En fase 2a-studie av ALN-PNP med og uten en GLP1R-agonist hos voksne pasienter med homozygot PNPLA3-relatert MASLD
En to-delt, fase 2a, randomisert, dobbelblind, placebo-kontrollert studie av ALN-PNP med og uten en GLP1R-agonist hos voksne med homozygot PNPLA3-relatert metabolsk dysfunksjons-assosiert steatotisk leversykdom (MASLD)
This study will test a study drug called ALN-PNP with and without another drug that is used for controlling blood sugar, appetite, and weight (for example, tirzepatide), to see if it can help treat MASLD, also known as fatty liver disease. ALN-PNP reduces the amount of Patatin-like phospholipase domain-containing protein 3 (PNPLA3), a protein that liver cells make, which may help decrease liver fat if there is an abnormal PNPLA3 protein.
The goal of this study is to understand the effect of ALN-PNP with or without tirzepatide on reducing liver fat.
The study is looking at:
- How well ALN-PNP with and without tirzepatide works
- What side effects ALN-PNP might cause
- How much ALN-PNP is in the blood at different times
- How the body and the liver change after having ALN-PNP, which can help researchers understand why ALN-PNP works better in some people than others
Studieoversikt
Status
Intervensjon / Behandling
Studietype
Registrering (Antatt)
Fase
- Fase 2
Kontakter og plasseringer
Studiekontakt
- Navn: Clinical Trials Administrator
- Telefonnummer: 844-734-6643
- E-post: clinicaltrials@regeneron.com
Studiesteder
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-
Arizona
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Tucson, Arizona, Forente stater, 85712
- Rekruttering
- Arizona Liver Health - Tucson
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California
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Coronado, California, Forente stater, 92118
- Rekruttering
- Southern California Research Center
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Pasadena, California, Forente stater, 91105
- Rekruttering
- California Liver Research Institute
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Florida
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Jupiter, Florida, Forente stater, 33458
- Rekruttering
- Health Awareness, Inc
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Miami, Florida, Forente stater, 33122
- Rekruttering
- Evolution Clinical Trials
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Louisiana
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Metairie, Louisiana, Forente stater, 70006
- Rekruttering
- Tandem Clinical Research, Plaza II
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Texas
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Austin, Texas, Forente stater, 78757
- Rekruttering
- American Research Corporation
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Georgetown, Texas, Forente stater, 78628
- Rekruttering
- Advanced Medical Trials
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San Antonio, Texas, Forente stater, 78215
- Rekruttering
- American Research Corporation at The Texas Liver Institute
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-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
Key Inclusion Criteria:
Part A and Part B:
- Homozygous for the PNPLA3 p.I148M genotype
- Liver fat by Magnetic Resonance Imaging-Proton Density Fat Fraction (MRI-PDFF) ≥15% at visit 3
- Has a Body Mass Index (BMI) ≥30 to <45 kg/m^2 at visit 2
Part A: To be eligible for randomization on study day 1:
- Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) ≤3 × Upper Limit of Normal (ULN) as described in the protocol
- On a stable dose of tirzepatide at randomization (≥5 mg weekly)
Key Exclusion Criteria:
- Evidence or diagnosis of portal hypertension or cirrhosis from any cause, including cirrhosis due to MASH, as determined by the investigator, based on medical history, clinical assessment, imaging, and/or liver biopsy
- Known chronic liver disease other than MASLD, as determined by the investigator, as defined in the protocol
- Contraindications to MRI examinations, including but not limited to persons with MRI-incompatible cardiac pacemaker and implants made of metal, severe claustrophobia, size restrictions
- Any contraindication listed in the Zepbound United States Prescribing Information (USPI), as defined in the protocol
NOTE: Other protocol-defined inclusion/exclusion criteria apply.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Firemannsrom
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: Del A: A1
|
Administered per the protocol Administered per the protocol
Andre navn:
|
|
Eksperimentell: Del A: A2
|
Administered per the protocol
Andre navn:
Administered per the protocol Placebo matching ALN-PNP |
|
Eksperimentell: Del B: B1
|
Administered per the protocol Administered per the protocol
Andre navn:
|
|
Eksperimentell: Del B: B2
|
Administered per the protocol
Andre navn:
Administered per the protocol Placebo matching ALN-PNP |
|
Eksperimentell: Del B: B3
|
Administered per the protocol |
|
Placebo komparator: Del B: B4
|
Administered per the protocol Placebo matching ALN-PNP |
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Percent change in liver fat
Tidsramme: From baseline at week 24
|
Part A |
From baseline at week 24
|
|
Percent change in liver fat
Tidsramme: From baseline at week 48
|
Part B |
From baseline at week 48
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
Oppnåelse av leverfett <5%
Tidsramme: Ved uke 24 og 48
|
Ved uke 24 og 48
|
|
Occurrence of Treatment-Emergent Adverse Events (TEAEs)
Tidsramme: Through week 72
|
Through week 72
|
|
Severity of TEAEs
Tidsramme: Through week 72
|
Through week 72
|
Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Clinical Trial Management, Regeneron Pharmaceuticals
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
- Sykdommer i fordøyelsessystemet
- Leversykdommer
- Fettlever
- Ikke-alkoholisk fettleversykdom
- Aminosyrer, peptider og proteiner
- Proteiner
- Glukagonlignende peptid-1 reseptor
- Glukagonlignende peptidreseptorer
- Reseptorer, G-protein-koblet
- Reseptorer, celleoverflate
- Membranproteiner
- Reseptorer, gastrointestinal hormon
- Reseptorer, peptid
- Tirzepatid
Andre studie-ID-numre
- ALN-PNP-MASLD-2543
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.
IPD-delingstidsramme
When Regeneron has:
- received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development
- made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry)
- the legal authority to share the data, and
- ensured the ability to protect participant privacy
Tilgangskriterier for IPD-deling
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
IPD-deling Støtteinformasjonstype
- STUDY_PROTOCOL
- SEVJE
- ICF
- ANALYTIC_CODE
- CSR
Legemiddel- og utstyrsinformasjon, studiedokumenter
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