- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT01230801
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
8. maj 2018 opdateret af: BioMarin Pharmaceutical
A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease
A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.
Studieoversigt
Undersøgelsestype
Interventionel
Tilmelding (Faktiske)
22
Fase
- Fase 2
- Fase 1
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
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Adelaide, SA
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Adelaide, Adelaide, SA, Australien, 5006
- Royal Adelaide Hospital, SA Pathology
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Birmingham, Det Forenede Kongerige, B15 2TH
- Old Queen Elizabeth Hospital, Department of Medicine
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London, Det Forenede Kongerige, NW3 2QG
- Royal Free Hospital
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Salford, Det Forenede Kongerige, M6 8HD
- Salford Royal Hospital NHS Trust
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California
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La Jolla, California, Forenede Stater, 92103-8765
- Univ of California San Diego School of Medicine
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Florida
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Gainesville, Florida, Forenede Stater, 32610
- University of Florida College of Medicine
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Kansas
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Kansas City, Kansas, Forenede Stater, 66160
- University of Kansas Medical Center
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Nice, Frankrig, 06202
- Hôpital de I´Archet- Centre Hospitalier Universitaire Nice
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Paris Cedex 13, Frankrig, 75651
- Hôpital Pitié-Salpêtrière
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Rheinland-pfalz
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Mainz, Rheinland-pfalz, Tyskland, 55131
- Zentrum für Kinder- und Jugenmedizin
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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
13 år og ældre (Barn, Voksen, Ældre voksen)
Tager imod sunde frivillige
Ingen
Køn, der er berettiget til at studere
Alle
Beskrivelse
Inclusion criteria:
- Patient has been diagnosed with Pompe Disease prior to or during the screening period based on 2 GAA gene mutations and either: endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed in cultured skin fibroblasts -or- endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed by dried blood spot or whole blood assay;
- Patient is male or female and 13 years of age or older at the time of enrollment in the study;
- Sexually active patients must be willing to use an acceptable method of contraception while participating in the study and for at least 4 months following the last dose of BMN 701;
- If patient is female and not considered to be of childbearing potential, she is at least 2 years post-menopausal or had tubal ligation at least 1 year prior to screening, or who have had total hysterectomy;
- If patient is female and of childbearing potential, she has negative urine pregnancy tests during the Screening Period and at the Baseline visit and be willing to have additional pregnancy tests during the study;
- Patient has ≥30% predicted upright FVC and either <80% predicted upright FVC, or >10% reduction in supine FVC compared to upright FVC during the Screening Period;
- Patient is naïve to Enzyme Replacement Therapy (ERT) with rhGAA;
- Patient must be able to ambulate at least 40 meters (131.2 feet) on the 6MWT conducted at the Screening visit (use of assistive devices such as walker, cane, or crutches, is permitted); and
- If subject was female, she was not lactating
Exclusion criteria:
- Patient has a history of diabetes or other disease known to cause hypoglycemia and is currently receiving, or might anticipate receiving, hypoglycemic agents during the course of the study;
- Patient has been on any immunosuppressive medication other than glucocorticosteroids within 1 year prior to enrollment into this study;
- Patient requires invasive ventilatory assistance at the time of enrollment into the study;
- Patient has received any investigational medication within 30 days prior to the first dose of study drug or is scheduled to receive any investigational drug other than BMN 701 during the course of the study;
- Patient has previously been admitted to the study;
- Patient is breastfeeding at screening or planning to become pregnant (self or partner) at any time during the study;
- Patient has a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the patient's ability to comply with the protocol requirements or compromise the patient's well being or safety;
- Patient has any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
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: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
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Eksperimentel: BMN 701
IV infusion
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GILT-mærket rekombinant human GAA
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Number of Participants With Adverse Events
Tidsramme: 24 weeks
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Number of Participants with Adverse Events as a Measure of Safety and Tolerability
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24 weeks
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Change From Baseline in Six Minutes Walk Test
Tidsramme: Baseline up to 24 weeks
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Change from Baseline in Six Minutes Walk Test.
