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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 52 weeks

Awards & highlights

Study 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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 52 weeks

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.

0 / 5Eligibility criteria met

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are persons of advanced age precluded from enrolling in this experiment?

"This trial's enrollment criteria mandates that subjects must be aged between 2 and 10 years old. Furthermore, there are 12 clinical trials for minors and 3 specifically tailored to those over 65."

Answered by AI

Does this medication have the approval of the U.S. Food and Drug Administration?

"As this is an early-stage Phase 2 trial, the safety rating of this medication was set to a score of 2 as there is only limited data on its efficacy."

Answered by AI

Where can I access this clinical trial currently?

"Currently, 8 facilities are participating in this study. These sites can be found in Houston, Saint Paul and Seattle as well as 5 other cities. To minimize travel needs if you intend to join the trial, it is recommended that you select a location nearest you."

Answered by AI

Do I meet the qualifications necessary to join this research endeavor?

"This trial is looking to recruit 60 individuals with achondroplasia, ranging in age from 2 to 10 years old. To be considered for enrolment, participants must have not entered puberty and be able to stand without assistance."

Answered by AI

Is this investigation currently open to new participants?

"The trial hosted on clinicaltrials.gov is not currently enrolling patients, having been first posted on June 24th 2020 and last updated July 29th 2022. However, there are still 12 other medical studies searching for participants at the present time."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia

2020. "A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With Achondroplasia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04085523.

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