QED - Propel trial (21st Oct 2019 update)
Prospective Clinical Assessment Study in Children With Achondroplasia - The PROPEL trial
Update - 21st Oct 2019
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.
All content in ClinicalTrials.gov identifier (NCT number): NCT04035811
Study Type : | Observational (no drug is administered) |
Estimated Enrollment : | 200 participants |
Observational Model: | Case-Only |
Time Perspective: | Prospective |
Official Title: | Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial |
Actual Study Start Date : | August 12, 2019 |
Estimated Primary Completion Date : | June 2026 |
Estimated Study Completion Date : | June 2026 |
Primary Outcome Measures (main objective)
- Annualized height velocity [ Time Frame: Up to 2 years ]
Secondary Outcome Measures :
- Collection of the 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 ]
Biospecimen Retention: Samples With DNA
Collect blood for central assessment of blood biomarkers
Eligibility criteria
Ages Eligible for Study: | 30 Months (2,5years) to 10 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Probability Sample |
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
Contacts
QED Therapeutics Senior Medical Director | 650-231-4088 | This email address is being protected from spambots. You need JavaScript enabled to view it. |
Clinical centers where the trial is taking place
United States, California | |
Benioff Children's Hospital Oakland | Not yet recruiting |
Oakland, California, United States, 94609 | |
United States, Ohio | |
Cincinnati Children's Hospital | Not yet recruiting |
Cincinnati, Ohio, United States, 45229 | |
Australia | |
Murdoch Children's Research Institute | Recruiting |
Parkville, Australia | |
United Kingdom | |
Birmingham Children's Hospital | Recruiting |
Birmingham, United Kingdom | |
Queen Elizabeth University Hospital | Not yet recruiting |
Glasgow, United Kingdom | |
Guy's and Saint Thomas' NHS Foundation Trust | Not yet recruiting |
London, United Kingdom | |
Manchester University Children's Hospital | Recruiting |
Manchester, United Kingdom | |
Sheffield Children's Hospital | Recruiting |
Sheffield, United Kingdom |