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.
Studieoversigt
Status
Betingelser
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Fase 3
Kontakter og lokationer
Studiekontakt
- Navn: Ascendis Registry Inquiries
- Telefonnummer: +45 61242484
- E-mail: asnd_registryinquiries@ascendispharma.com
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
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
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Firedobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: Once weekly navepegritide SC and lonapegsomatropin SC
|
Administered weekly as two separate SC injections. Treatment duration of up to 104 weeks |
|
Eksperimentel: 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. |
|
Eksperimentel: 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. |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Annualized Growth Velocity (AGV)
Tidsramme: 52 weeks
|
To evaluate the AGV, cm per year, of dual therapy with navepegritide and lonapegsomatropin compared to navepegritide alone |
52 weeks
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Treatment-emergent Adverse Events (AE)
Tidsramme: Throughout trial duration 104 weeks
|
Number of events |
Throughout trial duration 104 weeks
|
|
Annualized Growth Velocity (AGV)
Tidsramme: 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
Tidsramme: 52 weeks
|
To evaluate the change from baseline in AGV, cm per year |
52 weeks
|
|
ACH-specific height Z-score
Tidsramme: 52 weeks
|
To evaluate the change from baseline in ACH-specific height Z-score |
52 weeks
|
|
CDC-based height Z-score
Tidsramme: 52 weeks
|
To evaluate the change from baseline in CDC-based height Z-score |
52 weeks
|
|
Bone safety
Tidsramme: Throughout trial duration 104 weeks
|
Qualitative assessment with radiology |
Throughout trial duration 104 weeks
|
|
Arm span Z-score
Tidsramme: 52 weeks
|
Change from baseline in arm span Z-score. |
52 weeks
|
|
Upper to lower body segment ratio
Tidsramme: 52 weeks
|
Change from baseline in upper to lower body segment ratio. |
52 weeks
|
|
Arm span
Tidsramme: 52 weeks
|
Change from baseline in arm span, cm. |
52 weeks
|
|
Interpedicular distance (IDP)
Tidsramme: 52 weeks
|
Change from baseline in IDP (L1-L4 average), mm |
52 weeks
|
Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Studieleder: Medical Director, MD, Ascendis Pharma A/S
Datoer for undersøgelser
Studer store datoer
Studiestart (Anslået)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- ASND0053
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
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