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Compares treatment options for Hypochondroplasia
Official title A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
ClinicalTrials.gov ID: NCT07126262
What this study is testing
What is Vosoritide?
Vosoritide is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for hypochondroplasia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 36
You may be able to join if
- Participants must be 0 to \< 36 months of age at randomization.
- Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated...
- Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of...
- Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg. Key
You likely can't join if
- Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
- Have an unstable medical condition likely to require surgical intervention during the study period.
- Taking any of the prohibited medications.
- Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term...
- Require any investigational agent prior to completion of study period.
- Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
See the full eligibility criteria
- Participants must be 0 to \< 36 months of age at randomization.
- Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
- Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and...
- Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg. Key
- Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
- Have an unstable medical condition likely to require surgical intervention during the study period.
- Taking any of the prohibited medications.
- Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time.
- Require any investigational agent prior to completion of study period.
- Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
- Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.
- Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
- Have known hypersensitivity to vosoritide or its excipients.
- Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study.
- Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- Los Angeles, California, United States
- Oakland, California, United States
- Washington D.C., District of Columbia, United States
- Chicago, Illinois, United States
- Baltimore, Maryland, United States
- Minneapolis, Minnesota, United States
- Columbia, Missouri, United States
- Fort Worth, Texas, United States
- Neenah, Wisconsin, United States
- South Brisbane, Queensland, Australia
- Parkville, Victoria, Australia
- Le Kremlin-Bicêtre, Paris, France
- Bron, Rhone-Alps, France
- Cologne, North Rhine-Westphalia, Germany
- Homburg, Saarland, Germany
- Magdeburg, Saxony-Anhalt, Germany
- Roma, Rome, Italy
- Genova, Italy
- Kumamoto, Japan
+ 4 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 months to 36 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Phoenix, Arizona, United States; Los Angeles, California, United States; Oakland, California, United States; Washington D.C., District of Columbia, United States; Chicago, Illinois, United States; Baltimore, Maryland, United States and 18 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.