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A Study of the Pharmacokinetics, Pharmacodynamics, and Safety of ARC1779 Injection in Patients With Von Willebrand Disease Type 2B

20. august 2009 oppdatert av: Archemix Corp.
To evaluate the effect of ARC1779, a therapeutic oligonucleotide ("aptamer") in patients with Type2B von Willebrand Disease.

Studieoversikt

Status

Tilbaketrukket

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

ARC1779 will be investigated in an open-label, uncontrolled study in up to 3 vWD-2B patients. Patients with vWD-2B will be screened for eligibility based primarily upon a single major criterion, i.e., presence of any degree of chronic thrombocytopenia. Eligible patients will be treated by intravenous infusion of ARC1779 for 72 hours.

Studietype

Intervensjonell

Registrering (Forventet)

2

Fase

  • Fase 2

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

16 år til 75 år (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

Inclusion Criteria:

  • Male or female patients;
  • ≥ 16 to ≤ 75 years of age;
  • Diagnosis of VWD-2B according to national expert guidelines for the USA [1] and Europe [2] based on medical history and findings from a matrix of laboratory assays which may include: platelet count, concentration of VWF antigen (VWF:Ag), VWF ristocetin cofactor activity (VWF:RCo), Factor VIII (FVIII) activity, ristocetin-induced platelet aggregation (RIPA), platelet function analyzer (PFA-100®) closure time, bleeding time (BT), VWF multimer test, VWF: platelet-binding (VWF:PB) activity, etc.)
  • Thrombocytopenia (defined as a platelet count < 100 per nL on at least 2 occasions within the month preceding enrollment;
  • Female patients of reproductive age must be enrolled within 1 to 7 days of the cessation of preceding menses;
  • Female patients must be non-pregnant and willing to use effective, redundant methods of contraception (i.e., for both self and male partner) throughout the study and for at least 30 days after discontinuation of study drug treatment;
  • Male patients must agree to use a medically acceptable contraceptive (abstinence or use of a condom with spermicide) throughout the study and for at least 30 days after discontinuation of study drug treatment;
  • All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document.

Exclusion Criteria:

  • Patients with a possible co-existing or alternative hematologic diagnosis which can account for the laboratory findings of thrombocytopenia, etc.;
  • Any significant medical co-morbidity which would pose an increased risk of bleeding (e.g., recent trauma or surgery, a history of gastrointestinal ulcers, etc.) or thrombosis (e.g., history of recurrent deep vein thrombosis (DVT).

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Group NT3
Patients will receive a total dose of approximately 1.7 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 3 mcg/mL
Legemiddel: ARC1779

Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min.

Group "NT3"

Legemiddel: ARC1779

Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours.

Group "T3"

Legemiddel: ARC1779

Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min.

Group "NT6"

Legemiddel: ARC1779

Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours.

Group "T6"
Eksperimentell: Group T3
Patients will receive a total dose of approximately 1.4 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 3 mcg/mL. The infusion of ARC1779 will be tapered by 50% from 48 hours to 60 hours and again by 50% from 60 hours to 72 hours.
Legemiddel: ARC1779

Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min.

Group "NT3"

Legemiddel: ARC1779

Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours.

Group "T3"

Legemiddel: ARC1779

Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min.

Group "NT6"

Legemiddel: ARC1779

Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours.

Group "T6"
Eksperimentell: Group NT6
Patients will receive a total dose of approximately 3.9 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 6 mcg/mL.
Legemiddel: ARC1779

Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min.

Group "NT3"

Legemiddel: ARC1779

Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours.

Group "T3"

Legemiddel: ARC1779

Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min.

Group "NT6"

Legemiddel: ARC1779

Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours.

Group "T6"
Eksperimentell: Group T6
Patients will receive a total dose of approximately 3.1 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 6 mcg/mL. The infusion of ARC1779 will be tapered by 50% from 48 hours to 60 hours and again by 50% from 60 hours to 72 hours.
Legemiddel: ARC1779

Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min.

Group "NT3"

Legemiddel: ARC1779

Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours.

Group "T3"

Legemiddel: ARC1779

Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min.

Group "NT6"

Legemiddel: ARC1779

Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours.

Group "T6"

Hva måler studien?

Primære resultatmål

Resultatmål
Tidsramme
To evaluate the effect of ARC1779 Injection on platelet counts in vWD-2B patients who have thrombocytopenia at baseline.
Tidsramme: 10 days
10 days

Sekundære resultatmål

Resultatmål
Tidsramme
To assess the concentration-response relationships among ARC1779 pharmacokinetic (PK) and pharmacodynamic (PD) parameters
Tidsramme: 10 days
10 days

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Archemix Corp.

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart

1. oktober 2008

Primær fullføring (Forventet)

1. juni 2009

Datoer for studieregistrering

Først innsendt

6. juni 2008

Først innsendt som oppfylte QC-kriteriene

9. juni 2008

Først lagt ut (Anslag)

10. juni 2008

Oppdateringer av studieposter

Sist oppdatering lagt ut (Anslag)

21. august 2009

Siste oppdatering sendt inn som oppfylte QC-kriteriene

20. august 2009

Sist bekreftet

1. august 2009

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • ARC1779-010

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Kliniske studier på Von Willebrands sykdom

Kliniske studier på ARC1779

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Sponsorer og samarbeidspartnere

Medisinsk tilstand

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Forhold

Sjeldne sykdommer

Legemiddelintervensjoner

Kosttilskudd

Sponsor / samarbeidspartnere

Steder

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