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

6. august 2018 oppdatert av: 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.

Studieoversikt

Status

Avsluttet

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Faktiske)

76

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

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

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

6 år til 35 år (Barn, Voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

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

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: Parallell tildeling
  • Masking: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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 navn:
  • Prolastin-C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
Eksperimentell: 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 navn:
  • 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
Eksperimentell: 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 navn:
  • Prolastin-C
  • Alpha-1 MP
  • Alpha1-antitrypsin
  • Alpha1-Proteinase Inhibitor (human), Modified Process
Eksperimentell: 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 navn:
  • 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

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
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
Tiltaksbeskrivelse
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

Samarbeidspartnere og etterforskere

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

Sponsor

Grifols Therapeutics LLC

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

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. mars 2014

Primær fullføring (Faktiske)

1. januar 2017

Studiet fullført (Faktiske)

1. juni 2017

Datoer for studieregistrering

Først innsendt

11. mars 2014

Først innsendt som oppfylte QC-kriteriene

18. mars 2014

Først lagt ut (Anslag)

20. mars 2014

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

5. september 2018

Siste oppdatering sendt inn som oppfylte QC-kriteriene

6. august 2018

Sist bekreftet

1. august 2018

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • GTI1302

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