DiabetesPhase 1NCT01319331
The Effects of Alpha-1 Antitrypsin (AAT) on the Progression of Type 1 Diabetes
A Phase 1 study testing Alpha 1-Antitrypsin (AAT, Aralast NP) in people with Diabetes and Type 1 Diabetes, run by University of Colorado, Denver at 1 site in United States.
Trial summary
The purpose of this study is to determine if the drug Alpha-1 Antitrypsin (AAT, Aralast NP) will preserve beta-cell function and help slow the progression of type 1 diabetes. Read more about this study
- Who is it for?
- Ages 6 to 45 years
- Conditions the study is for
- Diabetes, Type 1 Diabetes
- What is being tested?
- Alpha 1-Antitrypsin (AAT, Aralast NP)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2010to May 2016
- Phase
- Phase 1
- 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 6 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of Type 1 Diabetes Mellitus based on ADA Criteria for fewer than 5 years but more than 100 days
- 6-45 years of age, inclusive. To assess safety, we will initially enroll 8 patients over the age of 16. Following the last infusion of the 8th patient, we will assess adverse events. As long as there are no stopping criteria met for these 8 patients we will decrease the age criteria down to 6 years old.
- C-peptide increase during screening mixed meal tolerance test with a minimal stimulated value of ≥ 0.2 pmol/mL.
- Positive for antibodies to insulin (if insulin autoantibody positive only, determination must be within two weeks of insulin initiation), GAD-65, IA-2 or ZnT8
- Agree to intensive management of diabetes with an HgbA1c goal of < 7.0%
- If female, (a) surgically sterile or (b) postmenopausal or (c) if of reproductive potential, willing to use medically acceptable birth control (e.g. female hormonal contraception, barrier methods or sterilization. ) until 3 months after completion of any treatment period
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- If male and of reproductive potential, willing to use medically acceptable birth control until 3 months after completion of any treatment period, unless the female partner is postmenopausal or surgically sterile
- Serum creatinine ≤ 1.5 x upper limit of normal
- AST < 2 times the upper limit of normal
- Hematology:WBC > 3000 x 109/L; platelets > 100 x 109/L; hemoglobin > 10.0 g/dL.
Who cannot join
- Unable or unwilling to comply with the requirements of the study protocol
- Body Mass Index (BMI) > 30 kg/m2
- Unstable blood sugar control defined as one or more episodes of severe hypoglycemia (defined as hypoglycemia that required the assistance of another person) within the last 30 days
- Previous immunotherapy for T1D
- Administration of an experimental agent for T1D at any time or use of an experimental device for T1D within 30 days of screening, unless approved by the study PI
- History of any organ transplant, including islet cell transplant
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- Active autoimmune or immune deficiency disorder (e.g. sarcoidosis, rheumatoid arthritis)
- Serum bilirubin > ULN, except those subjects whose abnormal values were attributed to any stable, benign condition (such as Gilbert's Syndrome) may be included
- TSH outside the normal range at screening, except those subjects on stable doses of thyroid hormone replacement therapy may be included
- Known HIV positivity, active hepatitis B or active hepatitis C infection
- Anticipated pregnancy during active dosing or within 3 months after completion of active dosing phase
- History of a malignant neoplasm within the previous 5 years (except in situ cervical cancer and curable non-melanoma skin malignancy)
- Any social condition or medical condition that would, in the opinion of the investigator, prevent complete participation in the study or that would pose a significant hazard to the subjects' participation
- History of active substance abuse within 12 months of screening
- A psychiatric or medical disorder that would prevent giving informed consent
- Individuals with a history of IgA deficiency
- Individuals with a history of hypersensitivity to AAT
Trial locations and contacts
1 site in 1 country.
United States1 site
Barbara Davis Center for Childhood Diabetes
Aurora, Colorado, 80045
About this study
Official title: The Effects of Open Label Alpha-1 Antitrypsin on the Progression of Type 1 Diabetes in Subjects With Detectable C-peptide
The purpose of this study is to determine if the drug Alpha-1 Antitrypsin (AAT, Aralast NP) will preserve beta-cell function and help slow the progression of type 1 diabetes.
- Conditions
- Diabetes, Type 1 Diabetes
- Being tested
- Alpha 1-Antitrypsin (AAT, Aralast NP)
- Keywords
- diabetes, type 1 diabetes, AAT, Alpha-1 Antitrypsin
- Medical subject headings
- Diabetes Mellitus, Diabetes Mellitus, Type 1, alpha 1-Antitrypsin Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Alpha 1-Antitrypsin (AAT, Aralast NP)Drug
Also called Alpha-1 Antitrypsin, AAT, Aralast NP
Eligible subjects will be treated once a week for 8 weeks (8 total treatments).
| Study group | Role | What they receive |
|---|---|---|
| Alpha-1 Antitrypsin (AAT, Aralast NP) | Experimental | Alpha-1 Antitrypsin (AAT, Aralast NP) as prescribed for study duration Receives: Drug: Alpha 1-Antitrypsin (AAT, Aralast NP) |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess participant safety & feasibility of study drug administration
Measured: Study duration is 2 years
Secondary outcomes (2)
To assess AAT treatment on the maintenance of c-peptide production
Measured: Stimulated c-peptide at year one and two.
