A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combi… (NCT06433557) | Clinical Trial Compass
Active — Not RecruitingPhase 2
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia
Denmark, Ireland, United Kingdom22 participantsStarted 2024-07-26
Plain-language summary
This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.
Who can participate
Age range2 Years – 11 Years
SexALL
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Inclusion criteria
✓. Written, signed informed consent and/or assent (if applicable) by the parent(s) or legal representative(s) of the participant, and as required by the IRB/HREC/IEC.
✓. Male or female between 2 to 11 years of age (inclusive) at the time of Visit 1.
✓. Clinical diagnosis of ACH with genetic confirmation of heterozygote genotype present at Visit 1. Documentation of historic test results are acceptable for proof of diagnosis.
✓. Able to stand without assistance.
✓. Parent(s)/caregiver(s)/legal guardian(s) willing and able to administer weekly SC injections of IMP and comply with all protocol requirements.
✓. At least 6 months of growth and disease history from TCC-NHS-01 or TCC-201 or comparable growth and disease history available from medical records.
✓. No intracranial pathology as confirmed by brain MRI (historical MRI obtained within 2 years prior to screening allowable).
Exclusion criteria
✕. Participation in any interventional clinical trial within three months prior to screening (except TCC-201 or ASND0039).
✕. Closed epiphysis at screening.
✕. History of or suspected hypersensitivity to the IMP or related products.
✕. Findings on fundoscopy at screening consistent with intracranial hypertension, papilledema, or evidence of any other retinal disease for which GH therapy is contraindicated.
✕. Have a growth disorder or medical condition other than ACH that results in short stature or abnormal growth such as severe ACH with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, GHD, Turner syndrome, pseudoachondroplasia, inflammatory bowel disease, celiac disease, hypothyroidism, hyperthyroidism, pre-diabetes, or diabetes mellitus.
✕. Have received any dose of prescription and/or investigational medications or device intended to affect stature, growth, or body proportionality at any time prior to screening.
✕. Receiving concurrent treatment with any agent that might influence growth or interfere with GH secretion or action:
✕. Inhaled corticosteroid therapy at a dose of \>400 µg/day of inhaled budesonide or equivalent for more than 28 consecutive days total over the course of 12 months prior to screening.