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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06410976




Registration number
NCT06410976
Ethics application status
Date submitted
8/05/2024
Date registered
13/05/2024
Date last updated
25/06/2024

Titles & IDs
Public title
Prospective Clinical Assessment Study in Children With Hypochondroplasia
Scientific title
Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL
Secondary ID [1] 0 0
QBGJ398-004
Universal Trial Number (UTN)
Trial acronym
HCH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypochondroplasia 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Normal musculoskeletal and cartilage development and function
Reproductive Health and Childbirth 0 0 0 0
Complications of newborn

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annualized height velocity
Timepoint [1] 0 0
up to 2 years
Secondary outcome [1] 0 0
Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio
Timepoint [1] 0 0
up to 2 years
Secondary outcome [2] 0 0
Cognitive functions
Timepoint [2] 0 0
up to 2 years
Secondary outcome [3] 0 0
HCH-related medical events reported as medical history or NT-AEs
Timepoint [3] 0 0
up to 2 years
Secondary outcome [4] 0 0
HCH-related surgical procedures
Timepoint [4] 0 0
up to 2 years
Secondary outcome [5] 0 0
Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form.
Timepoint [5] 0 0
up to 2 years

Eligibility
Key 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.
Minimum age
30 Months
Maximum age
16 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/06/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2026
Actual
Sample size
Target
150
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
Tennessee
Country [7] 0 0
United States of America
State/province [7] 0 0
Wisconsin
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
France
State/province [10] 0 0
Bron
Country [11] 0 0
France
State/province [11] 0 0
Paris
Country [12] 0 0
France
State/province [12] 0 0
Toulouse
Country [13] 0 0
Germany
State/province [13] 0 0
Baden-Wurtemberg
Country [14] 0 0
Germany
State/province [14] 0 0
Sachsen-Anhalt
Country [15] 0 0
Norway
State/province [15] 0 0
Bergen
Country [16] 0 0
Norway
State/province [16] 0 0
Oslo
Country [17] 0 0
Portugal
State/province [17] 0 0
Coimbra
Country [18] 0 0
Singapore
State/province [18] 0 0
Singapore
Country [19] 0 0
Spain
State/province [19] 0 0
Vitoria-gasteiz
Country [20] 0 0
Sweden
State/province [20] 0 0
Solna
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Bristol
Country [22] 0 0
United Kingdom
State/province [22] 0 0
London
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Manchester
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
QED Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).
Trial website
https://clinicaltrials.gov/study/NCT06410976
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
QED Therapeutics, Inc
Address 0 0
Country 0 0
Phone 0 0
1-877-280-5655
Fax 0 0
Email 0 0
medinfo@qedtx.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06410976