Could a daily shot help kids with rare bone diseases grow?
NCT ID NCT05845749
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early study tests a daily injection of vosoritide (Voxzogo) in 6 children aged 5 to 10 with MPS IVA or VI, rare conditions that cause growth problems. The main goal is to see if the drug is safe and tolerable over 96 weeks. Researchers will also look at changes in height and bone growth markers.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Vosoritide (Voxzogo) injection
- What this could lead to
- If it works, this could point toward a treatment to help children with MPS IVA and VI grow taller and improve bone health.
- What could go wrong
- This is a very small, early-phase study with only 6 participants, so results may not apply to everyone. It is designed mainly to check safety, not to prove effectiveness.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
-
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.