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AchondroplasiaPhase 2NCT05246033

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

A Phase 2 study testing TransCon CNP in people with Achondroplasia, run by Visen Pharmaceuticals (Shanghai) Co., Ltd. at 5 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Purpose of the study: The main purpose of this study is to determine the safety and evaluate the effect of a once weekly dose of TransCon CNP in prepubertal children with achondroplasia in China. Read more about this study

Who is it for?
Ages 2 to 10 years
Conditions the study is for
Achondroplasia
What is being tested?
TransCon CNP
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
24 participants
Where
5 sitesChina
Study dates
5 Jan 2022to 23 Apr 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 10 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Diagnosis of achondroplasia confirmed by genetic testing
  • Age criteria: between ages 2 to 10 years old (inclusive) at Screening Visit
  • Tanner stage 1 breast development for females or testicular volume < 4ml for males at Screening
  • Able to stand without assistance
  • Parent/ legal guardian willing and able to administer subcutaneous injections of study medication
  • 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)

Who cannot join

  • 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
  • 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)
  • Have received any investigational medicinal product or device intended to affect stature or body proportionality at any time
Show all 36Show fewer
  • History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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
  • 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)
  • 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.
  • Ongoing treatment with any medication that affects blood pressure or heart rate
  • Known hypersensitivity to the components of the investigational medicinal product (trehalose, tris(hydroxymethyl)aminomethane, succinate and PEG)
  • 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

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

China5 sites

  • Ascendis Pharma Investigational Site

    Beijing, 100045

  • Ascendis Pharma Investigational Site

    Guangzhou, 510080

  • Ascendis Pharma Investigational Site

    Hangzhou, 310053

  • Ascendis Pharma Investigational Site

    Shanghai, 20082

  • Ascendis Pharma Investigational Site

    Wuhan, 430030

Other trials in China

About this study

Official title: ACcomplisH China: A Phase 2, Multicenter, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia

Purpose of the study:

The main purpose of this study is to determine the safety and evaluate the effect of a once weekly dose of TransCon CNP in prepubertal children with achondroplasia in China.

Read more

Study Treatments:

TransCon CNP is an investigational (new) drug, which means that it is currently being tested, and therefore is considered experimental. TransCon CNP is designed to provide a sustained exposure of active CNP by subcutaneous (under the skin) injection once weekly.

The Randomized Period of this study is a double-blinded and placebo-controlled. "Placebo-controlled" means that some participants will receive injections that don't contain any TransCon CNP (placebo injection - no active ingredient). "Double-blinded" means that neither the participant nor the study doctor will know which treatment the participant will be receiving, except in an emergency.

After completion of the Randomized Period the trial participant may be invited to take part of the Open-Label Period of this study.

"Open-label" means that all participants will receive injections that contain TransCon CNP; regardless of which treatment (TransCon CNP or placebo) was assigned during the 52 weeks (1 year) Randomized Period/blinded treatment period. It also means that both the participant and the study doctor will know which treatment, and which dose the participant receives.

Conditions
Achondroplasia
Being tested
TransCon CNP, Placebo for TransCon CNP
Keywords
Achondroplasia, Dwarfism
Medical subject headings
Achondroplasia, Dwarfism

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TransCon CNPDrug

    TransCon CNP drug product is a lyophilized powder in a single-use vial containing either TransCon CNP 3.9 mg CNP-38/vial or TransCon CNP 0.80 mg CNP-38/vial. Prior to use, the lyophilized powder is reconstituted with sterile water for injection and administered by subcutaneous injection via syringe and needle.

  • Placebo for TransCon CNPDrug

    Weekly subcutaneously injection of placebo.

Study groups in NCT05246033
Study groupRoleWhat they receive
TransCon CNP 50 mcgExperimentalTransCon CNP 50 mcg CNP/kg or placebo mimicking TransCon CNP 50 mcg delivered once weekly by subcutaneous injection
Receives: Drug: TransCon CNP
TransCon CNP 100 mcgExperimentalTransCon CNP 100 mcg CNP/kg or placebo mimicking TransCon CNP 100 mcg delivered once weekly by subcutaneous injection
Receives: Drug: TransCon CNP
PlaceboPlacebo comparatorPlacebo delivered once weekly by subcutaneous injection
Receives: Drug: Placebo for TransCon CNP

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)

    Safety and tolerability of once weekly TransCon CNP treatment or placebo

    Measured: 52 weeks

  2. Annualized height velocity (centimeters/year) at 52 weeks

    Annualized height velocity measured in centimeters over 52 weeks for TransCon CNP or placebo

    Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Feb 2022
  2. Study start5 Jan 2022
  3. Primary completion15 Mar 2023
  4. Study completion23 Apr 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
There are 2 cohorts enrolling 24 subjects who will be randomized to receive either TransCon CNP or Placebo in a 3:1 ratio
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
24 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05246033
Sponsor's protocol ID
TCC-204
Status last verified
Aug 2026
First submitted
9 Feb 2022
Last updated
10 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing TransCon CNP in people with Achondroplasia, run by Visen Pharmaceuticals (Shanghai) Co., Ltd. at 5 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 36 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) (time frame: 52 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05246033.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Aug 2026.

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