Alpha 1-Antitrypsin DeficiencyPhase 3STAMPNCT00301366
The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency
A Phase 3 study testing alpha-1 proteinase inhibitor (human) in people with Alpha 1-Antitrypsin Deficiency, run by Grifols Therapeutics LLC at 10 sites in 2 countries.
Trial summary
The purpose of this clinical study is to assess the safety and tolerability of Alpha-1 MP in adult Alpha1-antitrypsin deficient patients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency
- What is being tested?
- alpha-1 proteinase inhibitor (human)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 10 sitesin 2 countries
- Study dates
- Jun 2006to Mar 2007
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Documented diagnosis of congenital Alpha1-antitrypsin deficiency
- Documented forced expiratory volume in 1 second (FEV1 ) between 20% - 80% of predicted value within last 6 months.
- Signed written informed consent prior to initiation of any study related procedures.
Who cannot join
- Females who are pregnant, breast feeding, or if of child-bearing potential, unwilling to practice adequate contraception throughout the study
- Use of systemic steroids within the 2 weeks prior to receiving study treatment (this does not include the use of inhaled steroids used on a routine or as needed basis).
- Subjects who have had exacerbations of their disease within one month of trial entry
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States8 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Cleveland Clinic Foundation
Cleveland, Ohio, 44122
National Jewish Medical and Research Center
Denver, Colorado, 80206
University of Florida College of Medicine
Gainesville, Florida, 32610-0225
University of Miami School of Medicine
Miami, Florida, 33101
St Lukes-Roosevelt Hospital Center, New York
New York, New York, 10019
Temple University Hospital
Philadelphia, Pennsylvania, 19140
University of Texas Health Center at Tyler
Tyler, Texas, 75708-3154
United Kingdom2 sites
University of Cambridge - Cambridge Institute for Medical Research
Cambridge, England, CB2 2XY
University Teaching Hospital of Edinburgh
Edinburgh, Scotland, EH8 9AG
About this study
Official title: Multi-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) Deficiency
The purpose of this clinical study is to assess the safety and tolerability of Alpha-1 MP in adult Alpha1-antitrypsin deficient patients.
Read the detailed description
The objective of this clinical trial (STAMP: Safety and Tolerability of Alpha-1 Modified Process) is to study the safety and tolerability of Alpha-1 MP in adult Alpha 1-antitrypsin deficient subjects as reported over 20 weeks of therapy. The primary objective is to describe the nature and frequency of treatment-emergent adverse events with "treatment-emergent" defined as any adverse event occurring after the start of the first study drug infusion.
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Being tested
- alpha-1 proteinase inhibitor (human)
- Keywords
- alpha 1-Antitrypsin Deficiency, alpha 1-Antitrypsin, pulmonary emphysema
- Medical subject headings
- alpha 1-Antitrypsin Deficiency, Pulmonary Emphysema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
alpha-1 proteinase inhibitor (human)Drug
Also called Prolastin, Alpha-1 antitrypsin (AAT), BAY x 5747, BAY 10-5233
60 mg/kg weekly for 20 weeks
| Study group | Role | What they receive |
|---|---|---|
| Alpha-1 Proteinase Inhibitor (Human), modified process | Experimental | Study the safety and tolerability of weekly infusions of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP, 60 mg/kg) over 20 weeks of therapy in adult Alpha-1 antitrypsin deficient subjects. Receives: Drug: alpha-1 proteinase inhibitor (human) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.
An adverse event is any untoward medical occurrence in a subject or clinical investigation subject administered with a pharmaceutical product. The adverse event does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the medicinal product.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2006 (estimated)
- Study startJun 2006
- Primary completionMar 2007
- Study completionMar 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Alpha 1-Antitrypsin DeficiencyStudies 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grifols Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kim Hanna, MSc, Study director, Grifols Therapeutics LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00301366
- Sponsor's protocol ID
- 11815
- Acronym
- STAMP
- Status last verified
- Jul 2014
- First submitted
- 8 Mar 2006
- Last updated
- 19 Aug 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med. 1987 Apr 23;316(17):1055-62. doi: 10.1056/NEJM198704233161704.
PubMed 3494198Gadek JE, Klein HG, Holland PV, Crystal RG. Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Invest. 1981 Nov;68(5):1158-65. doi: 10.1172/jci110360.
