Alpha 1-Antitrypsin DeficiencyPhase 3ChAMPNCT00295061
Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults
A Phase 3 study testing Alpha-1 MP and alpha-1 proteinase inhibitor (human) in people with Alpha 1-Antitrypsin Deficiency, run by Grifols Therapeutics LLC at 8 sites in United States.
Trial summary
The purpose of this clinical study (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP) and Prolastin 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 MP and alpha-1 proteinase inhibitor (human)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 8 sitesUnited States
- Study dates
- May 2006to Feb 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
- Must be receiving augmentation therapy with plasma-derived (human) Alpha1-Proteinase Inhibitor (Prolastin®) for at least one month prior to study entry.
- 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
8 sites in 1 country.
8 sites in 1 country
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
About this study
Official title: Multi-center, Randomized, Double-blind, Crossover Trial to Evaluate the Pharmacokinetic Comparability of Alpha-1 MP to Prolastin in Subjects With Alpha1-antitrypsin Deficiency.
The purpose of this clinical study (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP) and Prolastin in adult Alpha1-antitrypsin deficient patients. Patients will be infused intravenously with study drug on a weekly schedule for 24 weeks.
Read the detailed description
The objective of this study is to demonstrate the pharmacokinetic comparability of Alpha-1 MP to Prolastin® in subjects with Alpha1-antitrypsin deficiency.
This study is divided into three 8-week treatment sequences including an initial 8-week double-blind treatment period (with one of the 2 study drugs), a second 8-week double-blind treatment period (with the other study drug), and a third 8-week open-label treatment period (with Alpha-1 MP).
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Being tested
- Alpha-1 MP, 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 MPDrug
Also called Alpha-1 antitrypsin (AAT), TAL6004
alpha-1 proteinase inhibitor (human), 60 mg/kg body weight
alpha-1 proteinase inhibitor (human)Drug
Also called Alpha-1 antitrypsin (AAT), BAY x 5747, BAY 10-5233, TAL-05-00007
Prolastin
| Study group | Role | What they receive |
|---|---|---|
| 1 Alpha-1 MP | Experimental | Sequential, blinded treatment periods of Alpha-1 MP (experimental), then crossed-over to Prolastin (active comparator), followed by open-label Alpha-1 MP Receives: Drug: Alpha-1 MP |
| 2 Prolastin | Active comparator | Sequential, blinded treatment periods of Prolastin (active comparator), then crossed-over to Alpha-1 MP (experimental), followed by open-label Alpha-1 MP Receives: Drug: alpha-1 proteinase inhibitor (human) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Alpha-1 MP vs. Prolastin® of Area Under the Curve (AUC) From Day 0 to Day 7
The primary objective of this study was to demonstrate the pharmacokinetic comparability (geometric least square mean ratio of AUC between the Alpha-1 MP vs. Prolastin®, 90% confidence interval falls within 0.80-1.25, FDA Guidance as being "bioequivalent" between two treatments) of Alpha-1 MP to Prolastin® in subjects with alpha-1-anti-trypsin (AAT) deficiency by comparing AUC from Day 0 to Day 7 of plasma Alpha1-PI measured by the functional activity (potency) assay. AUC from Day 0 to Day 7 was calculated at steady state at the end of the first and second 8-week treatment periods during the 16-week double-blind, crossover phase.
Measured: Day 0 to Day 7
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2006 (estimated)
- Study startMay 2006
- Primary completionFeb 2007
- Study completionFeb 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 (1)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
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grifols Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Kim Hanna, MSc, Study director, Grifols Therapeutics LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00295061
- Sponsor's protocol ID
- 11816
- Acronym
- ChAMP
- Status last verified
- Aug 2014
- First submitted
- 20 Feb 2006
- Last updated
- 9 Sep 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Stocks JM, Brantly ML, Wang-Smith L, Campos MA, Chapman KR, Kueppers F, Sandhaus RA, Strange C, Turino G. Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha(1)-antitrypsin deficiency: a randomized study. BMC Clin Pharmacol. 2010 Sep 30;10:13. doi: 10.1186/1472-6904-10-13.
