Could a daily injection spare babies with dwarfism from risky neck surgery?
NCT ID NCT04554940
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called vosoritide in infants with achondroplasia (the most common form of dwarfism) who are at high risk of needing a surgery to relieve pressure on the spinal cord at the base of the skull. The main goal is to see if the drug is safe and if it can help enlarge the skull opening, potentially reducing the need for surgery. About 20 babies under 12 months old will receive the drug or standard care, and their health will be closely monitored.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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20 people
The number who actually took part.
- Started
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Oct 2020
- Expected to finish
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Dec 2027
An estimate. End dates often move.
- Lead sponsor
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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
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0 to 12 months
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: * Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research related procedure. * Have ACH, documented by genetic testing. * Are willing and able to perform all study procedures as physically possible. * Age 0 to ≤ 12 months, at study entry (Day 1). Given that any potential impact of vosoritide therapy on the foramen magnum is dependent on treating as early as possible and as long as possible while the synchondroses at the base of the skull are still open. For subjects \> 6 months of age at enrollment, a discussion between the investigator and the Medical Monitor should occur with the goal of limiting the number of subjects in the range of \> 6 months to ≤ 12 months of age. * Parent(s) or caregiver(s) are willing to administer daily injections to the subject and complete the required training. * Have evidence of CMC that "may" require surgical intervention Exclusion Criteria: * Have hypochondroplasia or short-stature condition other than achondroplasia (eg, trisomy 21, pseudoachondroplasia, etc). * Have CMC that either does not require surgical intervention (for example foramen magnum narrowing with preservation of the cerebrospinal fluid space) or does require immediate surgical intervention . * Have any of the following: Untreated congenital hypothyroidism or maternal history of hyperthyroidism, Insulin-requiring neonatal diabetes mellitus, Autoimmune inflammatory disease, Inflammatory bowel disease, Autonomic neuropathy. * Have a history of any of the following:Renal insufficiency, Chronic anemia,Baseline systolic blood pressure below age and gender specified normal range or recurrent symptomatic hypotension (defined as episodes of low blood pressure generally accompanied by symptoms eg, pallor, cyanosis, irritability, poor feeding) and Cardiac or vascular disease. * Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or QTc-F ≥ 450 msec on screening ECG. * Have been treated with growth hormone, insulin-like growth factor 1, or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time. * Have ever had prior cervicomedullary decompression surgery. * Have had a fracture of the long bones or spine within 6 months prior to Screening.
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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
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Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
London, SE1 7EH, United Kingdom
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Murdoch Children's Research Institute
Parkville, Victoria, 3052, Australia
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Sheffield Children's NHS Foundation Trust
Sheffield, S10 2TH, United Kingdom
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a weekly shot help children with achondroplasia grow taller?
- Can growth hormone boost height in children with dwarfism?
- Weekly shot may boost growth in kids with dwarfism
- Experimental achondroplasia drug trial halted early
- No treatment, just tracking: PROPEL study watches how kids with achondroplasia grow
- New drug aims to boost height in kids with dwarfism