Denne side blev automatisk oversat, og nøjagtigheden af ​​oversættelsen er ikke garanteret. Der henvises til engelsk version for en kildetekst.

Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

6. august 2018 opdateret af: Grifols Therapeutics LLC

A Multicenter, Randomized, Partial-Blinded, Placebo-Controlled Study to Evaluate the Safety and Efficacy of a Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus

This is a multicenter, randomized, partial-blinded, five-arm, placebo-controlled study of human plasma-derived alpha1-proteinase inhibitor (alpha1-PI) in children (ages 6-11 years old) and teens/adults (ages 12-35 years old) with new onset Type 1 Diabetes Mellitus (T1DM). Currently enrolling ages 12-35 only. Once 25 patients are randomized and data is reviewed enrollment will be opened to the child cohort. The purpose of this study is to evaluate the safety and efficacy of four dosing regimens of human plasma-derived alpha1-PI in T1DM.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

76

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
    • Arizona
      • Tucson, Arizona, Forenede Stater, 85724
        • University of Arizona
    • California
      • Los Angeles, California, Forenede Stater, 90015
        • Clinica Medica San Miguel
      • Orange, California, Forenede Stater, 92868
        • Diabetes Associates Medical Group
      • San Diego, California, Forenede Stater, 92123
        • Rady Children's Hospital San Diego
      • Tarzana, California, Forenede Stater, 91356
        • Metabolic Institute of America
      • Ventura, California, Forenede Stater, 93003
        • Ronald H Chochinov MD
    • Connecticut
      • New Haven, Connecticut, Forenede Stater, 06511
        • Yale New Haven Hospital
    • Delaware
      • Newark, Delaware, Forenede Stater, 19713
        • Christiana Care Health Services
    • Florida
      • Jacksonville, Florida, Forenede Stater, 32258
        • Solutions Through Advanced Research Inc.
      • Miami, Florida, Forenede Stater, 33133
        • CCM Clinical Research
      • Miami, Florida, Forenede Stater, 33136
        • Advanced Pharma CR LLC
    • Georgia
      • Atlanta, Georgia, Forenede Stater, 30309
        • Atlanta Diabetes Associates
    • Idaho
      • Idaho Falls, Idaho, Forenede Stater, 83404
        • Rocky Mountain Diabetes and Osteoporosis Center
    • Illinois
      • Chicago, Illinois, Forenede Stater, 60612
        • Cook County Hospital
    • Indiana
      • Indianapolis, Indiana, Forenede Stater, 46202
        • Methodist Research Institute
    • Iowa
      • Iowa City, Iowa, Forenede Stater, 52242
        • University of Iowa Hospitals and Clinics
    • Kentucky
      • Louisville, Kentucky, Forenede Stater, 40202
        • University of Louisville
    • Maryland
      • Baltimore, Maryland, Forenede Stater, 21229
        • Barry J. Reiner MD, LLC.
    • Massachusetts
      • Worcester, Massachusetts, Forenede Stater, 01655
        • UMass Memorial Medical Center
    • Michigan
      • Detroit, Michigan, Forenede Stater, 48201
        • Wayne State University
    • Minnesota
      • Saint Paul, Minnesota, Forenede Stater, 55102
        • Children's Hospitals and Clinics of Minnesota
    • New Jersey
      • Morristown, New Jersey, Forenede Stater, 07962
        • Morristown Medical Center
    • New Mexico
      • Albuquerque, New Mexico, Forenede Stater, 87131-0001
        • University of New Mexico, Health Sciences Center
    • New York
      • Buffalo, New York, Forenede Stater, 14222
        • Women and Children's Hospital
    • North Carolina
      • Raleigh, North Carolina, Forenede Stater, 27610
        • WakeMed Children's Hospital
    • Ohio
      • Columbus, Ohio, Forenede Stater, 43210
        • Ohio State University
      • Columbus, Ohio, Forenede Stater, 43201
        • Endocrinology Associates Inc
    • Oklahoma
      • Oklahoma City, Oklahoma, Forenede Stater, 73112
        • Pediatric Endocrinology, Genetics & Metabolism
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forenede Stater, 19106
        • Children's Hospital of Philadelphia
      • Pittsburgh, Pennsylvania, Forenede Stater, 15224
        • Children's Hospital of Pittsburgh
    • South Dakota
      • Rapid City, South Dakota, Forenede Stater, 57701
        • Rapid City Regional Hospital/Health Clinical Research
    • Texas
      • Dallas, Texas, Forenede Stater, 75390
        • University of Texas Southwestern Medical Center
      • Dallas, Texas, Forenede Stater, 75231
        • Research Institute Of Dallas
      • San Antonio, Texas, Forenede Stater, 78229
        • University of Texas Health Science Center
      • San Antonio, Texas, Forenede Stater, 78154
        • Northeast Clinical Research of San Antonio LLC
      • San Antonio, Texas, Forenede Stater, 78232
        • Consano Clinical Research

