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

Primary Purpose

Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease, Graft-versus-host-disease

Status

No longer available

Phase

Locations

Study Type

Expanded Access

Intervention

Glassia

About this trial

This is an expanded access trial for Acute-graft-versus-host Disease

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All Sexes

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

Sites / Locations

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

NCT ID

NCT03172455

First Posted

May 29, 2017

Last Updated

September 30, 2020

Sponsor

Impatients N.V. trading as myTomorrows

Collaborators

Kamada, Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT03172455

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)

Study Type

Expanded Access

2. Study Status

Record Verification Date

September 2020

Overall Recruitment Status

No longer available

Study Start Date

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Primary Completion Date

undefined (undefined)

Study Completion Date

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3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Impatients N.V. trading as myTomorrows

Collaborators

Kamada, Ltd.

4. Oversight

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease, Graft-versus-host-disease, Graft Vs Host Disease, Alpha 1-Antitrypsin Deficiency, Alpha-1 Proteinase Inhibitor, Alpha-1 Protease Inhibitor Deficiency, Acute Graft-Versus-Host Reaction Following Bone Marrow Transplant

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type

Drug

Intervention Name(s)

Glassia

Other Intervention Name(s)

Alpha 1 antitrypsin

Intervention Description

Application 90 mg/kg loading dose day 1, 30 mg/kg every other day (day 3 - day 15)

10. Eligibility

Sex

All

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

12. IPD Sharing Statement

Citations:

PubMed Identifier

3886224

Citation

Breit SN, Wakefield D, Robinson JP, Luckhurst E, Clark P, Penny R. The role of alpha 1-antitrypsin deficiency in the pathogenesis of immune disorders. Clin Immunol Immunopathol. 1985 Jun;35(3):363-80. doi: 10.1016/0090-1229(85)90097-2.

Results Reference

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PubMed Identifier

21600901

Citation

Toldo S, Seropian IM, Mezzaroma E, Van Tassell BW, Salloum FN, Lewis EC, Voelkel N, Dinarello CA, Abbate A. Alpha-1 antitrypsin inhibits caspase-1 and protects from acute myocardial ischemia-reperfusion injury. J Mol Cell Cardiol. 2011 Aug;51(2):244-51. doi: 10.1016/j.yjmcc.2011.05.003. Epub 2011 May 12.

Results Reference

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PubMed Identifier

18852465

Citation

Lewis EC, Mizrahi M, Toledano M, Defelice N, Wright JL, Churg A, Shapiro L, Dinarello CA. alpha1-Antitrypsin monotherapy induces immune tolerance during islet allograft transplantation in mice. Proc Natl Acad Sci U S A. 2008 Oct 21;105(42):16236-41. doi: 10.1073/pnas.0807627105. Epub 2008 Oct 13.

Results Reference

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PubMed Identifier

11502863

Citation

Churg A, Dai J, Zay K, Karsan A, Hendricks R, Yee C, Martin R, MacKenzie R, Xie C, Zhang L, Shapiro S, Wright JL. Alpha-1-antitrypsin and a broad spectrum metalloprotease inhibitor, RS113456, have similar acute anti-inflammatory effects. Lab Invest. 2001 Aug;81(8):1119-31. doi: 10.1038/labinvest.3780324.

Results Reference

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

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