Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)

Brief Title

Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)

Official Title

Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)

Brief Summary

      An Early Access Program for patients with steroid refractory acute GvHD after hematopoietic
      stem cell transplantation.

      This Program is available for female and male who are recipients of allogenic HSCT and who
      have been newly diagnosed with acute GvHD.

Detailed Description

      GLASSIA (human alpha-1 proteinase inhibitor (A1PI), also known as human alpha-1 antitrypsin,
      Kamada-AAT or Kamada-API) is a, liquid, ready-to-use preparation of human A1PI. Alpha-1
      proteinase inhibitor belongs to the family of serine proteinase inhibitors and is primarily
      produced in the liver and secreted into the circulation. In addition to its anti-proteinase
      activity, A1PI showed to have anti-inflammatory, anti-apoptotic and immunomodulatory
      properties (1-4).

      GLASSIA is an injection solution prepared from human plasma collected from healthy volunteer
      blood donors in accordance with Food and Drug Administration (FDA) and European Medicines
      Agency (EMA) regulations. GLASSIA was approved in the United States (US) in July 2010 and is
      indicated for chronic augmentation and maintenance therapy in adults with clinically evident
      emphysema due to severe hereditary deficiency of Alpha1-PI (alpha1-antitrypsin deficiency).

Study Type

Expanded Access

Condition

Acute-graft-versus-host Disease

Intervention

Glassia

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

Drug

Eligibility Criteria

        Inclusion Criteria:

          -  This program is available for patients who suffer from steroid refractory acute GvHD
             after HSCT, or for whom, in the opinion of their treating physician, other treatment
             options or clinical trials in this indication are unsuitable.

          -  The disease indication for which the participant required HSCT must be in remission

          -  Acute graft-versus-host disease (aGvHD), including lower GI involvement (modified

          -  International Bone Marrow Transplant Registry (IBMTR) Severity Stage 1 to 4 [>500 mL
             diarrhea/day]), with or without other organ system involvement.

          -  For women of childbearing potential, had a negative serum or urine pregnancy test
             within 14 days prior to enrolment.

        Exclusion Criteria:

          -  Participant with manifestations of chronic GvHD

          -  Participant with acute/chronic GvHD overlap syndrome

          -  Participant whose GvHD developed after donor lymphocyte infusion

          -  Participant with severe sepsis involving at least 1 organ failure

          -  Participant who is seropositive or positive in the nucleic acid test for human
             immunodeficiency virus (HIV)

          -  Participant with active hepatitis B or C

          -  If female, participant is pregnant or lactating at the time of enrollment, or has
             plans to become pregnant during the program

Gender

All

Ages

N/A - N/A

Contacts

, +31 885253888, [email protected]

Administrative Informations

NCT ID

NCT03172455

Organization ID

2017-AATGLAKAM-EU

Responsible Party

Sponsor

Study Sponsor

Impatients N.V. trading as myTomorrows

Collaborators

 Kamada, Ltd.

Study Sponsor

, ,

Verification Date

February 2020