Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

• ClinicalTrials.gov Identifier: NCT04035811
• Recruitment Status: Recruiting
• First Posted: July 29, 2019
• Last Update Posted: April 8, 2022
• Sponsor: QED Therapeutics, Inc.
• Information provided by (Responsible Party): QED Therapeutics, Inc.

Tracking Information

• First Submitted Date: July 20, 2019
• First Posted Date: July 29, 2019
• Last Update Posted Date: April 8, 2022
• Actual Study Start Date: August 12, 2019
• Estimated Primary Completion Date: June 2026 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: July 24, 2019): Annualized height velocity [ Time Frame: Up to 2 years ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: July 24, 2019)

• Collection of natural history of achondroplasia symptoms in children with achondroplasia [ Time Frame: Up to 2 years ]
• To characterize achondroplasia symptoms in children with achondroplasia [ Time Frame: Up to 2 years ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
• Official Title: Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
• Brief Summary: This is a long-term, multi-center, observational study in children 2.5 to 10 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications and treatments of study participants. No study medication will be administered.
• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Case-Only; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided

Biospecimen

Retention:   Samples With DNA

Description:

Collect blood for central assessment of blood biomarkers

• Sampling Method: Probability Sample
• Study Population: Children with achondroplasia
• Condition: Achondroplasia
• Intervention: Not Provided
• Study Groups/Cohorts: Not Provided
• 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.

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: July 24, 2019): 200
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: June 2026
• Estimated Primary Completion Date: June 2026 (Final data collection date for primary outcome measure)

Eligibility Criteria

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 10 years (inclusive) at study entry
• Diagnosis of ACH
• Ambulatory and able to stand without assistance
• Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures

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
• 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 received a C-type natriuretic peptide (CNP) analog or treatment targeting fibroblast growth factor receptor (FGFR) inhibition at any time
• Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
• Have used any other investigational product or investigational medical device for the treatment of ACH or short stature
• Have had previous limb-lengthening surgery

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 30 Months to 10 Years (Child)
• Accepts Healthy Volunteers: No

Contacts

Contact: QED Therapeutics; 1-877-280-5655; PROPELstudyinfo@QEDTX.com

• Listed Location Countries: Australia, Canada, France, Spain, United Kingdom, United States

Administrative Information

• NCT Number: NCT04035811
• Other Study ID Numbers: QBGJ398-001
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: QED Therapeutics, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: QED Therapeutics, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: QED Therapeutics VP, Clinical Development; QED Therapeutics

• PRS Account: QED Therapeutics, Inc.
• Verification Date: April 2022