Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Achondroplasia Clinical Trial

Official title:

Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04035811
• Other study ID #: QBGJ398-001
• Status: Recruiting
• Start date: August 12, 2019
• Est. completion date: June 2026

Study information

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

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: June 2026
• Est. primary completion date: June 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 30 Months to 17 Years

Eligibility

Key Inclusion Criteria:

• 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 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.

Related Conditions & MeSH terms

• Achondroplasia

Locations

Country	Name	City	State
Argentina	Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan	Buenos Aires
Australia	Murdoch Children's Research Institute	Parkville
Canada	Stollery Children's Hospital	Edmonton	Alberta
Canada	Children's Hospital - London Health Sciences Center	London	Ontario
Canada	University of Montreal	Montréal	Quebec
Canada	University of Ottawa	Ottawa	Ontario
France	Hopital Femme Mere Enfant	Lyon
France	Hopital Necker-Enfants Malades	Paris
France	Hopital des Enfants	Toulouse
Germany	Otto-von-Guericke-University Magdeburg Medical Fakulty	Magdeburg
Italy	Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS	Rome
Norway	Haukeland Universitetssjukehus	Bergen
Norway	Oslo Universitetssykehus	Oslo
Singapore	KK Women's and Children's Hospital	Singapore
Spain	Vithas Hospital San Jose	Barcelona
Spain	Hospital Universitario La Paz	Madrid
Spain	Hospital Universitario Virgen de la Victoria	Málaga
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	Bristol Royal Hospital for Children	Bristol	England
United Kingdom	Queen Elizabeth University Hospital	Glasgow
United Kingdom	St. Thomas' Hospital	London
United Kingdom	Manchester University Children's Hospital	Manchester
United Kingdom	Sheffield Children's Hospital	Sheffield
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Johns Hopkins School of Medicine	Baltimore	Maryland
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	University of Missouri	Columbia	Missouri
United States	Cook's Children Medical Center	Dallas	Texas
United States	University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic	Madison	Wisconsin
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Benioff Children's Hospital Oakland	Oakland	California
United States	Nemours Alfred I. Dupont Hospital for Children	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
QED Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Canada,  France,  Germany,  Italy,  Norway,  Singapore,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change from baseline in height Z score		Up to 2 years
Other	Change from baseline in upper to lower body ratio (cm)		Up to 2 years
Other	Change from baseline in upper arm to forearm ratio (cm)		Up to 2 years
Other	Change from baseline in upper leg to lower leg ratio (cm)		Up to 2 years
Other	Bone biomarkers (blood)		Up to 2 years
Other	ACH-related NT-AEs		Up to 2 years
Other	ACH-related surgical procedures		Up to 2 years
Other	Changes in health-related quality of life as assessed by Pediatric Quality of Life Inventory (PedsQoL)		Up to 2 years
Other	Changes in health-related quality of life as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)		Up to 2 years
Other	Changes in overall body pain as assessed by Numeric Rating Scale for pain (Pain-NRS)		Up to 2 years
Other	Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)		Up to 2 years
Other	Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test)		Up to 2 years
Other	Change in attention assessed by age-appropriate computerized tests (Identification Test)		Up to 2 years
Other	Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)		Up to 2 years
Other	Change in working memory assessed by age-appropriate computerized tests (One Back Test)		Up to 2 years
Primary	Annualized height velocity (cm/year)		Up to 2 years

External Links

•   Related Info