Navepegritide for Achondroplasia

(teACH Trial)

Recruiting at 3 trial locations

Overseen By Ascendis Registry Inquiries

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Ascendis Pharma A/S

Must not be taking: Systemic corticosteroids

Disqualifiers: Growth disorders, Severe FGFR3 mutation, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.

Who Is on the Research Team?

Claus Strange

Principal Investigator

Ascendis Pharma A/S

Are You a Good Fit for This Trial?

This trial is for adolescents aged 12-18 with Achondroplasia. Participants need a guardian to administer weekly injections, have a historical height measurement from the past 6-15 months, and provide consent. A confirmed genetic diagnosis of Achondroplasia is required.

Inclusion Criteria

My guardian can give me weekly shots and follow the study rules.

I have a height measurement in my medical records from 6 to 15 months ago.

I (or my guardian) have signed the consent form for this trial.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive once weekly subcutaneous doses of navepegritide 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

• Navepegritide

Trial Overview The trial tests Navepegritide's effectiveness and safety in increasing growth velocity over a year compared to placebo. Adolescents will receive weekly subcutaneous doses of either Navepegritide or an inactive drug.

How Is the Trial Designed?

Group I: Navepegritide Experimental Treatment 1 Intervention

Once weekly double-blinded treatment with SC injection of 100 µg/kg of navepegritide for 52 weeks

Group II: Placebo for Navepegritide Placebo Group 1 Intervention

Once weekly double-blinded treatment with SC injection of 100 µg/kg of placebo for navepegritide for 52 weeks

Who Is Running the Clinical Trial?

Ascendis Pharma A/S

Lead Sponsor

Trials

15

Recruited

1,600+