โšกYour ready-to-use recruitment solution: IRB-compliant, SEO-optimized website that automatically routes qualified patient leads to your clinical study (14-day free trial, then $44/mo)

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

Sponsored by BioMarin Pharmaceutical

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...

Current Status

Recruiting

Auto-verified from ClinicalTrials.gov

Verified Aug 17, 2025

Phase

Clinical Trial

Sponsor

BioMarin Pharmaceutical

Enrollment Target

60

Start Date

Jul 2025

๐Ÿ“Š
1 of 60 participants interested
2% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.

Who Can Participate

Inclusion Criteria

Participants must be 0 to \< 36 months of age at randomization. 2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH). 3. Participants aged 0 to \< 12 months must have a height Z-score of โ‰ค -1.0 SDS andparticipants aged โ‰ฅ 12 to \< 36 months must have a height Z-score of โ‰ค -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts. 4. Participant's weight at the Day 1 visit (pre-treatment) must be โ‰ฅ 3 kg. Key

Exclusion Criteria

Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia). 2. Have an unstable medical condition likely to require surgical intervention during the study period. 3. Taking any of the prohibited medications. 4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroi

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

๐Ÿ“‹

Study Type

INTERVENTIONAL

๐Ÿ‘ฅ

Target Enrollment

60 participants

๐ŸŽ‚

Age Range

0 Months - 36 Months

โšง

Gender

ALL

๐Ÿข

Sponsor

BioMarin Pharmaceutical

Study Locations

Choose your preferred location or select flexible during enrollment

NOT_YET_RECRUITING

Phoenix

Arizona

Location available
View Phoenix location page
NOT_YET_RECRUITING

Los Angeles

California

Location available
View Los Angeles location page
NOT_YET_RECRUITING

Oakland

California

Location available
View Oakland location page
RECRUITING

Washington D.C.

District of Columbia

Location available
View Washington D.C. location page
NOT_YET_RECRUITING

Chicago

Illinois

Location available
View Chicago location page
NOT_YET_RECRUITING

Baltimore

Maryland

Location available
View Baltimore location page
NOT_YET_RECRUITING

Minneapolis

Minnesota

Location available
View Minneapolis location page
NOT_YET_RECRUITING

Columbia

Missouri

Location available
View Columbia location page
NOT_YET_RECRUITING

Neenah

Wisconsin

Location available
View Neenah location page

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Find More Ankylosing Spondylitis Trials by City

Browse all ankylosing spondylitis clinical trials in these cities โ€” not just this study.

Looking for Hypochondroplasia Treatment in Phoenix?

Join others in Arizona exploring innovative treatment options through clinical research

Hypochondroplasia Treatment Options in Phoenix, Arizona

If you're searching for Hypochondroplasia treatment in Phoenix, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Phoenix, Los Angeles, Oakland and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Hypochondroplasia. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Arizona
Now Enrolling
Up to 60 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Hypochondroplasia?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Hypochondroplasia

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Hypochondroplasia Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT07126262. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.