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

6 år til 35 år (Barn, Voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Inclusion Criteria:

  • Diagnosis of T1DM according to the ADA criteria.

  • Current use of injected insulin therapy and one positive result on testing for any of the following antibodies (If not currently on insulin therapy, must have positive result for at least two of the below antibodies):

    • Anti-islet-cell antibodies (islet cell antigen 512, insulinoma associated protein 2),
    • Anti-glutamic acid decarboxylase antibodies, or
    • Anti-insulin antibodies (unless received insulin therapy for > 7 days).
  • Body Mass Index (BMI) ≤ 28 kg/m2 for adults (≥ 20 years of age) OR ≤ 90th percentile in accordance with the Centers for Disease Control BMI assessment for children and teens (2 through 19 years old).

Exclusion Criteria:

  • History of or current diabetic retinopathy, neuropathy, or nephropathy.
  • Known thrombophilia or history of thrombosis.
  • Malignant disease (including malignant melanoma; however, other forms of skin cancer are allowed) within five years of randomization.
  • Active Hepatitis A virus, Hepatitis B virus, Hepatitis C virus, or Human Immunodeficiency Virus infection.
  • History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
  • Known selective or severe Immunoglobulin A deficiency.
  • Elevated liver enzymes (aspartate transaminase, alanine aminotransferase, and alkaline phosphatase) equal to or greater than 2.5 times the upper limit of normal.
  • Therapy with exenatide or any other agents that stimulate pancreatic β cell regeneration or insulin secretion, or any antidiabetic agents (oral or parenteral) other than insulin within one month prior to screening.
  • Use of omega-3 fatty acid supplements, including fish oil, within seven days prior to screening.
  • Current or planned therapy with inhaled insulin, if it becomes available.
  • Chronic use of systemic steroids, with the exception of inhaled steroids, above a stable dose equivalent to 5 mg/day prednisone (e.g., 10 mg every 2 days) within 4 weeks prior to randomization. It is recommended to maintain the same dose throughout the study. (Note: Subjects with autoimmune conditions (i.e., asthma) necessitating treatment with systemic short-term corticosteroids and administered a rapid taper are eligible per protocol with the caveat that the tapering is complete or decreased to the minimum requirement (i.e., 5 mg/day) at least 1 week prior to the Baseline visit (when randomization occurs) to ensure the subject is stable. For longer term steroid usage, please consult the Grifols Medical Monitor before considering the subject for study participation.)
  • Treatment with immunosuppressants or cytostatic agents within 6 months of randomization.

