Vosoritide for Hypochondroplasia

(CANOPY-HCH-EXT Trial)

Enrolling by invitation at 22 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: BioMarin Pharmaceutical

Must not be taking: Prohibited medications

Disqualifiers: Malignancy, Pregnancy, Growth issues, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Annually from enrollment through Final Adult Height

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the long-term safety and effectiveness of vosoritide for individuals with hypochondroplasia, a condition affecting bone growth. Participants will take vosoritide daily to determine if it improves growth over time. The trial seeks individuals who have participated in a previous study on this condition and can receive daily injections. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it mentions that you cannot take any prohibited medications. It's best to discuss your current medications with the study team to see if they are allowed.

Is there any evidence suggesting that vosoritide is likely to be safe for humans?

Research has shown that vosoritide is generally safe for people. In earlier studies, children with hypochondroplasia who took vosoritide grew faster and experienced only mild side effects, indicating that the side effects were not serious. Another study found that vosoritide helped reduce bowing of the leg bones in children compared to those who received a placebo. These findings suggest that vosoritide is well-tolerated by patients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for hypochondroplasia, which primarily focus on managing symptoms, vosoritide offers a novel approach by directly targeting the underlying cause of the condition. Vosoritide works by enhancing bone growth through its action on the natriuretic peptide receptor-B (NPR-B), which is a new mechanism compared to traditional therapies. Researchers are excited about vosoritide because it has shown potential to improve height outcomes in a way that existing treatments have not been able to achieve, offering new hope for patients with hypochondroplasia.

What evidence suggests that vosoritide might be an effective treatment for hypochondroplasia?

Research has shown that vosoritide, the treatment under study in this trial, can help children with hypochondroplasia grow taller. Studies have found that children who took vosoritide grew faster, with one study reporting an average height increase of 11.86 cm over two years. Vosoritide also helps straighten the shin bone, benefiting children with bone conditions. These results suggest that vosoritide could be a promising treatment to help children with hypochondroplasia grow. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for children with Hypochondroplasia who have completed a prior study (111-303 or 111-212) and still have growing bones. They must be willing to use contraception if sexually active, not planning pregnancy, able to perform study tasks, and their guardians must consent and administer daily injections.

Inclusion Criteria

If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study

Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays

Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure

Exclusion Criteria

Permanently discontinued study treatment in the studies 111-303 or 111-212

Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care

Require any investigational agent prior to completion of study period

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily doses of vosoritide to evaluate long-term safety and efficacy

Annually from enrollment through Final Adult Height

Follow-up

Participants are monitored for safety and effectiveness after treatment

Annually from enrollment through Final Adult Height

Open-label extension

Participants continue to receive vosoritide treatment to assess long-term outcomes

Long-term until final adult height

What Are the Treatments Tested in This Trial?

Interventions

• Vosoritide

Trial Overview The trial is testing the long-term safety and effectiveness of daily doses of Vosoritide in treating Hypochondroplasia. It's an extension study for participants who've already been receiving the treatment.

How Is the Trial Designed?

Group I: Vosoritide Experimental Treatment 1 Intervention

Open-label active drug

Who Is Running the Clinical Trial?

BioMarin Pharmaceutical

Lead Sponsor

Trials

162

Recruited

115,000+

Alexander Hardy

BioMarin Pharmaceutical

Chief Executive Officer since 2023

MBA from INSEAD

Greg Friberg

BioMarin Pharmaceutical

Chief Medical Officer

MD from New York Medical College

Citations

1. thelancet.com thelancet.com/journals/eclinm/article/PIIS2589-5370(24)00170-6/fulltext

Vosoritide treatment for children with hypochondroplasia Vosoritide was safe and effective in increasing growth velocity in children with hypochondroplasia. Efficacy was similar to what has been ...

2. investors.biomarin.com investors.biomarin.com/news/news-details/2025/BioMarin-Presents-New-Data-for-VOXZOGO-vosoritide-in-Children-with-Achondroplasia-and-Other-Skeletal-Conditions-at-Two-International-Endocrinology-Meetings/default.aspx

BioMarin Presents New Data for VOXZOGO® (vosoritide) ... Children who received VOXZOGO had a significant reduction in the magnitude of tibial bowing compared to children who received placebo.

3. sciencedirect.com sciencedirect.com/science/article/pii/S2949774424001584

Vosoritide increases growth velocity in hypochondroplasia ... The primary outcomes are rate of adverse events and change in AGV and height SD from baseline. Secondary outcomes included measurement of body proportions and ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT07126262

NCT07126262 | A Study of Vosoritide Versus Placebo in ... Study Overview. The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH ...

5. rarediseaseadvisor.com rarediseaseadvisor.com/news/real-world-studies-demonstrated-vosoritides-growth-benefits-achondroplasia/

Real-World Data Support Efficacy of Vosoritide in Children ... Those treated for 24 months exhibited even greater growth, with an average height gain of 11.86 cm and a Z-score improvement of 1.15. These ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11133798/

Vosoritide treatment for children with hypochondroplasia Vosoritide was safe and effective in increasing growth velocity in children with hypochondroplasia. Efficacy was similar to what has been reported in children ...

7. nature.com nature.com/articles/s41574-024-01074-9

International consensus guidelines on the implementation ... Clinical trial data published early in 2024 demonstrated height Z-score improvements with a mild adverse event profile in children aged 3–59 ...