Early Access Program Using Alpha 1 Antitrypsin Infusion f... | NCT03172455

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

NCT03172455

Sponsor

Impatients N.V. trading as myTomorrows

Status

No longer available

Last Update Posted

Oct 5, 2020Actual

Enrollment

Not provided

Phase

Not provided

Conditions

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

Interventions

Glassia

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Status Module

Record Verification Date

Sep 2020

Overall Recruitment Status or Expanded Access Status

No longer available

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

May 29, 2017

First Submission Date that Met QC Criteria

May 29, 2017

First Posted Date

Jun 1, 2017Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 30, 2020

Last Update Posted Date

Oct 5, 2020Actual

Name	Class
Kamada, Ltd.	INDUSTRY

Description Module

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).

Conditions Module

Conditions

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

Keywords

Not provided

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Name	Type	Description	Arm Group Labels	Other Names
Glassia	Drug	Application 90 mg/kg loading dose day 1, 30 mg/kg every other day (day 3 - day 15)		Alpha 1 antitrypsin

Eligibility Module

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

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
3886224	Background	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.
21600901	Background	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.

ID	Term
D006086	Graft vs Host Disease
D019896	alpha 1-Antitrypsin Deficiency
C566273	alpha-1-Antitrypsin Deficiency, Autosomal Recessive

ID	Term
D007154	Immune System Diseases
D008107	Liver Diseases
D004066	Digestive System Diseases
D008171	Lung Diseases
D012140	Respiratory Tract Diseases
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D013352	Subcutaneous Emphysema
D004646	Emphysema
D010335	Pathologic Processes
D013568	Pathological Conditions, Signs and Symptoms

ID	Term
D006023	Glycoproteins
D006001	Glycoconjugates
D002241	Carbohydrates
D015843	Serpins
D010455	Peptides
D000602	Amino Acids, Peptides, and Proteins
D000209	Acute-Phase Proteins
D001798	Blood Proteins
D011506	Proteins
D000510	Alpha-Globulins
D012712	Serum Globulins
D005916	Globulins