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Long-Term Extension Study of Vosoritide to Treat Children with Hypochondroplasia
EU CTIS ID: 2024-517238-16-00
What this study is testing
To evaluate the long-term safety, tolerability, and efficacy of vosoritide treatment until final adult height (FAHa) in patients from studies 111-303 and 111-212, respectively.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Participants must have completed the Week 52 visit for 111 303 or 111-212 and have open epiphyses as assessed by left hand antero posterior (AP) X-rays, by the Greulich and Pyle method (Greulich 1971) as per standard of care
- 2. Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure.
- 3. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study.
- 4. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.
- 5. Participants are willing and able to perform all study procedures as physically possible.
- 6. Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training.
You likely can't join if
- 1. Permanently discontinued study treatment in the studies 111-303 or 111-212.
- 10. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
- 2. Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care.
- 3. Taking or planning to take any of the prohibited medications listed in Section 6.4.
- 4. Planned or expected to have limb-lengthening surgery during the study period.
- 5. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
See the full eligibility criteria
- 1. Participants must have completed the Week 52 visit for 111 303 or 111-212 and have open epiphyses as assessed by left hand antero posterior (AP) X-rays, by the Greulich and Pyle method (Greulich 1971) as per standard of care
- 2. Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure.
- 3. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study.
- 4. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.
- 5. Participants are willing and able to perform all study procedures as physically possible.
- 6. Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training.
- 1. Permanently discontinued study treatment in the studies 111-303 or 111-212.
- 10. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
- 2. Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care.
- 3. Taking or planning to take any of the prohibited medications listed in Section 6.4.
- 4. Planned or expected to have limb-lengthening surgery during the study period.
- 5. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
- 6. Require any investigational agent prior to completion of study period.
- 7. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy
- 8. Have known hypersensitivity to vosoritide or its excipients.
- 9. Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- United States
- Australia
- Canada
- Japan
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, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; United States; Australia; Canada; Japan. 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 EU CTIS; the study team decides eligibility.