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

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Brief Title

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

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

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.
    

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 Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

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


Condition

Alpha1-Antitrypsin Deficiency

Intervention

Alpha-1 MP

Study Arms / Comparison Groups

 Alpha-1 MP
Description:  Alpha-1 MP 60 mg/kg administered weekly via intravenous administration

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

4

Start Date

October 2016

Completion Date

January 20, 2023

Primary Completion Date

January 20, 2023

Eligibility 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.
      

Gender

All

Ages

20 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Japan

Location Countries

Japan

Administrative Informations


NCT ID

NCT02870348

Organization ID

GTI1401-OLE


Responsible Party

Sponsor

Study Sponsor

Grifols Therapeutics LLC

Collaborators

 Grifols Japan K.K.

Study Sponsor

, , 


Verification Date

May 2019