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: Parallel tildeling
  • Maskning: Firedobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Alpha1-PI 180 mg/kg/wk, 26 weeks
180 mg/kg weekly infusions of Alpha1-PI for 26 weeks.
Biologisk: 180 mg/kg Alpha1-PI
Andre navne:
  • Prolastin-C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
Eksperimentel: 90 mg/kg/wk Alpha1-PI, 26 weeks
90 mg/kg weekly infusions of Alpha1-PI for 26 weeks.
Biologisk: 90 mg/kg Alpha1-PI
Andre navne:
  • Prolastin-C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
Placebo komparator: Placebo, 26 weeks
Weekly infusions of placebo for 26 weeks.
Biologisk: Placebo
Eksperimentel: 180 mg/kg/wk Alpha1-PI, 13 weeks
180 mg/kg weekly infusions of Alpha1-PI for 13 weeks.
Biologisk: 180 mg/kg Alpha1-PI
Andre navne:
  • Prolastin-C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
Eksperimentel: 90 mg/kg/wk Alpha1-PI, 13 weeks
90 mg/kg weekly infusions of Alpha1-PI for 13 weeks
Biologisk: 90 mg/kg Alpha1-PI
Andre navne:
  • Prolastin-C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
Placebo komparator: Placebo, 13 weeks
Weekly infusions of placebo for 13 weeks.
Biologisk: Placebo

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change From Baseline in Mixed Meal Tolerance Test (MMTMT) Stimulated C-peptide 2 Hour Area Under the Concentration-time Curve (AUC)
Tidsramme: Baseline, Week 52 (pre-high protein drink and 15, 30, 60, 90, 120 minutes post-drink)
C-peptide concentration during MMTT with high protein energy drink. "Dose" for time frame refers to intake of high protein energy drink.
Baseline, Week 52 (pre-high protein drink and 15, 30, 60, 90, 120 minutes post-drink)

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Change From Baseline for MMTT Stimulated C-peptide 2h AUC
Tidsramme: Baseline, Weeks 14, 27, 39, 69, 87, and 104 (pre-high protein drink and 15, 30, 60, 90, 120 minutes post-drink)
Baseline, Weeks 14, 27, 39, 69, 87, and 104 (pre-high protein drink and 15, 30, 60, 90, 120 minutes post-drink)
Change From Baseline for HbA1c Levels
Tidsramme: Baseline, Weeks 14, 27, 39, 52, 69, 87, and 104
Baseline, Weeks 14, 27, 39, 52, 69, 87, and 104
Number of Subjects With Overall Severe Hypoglycemic Episodes
Tidsramme: 104 weeks
Severe hypoglycemia defined according the ADA Workgroup on Hypoglycemia definition, as follows: An event requiring assistance of another person to actively administer carbohydrate, glucagons, or other resuscitative actions.
104 weeks
Change From Baseline for Mean Daily Insulin Dose Requirements
Tidsramme: Baseline, Weeks 2, 4, 14, 27, 39, 52, 69, 87, and 104
Baseline, Weeks 2, 4, 14, 27, 39, 52, 69, 87, and 104
Change From Baseline for Mean Daily Glucose Levels Prior to Meals and Bedtime
Tidsramme: Baseline, Weeks 2, 4, 14, 27, 39, 52, 69, 87, and 104
For each visit, the mean daily glucose levels were calculated over the previous 3-7 days prior to the study visit from blood glucose levels recorded daily prior to meals and bedtime.
Baseline, Weeks 2, 4, 14, 27, 39, 52, 69, 87, and 104

Samarbejdspartnere og efterforskere

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

Sponsor

Grifols Therapeutics LLC

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Generelle publikationer

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

1. marts 2014

Primær færdiggørelse (Faktiske)

1. januar 2017

Studieafslutning (Faktiske)

1. juni 2017

Datoer for studieregistrering

Først indsendt

11. marts 2014

Først indsendt, der opfyldte QC-kriterier

18. marts 2014

Først opslået (Skøn)

20. marts 2014

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

5. september 2018

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

6. august 2018

Sidst verificeret

1. august 2018

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • GTI1302

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 Type 1 diabetes mellitus

Kliniske forsøg med Placebo

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in France

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

Abonner