Vosoritide for Dwarfism

(CANOPY-HCH-3 Trial)

Recruiting at 24 trial locations

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: BioMarin Pharmaceutical

Must not be taking: Growth hormone, IGF-1, Anabolic steroids

Disqualifiers: Short stature, Unstable condition, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

80 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called vosoritide to determine if it can help children with hypochondroplasia (HCH), a form of dwarfism, grow taller. The study compares vosoritide injections to placebo injections (inactive treatment) to assess any differences. Children with a confirmed genetic diagnosis of HCH and shorter height compared to peers might be suitable candidates for this trial. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it does exclude participants who are taking certain prohibited medications or have been treated with growth hormone, IGF-1, or anabolic steroids.

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

Research has shown that vosoritide is generally safe. In earlier studies, only 3% of patients experienced a temporary drop in blood pressure, sometimes accompanied by vomiting or dizziness. These issues were mild and short-lived. The FDA has already approved vosoritide for children with achondroplasia, a condition similar to hypochondroplasia. This approval suggests the treatment is generally safe for young patients. The drug has demonstrated an ability to increase height without major side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hypochondroplasia?

Vosoritide is unique because it targets the underlying cause of dwarfism by directly affecting bone growth. Unlike standard treatments that mostly focus on managing symptoms, vosoritide acts on the growth plates in bones to promote lengthening. This mechanism of action offers a potential breakthrough for increasing height in individuals with dwarfism, which is why researchers are so eager about its possibilities.

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

Studies have shown that vosoritide works well for children with achondroplasia, a condition similar to hypochondroplasia. In past research, vosoritide helped children grow and move better over a year. Long-term studies indicate that these growth benefits can last up to six years. The treatment is generally safe, with most children experiencing no serious side effects. In this trial, participants will receive either a vosoritide injection or a placebo injection. Although this trial focuses on hypochondroplasia, the positive results observed in achondroplasia offer hope for similar outcomes. ^{6 7 8 9 10}

Who Is on the Research Team?

Medical Director, MD

Principal Investigator

BioMarin Pharmaceutical

Are You a Good Fit for This Trial?

This trial is for children with hypochondroplasia, a form of short-limbed dwarfism. Participants should have conditions like dwarfism, lordosis (curved lower back), or congenital limb deformities related to HCH.

Inclusion Criteria

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

I am a female over 10 or have started my periods and can take pregnancy tests.

My height is much shorter than average for my age and sex.

Exclusion Criteria

Have known hypersensitivity to vosoritide or its excipients

Short stature condition other than HCH

I might need surgery for an unstable condition during the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily subcutaneous injections of vosoritide or placebo for 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Vosoritide

Trial Overview The study is testing Vosoritide's effectiveness compared to a placebo in treating children with HCH. It's a Phase 3 trial, which means it's looking at how well the drug works and monitoring its safety.

How Is the Trial Designed?

Group I: vosoritide injection with vial and syringe Experimental Treatment 1 Intervention

Group II: Placebo injection with vial and syringe Placebo Group 1 Intervention

Vosoritide is already approved in United States for the following indications:

Approved in United States as Voxzogo for:

• To increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses
• to increase linear growth in pediatric patients less than 5 years of age with achondroplasia with open epiphyses

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

Published Research Related to This Trial

Vosoritide (Voxzogo) has received accelerated approval from the FDA for treating children aged five and older with achondroplasia, a common form of dwarfism.

To ensure safety and prevent hypotensive episodes, children taking vosoritide should consume a meal and 240 to 300 mL of fluid within an hour before administration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

New Drug Treats Pediatric Achondroplasia. Aschenbrenner, DS.[2023]

Vosoritide (VOXZOGO®) is a modified C-type natriuretic peptide that effectively treats achondroplasia by inhibiting the overactive FGFR3 gene, which negatively regulates bone growth.

Approved in the EU for patients aged 2 years and older with open epiphyses, vosoritide represents a significant advancement in achondroplasia treatment, with ongoing regulatory reviews in the USA and further clinical development worldwide.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vosoritide: First Approval. Duggan, S.[2022]

Vosoritide is the first approved treatment for achondroplasia, and parents of children using it reported a strong motivation to manage the daily injectable treatment for potential long-term benefits, such as improved height and independence.

