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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04035811




Registration number
NCT04035811
Ethics application status
Date submitted
20/07/2019
Date registered
29/07/2019
Date last updated
28/05/2024

Titles & IDs
Public title
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
Scientific title
Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
Secondary ID [1] 0 0
QBGJ398-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Achondroplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

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 (cm/year)
Timepoint [1] 0 0
Up to 2 years

Eligibility
Key inclusion criteria
Key

* Signed informed consent by study participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the study participant (when applicable)
* Aged 2.5 to <17 years at study entry
* Diagnosis of ACH
* Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures

Key
Minimum age
30 Months
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Have hypochondroplasia or short stature condition other than ACH (e.g. trisomy 21, pseudoachondroplasia, psychosocial short stature)
* In females, having had their menarche
* Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH
* Annualized height growth velocity =1.5 cm/year over a period =6 months prior to screening
* Current evidence of corneal or retinal disorder/keratopathy
* Current evidence of endocrine alterations of calcium/phosphorus homeostasis
* Have a concurrent disease or condition that in the view of the Investigator and/or Sponsor, may impact growth or where the treatment is known to impact growth.
* Significant abnormality in screening laboratory results.
* Have been treated with growth hormone, insulin-like growth factor 1 (IGF 1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time
* Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
* Have had previous guided growth surgery or limb-lengthening surgery within 12 months prior to screening.

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
12/08/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2026
Actual
Sample size
Target
250
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
- Parkville
Recruitment outside Australia
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United States of America
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California
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United States of America
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Colorado
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United States of America
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Delaware
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United States of America
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Maryland
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United States of America
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Missouri
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United States of America
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Ohio
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United States of America
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Tennessee
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United States of America
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Texas
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United States of America
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Wisconsin
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Argentina
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Buenos Aires
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Canada
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Alberta
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Canada
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Ontario
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Canada
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Quebec
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France
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Lyon
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France
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Paris
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France
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Toulouse
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Germany
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Magdeburg
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Italy
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Rome
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Norway
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Bergen
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Norway
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Oslo
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Singapore
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Singapore
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Spain
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Barcelona
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Spain
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Madrid
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Spain
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Málaga
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United Kingdom
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England
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United Kingdom
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Birmingham
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United Kingdom
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Glasgow
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United Kingdom
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London
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United Kingdom
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Manchester
Country [30] 0 0
United Kingdom
State/province [30] 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, multi-center, observational study in children 2.5 to \<17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.
Trial website
https://clinicaltrials.gov/study/NCT04035811
Trial related presentations / publications
Savarirayan R, De Bergua JM, Arundel P, McDevitt H, Cormier-Daire V, Saraff V, Skae M, Delgado B, Leiva-Gea A, Santos-Simarro F, Salles JP, Nicolino M, Rossi M, Kannu P, Bober MB, Phillips J 3rd, Saal H, Harmatz P, Burren C, Gotway G, Cho T, Muslimova E, Weng R, Rogoff D, Hoover-Fong J, Irving M. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. Ther Adv Musculoskelet Dis. 2022 Mar 21;14:1759720X221084848. doi: 10.1177/1759720X221084848. eCollection 2022.
Public notes

Contacts
Principal investigator
Name 0 0
QED Therapeutics, Inc. VP, Clinical Development
Address 0 0
QED Therapeutics
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
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04035811