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Trial record 6 of 20 for:    achondroplasia

Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

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

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
Achondroplasia

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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial
Estimated Study Start Date : July 2019
Estimated Primary Completion Date : June 2026
Estimated Study Completion Date : June 2026





Primary Outcome Measures :
  1. Annualized height velocity [ Time Frame: Up to 2 years ]

Secondary Outcome Measures :
  1. Collection of natural history of achondroplasia symptoms in children with achondroplasia [ Time Frame: Up to 2 years ]
  2. 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


Information from the National Library of Medicine

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Ages Eligible for Study:   30 Months to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Children with achondroplasia
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04035811


Contacts
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Contact: QED Therapeutics Senior Medical Director 650-231-4088 PROPELtrialinfo@QEDTX.com

Locations
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Australia
Murdoch Children's Research Institute Recruiting
Parkville, Australia
Sponsors and Collaborators
QED Therapeutics, Inc.
Investigators
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Study Director: QED Therapeutics Senior Medical Director QED Therapeutics

Additional Information:
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Responsible Party: QED Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04035811     History of Changes
Other Study ID Numbers: QBGJ398-001
First Posted: July 29, 2019    Key Record Dates
Last Update Posted: July 29, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Keywords provided by QED Therapeutics, Inc.:
Skeletal dysplasia
Endochondral ossification
ACH
Shortened proximal limbs
Fibroblast growth factor receptor 3
FGFR3
Endochondral bone formation
Short-limb disproportionate dwarfism
Bone disease
Dwarfism
Bone diseases
Musculoskeletal diseases
Osteochondrodysplasia
Genetic diseases
Inborn

Additional relevant MeSH terms:
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Achondroplasia
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Osteochondrodysplasias
Genetic Diseases, Inborn