MPS IVAPhase 1/2NCT05845749
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
A Phase 1/2 study testing Vosoritide Injection [Voxzogo] in people with MPS IVA and MPS VI, run by University of California, San Francisco at 1 site in United States.
Trial summary
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI. Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety profiles and… Read more about this study
- Who is it for?
- Ages 5 to 10 years
- What is being tested?
- Vosoritide Injection [Voxzogo]
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Sep 2023to 31 Dec 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age >= 5 years and < 10 years
- Tanner stage 1
- Clinical Diagnosis of MPS IVA or VI
- Subjects will be stratified into 2 groups:
- MPS IVA (3 patients)
- MPS VI (3 patients)
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- 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
- Currently receiving ERT [elosulfase alfa (Vimizim®) or galsulfase (NAGLAZYME®)] for minimum of 12 months prior to study entry
- HSCT greater than 3 years before entry
- Height Z-score <-2.0 or less than 2 cm change in height velocity over the last 1 year
- Willing to consent to the study and comply with all study procedures and assessments
- Able to stand independently without hand support for minimum of one minute
- Guardians able to successfully administer investigational drug daily/SQ
Who cannot join
- ERT naïve
- Poor compliance with ERT (<75% in 6 month period)
- Diagnosis with growth hormone deficiency (defined by IGF-1 SDS <-1.0 according to age, gender and tanner stage)
- Hypothyroidism, untreated (TSH >4.0 mU/L)
- Receiving or has received growth hormone therapy, IGF-1 therapy, anti-TNF alpha therapy, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium channel blockers, cardiac glycosides, systemic anticholinergic agents, any medication that may impair or enhance compensatory tachycardia, diuretics or other drugs known to alter renal or tubular function within the previous 6 months.
- Receiving or has previously received a GnRH analog (e.g. leuprolide acetate, histrelin)
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- History of malignancy
- History of chronic inflammatory condition not related to MPS
- History of conditions/medical therapies that might affect the interpretation of growth results such as anemia, celiac disease, diabetes, inflammatory bowel disease, and cystic fibrosis
- QTC (Fridericia) > 450 msec
- Malnutrition (BMI <5th percentile)
- History of gene therapy
- Concurrent participation on an investigational drug trial
- Investigational drug washout minimum of 5 half-lives of the drug or 1 month whichever is longer
- Previous or current treatment with the investigational drug (vosoritide)
- Known or suspected allergy to the investigational drug (vosoritide)
- Bone fracture within the previous 6 months
- Skeletal surgery within the previous 6 months, or anticipated significant surgery (in the view of the investigator) during course of the study
- Any history of bone lengthening surgeries or spine fixation surgery
- Spine curvature (scoliosis) on previous x-ray greater than 25 degrees
- Untreated severe sleep apnea
- History of chronic renal insufficiency, defined previously as an eGFR <60 mL/min/1.73m2
- Illness that could affect blood pressure / orthostatic problems
- Treated with medications known to affect QC/QTc
- LV Ejection fraction <40%; LVEF=[SV/EDV] x100 (American Society Echocardiography)
- Treated with chronic oral steroids in previous 6 months
- Mean SpO2 of < 92% at baseline, taken from average of 3 measurements in each hand
- Concurrent disease or condition that in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
Trial locations and contacts
1 site in 1 country.
United States1 site
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
About this study
Official title: A Proof of Concept Study to Evaluate the Safety and Efficacy of Voxzogo (Vosoritide) for the Treatment of Growth Deficits in MPS IVA and VI
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI. Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety profiles and determine annualized growth velocity. The primary study endpoint is the determination of safety and tolerability of daily vosoritide treatment in MPS. Exploratory endpoints include changes in linear and segmental growth as well as biomarkers of growth and bone metabolism.
Read the detailed description
The investigators propose to conduct a single arm phase I/II study of Vosoritide (also called VOXZOGO® and BMN111) in 6 pediatric patients with mucopolysaccaridosis (MPS) types IVA and VI; 3 patients with each disease. This will be a single center study performed at UCSF Children's Hospital, Oakland, under the direction of Dr. Paul Harmatz, Professor in Residence in the Department of Pediatric Gastroenterology.
Mucopolysaccharidoses (MPS) are a group of ultra rare genetic lysosomal storage diseases caused by deficiency in various enzymes responsible for the breakdown of glycosaminoglycans (GAGs), leading to progressive accumulations of GAGs in the tissues and organs. Patients with MPS have severe growth deficits and growth-related decreased quality of life. In this study, the MPS disorders which have the most severe growth deficits will be the focus, MPS IVA and VI.
