Alpha 1-Antitrypsin DeficiencyPhase 2NCT00067756
4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?
A Phase 2 study testing 4-PBA in people with Alpha 1-Antitrypsin Deficiency, run by University of Florida at 1 site in United States.
Trial summary
The purpose of this study is to find out whether 4-PBA will increase the level of AAT in persons with AAT deficiency whether or not they have liver disease. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Alpha 1-Antitrypsin Deficiency
- What is being tested?
- 4-PBA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2001to Oct 2003
- Phase
- Phase 2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18-65
- Serum A1-PI levels <11uM an appropriate genetic phenotype/genotype
- 5 of 10 subjects must have documented laboratory evidence of liver disease
- Willingness to withhold Prolastin therapy for 6 weeks prior to screening and throughout the 4-PBA dosing period (up to 3 months)
Who cannot join
- Any cause of liver disease other than Alpha-1 Antitrypsin deficiency
- Evidence of advanced liver disease
- HIV positive
- Use of systemic steroids, ursodeoxycholic acid (Actigall, Urso), or herbs in the prior 6 months
Trial locations and contacts
1 site in 1 country.
About this study
Official title: "4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals"
The purpose of this study is to find out whether 4-PBA will increase the level of AAT in persons with AAT deficiency whether or not they have liver disease.
Read the detailed description
The purpose of this study is to determine whether 4-PBA will significantly increase serum Z AAT levels in AAT-deficient individuals with and without evidence of hepatocellular injury and to assess its effects on liver injury.
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Being tested
- 4-PBA
- Keywords
- Pulmonary Disease, Liver Disease
- Medical subject headings
- alpha 1-Antitrypsin Deficiency, Lung Diseases, Liver Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4-PBADrug
Also called 4-phenyl butyric acid
During the first 3 days of this phase baseline serum AAT levels will be determined. The participants will be then given increased amounts of 4-PBA orally in 6 divided doses (day 4-6, 30 g/day and day 7-9, 40/day
| Study group | Role | What they receive |
|---|---|---|
| 4-PBA | Experimental | The study will involve a 4-PBA dose escalation and pharmacokinetics component The study group will be comprised of a total of at least 10 AAT-deficient,(phenotype ZZ referred to as PiZZ) patients. These patients will be divided into two groups: with and without clinical evidence of mild to moderate hepatocellular injury. Receives: Drug: 4-PBA |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.
Measured: 10 days
Secondary outcomes (1)
To determine the pharmacokinetics of 4-PBA
Measured: 10 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Aug 2003 (estimated)
- Study startNov 2001
- Primary completionOct 2003
- Study completionOct 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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.
- More trials run by University of FloridaThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Alpha-1 Foundation, Brantly, Mark L., M.D.
- Responsible party
- Sponsor
- Investigators
- Mark L Brantly, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00067756
- Sponsor's protocol ID
- 87-2001
- Status last verified
- Apr 2013
- First submitted
- 26 Aug 2003
- Last updated
- 3 Jan 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Gonzalez-Peralta RP, Leonard S, Harvey R, Schreck P, Vironosvkaya N, Brantly ML. 4-PHENYL BUTYRATE MEDIATED SECRETION RESCUE IN PATIENTS WITH ALPHA 1-ANTITRYPSIN (AAT) DEFICIENCY: A PILOT STUDY HEPATOLOGY, Vol. 44, No. 4, Suppl. 1, 2006 AASLD ABSTRACTS, p. 211A, 61
Study at a glance
From the registry record, last updated by the sponsor on 3 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- "4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals"
- ClinicalTrials.gov ID
- NCT00067756
- Acronym / sponsor ID
- 87-2001
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Keywords
- Pulmonary Disease, Liver Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Alpha-1 Foundation, Brantly, Mark L., M.D.
- Study start
- Nov 2001
- Primary completion
- Oct 2003 (actual)
- Study completion
- Oct 2003 (actual)
- First posted
- 27 Aug 2003 (estimated)
- 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.
The purpose of this study is to find out whether 4-PBA will increase the level of AAT in persons with AAT deficiency whether or not they have liver disease.
Detailed description
The purpose of this study is to determine whether 4-PBA will significantly increase serum Z AAT levels in AAT-deficient individuals with and without evidence of hepatocellular injury and to assess its effects on liver injury.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 4-PBA | Experimental | Drug: 4-PBAThe study will involve a 4-PBA dose escalation and pharmacokinetics component The study group will be comprised of a total of at least 10 AAT-deficient,(phenotype ZZ referred to as PiZZ) patients. These patients will be divided into two groups: with and without clinical evidence of mild to moderate hepatocellular injury. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Age 18-65
- Serum A1-PI levels <11uM an appropriate genetic phenotype/genotype
- 5 of 10 subjects must have documented laboratory evidence of liver disease
- Willingness to withhold Prolastin therapy for 6 weeks prior to screening and throughout the 4-PBA dosing period (up to 3 months)
Key exclusion criteria (4 listed)
- Any cause of liver disease other than Alpha-1 Antitrypsin deficiency
- Evidence of advanced liver disease
- HIV positive
- Use of systemic steroids, ursodeoxycholic acid (Actigall, Urso), or herbs in the prior 6 months
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 |
|---|---|---|
| 4-PBAAlso: 4-phenyl butyric acidDuring the first 3 days of this phase baseline serum AAT levels will be determined. The participants will be then given increased amounts of 4-PBA orally in 6 divided doses (day 4-6, 30 g/day and day 7-9, 40/day | Drug | 4-PBA |
Primary outcome
To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.
Time frame: 10 days
1 secondary outcome
To determine the pharmacokinetics of 4-PBA
Time frame: 10 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jan 2014.
- Results publicationGonzalez-Peralta RP, Leonard S, Harvey R, Schreck P, Vironosvkaya N, Brantly ML. 4-PHENYL BUTYRATE MEDIATED SECRETION RESCUE IN PATIENTS WITH ALPHA 1-ANTITRYPSIN (AAT) DEFICIENCY: A PILOT STUDY HEPATOLOGY, Vol. 44, No. 4, Suppl. 1, 2006 AASLD ABSTRACTS, p. 211A, 61
- Alpha-1 Foundation Alpha-1 Research ProgramLink 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.
Mark L Brantly, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 4-PBA in people with Alpha 1-Antitrypsin Deficiency, run by University of Florida at 1 site 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 4 inclusion and 4 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 3 Jan 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease. (time frame: 10 days). 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 NCT00067756, 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 3 Jan 2014.