PubMed 7028785Gadek JE, Fells GA, Zimmerman RL, Rennard SI, Crystal RG. Antielastases of the human alveolar structures. Implications for the protease-antiprotease theory of emphysema. J Clin Invest. 1981 Oct;68(4):889-98. doi: 10.1172/jci110344.
PubMed 6169740
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) Deficiency
- ClinicalTrials.gov ID
- NCT00301366
- Acronym / sponsor ID
- STAMP / 11815
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Keywords
- alpha 1-Antitrypsin Deficiency, alpha 1-Antitrypsin, pulmonary emphysema
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- Jun 2006
- Primary completion
- Mar 2007 (actual)
- Study completion
- Mar 2007 (actual)
- First posted
- 10 Mar 2006 (estimated)
- Results
- Posted 19 Aug 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this clinical study is to assess the safety and tolerability of Alpha-1 MP in adult Alpha1-antitrypsin deficient patients.
Detailed description
The objective of this clinical trial (STAMP: Safety and Tolerability of Alpha-1 Modified Process) is to study the safety and tolerability of Alpha-1 MP in adult Alpha 1-antitrypsin deficient subjects as reported over 20 weeks of therapy. The primary objective is to describe the nature and frequency of treatment-emergent adverse events with "treatment-emergent" defined as any adverse event occurring after the start of the first study drug infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Alpha-1 Proteinase Inhibitor (Human), modified process | Experimental | Drug: alpha-1 proteinase inhibitor (human)Study the safety and tolerability of weekly infusions of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP, 60 mg/kg) over 20 weeks of therapy in adult Alpha-1 antitrypsin deficient subjects. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Documented diagnosis of congenital Alpha1-antitrypsin deficiency
- Documented forced expiratory volume in 1 second (FEV1 ) between 20% - 80% of predicted value within last 6 months.
- Signed written informed consent prior to initiation of any study related procedures.
Key exclusion criteria (3 listed)
- Females who are pregnant, breast feeding, or if of child-bearing potential, unwilling to practice adequate contraception throughout the study
- Use of systemic steroids within the 2 weeks prior to receiving study treatment (this does not include the use of inhaled steroids used on a routine or as needed basis).
- Subjects who have had exacerbations of their disease within one month of trial entry
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 |
|---|---|---|
| alpha-1 proteinase inhibitor (human)Also: Prolastin, Alpha-1 antitrypsin (AAT), BAY x 5747, BAY 10-523360 mg/kg weekly for 20 weeks | Drug | Alpha-1 Proteinase Inhibitor (Human), modified process |
Primary outcome
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.
Time frame: 24 weeks
An adverse event is any untoward medical occurrence in a subject or clinical investigation subject administered with a pharmaceutical product. The adverse event does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the medicinal product.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 3494198Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med. 1987 Apr 23;316(17):1055-62. doi: 10.1056/NEJM198704233161704.
- Background: PubMed 7028785Gadek JE, Klein HG, Holland PV, Crystal RG. Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Invest. 1981 Nov;68(5):1158-65. doi: 10.1172/jci110360.
- Background: PubMed 6169740Gadek JE, Fells GA, Zimmerman RL, Rennard SI, Crystal RG. Antielastases of the human alveolar structures. Implications for the protease-antiprotease theory of emphysema. J Clin Invest. 1981 Oct;68(4):889-98. doi: 10.1172/jci110344.
- The "Alpha-1 Foundation", dedicated to providing the leadership and resources that will result in increased research, improved health, worldwide detection and a cure for Alpha-1 Antitrypsin DeficiencyLink provided by the sponsor.
- AlphaNet, Inc devoted to improving the lives of individuals with Alpha-1 antitrypsin deficiency through comprehensive disease management services, clinical research administration, and consultative servicesLink provided by the sponsor.
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Alpha 1 Antitrypsin Deficiency
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.
Kim Hanna, MSc study director
Grifols Therapeutics LLC
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing alpha-1 proteinase inhibitor (human) in people with Alpha 1-Antitrypsin Deficiency, run by Grifols Therapeutics LLC at 10 sites in 2 countries.
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 3 inclusion and 3 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: finished, as of the sponsor's last update on 19 Aug 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 19 Aug 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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.
What does the primary outcome measure?
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00301366, which also lists 3 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 19 Aug 2014.