Enzyme replacement therapies (ERT) have been developed and approved for use in MPS. Though ERT has improved functional outcomes it does not lead to complete reversal of disease progression. Patients maintained on ERT continue to experience significant growth deficits.
Vosoritide, a CNP analog and recently approved FDA drug, has been shown to improve linear growth in patients with achondroplasia.
This proposal is for a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI. Subjects will be included if they are > 5 years and < 10 years, Tanner pubertal stage 1 with a height Z-score of <-2.0 or less than 2 cm change in height velocity over the year prior to screening. Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety profiles and determine pre-treatment (baseline) annualized growth velocity. The primary study endpoint is the determination of safety and tolerability of daily vosoritide treatment in MPS. Segmental growth, other functional assessments, inflammation, and bone/collagen markers, as well as quality of life will also be assessed.
- Being tested
- Vosoritide Injection [Voxzogo]
- Keywords
- Growth, Vosoritide, Children, Height, Voxzogo
- Medical subject headings
- Mucopolysaccharidosis IV, Mucopolysaccharidosis VI
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vosoritide Injection [Voxzogo]Drug
Also called Vosoritide, BMN111
Vosoritide will be given via a once daily subcutaneous injection at a dose of 15 ug/kg/day, at approximately the same time each day when feasible. Vosoritide will be supplied to the subject as 0.4 mg vial, 0.56 mg vial or 1.2 mg vials to be reconstituted with sterile water up to 0.8 mg/mL or 2 mg/mL concentrations for injection. The volume to be administered (injection volume) will be based on the subject's body weight and the concentration of vosoritide. All supplies will be provided to the subject for home based administration after training at the study site.
| Study group | Role | What they receive |
|---|---|---|
| Vosoritide | Other | This is a single arm open label study of daily SQ dose of vosoritide Receives: Drug: Vosoritide Injection [Voxzogo] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability: Incidence of adverse events while treated with vosoritide
Incidence of treatment-emergent adverse events as assessed by the evaluation of vital signs, pulse oximetry, pulmonary function, ECG (cardiac arrhythmia), ECHO (doppler of aortic velocity for stenosis, aortic valve area, and qualitative assessment of aortic valve thickness), spinal X-rays (worsening scoliosis, lordosis or kyphosis), standing lower extremity X-rays (worsening of genu valgum), decrease in six-minute walk distance and linear and segmental growth for determination of excessive or disproportionate growth. All safety assessments will be performed at a minimum at the beginning of the intervention (Visit 1) and the end of the intervention (Visit 3) in patients with MPS IVA and VI
Measured: 48 weeks
Secondary outcomes (1)
Change in height velocity while treated with vosoritide
Explore the change from baseline (0-24 weeks pre-intervention) in age-sex annualized height velocity after 96 weeks of daily subcutaneous vosoritide therapy in patients with MPS IVA and VI
Measured: 120 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2023
- Study start25 Sep 2023
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MPS IVAStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a 24 week natural history study followed by a 96 week open label single arm intervention with vosoritide
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Paul Harmatz, MD, University of California, San Francisco
- Investigators
- Ellen B Fung, PhD, Principal investigator, University of California, San Francisco
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05845749
- Sponsor's protocol ID
- 22-37966
- Status last verified
- Mar 2026
- First submitted
- 14 Apr 2023
- Last updated
- 10 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Proof of Concept Study to Evaluate the Safety and Efficacy of Voxzogo (Vosoritide) for the Treatment of Growth Deficits in MPS IVA and VI
- ClinicalTrials.gov ID
- NCT05845749
- Acronym / sponsor ID
- 22-37966
- Keywords
- Growth, Vosoritide, Children, Height, Voxzogo
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 6 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Study start
- 25 Sep 2023 (actual)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 6 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI. Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety profiles and determine annualized growth velocity. The primary study endpoint is the determination of safety and tolerability of daily vosoritide treatment in MPS. Exploratory endpoints include changes in linear and segmental growth as well as biomarkers of growth and bone metabolism.
Detailed description
The investigators propose to conduct a single arm phase I/II study of Vosoritide (also called VOXZOGO® and BMN111) in 6 pediatric patients with mucopolysaccaridosis (MPS) types IVA and VI; 3 patients with each disease. This will be a single center study performed at UCSF Children's Hospital, Oakland, under the direction of Dr. Paul Harmatz, Professor in Residence in the Department of Pediatric Gastroenterology.
