Acute-graft-versus-host DiseaseNCT03172455
Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)
A study testing Glassia in people with Acute-graft-versus-host Disease and Steroid Refractory Acute Graft Versus Host Disease, run by Impatients N.V. trading as myTomorrows.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease, Graft-versus-host-disease
- What is being tested?
- Glassia
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
About this study
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.
Read the 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
- 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
- Being tested
- Glassia
- Medical subject headings
- Graft vs Host Disease, alpha 1-Antitrypsin Deficiency, alpha-1-Antitrypsin Deficiency, Autosomal Recessive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GlassiaDrug
Also called Alpha 1 antitrypsin
Application 90 mg/kg loading dose day 1, 30 mg/kg every other day (day 3 - day 15)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Acute-graft-versus-host DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Impatients N.V. trading as myTomorrows
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Kamada, Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03172455
- Sponsor's protocol ID
- 2017-AATGLAKAM-EU
- Status last verified
- Sep 2020
- First submitted
- 29 May 2017
- Last updated
- 5 Oct 2020
- Results posted
- No
Publications
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.
PubMed 3886224Toldo 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.
PubMed 21600901Lewis 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.
PubMed 18852465Churg 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.
PubMed 11502863
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)
- ClinicalTrials.gov ID
- NCT03172455
- Acronym / sponsor ID
- 2017-AATGLAKAM-EU
- 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
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Impatients N.V. trading as myTomorrowsIndustry (pharma, biotech, devices)
- Collaborators
- Kamada, Ltd.
- First posted
- 1 Jun 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (7 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GlassiaAlso: Alpha 1 antitrypsinApplication 90 mg/kg loading dose day 1, 30 mg/kg every other day (day 3 - day 15) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2020.
- Background: PubMed 3886224Breit 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.
- Background: PubMed 21600901Toldo 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.
- Background: PubMed 18852465Lewis 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.
- Background: PubMed 11502863Churg 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Glassia in people with Acute-graft-versus-host Disease and Steroid Refractory Acute Graft Versus Host Disease, run by Impatients N.V. trading as myTomorrows.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Oct 2020. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03172455, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2020.