Prospective Clinical Assessment Study in Children With Achondroplasia: The PROPEL Trial

Activo, no reclutaClinicalTrials.gov

ID: NCT04035811Tipo: OBSERVATIONALInicio: 12 de ago de 2019Fin estimado: 1 de jun de 2026

Traducción no disponible, mostrando original

Resumen

This is a long-term, multi-center, observational study in children 2.5 to \<17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life, body pain, functional abilities, cognitive functions, and treatments of study participants. No study medication will be administered.

Elegibilidad

Edad mínima: 30 MonthsEdad máxima: 17 YearsSexo: ALL

Criterios de inclusión

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 \<17 years at study entry
Diagnosis of ACH
Study participants and parent(s) or LAR(s) are willing and able to comply with study visits and study procedures

Criterios de exclusión

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
Current evidence of corneal or retinal disorder/keratopathy
Current evidence of endocrine alterations of calcium/phosphorus homeostasis
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 had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
Have had previous guided growth surgery or limb-lengthening surgery within 12 months prior to screening.

Ubicaciones

Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan

Buenos Aires, Argentina

Patrocinadores

PrincipalQED Therapeutics, a BridgeBio company

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.

Prospective Clinical Assessment Study in Children With Ac... | EligiMed