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Sikkerhed, tolerabilitet, farmakokinetik og foreløbig effektivitet af SPL84 hos patienter med cystisk fibrose

11. maj 2026 opdateret af: SpliSense Ltd.

En fase 2a, randomiseret, placebokontrolleret, dobbeltblind multipel stigende dosisundersøgelse i patienter med cystisk fibrose, der bærer 3849 +10 Kb C->T-mutationen for at evaluere sikkerheden, tolerabiliteten, farmakokinetikken og den foreløbige effektivitet af SPL84

Målet med dette kliniske forsøg er at lære, om lægemidlet SPL84 er sikkert for voksne patienter med cystisk fibrose (CF). Det vil også lære, om lægemidlet virker til at behandle værker til at behandle CF med en specifik mutation.

Formålet med denne undersøgelse er at:

  • test sikkerheden og effektiviteten af ​​flere doser af undersøgelseslægemidlet, SPL84
  • teste, hvordan flere doser af lægemidlet behandles af kroppen

Forskere vil sammenligne lægemidlet SPL84 med et placebo (et stof, der ligner hinanden, der ikke indeholder noget lægemiddel) for at se, om lægemidlet SPL84 er sikkert, og om det virker til at behandle CF.

Deltagerne vil:

Tag lægemidlet SPL84 eller placebo ved inhalation hver uge i 9 uger måneder Besøg klinikken cirka 14 gange i løbet af 17,5 uger for kontrol og test

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

64

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Forenede Stater
    • California
      • Los Angeles, California, Forenede Stater, 90033
        • Rekruttering
        • University of Southern California
        • Kontakt:
    • Colorado
      • Denver, Colorado, Forenede Stater, 80206
        • Rekruttering
        • National Jewish Health
        • Kontakt:
    • Massachusetts
      • Boston, Massachusetts, Forenede Stater, 02115

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

  • Diagnose af CF og to CF-fremkaldende mutationer; 3849+10 Kb C->T-mutation på én allel i CF-transmembrankonduktansregulatorgenet (CFTR) (homozygot eller sammensat heterozygot). Kildedokumentation fra et certificeret genetisk laboratorium er påkrævet.
  • Body mass index (BMI) på ≥ 17 kg/m2.
  • FEV1 40-90% forudsagt ved screening.
  • Ikke-rygere eller vapers i mindst 180 dage (6 måneder) før screening, pr. deltagerrapport.

Ekskluderingskriterier:

  • Brug af Kalydeco, Orkambi, Symdeko/Symkevi eller Trikafta/Kaftrio inden for 30 dage efter første dosis med undersøgelsesintervention.
  • Brug af ethvert forsøgslægemiddel (bortset fra SPL84) eller udstyr inden for 30 dage efter første dosis med undersøgelsesintervention.
  • Brug af systemiske steroider over 3 på hinanden følgende måneder inden for de sidste 6 måneder før screening, eller brug af systemiske steroider i den sidste måned før screening. Brug af inhalerede steroider over 1 mg.
  • Brug af CF-medicin, f.eks. inhalerede antibiotika, dornase alfa (Pulmozyme), hypertonisk saltvand og fysioterapi bør være på stabilt regime i perioden 28 dage før screening; de deltagere, der tager inhalationsantibiotika til profylakse, skal have et stabilt regime af disse lægemidler i mindst 90 dage før første dosis med undersøgelsesintervention.
  • Enhver akut infektion, herunder akutte øvre eller nedre luftvejsinfektioner, pulmonal eksacerbation, ændringer i behandling for lungesygdom eller enhver ikke-CF-relateret sygdom, som resulterer i initiering af enhver ny behandling inden for 14 dage før første dosis med undersøgelsesintervention.
  • Hæmoptyse på mere end 30 ml inden for 90 dage før dag 1 eller hospitalsindlæggelse for hæmotyse inden for 6 måneder efter første dosis med undersøgelsesintervention.
  • Leversygdom karakteriseret ved klinisk signifikant cirrose og/eller dokumenteret portal hypertension.
  • Historie om enhver organtransplantation.
  • Dokumenteret coronavirus sygdom (COVID-19) infektion inden for 4 uger før dosering.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Sekventiel tildeling
  • Maskning: Dobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Placebo komparator: Placebo
Andet: Placebo
Placeboopløsning til forstøvning
Aktiv komparator: SPL84
Medicin: SPL84
SPL84-løsning til forstøver