Mucopolysaccharidoses (MPS) are a group of ultra rare genetic lysosomal storage diseases caused by deficiency in various enzymes responsible for the breakdown of glycosaminoglycans (GAGs), leading to progressive accumulations of GAGs in the tissues and organs. Patients with MPS have severe growth deficits and growth-related decreased quality of life. In this study, the MPS disorders which have the most severe growth deficits will be the focus, MPS IVA and VI.
Enzyme replacement therapies (ERT) have been developed and approved for use in MPS. Though ERT has improved functional outcomes it does not lead to complete reversal of disease progression. Patients maintained on ERT continue to experience significant growth deficits.
Vosoritide, a CNP analog and recently approved FDA drug, has been shown to improve linear growth in patients with achondroplasia.
This proposal is for a Phase I/II, single arm, open label study of vosoritide therapy provided subcutaneously at 15 ug/kg/day for 96 weeks to 6 patients with MPS IVA or VI. Subjects will be included if they are > 5 years and < 10 years, Tanner pubertal stage 1 with a height Z-score of <-2.0 or less than 2 cm change in height velocity over the year prior to screening. Prior to enrollment in the interventional arm of study, subjects will be followed for a minimum of 24 weeks to gather information on safety profiles and determine pre-treatment (baseline) annualized growth velocity. The primary study endpoint is the determination of safety and tolerability of daily vosoritide treatment in MPS. Segmental growth, other functional assessments, inflammation, and bone/collagen markers, as well as quality of life will also be assessed.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a 24 week natural history study followed by a 96 week open label single arm intervention with vosoritide
| Arm | Type | Receives |
|---|---|---|
| Vosoritide | Other | Drug: Vosoritide Injection [Voxzogo]This is a single arm open label study of daily SQ dose of vosoritide |
Who may qualify
- Ages
- 5 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (15 listed)
- Age >= 5 years and < 10 years
- Tanner stage 1
- Clinical Diagnosis of MPS IVA or VI
- MPS IVA (3 patients)
- MPS VI (3 patients)
Key exclusion criteria (28 listed)
- ERT naïve
- Poor compliance with ERT (<75% in 6 month period)
- Diagnosis with growth hormone deficiency (defined by IGF-1 SDS <-1.0 according to age, gender and tanner stage)
- Hypothyroidism, untreated (TSH >4.0 mU/L)
- Receiving or has received growth hormone therapy, IGF-1 therapy, anti-TNF alpha therapy, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium channel blockers, cardiac glycosides, systemic anticholinergic agents, any medication that may impair or enhance compensatory tachycardia, diuretics or other drugs known to alter renal or tubular function within the previous 6 months.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vosoritide Injection [Voxzogo]Also: Vosoritide, BMN111Vosoritide will be given via a once daily subcutaneous injection at a dose of 15 ug/kg/day, at approximately the same time each day when feasible. Vosoritide will be supplied to the subject as 0.4 mg vial, 0.56 mg vial or 1.2 mg vials to be reconstituted with sterile water up to 0.8 mg/mL or 2 mg/mL concentrations for injection. The volume to be administered (injection volume) will be based on the subject's body weight and the concentration of vosoritide. All supplies will be provided to the subject for home based administration after training at the study site. | Drug | Vosoritide |
Primary outcome
Safety and tolerability: Incidence of adverse events while treated with vosoritide
Time frame: 48 weeks
Incidence of treatment-emergent adverse events as assessed by the evaluation of vital signs, pulse oximetry, pulmonary function, ECG (cardiac arrhythmia), ECHO (doppler of aortic velocity for stenosis, aortic valve area, and qualitative assessment of aortic valve thickness), spinal X-rays (worsening scoliosis, lordosis or kyphosis), standing lower extremity X-rays (worsening of genu valgum), decrease in six-minute walk distance and linear and segmental growth for determination of excessive or disproportionate growth. All safety assessments will be performed at a minimum at the beginning of the intervention (Visit 1) and the end of the intervention (Visit 3) in patients with MPS IVA and VI
1 secondary outcome
Change in height velocity while treated with vosoritide
Time frame: 120 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2026.
- Individual participant dataSharing planned: No
Related trials for MPS IVA
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Non-invasive Functional Assessment and Pathogenesis of Morquio A
Mucopolysaccharidosis IV Type A, Morquio A Syndrome
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ellen B Fung, PhD principal investigator
University of California, San Francisco
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Vosoritide Injection [Voxzogo] in people with MPS IVA and MPS VI, run by University of California, San Francisco at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 28 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 10 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability: Incidence of adverse events while treated with vosoritide (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2026; AzTrials last refreshed its copy on 8 Nov 2026.