Authorised Therapeutic exploratory (Phase II) Hypochondroplasia

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children with Hypochondroplasia, Aged 0 to < 36 Months

EU CTIS ID: 2024-512261-14-00

What this study is testing

To evaluate the safety and tolerability of vosoritide versus placebo in children with HCH aged 0 to < 36 months To evaluate the effect on linear growth of vosoritide versus placebo

Therapeutic exploratory (Phase II)

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 be 0 to < 36 months of age at randomization.
2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH). Genetic confirmation of disease can be obtained either in Study 111-902 or during the Screening period of 111-212 (Appendix 4).
3. Participants aged 0 to < 12 months must have a height Z-score of ≤ −1.0 SDS and participants 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 age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts (https://www.cdc.gov/growthcharts/zscore.htm) as assessed at Screening.
4. Participant’s weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.
5. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure.
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. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
18. Have known hypersensitivity to vosoritide or its excipients.
19. Have a history of hip surgery or severe hip dysplasia.
2. Have any of the following: • Hypothyroidism or hyperthyroidism, growth hormone deficiency, hypercortisolism or hypopituitarism, or other endocrine cause of short stature. • Insulin-requiring diabetes mellitus. • Autoimmune inflammatory disease (e.g., systemic lupus erythematosus, juvenile dermatomyositis, scleroderma). • Other chronic diseases that per investigator determination may be causative of a participant’s short stature, including conditions causing malnutrition (e.g., inflammatory bowel disease, cystic fibrosis, celiac disease, eating disorders). • Autonomic neuropathy.
20. Have a history of clinically significant hip injury in the 30 days prior to Screening.
21. Have a history of slipped capital femoral epiphysis or avascular necrosis of the femoral head.

See the full eligibility criteria

Who can join

1. Participants must be 0 to < 36 months of age at randomization.
2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH). Genetic confirmation of disease can be obtained either in Study 111-902 or during the Screening period of 111-212 (Appendix 4).
3. Participants aged 0 to < 12 months must have a height Z-score of ≤ −1.0 SDS and participants 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 age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts (https://www.cdc.gov/growthcharts/zscore.htm) as assessed at Screening.
4. Participant’s weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.
5. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure.
6. Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training.

What rules you out

1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
18. Have known hypersensitivity to vosoritide or its excipients.
19. Have a history of hip surgery or severe hip dysplasia.
2. Have any of the following: • Hypothyroidism or hyperthyroidism, growth hormone deficiency, hypercortisolism or hypopituitarism, or other endocrine cause of short stature. • Insulin-requiring diabetes mellitus. • Autoimmune inflammatory disease (e.g., systemic lupus erythematosus, juvenile dermatomyositis, scleroderma). • Other chronic diseases that per investigator determination may be causative of a participant’s short stature, including conditions causing malnutrition (e.g., inflammatory bowel disease, cystic fibrosis, celiac disease, eating disorders). • Autonomic neuropathy.
20. Have a history of clinically significant hip injury in the 30 days prior to Screening.
21. Have a history of slipped capital femoral epiphysis or avascular necrosis of the femoral head.
22. Have abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant.
23. Have a condition or circumstance that places the participant at high risk for poor treatment compliance or for not completing the study.
24. Have any concurrent disease or condition that will interfere with study participation or safety evaluations, for any reason.
3. Have a history of any of the following: • Renal insufficiency defined as estimated glomerular filtration rate (eGFR) of < 60 ml/min/1.73 m2 using the revised Schwartz Pediatric Bedside eGFR formula. • Chronic anemia or hemoglobin (Hgb) < 10.0 g/dL (Screening lab test). • Recurrent symptomatic hypotension (i.e., dizziness, fainting, postural tachycardia) or recurrent symptomatic orthostatic hypotension.
4. History of cardiac or vascular disease, including the following: • Cardiac dysfunction • Hypertrophic cardiomyopathy • Pulmonary hypertension • Congenital heart disease with ongoing cardiac dysfunction • Cerebrovascular disease • Aortic insufficiency or other clinically significant valvular dysfunction • Clinically significant atrial or ventricular arrhythmia
10. Have had regular long-term treatment (> 1 month) with oral corticosteroids in the 12 months prior to Screening.
5. Have an unstable medical condition likely to require surgical intervention during the study period.
6. Have documented uncorrected vitamin D deficiency: 25-hydroxy-vitamin D ≤ 15 ng/mL (37.5 nmol/L). Note: participants with deficiency may receive supplementation and re-screen after 8 weeks.
7. Taking any of the prohibited medications.
8. Require current chronic therapy with antihypertensive medication or any medication that may compromise the safety or ability of the participant to participate in this clinical study.
9. 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.
Please see protocol for complete details.
11. Have condition(s) requiring a daily inhaled steroid dose > 400 µg of inhaled budesonide per day or equivalent. Low-dose ongoing inhaled steroids for asthma, or intranasal steroids, are acceptable.
12. Have had a fracture of the long bones or spine within 6 months prior to Screening.
13. Require any investigational agent prior to completion of study period.
14. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
15. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time
16. Have aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥ 3 × upper limit of normal (ULN or total bilirubin ≥ 1.5 × ULN at screening (except for participants with a known history of Gilberts).
17. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.

The study team makes the final eligibility decision.

Where it's taking place

United Kingdom
Australia
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; Australia; Japan. Enter your location above to see the nearest site and check your eligibility.

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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.