At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Infigratinib is provided as a single dose of minitablets for oral administration (drug), Infigratinib is provided as sprinkle capsules for daily oral administration (drug), Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration (drug), Infigratinib is provided as sprinkle capsules for daily oral administration (drug)
- How long the study runs
- Study runs about 76 months (dates as stated)
- About the drug or intervention
- Infigratinib is provided as a single dose of minitablets for oral administration — drug: * The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria. · Infigratinib is provided as sprinkle capsules for daily oral administration — drug: * The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group. · Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration — drug: * The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group. · Infigratinib is provided as sprinkle capsules for daily oral administration — drug: * The dose of infigratinib will be the dose confirmed in the Phase 2 portion and used in the Phase 2b portion for the age group.
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a medicine called infigratinib in babies and toddlers under 3 years old who have achondroplasia, a genetic condition that affects bone growth and causes short stature. The study is being run by QED Therapeutics, a BridgeBio company. Its purpose is stated in the title as an interventional study of infigratinib in children with achondroplasia; further details about the study's aims are not stated — ask the trial team.
Who can take part
- Diagnosis of achondroplasia confirmed by genetic testing from a certified laboratory
- Aged 0 to 32 months (2 years and 8 months) at screening
- Parent or legal guardian has given informed consent
- Parents or guardians can attend all study visits and follow all study requirements
- Parents or guardians can follow routine care guidance for infants and young children with achondroplasia
- Able to swallow age-appropriate medicine given by mouth
- Babies under 1 year old must be taking recommended vitamin D supplements (5–10 micrograms a day or more, or as advised by national guidelines)
Who may not be able to
- Hypochondroplasia, another genetic condition, or any condition that can affect growth
- Born before 37 weeks of pregnancy and/or weighing under 2,500 grams at birth
- Severe reflux (gastro-oesophageal reflux disease) needing more than 1 week of treatment with medicines not allowed in the study
- Evidence of compression at the top of the spine (cervicomedullary compression), based on an MRI scan (magnetic resonance imaging), whether or not there are symptoms
- A broken long bone or spine bone within 6 months before screening
- Any other significant illness or condition that, in the view of the researcher or sponsor, could affect the study results, need treatments not allowed in the study, or make it hard to complete the study
- Having had, or planning to have, any other investigational or approved treatment for achondroplasia or short stature — including growth hormone, IGF-1 (insulin-like growth factor 1), CNP (C-type natriuretic peptide) analogues, FGF (fibroblast growth factor) ligand traps, or treatments targeting FGFR (fibroblast growth factor receptor)
- Long-term treatment with high-dose steroid medicines (glucocorticoids), or anti-inflammatory steroid doses for over 3 weeks within 6 months of screening — note that low-dose steroid creams, inhalers, or nasal sprays are acceptable
- Significantly abnormal results on screening blood tests
- Allergy or hypersensitivity to any part of the study medicine
What taking part involves
- • Taking infigratinib, a medicine given by mouth
- • Details about the dose, how often it is taken, and the treatment length are not stated — ask the trial team
Time commitment: Not stated — ask the trial team about how many visits there are, how long the study lasts, and what taking part involves; parents or guardians must be able to attend all study visits and comply with all study requirements.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 0 Years to 32 Months
- Who
- All
- Number of participants
- 77
- Started
- 2025-11-19
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for achondroplasia
- • Phase2 - 77 participants
- • This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH
Who can take part?
- • Ages 0 Years to 32 Months
- • Diagnosed with achondroplasia
Where?
- • Glasgow - NHS Greater Glasgow and Clyde
- • Manchester - Manchester University NHS Foundation Trust
- • Sheffield - Sheffield Children's NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \< 3 years old with ACH at the selected dose.
More detail
PROPEL Infant \& Toddler (I\&T) is a Phase 2, multicenter, randomized, placebo-controlled study that comprises 4 portions: the single ascending dose (SAD) portion (open-label), the Phase 2 portion (open-label), the Phase 2b portion (placebo-controlled), and an Extension Portion (open-label). The study will evaluate children with ACH \< 3 years old being administered oral infigratinib.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 0 Years - 32 Months
- Who can join: All genders
Biomarkers mentioned
What the study is looking for
- ✓Age 0 to 32 months (2 years and 8 months) at screening.
- ✓Signed agreement to take part, which must be obtained from each participant's parent(s) or legal guardian.
- ✓Parent(s)/Guardian(s) willing and able to attend all study visits and comply with all study requirements.
- ✓Parent(s)/Guardian(s) willing and able to comply with the routine care of the study participants according to local...
- ✓Able to swallow age-appropriate oral medication.
Who cannot take part
- ✗Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition...
- ✗Gestational age at birth \<37 weeks and/or birth weight \<2500 grams.
- ✗Gastroesophageal reflux disease requiring prolonged treatment (\>1 week) with prohibited medications.
- ✗History of fracture of a long bone or spine within 6 months prior to screening.
- ✗Significant abnormality in screening laboratory results,
See the full criteria
Where Is This Study? (3 UK sites)
NHS Greater Glasgow and Clyde
Glasgow G3 8SJ, United Kingdom
Manchester University NHS Foundation Trust
Manchester M13 9WL, United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield S10 2TH, United Kingdom
How to Get in Touch
QED Therapeutics Inc.
Sponsor contactCONTACT