The 6MWT measured the maximum distance the subject could walk on a flat, hard surface in a period of 6 minutes
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Baseline up to 24 weeks
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Andre resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
---|---|---|
Change From Baseline in Percent Predicted Upright Forced Vital Capacity
Tidsramme: Baseline up to 24 week
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Change from Baseline in Percent Predicted Upright Forced Vital Capacity.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 week
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Change From Baseline in Percent Predicted Supine Forced Vital Capacity
Tidsramme: Baseline up to 24 weeks
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Change from Baseline in Percent Predicted Supine Forced Vital Capacity.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 weeks
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Change From Baseline in Percent Predicted Upright Maximum Expiratory Pressure
Tidsramme: Baseline up to 24 weeks
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Change from Baseline in Percent Predicted Upright Maximum Expiratory Pressure.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 weeks
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Change From Baseline in Percent Predicted Upright Maximum Inspiratory Pressure
Tidsramme: Baseline up to 24 weeks
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Change from Baseline in Percent Predicted Upright Maximum Inspiratory Pressure.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 weeks
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Change From Baseline in Upright Maximum Ventilatory Volume
Tidsramme: Baseline up to 24 weeks
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Change from Baseline in Upright Maximum Ventilatory Volume.
Changes in respiratory function were assessed by measurement of MEP, MIP and MVV; and percent predicted upright and supine FVC.
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Baseline up to 24 weeks
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Efterforskere
- Studieleder: Medical Monitor, BioMarin Pharmaceutical
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)
17. januar 2011
Primær færdiggørelse (Faktiske)
6. marts 2013
Studieafslutning (Faktiske)
6. marts 2013
Datoer for studieregistrering
Først indsendt
27. oktober 2010
Først indsendt, der opfyldte QC-kriterier
28. oktober 2010
Først opslået (Skøn)
29. oktober 2010
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
11. juni 2018
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
8. maj 2018
Sidst verificeret
1. maj 2018
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Metaboliske sygdomme
- Hjernesygdomme
- Sygdomme i centralnervesystemet
- Sygdomme i nervesystemet
- Genetiske sygdomme, medfødte
- Kulhydratmetabolisme, medfødte fejl
- Metabolisme, medfødte fejl
- Lysosomale opbevaringssygdomme
- Hjernesygdomme, metaboliske
- Hjernesygdomme, metaboliske, medfødte
- Lysosomale opbevaringssygdomme, nervesystemet
- Glykogenopbevaringssygdom
- Glykogenopbevaringssygdom type II
Andre undersøgelses-id-numre
- POM-001
- 2010-023561-22 (EudraCT nummer)
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Ja
Studerer et amerikansk FDA-reguleret enhedsprodukt
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 BMN 701
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BioMarin PharmaceuticalAfsluttetPompes sygdomForenede Stater, Det Forenede Kongerige, Australien, Frankrig, Tyskland, New Zealand
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BioMarin PharmaceuticalAfsluttetSent debuterende Pompes sygdomForenede Stater, Det Forenede Kongerige, Tyskland, Frankrig, Italien, Portugal, Belgien, Holland
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CephalonAfsluttet
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Terns, Inc.Ikke rekrutterer endnuKronisk myeloid leukæmi | Kronisk myeloid leukæmi, kronisk faseForenede Stater
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AmgenTrukket tilbageRecidiverende/Refraktær Myelom
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BioMarin PharmaceuticalGodkendt til markedsføringMorquio A syndrom | MPS IVA | Mucopolysaccharidosis IVAForenede Stater, Puerto Rico
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BioMarin PharmaceuticalRekrutteringArveligt angioødem | HAEForenede Stater, Spanien, Australien
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BioMarin PharmaceuticalAfsluttetMorquio A syndrom | MPS IV A | Mucopolysaccharidosis IVADet Forenede Kongerige
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Millennium Pharmaceuticals, Inc.AfsluttetAvancerede solide tumorerForenede Stater