The study highlighted the need for better support and consistent training for families, as experiences with treatment initiation varied significantly, impacting their ability to manage the treatment effectively at home.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parents' Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia. NiMhurchadha, S., Butler, K., Argent, R., et al.[2023]

Vosoritide, administered at a recommended dose of 15 μg/kg, was shown to be effective in increasing growth velocity in children with achondroplasia over a 52-week phase III study involving 60 patients, with no significant safety concerns noted.

The pharmacokinetics of vosoritide indicated rapid absorption and consistent exposure without accumulation, and the development of anti-vosoritide antibodies in 42% of patients did not adversely affect growth outcomes or drug exposure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. Chan, ML., Qi, Y., Larimore, K., et al.[2022]

Vosoritide is the first approved therapy specifically designed to increase growth velocity in children with achondroplasia, and early experiences from experts indicate a generally positive reception in clinical practice.

Effective management of vosoritide treatment requires multidisciplinary support, patient education on administration, and monitoring for adherence, as these factors significantly influence treatment outcomes and patient satisfaction.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice. Semler, O., Cormier-Daire, V., Lausch, E., et al.[2023]

Voxzogo (vosoritide) is the first drug approved by the FDA and EMA specifically for treating achondroplasia, a leading cause of dwarfism, marking a significant advancement in addressing this rare condition.

Despite the risk of decreased blood pressure as a severe adverse event, the benefits of Voxzogo in fulfilling an unmet medical need for patients with achondroplasia led to its accelerated approval in the U.S. as a treatment for a rare pediatric disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vosoritide, a miracle drug, covering unmet need in achondroplasia: A regulatory update. Simran, ., S, KDS., Dushantrao, SC., et al.[2023]

Citations

1. biomarin.com biomarin.com/news/press-releases/biomarin-presents-new-data-for-voxzogo-vosoritide-in-children-with-achondroplasia-and-other-skeletal-conditions-at-two-international-endocrinology-meetings/

BioMarin Presents New Data for VOXZOGO® (vosoritide) ... New data in children under 5 years with achondroplasia showed treatment with VOXZOGO was associated with improvement in tibial bowing, a common cause of pain ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12352272/

Vosoritide (Voxzogo) for Achondroplasia: A Review of ... Treatment with vosoritide may improve long-term outcomes for children with achondroplasia when compared with the current pharmacological ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40471380/

Real-World Safety and Effectiveness of Vosoritide in ... Vosoritide showed long-term effectiveness in a real-world Portuguese population of patients with achondroplasia. Vosoritide was also well tolerated.

4. academic.oup.com academic.oup.com/jes/article/9/5/bvaf041/8064591

Real-world Outcome of Vosoritide Treatment in Children With ... In a real-world setting, children with ACH showed significant improvements in growth and physical function after 12 months of treatment with vosoritide.

5. sciencedirect.com sciencedirect.com/science/article/pii/S2666634024004604

Sustained growth-promoting effects of vosoritide in children ... Vosoritide treatment was well tolerated and had sustained growth-promoting effects in children with achondroplasia treated for up to 6 years.

6. hcp.biomarin.com hcp.biomarin.com/en-us/voxzogo/safety-data/

Safety | VOXZOGO® (vosoritide) HCP VOXZOGO safety data. 2 of 60 VOXZOGO-treated patients (3%) each had one symptomatic episode of blood-pressure decrease with vomiting and/or dizziness ...

7. voxzogo.com voxzogo.com/en-us/

VOXZOGO® (vosoritide) | Learn More Here For You From The Start. Only VOXZOGO is FDA approved from birth for children with achondroplasia until growth plates close. Why Families Choose VOXZOGO.

8. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2023/214938s002lbl.pdf

VOXZOGO (vosoritide) for injection - accessdata.fda.gov The safety and effectiveness of VOXZOGO have been established in pediatric patients for the improvement in linear growth in patients with achondroplasia with ...

9. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39864410/

Real-World Safety and Effectiveness of Vosoritide in ... In clinical trials, vosoritide has shown a positive safety profile and increased height in children with achondroplasia.

10. clinicaltrials.gov clinicaltrials.gov/study/NCT04554940

Study Details | NCT04554940 | A Clinical Trial to Evaluate ... A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia · Study Overview · Contacts and Locations · Participation Criteria · Study ...