Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
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| ClinicalTrials.gov Identifier: NCT04035811 |
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Recruitment Status :
Recruiting
First Posted : July 29, 2019
Last Update Posted : April 8, 2022
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Sponsor:
QED Therapeutics, Inc.
Information provided by (Responsible Party):
QED Therapeutics, Inc.
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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 |
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| Achondroplasia |
| 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 |
| Actual Study Start Date : | August 12, 2019 |
| Estimated Primary Completion Date : | June 2026 |
| Estimated Study Completion Date : | June 2026 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Achondroplasia
Primary Outcome Measures :
- Annualized height velocity [ Time Frame: Up to 2 years ]
Secondary Outcome Measures :
- 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 ]
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
| Contact: QED Therapeutics | 1-877-280-5655 | PROPELstudyinfo@QEDTX.com |
Locations
Show 20 study locations
Sponsors and Collaborators
QED Therapeutics, Inc.
Investigators
| Study Director: | QED Therapeutics VP, Clinical Development | QED Therapeutics |
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | QED Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT04035811 |
| Other Study ID Numbers: |
QBGJ398-001 |
| First Posted: | July 29, 2019 Key Record Dates |
| Last Update Posted: | April 8, 2022 |
| Last Verified: | April 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by QED Therapeutics, Inc.:
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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 |