Open-Label, Long-Term, Extension Study 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]

Decision date (initial)

2026-04-15

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

QED Therapeutics Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Pharmacokinetic

To evaluate the safety and tolerability of long-term, once-daily oral administration of infigratinib for the treatment of participants living with HCH. To evaluate the efficacy of long-term, once-daily oral administration infigratinib in participants with HCH assessed by changes over time in standing height Z-score.

Secondary objectives 5

1. To evaluate changes over time in other indicators of growth and development.
2. To evaluate changes over time in X functions.
3. To evaluate changes over time in comorbidities associated with HCH.
4. To evaluate changes over time in Health- related Quality of Life.
5. To evaluate treatment benefit as assessed by qualitative interviews by the participant and caregiver.

Conditions and MedDRA coding

Hypochondroplasia

Study design 1 period

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 7

1. Inclusion Criteria for Participants Rolling Over from ACCEL 2/3: 1. Pediatric participants with HCH who have completed ACCEL 2/3.
2. Inclusion Criteria for Participants Rolling Over from ACCEL 2/3: 2. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
3. Inclusion Criteria for Participants Rolling Over from ACCEL: 1. Participant must be 3 to <18 years of age at screening and have growth potential (defined as X in females and X in males).
4. Inclusion Criteria for Participants Rolling Over from ACCEL: 2. Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
5. Inclusion Criteria for Participants Rolling Over from ACCEL: 3. Participants have at least a 6-month period of growth assessment in the ACCEL study before study entry.
6. Inclusion Criteria for Participants Rolling Over from ACCEL: 4. Participants are ambulatory and able to stand without assistance.
7. Inclusion Criteria for Participants Rolling Over from ACCEL: 5. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.

Exclusion criteria 14

1. Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 1. Participant has concurrent medical condition (ie, circumstance, syndrome, symptom, sign, etc) that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.
2. Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 2. Current evidence of clinically significant X
3. Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 3. Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication.
4. Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 4. Participants who prematurely discontinued ACCEL 2/3.
5. Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 5. Participants who have reached final height or near final height (AHV X for a minimum 6-month observation period and X for males and X for females).
6. Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 6. Current participation in an ongoing clinical study with a sponsor other than QED.
7. Exclusion Criteria for Participants Rolling Over from ACCEL: 1. Participants who have a clinically significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the participant or would interfere with study participation or safety evaluations. Complete list referenced in protocol.
8. Exclusion Criteria for Participants Rolling Over from ACCEL: 2. Participants who have a history and/or current evidence of extensive ectopic tissue calcification.
9. Exclusion Criteria for Participants Rolling Over from ACCEL: 3. Participants who have a history of malignancy.
10. Exclusion Criteria for Participants Rolling Over from ACCEL: 4. Currently receiving treatment with agents that are known strong X or prolonged treatment (>1 week) with medications that alter the pH of the gastrointestinal tract.
11. Exclusion Criteria for Participants Rolling Over from ACCEL: 5. Participants receiving medications which could increase X
12. Exclusion Criteria for Participants Rolling Over from ACCEL: 6. Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid therapy or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit.
13. Exclusion Criteria for Participants Rolling Over from ACCEL: 7. Having had a fracture of the long bones or spine within 12 months of screening.
14. Exclusion Criteria for Participants Rolling Over from ACCEL: 8. Current participation in an ongoing clinical study with a sponsor other than QED.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Incidence, type, severity, and causality of AEs; SAEs; AEs that require dose reduction or discontinuation per study protocol; and changes over time in vital signs, laboratory assessments and special investigations (ophthalmic and dental evaluations), and imaging (x-rays, , dual x-ray absorptiometry [DXA] scan).
2. Changes over time in standing height Z-score (in relation to both HCH and average height tables for age and sex).

Secondary endpoints 5

1. Changes over time in AHV and AHV Z-score in relation to average height tables for age and in body proportions including upper to lower body segment ratio, upper arm to forearm length ratio, upper leg to lower leg length ratio, arm span to standing height ratio, and head circumference to standing height ratio, in weight Z-score and BMI in relation to average height tables (and HCH tables, if and when they become available), age of puberty onset and time to X, in body composition
2. Change over time in various quality of life scales including Pediatric Quality of Life, Quality of Life in Short Stature Youth, Patient/ Parent Global Impression of Severity and Change
3. Treatment benefit as reported by parent and participant during qualitative interview.
4. Changes over time in body composition as assessed by dual x-ray absorptiometry (DXA) scans.
5. Changes over time in bone morphology/density by x-ray and DXA.

Investigational products

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

Test 5

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 12

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Results and documents

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

Documents 55 files

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

Application history

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