PubMed 20920295
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-center, Randomized, Double-blind, Crossover Trial to Evaluate the Pharmacokinetic Comparability of Alpha-1 MP to Prolastin in Subjects With Alpha1-antitrypsin Deficiency.
- ClinicalTrials.gov ID
- NCT00295061
- Acronym / sponsor ID
- ChAMP / 11816
- 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
- 24 participants (actual)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- May 2006
- Primary completion
- Feb 2007 (actual)
- Study completion
- Feb 2007 (actual)
- First posted
- 22 Feb 2006 (estimated)
- Results
- Posted 9 Sep 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 (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP) and Prolastin in adult Alpha1-antitrypsin deficient patients. Patients will be infused intravenously with study drug on a weekly schedule for 24 weeks.
Detailed description
The objective of this study is to demonstrate the pharmacokinetic comparability of Alpha-1 MP to Prolastin® in subjects with Alpha1-antitrypsin deficiency.
This study is divided into three 8-week treatment sequences including an initial 8-week double-blind treatment period (with one of the 2 study drugs), a second 8-week double-blind treatment period (with the other study drug), and a third 8-week open-label treatment period (with Alpha-1 MP).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 Alpha-1 MP | Experimental | Drug: Alpha-1 MPSequential, blinded treatment periods of Alpha-1 MP (experimental), then crossed-over to Prolastin (active comparator), followed by open-label Alpha-1 MP |
| 2 Prolastin | Active comparator | Drug: alpha-1 proteinase inhibitor (human)Sequential, blinded treatment periods of Prolastin (active comparator), then crossed-over to Alpha-1 MP (experimental), followed by open-label Alpha-1 MP |
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
- Must be receiving augmentation therapy with plasma-derived (human) Alpha1-Proteinase Inhibitor (Prolastin®) for at least one month prior to study entry.
- 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 MPAlso: Alpha-1 antitrypsin (AAT), TAL6004alpha-1 proteinase inhibitor (human), 60 mg/kg body weight | Drug | 1 Alpha-1 MP |
| alpha-1 proteinase inhibitor (human)Also: Alpha-1 antitrypsin (AAT), BAY x 5747, BAY 10-5233, TAL-05-00007Prolastin | Drug | 2 Prolastin |
Primary outcome
Alpha-1 MP vs. Prolastin® of Area Under the Curve (AUC) From Day 0 to Day 7
Time frame: Day 0 to Day 7
The primary objective of this study was to demonstrate the pharmacokinetic comparability (geometric least square mean ratio of AUC between the Alpha-1 MP vs. Prolastin®, 90% confidence interval falls within 0.80-1.25, FDA Guidance as being "bioequivalent" between two treatments) of Alpha-1 MP to Prolastin® in subjects with alpha-1-anti-trypsin (AAT) deficiency by comparing AUC from Day 0 to Day 7 of plasma Alpha1-PI measured by the functional activity (potency) assay. AUC from Day 0 to Day 7 was calculated at steady state at the end of the first and second 8-week treatment periods during the 16-week double-blind, crossover phase.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 20920295Stocks JM, Brantly ML, Wang-Smith L, Campos MA, Chapman KR, Kueppers F, Sandhaus RA, Strange C, Turino G. Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha(1)-antitrypsin deficiency: a randomized study. BMC Clin Pharmacol. 2010 Sep 30;10:13. doi: 10.1186/1472-6904-10-13.
- 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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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
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Alpha-1 MP and alpha-1 proteinase inhibitor (human) in people with Alpha 1-Antitrypsin Deficiency, run by Grifols Therapeutics LLC at 8 sites in United States.
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 9 Sep 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. 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 9 Sep 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?
Alpha-1 MP vs. Prolastin® of Area Under the Curve (AUC) From Day 0 to Day 7 (time frame: Day 0 to Day 7). 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 NCT00295061, which also lists 1 linked publication.
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 9 Sep 2014.