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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 72 weeks
Awards & highlights
Study Summary
This trial is testing a medication for MPS to assess its safety and how it affects growth and bone metabolism.
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.Check my eligibility
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.See study design
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 belowSelect...
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.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 72 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~72 weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Safety and tolerability: Incidence of adverse events while treated with vosoritide
Secondary outcome measures
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
Find a Location
Who is running the clinical trial?
University of California, San FranciscoLead Sponsor
2,507 Previous Clinical Trials
15,238,569 Total Patients Enrolled
UCSF Benioff Children's Hospital OaklandLead Sponsor
78 Previous Clinical Trials
24,519 Total Patients Enrolled
Paul Harmatz, MDPrincipal InvestigatorUniversity of California, San Francisco
5 Previous Clinical Trials
54 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility 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.I have been diagnosed with MPS IVA or VI.Your oxygen level is consistently below 92% when measured in both hands.I have taken my enzyme replacement therapy less than 75% of the time in the last 6 months.You have a spine curvature (scoliosis) greater than 25 degrees shown on a previous x-ray.I have undergone gene therapy in the past.I have had surgery to lengthen my bones or fix my spine.You are at the earliest stage of physical development.I have never received enzyme replacement therapy.I have not taken specific medications for my condition in the last 6 months.I have been diagnosed with growth hormone deficiency.I am currently taking or have taken hormone therapy for cancer.I have a medical history that could affect growth measurement results.I have a history of chronic kidney problems with low filtration rates.Your heart's pumping ability is less than 40%.My MPS diagnosis is confirmed by genetic tests or enzyme deficiency.I have not had major bone surgery in the last 6 months and do not expect any during the study.My guardian can give me the study drug every day.I have been on oral steroids for the past 6 months.You have untreated low thyroid hormone levels.I had a stem cell transplant more than 3 years ago.I can stand by myself for at least one minute without help.I have previously been treated with or am currently on vosoritide.I have a condition that can affect my blood pressure or cause dizziness when standing.I am between 5 and 9 years old.I have not taken any experimental drugs recently.I have had a bone fracture in the last 6 months.I am not allergic to vosoritide.I have had cancer before.Your heart takes longer than normal to recharge between beats.You are significantly shorter than other people your age, or your height hasn't changed much in the last year.I have been on ERT with Vimizim or Naglazyme for at least 12 months.I have a long-term inflammation condition not linked to MPS.
Research Study Groups:
This trial has the following groups:- Group 1: Vosoritide
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Would I be eligible to participate in this medical experiment?
"To take part in this medical experiment, participants must have been diagnosted with mucopolysaccharidosis and be between the age of 5 and 10 years old. The trial requires 6 volunteers to fill its ranks."
Answered by AI
Is the current trial open to participants aged 30 or older?
"The age range requirement for this study is between 5 to 10 years old."
Answered by AI
Are there any spaces remaining for people to join this experiment?
"Going by the clinicaltrials.gov information, this trial is currently not taking on any more participants. It was launched on July 1st 2023 and last updated on May 4th of that same year. Nonetheless, 3 other trials are actively enrolling patients at present."
Answered by AI
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