Open-Label followed by a Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Infigratinib in Children with Hypochondroplasia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Qed Therapeutics Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

14 Aug 2025 → ongoing

Decision date (initial)

2025-07-15

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Safety, Pharmacokinetic, Efficacy

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.

Secondary objectives 5

1. ACCEL 3 : To evaluate changes in other key indicators of growth and body proportions.
2. ACCEL 2 : To evaluate changes in cognitive functions.
3. ACCEL 2 : To evaluate changes in other indicators of growth and body proportions.
4. ACCEL 2 : To evaluate the PK profile of infigratinib and its active metabolites in children with HCH after administration of oral infigratinib.
5. ACCEL 2 : To evaluate CXM, an indicator of bone growth.

Conditions and MedDRA coding

Hypochondroplasia

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration, European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. Participants must have completed the Week 26 visit in the observational study (QBGJ398-004).
2. 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.
3. Signed informed consent.
4. ACCEL 2: Participants 5-11 years of age (inclusive).
5. Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
6. 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).
7. Participants are able to swallow oral medication.
8. Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
9. Participants are ambulatory and able to stand without assistance.
10. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.

Exclusion criteria 18

1. 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.
2. 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.
3. 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.
4. Participants receiving medications which could increase serum phosphorus and/or calcium concentrations
5. Clinically significant abnormality in any laboratory test result at screening as specified in the protocol.
6. Having had a fracture of the long bones or spine within 12 months prior to screening.
7. Females who have had their menarche (ACCEL 2 only).
8. Pregnant or breastfeeding at the screening visit.
9. Allergy to any components of the study drug.
10. Children with epilepsy who meet certain additional criteria.
11. 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.
12. Current evidence of clinically significant corneal or retinal disorder/keratopathy.
13. Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations.
14. History and/or current evidence of extensive ectopic tissue calcification.
15. History of malignancy.
16. Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature.
17. 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.
18. Current participation in any other ongoing clinical study with other sponsor.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. ACCEL 2: Change from baseline (BL) in height velocity (HV) at Week 26 (annualized to cm/year). ACCEL 3: Change from BL to Week 52 in AHV compared to placebo.

Secondary endpoints 2

1. ACCEL 2: Change from BL in height Z-score (in relation to both HCH and average height tables for age and sex) compared to placebo.
2. ACCEL 3: Change from BL to Week 52 in upper to lower body segment ratio, compared to placebo.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 5

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Qed Therapeutics Inc.

Sponsor organisation

Qed Therapeutics Inc.

Address

1800 Owens Street Ste C1200

City

San Francisco

Postcode

94158-2584

Country

United States

Scientific contact point

Organisation

Qed Therapeutics Inc.

Contact name

QED Clinical Development

Public contact point

Organisation

Qed Therapeutics Inc.

Contact name

Regulatory Affairs

Third parties 14

Locations

5 EU/EEA countries · 7 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 171 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications