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Peptide Hormone Analog

TransCon CNP for Achondroplasia

Phase 2
Waitlist Available
Research Sponsored by Ascendis Pharma A/S
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Able to stand without assistance
Age between 2 to 10 years old (inclusive) at Screening Visit
Must not have
Have received any study drug or device intended to affect stature or body proportionality at any time
Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights

Summary

This trial is testing a new drug for children with achondroplasia, a condition that causes dwarfism. The drug is given weekly and the trial will compare it to a placebo to see if it is effective.

Who is the study for?
This trial is for prepubertal children aged 2-10 with Achondroplasia, able to stand without assistance and have a caregiver willing to give injections. Kids who've had growth treatments or need surgery that could interfere with the study can't join.Check my eligibility
What is being tested?
The trial tests TransCon CNP, given weekly via injection, against a placebo in kids with Achondroplasia. It's randomized and double-blind, meaning neither the researchers nor participants know who gets the real treatment.See study design
What are the potential side effects?
While specific side effects are not listed here, they may include reactions at the injection site, general discomfort or other issues related to growth treatments which will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can stand up on my own without help.
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I am between 2 and 10 years old.
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I am prepubertal according to the screening visit.
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I have been diagnosed with ACH and it's genetically confirmed.
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My caregiver can give me shots under my skin as part of the study.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have never used drugs or devices to alter my height or body shape.
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I have taken medication to alter my height or body shape in the last 6 months.
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I have a condition affecting my bone growth, but it's not Achondroplasia.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Annualized Height Velocity (cm/Year) After 52 Weeks of Double-blind Treatment

Trial Design

6Treatment groups
Experimental Treatment
Placebo Group
Group I: TransCon CNP 6 mcgExperimental Treatment2 Interventions
TransCon CNP 6 mcg CNP/kg delivered once weekly by subcutaneous injection.
Group II: TransCon CNP 50 mcgExperimental Treatment2 Interventions
TransCon CNP 50 mcg CNP/kg delivered once weekly by subcutaneous injection.
Group III: TransCon CNP 20 mcgExperimental Treatment2 Interventions
TransCon CNP 20 mcg CNP/kg delivered once weekly by subcutaneous injection.
Group IV: TransCon CNP 100 mcgExperimental Treatment2 Interventions
TransCon CNP 100 mcg CNP/kg delivered once weekly by subcutaneous injection.
Group V: Open-Label Extension PeriodExperimental Treatment1 Intervention
Subjects who complete the 52-week blinded treatment period can continue into the 104-week open-label extension period and will receive treatment with TransCon CNP.
Group VI: PlaceboPlacebo Group1 Intervention
Placebo mimicking 6, 20, 50, or 100 mcg CNP/kg delivered once weekly by subcutaneous injection.

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
TransCon CNP is a sustained release formulation of C-type Natriuretic Peptide (CNP), which promotes bone growth by inhibiting the signaling pathway of fibroblast growth factor receptor 3 (FGFR3). FGFR3 is overactive in Achondroplasia, leading to impaired bone growth and short stature. By blocking this pathway, CNP helps to normalize bone growth and improve height outcomes in patients. This mechanism is crucial for Achondroplasia patients as it directly targets the underlying cause of their growth issues, offering a potential therapeutic approach to enhance their quality of life.

Find a Location

Who is running the clinical trial?

Ascendis Pharma A/SLead Sponsor
11 Previous Clinical Trials
1,500 Total Patients Enrolled
2 Trials studying Achondroplasia
284 Patients Enrolled for Achondroplasia
Marie-Louise Hartoft-Nielsen, MD, PhDStudy DirectorAscendis Pharma A/S
2 Previous Clinical Trials
284 Total Patients Enrolled
2 Trials studying Achondroplasia
284 Patients Enrolled for Achondroplasia
Adebola Giwa, MDStudy DirectorAscendis Pharma
2 Previous Clinical Trials
400 Total Patients Enrolled
2 Trials studying Achondroplasia
400 Patients Enrolled for Achondroplasia

Media Library

TransCon CNP (Peptide Hormone Analog) Clinical Trial Eligibility Overview. Trial Name: NCT04085523 — Phase 2
Achondroplasia Research Study Groups: Open-Label Extension Period, Placebo, TransCon CNP 20 mcg, TransCon CNP 100 mcg, TransCon CNP 50 mcg, TransCon CNP 6 mcg
Achondroplasia Clinical Trial 2023: TransCon CNP Highlights & Side Effects. Trial Name: NCT04085523 — Phase 2
TransCon CNP (Peptide Hormone Analog) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04085523 — Phase 2
~11 spots leftby Jul 2025