BridgeMD
Open-Label followed by a Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Infigratinib in Children with Hypochondroplasia
EU CTIS ID: 2024-516822-67-00
What this study is testing
ACCEL 2: To obtain preliminary evidence of the efficacy of infigratinib in pediatric participants 5 to 11 years of age with HCH who have short stature. ACCEL 2: To evaluate the safety and tolerability of infigratinib in children with HCH. ACCEL 3: To evaluate the efficacy of infigratinib in pediatric participants 3 to <18 years of age with HCH who have short stature with potential to grow.
- Phase II and Phase III (Integrated)
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
- Participants must have completed the Week 26 visit in the observational study (QBGJ398-004).
- Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
- If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception while taking study drug and for 30 days after the last dose of study drug.
- Signed informed consent.
- ACCEL 2: Participants 5-11 years of age (inclusive).
- Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
You likely can't join if
- Participants who have ACH or a short stature condition other than HCH. Participants with variants in FGFR3 known to cause ACH or other FGFR3-related conditions will be excluded.
- Children with epilepsy who meet certain additional criteria.
- Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib. Complete list referenced in protocol.
- Current evidence of clinically significant corneal or retinal disorder/keratopathy.
- Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations.
- History and/or current evidence of extensive ectopic tissue calcification.
See the full eligibility criteria
- Participants must have completed the Week 26 visit in the observational study (QBGJ398-004).
- Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
- If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception while taking study drug and for 30 days after the last dose of study drug.
- Signed informed consent.
- ACCEL 2: Participants 5-11 years of age (inclusive).
- Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
- AHV >X cm/year over a period ≥26 weeks prior to screening based on measurements obtained during observational Study QBJG398-004 (ACCEL) (Phase 2 portion only). NOTE: Visit window may be applied to the 26-week period of the observational ACCEL study as per the ACCEL SoA (ie. the Week 26 visit in ACCEL may occur slightly earlier than 26 weeks).
- Participants are able to swallow oral medication.
- Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
- Participants are ambulatory and able to stand without assistance.
- Participants who have ACH or a short stature condition other than HCH. Participants with variants in FGFR3 known to cause ACH or other FGFR3-related conditions will be excluded.
- Children with epilepsy who meet certain additional criteria.
- Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib. Complete list referenced in protocol.
- Current evidence of clinically significant corneal or retinal disorder/keratopathy.
- Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations.
- History and/or current evidence of extensive ectopic tissue calcification.
- History of malignancy.
- Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature.
- Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit.
- Current participation in any other ongoing clinical study with other sponsor.
- Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study; or previous guided growth surgery with plates still in place or removed within the 12 months prior to screening.
- Currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP)3A or prolonged treatment (>1 week) with medications that alter the pH of the gastrointestinal tract.
- Participants receiving medications which could increase serum phosphorus and/or calcium concentrations
- Clinically significant abnormality in any laboratory test result at screening as specified in the protocol.
- Having had a fracture of the long bones or spine within 12 months prior to screening.
- Females who have had their menarche (ACCEL 2 only).
- Pregnant or breastfeeding at the screening visit.
- Allergy to any components of the study drug.
The study team makes the final eligibility decision.
Where it's taking place
- Singapore
- Australia
- United Kingdom
- Canada
- United States
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 Singapore; Australia; United Kingdom; Canada; United States. 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.