Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

Sponsor

QED Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04035811

Collaborator

(none)

200

Enrollment

Locations

81.6

Anticipated Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Achondroplasia

Study Design

Study Type:

Observational

Anticipated 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 :

Aug 12, 2019

Anticipated Primary Completion Date :

Jun 1, 2026

Anticipated Study Completion Date :

Jun 1, 2026

Outcome Measures

Primary Outcome Measures

Annualized height velocity [Up to 2 years]

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

30 Months to 10 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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 and Locations

Locations

	Site	City	State	Country	Postal Code
1	Benioff Children's Hospital Oakland	Oakland	California	United States	94609
2	Nemours Alfred I. Dupont Hospital for Children	Wilmington	Delaware	United States	19803
3	Johns Hopkins School of Medicine	Baltimore	Maryland	United States	21211
4	Cincinnati Children's Hospital	Cincinnati	Ohio	United States	45229
5	Vanderbilt University Medical Center	Nashville	Tennessee	United States	37232
6	Cook's Children Medical Center	Dallas	Texas	United States	75207
7	Murdoch Children's Research Institute	Parkville		Australia
8	Stollery Children's Hospital	Edmonton	Alberta	Canada	T6G 2H7
9	Hopital Femme Mere Enfant	Lyon		France
10	Hopital Necker-Enfants Malades	Paris		France
11	Hopital des Enfants	Toulouse		France
12	Vithas Hospital San Jose	Barcelona		Spain	08305
13	Hospital Universitario La Paz	Madrid		Spain	24086
14	Hospital Universitario Virgen de la Victoria	Málaga		Spain
15	Bristol Royal Hospital for Children	Bristol	England	United Kingdom
16	Birmingham Children's Hospital	Birmingham		United Kingdom
17	Queen Elizabeth University Hospital	Glasgow		United Kingdom
18	St. Thomas' Hospital	London		United Kingdom
19	Manchester University Children's Hospital	Manchester		United Kingdom
20	Sheffield Children's Hospital	Sheffield		United Kingdom