What side effects are associated with this type of intervention?

BMN111, a C-type Natriuretic Peptide Analog, is being studied for its potential to promote endochondral ossification and bone growth in children with Achondroplasia. Common side effects for treatments in this category can include injection site reactions, headaches, and potential cardiovascular effects due to the nature of natriuretic peptides. As with any treatment promoting bone growth, there may also be concerns about disproportionate growth or other skeletal abnormalities. It is important to monitor these treatments closely under medical supervision.

What prior FDA approvals exist?

The provided context does not mention any specific prior FDA approvals for the treatment of Achondroplasia. However, BMN111 (vosoritide), a C-type Natriuretic Peptide Analog, is being studied for its efficacy in promoting endochondral ossification and bone growth in children with Achondroplasia. This type of treatment represents a novel approach targeting the underlying pathophysiology of the condition.

What other types of research exist?

Currently, there are no specific novel alternatives to BMN111 mentioned in the provided context for the treatment of Achondroplasia. Patients should consult with their healthcare provider to explore the latest research and clinical trials for potential new treatments.

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Peptide

BMN 111 for Achondroplasia

Phase 2

Waitlist Available

Research Sponsored by BioMarin Pharmaceutical

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Must not have

Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F > 450 msec

Timeline

Screening 3 weeks

Treatment Varies

Follow Up "through study completion, an average of 5 years"

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trial is testing BMN111, an injection, in children with Achondroplasia to help them grow taller. BMN111 has shown promise in promoting growth in children with achondroplasia.

Who is the study for?

This trial is for children with Achondroplasia who have completed a year in the BMN 111-206 study. They must be able to perform all study procedures and their parents or guardians agree to consent. Children can't join if they have serious heart issues, need other investigational drugs, have conditions affecting study participation or compliance, stopped BMN 111 early in the previous study, are on certain heart or kidney medications, or are pregnant.

What is being tested?

The trial tests long-term safety and effectiveness of BMN 111 given as a daily subcutaneous injection based on weight until near-adult height is reached. It's an open-label Phase 2 extension where around 70 subjects from a prior study continue with BMN 111 treatment.

What are the potential side effects?

While specific side effects aren't listed here, potential risks may include reactions at the injection site, general discomforts like headaches or nausea, and any unforeseen complications related to growth patterns due to drug action.

Eligibility Criteria

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

My heart's electrical activity is abnormal, or my QTc-F measurement is over 450 msec.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ "through study completion, an average of 5 years"

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~"through study completion, an average of 5 years"

This trial's timeline: 3 weeks for screening, Varies for treatment, and "through study completion, an average of 5 years" for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

Secondary study objectives

Potential Changes in health-related quality of life as measured by the quality of life in Short- statured youth

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Active BMN 111Experimental Treatment1 Intervention

Once daily subcutaneous injections of recommended dose of BMN 111 based on weight-band dosing.

0 / 1Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

BMN111, a C-type Natriuretic Peptide (CNP) analog, works by inhibiting the overactive fibroblast growth factor receptor 3 (FGFR3) pathway, which is responsible for the impaired bone growth seen in Achondroplasia. By promoting endochondral ossification and bone growth, BMN111 helps to normalize the growth of bones, addressing the primary cause of disproportionate short stature in Achondroplasia patients. This mechanism is crucial as it directly targets the underlying genetic issue, offering a potential therapeutic approach to improve height and skeletal development in affected individuals.

Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model.