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Can a weekly shot help children with achondroplasia grow taller?

NCT ID NCT05246033

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 10, 2026 · Last updated Aug 11, 2026 · Updated 1 time

Summary

This phase 2 trial is testing an experimental drug called TransCon CNP in children with achondroplasia, a common form of dwarfism. The study aims to see if a once-weekly injection under the skin can safely improve growth, measured by height velocity over a year. Participants will be randomly assigned to receive either the drug or a placebo, and neither they nor their doctors will know which they get. After the blinded year, all may receive the active drug in an open-label phase.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
TransCon CNP, an experimental drug designed to provide sustained exposure to C-type natriuretic peptide (CNP) via weekly subcutaneous injection
What this could lead to
If successful, this could lead to a new treatment option to improve growth and reduce complications in children with achondroplasia.
What could go wrong
This is an early-phase trial with a small number of participants, so results may not be conclusive or generalizable. The drug is experimental and may have side effects or fail to show significant benefit.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

24 people

The number who actually took part.

Started

Jan 2022

Finished

Apr 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 10 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Diagnosis of achondroplasia confirmed by genetic testing 2. Age criteria: between ages 2 to 10 years old (inclusive) at Screening Visit 3. Tanner stage 1 breast development for females or testicular volume \< 4ml for males at Screening 4. Able to stand without assistance 5. Parent/ legal guardian willing and able to administer subcutaneous injections of study medication 6. Written, signed informed consent of the parent(s) or legal guardian(s) of the participant and written assent of the participant as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC) Exclusion Criteria: 1. Clinically significant findings at Screening that: * Are expected to require surgical intervention during participation in the trial or * Are musculoskeletal in nature, such as Salter-Harris fractures or severe hip pain or * Otherwise are considered by the Investigator or Medical Monitor to make a participant unfit to receive investigational medicinal products or undergo trial related procedures 2. Have received any dose of prescription medications intended to affect stature or body proportionality (including human growth hormone) in the 6 months prior to Screening (excluding nutritional supplements) 3. Have received any investigational medicinal product or device intended to affect stature or body proportionality at any time 4. History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones 5. History of any bone-related surgery that affects growth potential of long bones, such as orthopedic reconstructive surgery, including but not limited to: * Foramen magnum decompression and laminectomy with full recovery are allowed with minimum of 6 months of bone healing * Osteotomy to correct bowing is allowed with 12 months of bone healing * Limb-lengthening with full recovery is allowed with a minimum of 12 months of bone healing * History of 8 plate epiphysiodesis is allowed, but the plates must have been removed prior to Screening with minimum of 4 weeks of healing 6. Have a growth disorder other than ACH that results in short stature or abnormal growth such as severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growth hormone deficiency, Turner syndrome, or pseudoachondroplasia 7. Have a medical condition that could result in short stature or abnormal growth such as inflammatory bowel disease, celiac disease, vitamin D deficiency, untreated hypothyroidism, or poorly controlled diabetes mellitus (HbA1c ≥8.0%), or other diabetic complications. Please note the following allowances: * Vitamin D deficiency or insufficiency treated with supplementation is allowed. Vitamin D deficiency is defined as 25(OH)D level \<20ng/mL (\<49.9 nmol/L), insufficiency is defined as 25(OH)D level \<20-30ng/mL (49.92 - 74.86 nmol/L). Participants with Vitamin D deficiency or insufficiency must be on Vitamin D regimen before enrollment * Participants with hypothyroidism must be clinically euthyroid for 3 months prior to enrollment and, in the opinion of the Investigator, have achieved any catch-up growth expected from thyroxine replacement * Participants with diabetes mellitus must have been on a stable medication regimen for 3 months prior to enrollment (dose adjustments are allowed but addition or discontinuation of medications in this time period is disallowed). In addition to HbA1c \<8.0%, any participant with diabetes must have adequate glycemic control in the opinion of the Investigator and Medical Monitor to be considered a good candidate for the trial 8. History or presence of malignant disease, other than basal cell epithelioma/carcinoma or completely resected squamous skin cancer with no recurrence for 12 months per medical records 9. History or presence of the following: * Chronic anemia (iron deficiency anemia that is resolved or adequately treated in the Investigator's opinion is allowed) * Significant cardiovascular disease per the judgement of the Investigator, such as congenital heart disease (uncomplicated patent ductus arteriosus and atrial or ventricular septal defect with repair are allowed), aortic insufficiency, clinically significant arrhythmias, congestive heart failure with NYHA class II and above or other conditions that impair regulation of blood pressure or heart rate * Condition that impacts hemodynamic stability (such as autonomic dysfunction, orthostatic intolerance) * Chronic renal insufficiency * Chronic or recurrent illness that can affect hydration or volume status. This may include conditions associated with decreased nutritional intake or increased volume loss * Bone fracture within 6 months prior to screening (within 2 months for fracture of digits) * Any disease or condition that, in the opinion of the Investigator or Medical Monitor, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or may present undue risk from receiving investigational product 10. Significant electrocardiogram abnormalities, including evidence of a previous myocardial infarction, left ventricular hypertrophy, flat T waves (particularly in the inferior leads) or more than minor non-specific ST-T wave changes or: * QRS \>90 milliseconds (msec) * QT interval corrected using Fridericia's formula (QTcF) \>440 msec * PR interval \>170 msec * Complete right or left bundle branch block 11. Requires, or anticipated to require, chronic (\> 4 weeks) or repeated treatment (more than twice/year) with oral corticosteroids during participation in the trial (low and mid-dose inhaled corticosteroids are allowed with Medical Monitor approval. High-dose inhaled corticosteroids are not allowed) 12. Use of medication known to prolong the QT/QTc interval (https://crediblemeds.org/. Note: Only medications on the Known Risk list are excluded, not those on the Possible or Conditional Risk lists). Prior use of such medications is allowed if participant has sufficient wash-out period (minimum 7 days or 5 half-lives, whichever was longer) and a normal QT/QTc interval on ECG. 13. Ongoing treatment with any medication that affects blood pressure or heart rate 14. Known hypersensitivity to the components of the investigational medicinal product (trehalose, tris(hydroxymethyl)aminomethane, succinate and PEG) 15. Any other reason that in the opinion of the Investigator or Medical Monitor would prevent the child from complying with the trial requirements, prevent successful completion of the trial, or prevent successful interpretation of trial data • This could include family situations, comorbid conditions, or medications that might impact safety or be considered confounding

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Ascendis Pharma Investigational Site

    Beijing, 100045, China

  • Ascendis Pharma Investigational Site

    Guangzhou, 510080, China

  • Ascendis Pharma Investigational Site

    Hangzhou, 310053, China

  • Ascendis Pharma Investigational Site

    Shanghai, 20082, China

  • Ascendis Pharma Investigational Site

    Wuhan, 430030, China

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