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Trial record 19 of 23 for:    Recruiting, Not yet recruiting, Available Studies | "alpha 1-Antitrypsin Deficiency"

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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ClinicalTrials.gov Identifier: NCT03172455
Expanded Access Status : Available
First Posted : June 1, 2017
Last Update Posted : August 8, 2018
Sponsor:
Collaborator:
Kamada, Ltd.
Information provided by (Responsible Party):
Impatients N.V. trading as myTomorrows

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.


Condition or disease Intervention/treatment
SR aGvHD 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 Drug: Glassia

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



Intervention Details:
  • Drug: Glassia
    Application 90 mg/kg loading dose day 1, 30 mg/kg every other day (day 3 - day 15)
    Other Name: Alpha 1 antitrypsin

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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.
  • Participants should be newly diagnosed.
  • For women of childbearing potential, had a negative serum or urine pregnancy test within 14 days prior to enrollment.

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

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): NCT03172455


Contacts
Contact: Michel van Harten, MD +31 885253888 michel.vanharten@mytomorrows.com
Contact: Alice Cardoso, MSc +31 885253888 alice.cardoso@mytomorrows.com

Sponsors and Collaborators
Impatients N.V. trading as myTomorrows
Kamada, Ltd.

Publications:
Responsible Party: Impatients N.V. trading as myTomorrows
ClinicalTrials.gov Identifier: NCT03172455     History of Changes
Other Study ID Numbers: 2017-AATGLAKAM-EU
First Posted: June 1, 2017    Key Record Dates
Last Update Posted: August 8, 2018
Last Verified: August 2018

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