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Looking for participantsPhase2

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

Sponsor: QED Therapeutics, a BridgeBio company

NCT ID: NCT07169279

View on ClinicalTrials.gov ↗

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.

Achondroplasia

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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

FGFR

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
Inclusion Criteria: * Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report from a certified laboratory, documenting the specific mutation. * Age 0 to 32 months (2 years and 8 months) at screening. * Signed informed consent, 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 guidance for the management of infants and young children with ACH. * Able to swallow age-appropriate oral medication. * In participants \<1 year old, be compliant with recommended vitamin D supplementation of 5 10 μg/day or higher (or as recommended by country specific guidelines). Exclusion Criteria: * Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition that can affect growth. * Gestational age at birth \<37 weeks and/or birth weight \<2500 grams. * Gastroesophageal reflux disease requiring prolonged treatment (\>1 week) with prohibited medications. * Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed during MRI done at screening or a previous MRI done at any time if the participant had not undergone decompression surgery. * History of fracture of a long bone or spine within 6 months prior to screening. * Any other significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib and/or would require treatment with a prohibited medication (per protocol), and/or would place the participant at high risk for poor treatment compliance or for failure to complete the study. * Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature, including (but not limited to) r-hGH, IGF-1, CNP analog, FGF ligand trap, or treatment targeting FGFR inhibition at any time. * Regular long-term (\>3 weeks; more than twice/year) treatment with supraphysiologic doses of glucocorticoid therapy (ie, \>15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (for over 3 weeks within 6 months of the screening visit. NOTE: Low-dose topical, inhaled, or intranasal corticosteroids are acceptable. * Significant abnormality in screening laboratory results, * Allergy or hypersensitivity to any components of the study drug.

Where Is This Study? (3 UK sites)

NHS Greater Glasgow and Clyde

Glasgow G3 8SJ, United Kingdom

Recruiting
Hospital R&D contact (matched)

Radek Penar

radoslaw.penar@nhs.scotn/a

Manchester University NHS Foundation Trust

Manchester M13 9WL, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

Sheffield Children's NHS Foundation Trust

Sheffield S10 2TH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Sarah Flude

scn-tr.research.governance@nhs.net0114 226 7980

How to Get in Touch

QED Therapeutics Inc.

Sponsor contact

CONTACT

18772805655 medinfo@qedtx.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-07