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Trial record 9 of 79 for:    "alpha-1 antitrypsin deficiency"

Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)

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ClinicalTrials.gov Identifier: NCT02870348
Recruitment Status : Enrolling by invitation
First Posted : August 17, 2016
Last Update Posted : May 7, 2018
Sponsor:
Collaborator:
Grifols Japan K.K.
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Brief Summary:
This is a multi-center, open-label study to evaluate the long-term safety of weekly IV infusions of 60 mg/kg Alpha-1 MP in adult subjects with AATD in Japan who have completed Study GTI1401.

Condition or disease Intervention/treatment Phase
Alpha1-Antitrypsin Deficiency Biological: Alpha-1 MP Phase 1 Phase 2

Detailed Description:

This is a multi-center, open-label study to evaluate the long-term safety of weekly IV infusions of 60 mg/kg Alpha-1 MP in adult subjects with AATD in Japan who have completed Study GTI1401. Study GTI1401 is being conducted to evaluate the safety and PK of Alpha-1 MP in subjects with AATD in Japan. In the current study, GTI1401-OLE, subjects will be administered 60 mg/kg Alpha-1 MP by weekly IV infusion for approximately 1 year (can be renewed annually with the consent of the subjects unless the sponsor informs of discontinuation of this OLE trial) to assess the long-term safety of Alpha-1 MP in subjects with AATD. This study will be conducted at up to 5 centers in Japan.

At the Week 9 Visit of Study GTI1401, after giving consent, on the same day, subjects will be assessed for eligibility at Screening/Extention (Ext) Week 1 Visit for this extension study, Study GTI1401-OLE. If eligible, subjects will be administered weekly IV infusions of 60 mg/kg Alpha-1 MP for approximately 1 year or longer. The Week 9 Visit of GTI1401 will be the End of Study Visit for the subjects who are enrolled in Study GTI1401-OLE.

Subjects in Study GTI1401-OLE will have the option to remain in Study GTI1401-OLE and continue to receive weekly IV infusions of 60 mg/kg Alpha-1 MP for another year or longer. If subjects plan to conclude their participation in the study (GTI1401-OLE) early (before Ext. Week 52), they will be asked to complete the End of Study Follow-Up Assessments, which will be scheduled 4 weeks after the last infusion.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi-Center, Open-Label Study to Evaluate the Long-term Safety of Weekly Intravenous Infusions of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency
Study Start Date : November 2016
Estimated Primary Completion Date : March 15, 2020
Estimated Study Completion Date : March 15, 2020


Arm Intervention/treatment
Experimental: Alpha-1 MP
Alpha-1 MP 60 mg/kg administered weekly via intravenous administration
Biological: Alpha-1 MP
Weekly intravenous infusions of 60 mg/kg Alpha-1 MP
Other Name: Prolastin-C




Primary Outcome Measures :
  1. Safety of 60 mg/kg Alpha-1 MP as assessed by Adverse events, ADRs, serious AEs (SAEs), discontinuations due to AEs or SAEs, and COPD exacerbations [ Time Frame: Week 1 to Week 56 ]
    Adverse events, ADRs, serious AEs (SAEs), discontinuations due to AEs or SAEs, and COPD exacerbations



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Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects who complete participation in Study GTI1401 (i.e., have completed the study through the Week 9 Visit).
  • Subjects who will and are able to provide written informed consent.

Exclusion Criteria:

  • Subjects with newly diagnosed severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis.
  • Subjects with newly diagnosed malignant tumor (including malignant melanoma; however, other forms of skin cancer are allowed).
  • Female subjects who are pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device [IUD] or intrauterine system [IUS], condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or abstinence) throughout the study or male subjects who have a partner who is of child-bearing potential and is unwilling to practice a highly effective method of contraception throughout the study.
  • Subjects with clinical signs and symptoms of active HAV, HBV, HCV, or HIV viral infection at the Week 9 Visit of Study GTI1401 and viral infection is further confirmed by testing.
  • Subjects with current evidence of smoking or has a positive urine cotinine test at the Week 9 Visit in Study GTI1401 that is due to smoking.
  • Subjects who currently participate in a study of another investigational product (other than Alpha-1 MP).
  • Subjects who have difficulty in adhering to the protocol or its procedures, in the opinion of the investigator.
  • Subjects who have medical conditions that may confound the results of this clinical trial or may endanger these subjects during their participation in this clinical trial in the opinion of the investigator.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02870348


Locations
Japan
Hiroskai University Hospital
Aomori, Japan, 036-8563
Hokkaido University Hospital
Hokkaido, Japan, 060-8648
Juntendo University Hospital
Tokyo, Japan, 113-8431
Sponsors and Collaborators
Grifols Therapeutics LLC
Grifols Japan K.K.

Responsible Party: Grifols Therapeutics LLC
ClinicalTrials.gov Identifier: NCT02870348     History of Changes
Other Study ID Numbers: GTI1401-OLE
First Posted: August 17, 2016    Key Record Dates
Last Update Posted: May 7, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes
Alpha 1-Antitrypsin
Protein C Inhibitor
Protease Inhibitors
Trypsin Inhibitors
Serine Proteinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action