Phase 3 Clinical Trial Evaluating the Efficacy and Safety of Navepegritide and Lonapegsomatropin in Achondroplasia (cAtCH)
A Phase 3, Randomized, Double-blind, Active Controlled, Multi-center Trial Investigating the Efficacy and Safety of Dual Therapy With Once-weekly Navepegritide and Once-weekly Lonapegsomatropin Compared to Navepegritide Monotherapy and Lonapegsomatropin Monotherapy in Children and Adolescents With Achondroplasia
This pivotal trial is being conducted to evaluate the efficacy, safety and tolerability of dual therapy with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 17 years.
Studieöversikt
Status
Betingelser
Studietyp
Inskrivning (Beräknad)
Fas
- Fas 3
Kontakter och platser
Studiekontakt
- Namn: Ascendis Registry Inquiries
- Telefonnummer: +45 61242484
- E-post: asnd_registryinquiries@ascendispharma.com
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
Tar emot friska volontärer
Kön som är behöriga för studier
Beskrivning
Inclusion Criteria:
- 1. Age 2 to <18 years at randomization
- 2. Clinical diagnosis of ACH with historical genetic confirmation
- 3. At least 1 height measurement available from medical records
Exclusion Criteria:
- 1. Treatment with growth hormone within 1 year prior to randomization
- 2. Prior treatment with certain medication(s) which potentially could have affected growth (prior treatment with vosoritide is not exclusive)
- 3. History of any surgery intended to affect stature, growth, or body proportionality except procedures for leg bowing
- 4. Unable to stand without assistance
- 5. Known or suspected medical conditions other than achondroplasia that could affect growth
- 6. Known history or presence of injury of the growth plates that affects growth potential of long bones
- 7. Known history or presence of malignant disease
Studieplan
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Randomiserad
- Interventionsmodell: Parallellt uppdrag
- Maskning: Fyrdubbla
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Experimentell: Once weekly navepegritide SC and lonapegsomatropin SC
|
Administered weekly as two separate SC injections. Treatment duration of up to 104 weeks |
|
Experimentell: Once weekly navepegritide SC and placebo for lonapegsomatropin SC
|
Administered as two separate SC injections. Treatment duration of up to 52 weeks of navepegritide and placebo for lonapegsomatropin followed by 52 weeks of navepegritide and lonapegsomatropin. |
|
Experimentell: Once weekly lonapegsomatropin SC and placebo for navepegritide SC
|
Administered as two separate SC injections. Treatment duration of up to 52 weeks of lonapegsomatropin and placebo for navepegritide followed by 52 weeks of navepegritide and lonapegsomatropin. |
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Annualized Growth Velocity (AGV)
Tidsram: 52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to navepegritide alone |
52 weeks
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Treatment-emergent Adverse Events (AE)
Tidsram: Throughout trial duration 104 weeks
|
Number of events |
Throughout trial duration 104 weeks
|
|
Annualized Growth Velocity (AGV)
Tidsram: 52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to lonapegsomatroptin alone |
52 weeks
|
|
Annualized Growth Velocity (AGV) change from baseline
Tidsram: 52 weeks
|
To evaluate the change from baseline in AGV, cm per year |
52 weeks
|
|
ACH-specific height Z-score
Tidsram: 52 weeks
|
To evaluate the change from baseline in ACH-specific height Z-score |
52 weeks
|
|
CDC-based height Z-score
Tidsram: 52 weeks
|
To evaluate the change from baseline in CDC-based height Z-score |
52 weeks
|
|
Bone safety
Tidsram: Throughout trial duration 104 weeks
|
Qualitative assessment with radiology |
Throughout trial duration 104 weeks
|
|
Arm span Z-score
Tidsram: 52 weeks
|
Change from baseline in arm span Z-score. |
52 weeks
|
|
Upper to lower body segment ratio
Tidsram: 52 weeks
|
Change from baseline in upper to lower body segment ratio. |
52 weeks
|
|
Arm span
Tidsram: 52 weeks
|
Change from baseline in arm span, cm. |
52 weeks
|
|
Interpedicular distance (IDP)
Tidsram: 52 weeks
|
Change from baseline in IDP (L1-L4 average), mm |
52 weeks
|
Samarbetspartners och utredare
Sponsor
Utredare
- Studierektor: Medical Director, MD, Ascendis Pharma A/S
Studieavstämningsdatum
Studera stora datum
Studiestart (Beräknad)
Primärt slutförande (Beräknad)
Avslutad studie (Beräknad)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- ASND0053
Läkemedels- och apparatinformation, studiedokument
Studerar en amerikansk FDA-reglerad läkemedelsprodukt
Studerar en amerikansk FDA-reglerad produktprodukt
Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .