- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT00989196
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of Human-cl rhFVIII, a Newly Developed Human Cell-line Derived Recombinant FVIII Concentrate in Previously Treated Patients With Severe Hemophilia A
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Studietype
Registrering (Faktiske)
Fase
- Fase 2
Kontakter og plasseringer
Studiesteder
-
-
-
Sofia, Bulgaria
- Prof. Lissitchkov
-
-
-
-
California
-
Los Angeles, California, Forente stater, 90007
- UCLA Orthodpedic Hospital
-
Sacramento, California, Forente stater, 95817
- University of California, Davis
-
-
Colorado
-
Denver, Colorado, Forente stater, 80045
- University of Colorado
-
-
District of Columbia
-
Washington, District of Columbia, Forente stater, 20057
- Georgetown University
-
-
Illinois
-
Chicago, Illinois, Forente stater, 60612
- Rush University Medical Center
-
-
New Jersey
-
New Brunswick, New Jersey, Forente stater, 08903
- University of Medicine and Dentistry
-
-
-
-
-
Berlin, Tyskland
- Vivantes Klinikum
-
-
Niedersachsen
-
Hannover, Niedersachsen, Tyskland
- Medizinische Hochschule
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
Inclusion Criteria:
- Severe hemophilia A (FVIII:C <= 1%)
- Male subjects between 12 and 65 years of age
- Body weight 25 kg to 110 kg
- Previously treated with FVIII concentrate for at least 150 EDs
Exclusion Criteria:
- Other coagulation disorder than hemophilia A
- Present or past FVIII inhibitor activity
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Crossover-oppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
---|---|
Eksperimentell: Human-cl rhFVIII
|
50 IU/kg for PK dose
|
Aktiv komparator: Kogenate FS
|
50 IU/kg for PK dose
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
---|---|---|
The Area Under the Concentration Curve for Human-cl rhFVIII Compared to Kogenate FS
Tidsramme: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion.
FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
|
At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
---|---|---|
Invivo Half-life (T1/2) for Human-cl rhFVIII Compared to Kogenate FS
Tidsramme: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion.
FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
|
At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
Maximum Plasma Concentration (Cmax) for Human-cl rhFVIII Compared to Kogenate FS
Tidsramme: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion.
FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
|
At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
Time to Reach Maximum Plasma Concentration (Tmax) for Human-cl rhFVIII Compared to Kogenate FS
Tidsramme: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion.
FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
|
At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
Mean Residence Time (MRT) for Human-cl rhFVIII Compared to Kogenate FS
Tidsramme: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion.
FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
|
At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
Volume of Distribution at Steady State (Vss) for Human-cl rhFVIII Compared to Kogenate FS
Tidsramme: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion.
FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
|
At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
Clearance (CL) for Human-cl rhFVIII Compared to Kogenate FS
Tidsramme: At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
After the infusion of 50IU/kg bw of Human-cl rhFVIII and Kogenate FS respectively, FVIII activity levels were measured at various time points before and after the infusion.
FVIII level results derived from the chromogenic FVIII assay were used to calculate the mean of the area under the curve normalized to the dose administered.
|
At baseline (prior to infusion), 0.25, 0.5, 0.75, 1, 3, 6, 9, 12, 24, 30 and 48 hours after the end of the infusion.
|
Efficacy of On-demand Treatment of Bleeding Episodes
Tidsramme: From 1st treatment after PK cycle 2 until study end.
|
After each infusion of IMP and at the end of a BE, the following efficacy assessment is made by the subject (together with the Investigator in case of on-site treatment): Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion. Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 - 12 hours after an infusion requiring up to 2 infusions for complete resolution. Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution. None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution. The assessment was made at the end of a BE in case more than one infusion was needed. |
From 1st treatment after PK cycle 2 until study end.
|
Immunogenicity (Number of Patients That Developed an Inhibitor During the Course of the Study)
Tidsramme: study entry, then immediately before both PK cycles, in the 48 hour sample of both PK cycles, after 10 to 15 EDs with human-cl rhFVIII, at the 3-month visit (± 2 weeks), then every 3 months (± 2 weeks) until study completion, and after >50 EDs (except for
|
Inhibitor activity was determined by the modified Bethesda assay (Nijmegen modification) at study entry, then immediately before both PK cycles, in the 48 hour sample of both PK cycles, after 10 to 15 EDs with human-cl rhFVIII, at the 3-month visit (± 2 weeks), then every 3 months (± 2 weeks) until study completion, and after >50 EDs (except for some patients who may finish the study before they achieve 50 EDs), with human-cl rhFVIII (i.e. at the study completion visit).
|
study entry, then immediately before both PK cycles, in the 48 hour sample of both PK cycles, after 10 to 15 EDs with human-cl rhFVIII, at the 3-month visit (± 2 weeks), then every 3 months (± 2 weeks) until study completion, and after >50 EDs (except for
|
Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Sigurd Knaub, PhD, Octapharma
Studierekorddatoer
Studer hoveddatoer
Studiestart
Primær fullføring (Faktiske)
Studiet fullført (Faktiske)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Anslag)
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
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- GENA-01
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .
Kliniske studier på Hemofili A
-
Cheikh Anta Diop University, SenegalInternational Atomic Energy AgencyHar ikke rekruttert ennå
-
National Institute of Environmental Health Sciences...TilbaketrukketBisfenol A
-
King Saud UniversityFullført
-
Medical University of ViennaFullførtImmunoglobulin AØsterrike
-
Ionis Pharmaceuticals, Inc.Akcea TherapeuticsFullført
-
University of California, DavisBill and Melinda Gates Foundation; Penn State University; Newcastle University og andre samarbeidspartnereFullførtVitamin A-statusFilippinene
-
Ionis Pharmaceuticals, Inc.FullførtForhøyet lipoprotein(a)Nederland, Storbritannia, Danmark, Tyskland, Canada
-
Arab American University (Palestine)FullførtA-PRF | ALLOGRAFTDet palestinske territoriet, okkupert
-
AmgenFullført
-
Shanghai Jiao Tong University School of MedicineFullførtBotulinumtoksin, type A
Kliniske studier på Human-cl rhFVIII
-
OctapharmaFullførtAlvorlig hemofili AForente stater, Frankrike, Canada, Storbritannia, India, Georgia, Moldova, Republikken, Polen, Ukraina
-
OctapharmaFullførtAlvorlig hemofili AFrankrike, Japan, Forente stater, Canada, Kroatia, Finland, Nederland, Nord-Makedonia, Slovenia
-
OctapharmaFullførtAlvorlig hemofili ACanada, Forente stater, Frankrike, Spania, Ukraina, Storbritannia, Hviterussland, Georgia, Tyskland, India, Italia, Moldova, Republikken, Marokko, Polen, Portugal, Den russiske føderasjonen, Slovenia
-
OctapharmaFullførtAlvorlig hemofili AØsterrike, Storbritannia, Bulgaria, Ungarn, Tyskland, Polen, Romania, Slovakia
-
OctapharmaFullførtAlvorlig hemofili ATyskland, Østerrike, Storbritannia, Bulgaria
-
OctapharmaAvsluttet
-
Boehringer IngelheimFullført
-
HeartWorks, Inc.RekrutteringMedfødt hjertesykdom | Univentrikulært hjerte | Hjertesvikt NYHA klasse III | Hjertesvikt NYHA klasse IVForente stater