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Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04035811
Recruitment Status : Recruiting
First Posted : July 29, 2019
Last Update Posted : March 31, 2020
Sponsor:
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 March 31, 2020
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
Change History
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 * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
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
Removed Location Countries  
 
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
Responsible Party QED Therapeutics, Inc.
Study Sponsor QED Therapeutics, Inc.
Collaborators Not Provided
Investigators
Study Director: QED Therapeutics Senior Medical Director QED Therapeutics
PRS Account QED Therapeutics, Inc.
Verification Date August 2019