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Safety and Tolerability of SPL84 as evaluated by number of subjects with at least one treatment-related adverse event (AE) or serious adverse event (SAEs)
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Incidence, nature, and severity of AEs and SAEs
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal heart rate
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal respiratory rate
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal systolic and diastolic blood pressure
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal oximetry
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal temperature
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal hematology lab test results
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal biochemistry lab test results
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal urinalysis lab test results
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal electrocardiogram (ECG) parameters
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
using an ECG machine that automatically calculates heart rate and measure PR, QRS, QT, and QTc intervals
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal physical examination findings
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Complete physical examinations include general appearance, head, ears, eyes, nose, throat, thyroid, chest (heart, lungs), abdomen, skin, neurological, extremities, back, neck, musculoskeletal, and lymph nodes.
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal pulmonary function tests results
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Pulmonary function tests will be performed according to the American Thoracic Society (ATS)/European Respiratory Society (ERS) and forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and forced mid-expiratory flow (FEF25-75) will be measured
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Safety and Tolerability of SPL84 as assessed by number of participants with abnormal immunogenicity results
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
assessment of anti-SPL84 antibodies will be performed both in serum and sputum
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Characterization of pharmacokinetics (PK) of SPL84: maximum serum concentration (Cmax)
Tidsramme: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Time to Cmax (Tmax)
Tidsramme: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: terminal elimination half-life (t1/2)
Tidsramme: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Area under the curve to the final sample (AUC0-t)
Tidsramme: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Area under the curve to infinity (AUC0-∞)
Tidsramme: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of PK of SPL84: Apparent clearance (CL/F)
Tidsramme: Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Cohort 1-3: Predose and 15 and 30 minutes and 1, 2, 4, 6, 8, and 24 hours postdose on Day 1 and Day 57; predose on Days 8 and 29; Days 64 and 87; Cohort 4: Predose and 1, 3, and 6 hours post dose on Days 1 and 78
Characterization of excretion of SPL84: concentration of SPL84 in urine
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or Day 85 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or Day 85 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in percent predicted FEV1
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Pulmonary function tests will be performed according to the ATS/ERS
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in percent predicted FEF25-75
Tidsramme: Day 1 to Day 87 (Cohort 1-3) or Day 108 (Cohort 4)
Day 1 to Day 87 (Cohort 1-3) or Day 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
The score for is standardized on a 0- to 100-point scale on which higher scores represent a higher quality of life
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Efficacy of SPL84 as assessed by change from baseline in body weight
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Preliminary efficacy of SPL84 as assessed by change from baseline of antibiotic treatment (Cohort 1-3 only)
Tidsramme: Day 1 through Day 87
Day 1 through Day 87
Cohort 1-3: Safety and Tolerability of SPL84 as assessed by number of participants with abnormal sputum microbiology results Cohort 4: Exploratory efficacy of SPL84 as assessed by number of participants with change in abnormal sputum microbiology result
Tidsramme: Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Sputum microbiology will be performed with a microbiology based assay; organism growth will be identified.
Day 1 through Day 87 (Cohort 1-3) or 108 (Cohort 4)
Exploratory efficacy of SPL84 as assessed by number of participants with change in Lung Clearance Index at 2.5% of starting concentration (LCI2.5)
Tidsramme: Day 1 to Day 85
Measured using multiple breath washout (at select study sites only)
Day 1 to Day 85

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

SpliSense Ltd.

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

24. juni 2024

Primær færdiggørelse (Anslået)

1. oktober 2027

Studieafslutning (Anslået)

31. december 2027

Datoer for studieregistrering

Først indsendt

15. maj 2024

Først indsendt, der opfyldte QC-kriterier

20. maj 2024

Først opslået (Faktiske)

24. maj 2024

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

14. maj 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

11. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • SPL84-002
  • 2024-511184-28 (EudraCT nummer)

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med Cystisk fibrose

Kliniske forsøg med SPL84

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