Research Study Groups: Vosoritide
Mucopolysaccharidosis Clinical Trial 2023: Vosoritide Highlights & Side Effects. Trial Name: NCT05845749 — Phase 1 & 2
Vosoritide (Growth Hormone Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05845749 — Phase 1 & 2
~3 spots leftby Dec 2025