Prospective Clinical Assessment Study in Children With Hypochondroplasia

Hypochondroplasia Clinical Trial

— HCH  

Official title:

Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06410976
• Other study ID #: QBGJ398-004
• Status: Recruiting
• Start date: June 5, 2024
• Est. completion date: October 2026

Study information

• Verified date: June 2024
• Source: QED Therapeutics, Inc.
• Contact: QED Therapeutics, Inc
• Phone: 1-877-280-5655
• Email: medinfo[@]qedtx.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).

Description:

The objective is to evaluate growth, HCH-related medical complications, health-related quality of life, functional abilities and cognitive functions of study participants. Data collected will contribute to the characterization of the natural history of children with HCH. No study medication will be administered.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: October 2026
• Est. primary completion date: October 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 30 Months to 16 Years

Eligibility

Inclusion Criteria:

Signed informed consent. Aged 2.5 to <17 years at study entry. Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test. Participants are ambulatory and able to stand without assistance. Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. Exclusion Criteria: Have ACH or short stature condition other than HCH. In females, having had their menarche. Annualized height growth velocity =1.5 cm/year over a period =6 months prior to screening. Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant. Clinically significant abnormality in any laboratory test result at screening Have been treated with growth hormone, IGF 1, or anabolic steroids the previous 6 months or long-term treatment (>3 months) at any time. Current evidence of corneal or retinal disorders. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature. Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable). Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening. Having had a fracture of the long bones or spine within 12 months of screening. History and/or current evidence of extensive ectopic tissue calcification. History of malignancy. Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation, and/or would place the participant at high risk for poor compliance with study activities or for not completing the study.

Related Conditions & MeSH terms

• Dwarfism
• Hypochondroplasia
• Lordosis

Locations

Country	Name	City	State
Australia	QED Investigative Site	Parkville	Victoria
Canada	QED Investigative Site	London	Ontario
Canada	QED Investigative Site	Montréal	Quebec
Canada	QED Investigative Site	Ottawa	Ontario
France	QED Investigative Site	Bron
France	QED Investigative Site	Paris
France	QED Investigative Site	Toulouse
Germany	QED Investigative Site	Freiburg	Baden-Wurtemberg
Germany	QED Investigative Site	Magdeburg	Sachsen-Anhalt
Norway	QED Investigative Site	Bergen
Norway	QED Investigative Site	Oslo
Portugal	QED Investigative Site	Coimbra
Singapore	QED Investigative Site	Singapore
Spain	QED Investigative Site	Vitoria-gasteiz
Sweden	QED Investigative Site	Solna
United Kingdom	QED Investigative Site	Bristol
United Kingdom	QED Investigative Site	London
United Kingdom	QED Investigative Site	Manchester
United Kingdom	QED Investigative Site	Sheffield	South Yorkshire
United States	QED Investigative Site	Aurora	Colorado
United States	QED Investigative Site	Baltimore	Maryland
United States	QED Investigative Site	Columbia	Missouri
United States	QED Investigative Site	Madison	Wisconsin
United States	QED Investigative Site	Nashville	Tennessee
United States	QED Investigative Site	Oakland	California
United States	QED Investigative Site	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
QED Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Germany,  Norway,  Portugal,  Singapore,  Spain,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change from BL in collagen X marker concentration (ug/L).		up to 2 years
Primary	Annualized height velocity		up to 2 years
Secondary	Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio		up to 2 years
Secondary	Cognitive functions		up to 2 years
Secondary	HCH-related medical events reported as medical history or NT-AEs		up to 2 years
Secondary	HCH-related surgical procedures		up to 2 years
Secondary	Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form.	Scale scores 0-100. Higher score=better Health-Related Quality of Llife	up to 2 years