Assess the effects of AAT on glycemic variability and A1c.
Measured: Continuous Glucose Monitoring at one and two years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2011 (estimated)
- Study startOct 2010
- Primary completionOct 2015
- Study completionMay 2016
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 DiabetesStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe 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 1
- 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 Colorado, Denver
- Sponsor type
- University, hospital or other
- Collaborators
- Omni Bio Pharmaceutical, Inc.
- Responsible party
- Sponsor
- Investigators
- Peter A Gottlieb, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01319331
- Sponsor's protocol ID
- 09-0667
- Status last verified
- Mar 2017
- First submitted
- 17 Mar 2011
- Last updated
- 24 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Ozeri E, Mizrahi M, Shahaf G, Lewis EC. alpha-1 antitrypsin promotes semimature, IL-10-producing and readily migrating tolerogenic dendritic cells. J Immunol. 2012 Jul 1;189(1):146-53. doi: 10.4049/jimmunol.1101340. Epub 2012 May 25.
PubMed 22634621
Study at a glance
From the registry record, last updated by the sponsor on 24 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effects of Open Label Alpha-1 Antitrypsin on the Progression of Type 1 Diabetes in Subjects With Detectable C-peptide
- ClinicalTrials.gov ID
- NCT01319331
- Acronym / sponsor ID
- 09-0667
- Conditions
- Diabetes, Type 1 Diabetes
- Keywords
- diabetes, type 1 diabetes, AAT, Alpha-1 Antitrypsin
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Collaborators
- Omni Bio Pharmaceutical, Inc.
- Study start
- Oct 2010
- Primary completion
- Oct 2015 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 21 Mar 2011 (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 determine if the drug Alpha-1 Antitrypsin (AAT, Aralast NP) will preserve beta-cell function and help slow the progression of type 1 diabetes.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Alpha-1 Antitrypsin (AAT, Aralast NP) | Experimental | Drug: Alpha 1-Antitrypsin (AAT, Aralast NP)Alpha-1 Antitrypsin (AAT, Aralast NP) as prescribed for study duration |
Who may qualify
- Ages
- 6 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Diagnosis of Type 1 Diabetes Mellitus based on ADA Criteria for fewer than 5 years but more than 100 days
- 6-45 years of age, inclusive. To assess safety, we will initially enroll 8 patients over the age of 16. Following the last infusion of the 8th patient, we will assess adverse events. As long as there are no stopping criteria met for these 8 patients we will decrease the age criteria down to 6 years old.
- C-peptide increase during screening mixed meal tolerance test with a minimal stimulated value of ≥ 0.2 pmol/mL.
- Positive for antibodies to insulin (if insulin autoantibody positive only, determination must be within two weeks of insulin initiation), GAD-65, IA-2 or ZnT8
- Agree to intensive management of diabetes with an HgbA1c goal of < 7.0%
Key exclusion criteria (17 listed)
- Unable or unwilling to comply with the requirements of the study protocol
- Body Mass Index (BMI) > 30 kg/m2
- Unstable blood sugar control defined as one or more episodes of severe hypoglycemia (defined as hypoglycemia that required the assistance of another person) within the last 30 days
- Previous immunotherapy for T1D
- Administration of an experimental agent for T1D at any time or use of an experimental device for T1D within 30 days of screening, unless approved by the study PI
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-Antitrypsin (AAT, Aralast NP)Also: Alpha-1 Antitrypsin, AAT, Aralast NPEligible subjects will be treated once a week for 8 weeks (8 total treatments). | Drug | Alpha-1 Antitrypsin (AAT, Aralast NP) |
Primary outcome
To assess participant safety & feasibility of study drug administration
Time frame: Study duration is 2 years
2 secondary outcomes
To assess AAT treatment on the maintenance of c-peptide production
Time frame: Stimulated c-peptide at year one and two.
Assess the effects of AAT on glycemic variability and A1c.
Time frame: Continuous Glucose Monitoring at one and two years.
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 24 Mar 2017.
- Derived from this study: PubMed 22634621Ozeri E, Mizrahi M, Shahaf G, Lewis EC. alpha-1 antitrypsin promotes semimature, IL-10-producing and readily migrating tolerogenic dendritic cells. J Immunol. 2012 Jul 1;189(1):146-53. doi: 10.4049/jimmunol.1101340. Epub 2012 May 25.
- Related InfoLink 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.
Peter A Gottlieb, MD principal investigator
University of Colorado, Denver
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Alpha 1-Antitrypsin (AAT, Aralast NP) in people with Diabetes and Type 1 Diabetes, run by University of Colorado, Denver 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 10 inclusion and 17 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 24 Mar 2017. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 assess participant safety & feasibility of study drug administration (time frame: Study duration is 2 years). 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 NCT01319331, 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 24 Mar 2017.