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Diabetes Mellitus, Type 1Phase 2RETAINNCT01183455

A Research Trial of Aralast NP in New Onset Diabetes (RETAIN) - Part II

A Phase 2 study testing Aralast NP in people with Diabetes Mellitus, Type 1, run by National Institute of Allergy and Infectious Diseases (NIAID) at 15 sites in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

Aralast NP (alpha-1 antitrypsin, AAT), an alpha-1 proteinase inhibitor (human), was the drug to be tested in this clinical trial. Aralast NP is an anti-inflammatory drug that affects the cells that are thought to be involved in the development of type 1 diabetes mellitus (T1DM, T1D). Read more about this study

Who is it for?
Ages 8 to 35 years
Conditions the study is for
Diabetes Mellitus, Type 1
What is being tested?
Aralast NP
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
0 participants
Where
15 sitesUnited States
Study dates
Oct 2010to Aug 2014
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 8 to 35 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Diagnosed with type 1 diabetes (T1D) within the past 100 days
  • Positive for at least one diabetes-related autoantibody (anti-GAD; anti-insulin, if obtained within 10 days of the onset of insulin therapy; IA-2 antibody and/or ICA, or ZnT8)
  • Peak stimulated C-peptide level greater than (>) 0.2 pmol/mL following a mixed meal tolerance test (MMTT)

Who cannot join

  • Severe active disease (hepatitis, cardiac or pulmonary disease, hypertension, immunodeficiency)
  • History of any bleeding or clotting factor deficiencies, or stroke
  • History of vascular disease or significant vascular abnormalities
  • Positive serology exposure to hepatitis B virus (HBV), hepatitis C virus (HCV), human immunodeficiency virus (HIV) or toxoplasmosis
  • Clinically active infection with Epstein-Barr virus (EBV), cytomegalovirus (CMV), or tuberculosis (TB)
  • Prior or current use of oral, inhaled or intranasal glucocorticoids, or any medication known to cause a significant, ongoing change in the course of T1D or immunologic status
Show all 14Show fewer
  • Prior treatment with alpha1-antitrypsin (AAT) or hypersensitivity to AAT or human plasma-derived products
  • Current or prior (within the last 30 days) use of metformin, sulfonylureas, glinides, thiazolidinediones, exenatide, liraglutide, DPP-IV inhibitors or amylin
  • Current use of any medication known to influence glucose tolerance (e.g., beta-blockers, angiotensin-converting enzyme inhibitors, interferons, quinidine anti-malarial drugs, lithium, niacin)
  • Females who are pregnant or lactating, or are unwilling to defer pregnancy during study participation
  • Immunoglobulin A (IgA) deficiency
  • Uncontrolled hypertension
  • Current life-threatening malignancy
  • Any condition that in the investigator's opinion may compromise study participation or may confound the interpretation of the study results

Trial locations and contacts

15 sites in 1 country.

15 sites in 1 country

United States15 sites

  • Atlanta Diabetes Associates

    Atlanta, Georgia, 30309

  • Emory University

    Atlanta, Georgia, 30322

  • Barbara Davis Center

    Aurora, Colorado, 80045

  • University of Maryland Medical Center

    Baltimore, Maryland, 21201

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Joslin Diabetes Center

    Boston, Massachusetts, 02215

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205

  • University of Iowa

    Iowa City, Iowa, 52242

  • University of California San Diego

    La Jolla, California, 92093

  • Yale University

    New Haven, Connecticut, 06511

  • Columbia University

    New York, New York, 10027

  • The Children's Hospital of Philadelphia

    Philadephia, Pennsylvania, 19104

  • Calvert Memorial Hospital

    Prince Frederick, Maryland, 20678

  • Cetero Research San Antonio

    San Antonio, Texas, 78229

  • University of Massachusetts Medical School

    Worchester, Massachusetts, 01655

Other trials in United States

About this study

Official title: Effect of Intravenous Alpha-1 Antitrypsin on Preserving Beta-cell Function in New-onset Type 1 Diabetes Mellitus (ITN041AI)- Part II

Aralast NP (alpha-1 antitrypsin, AAT), an alpha-1 proteinase inhibitor (human), was the drug to be tested in this clinical trial. Aralast NP is an anti-inflammatory drug that affects the cells that are thought to be involved in the development of type 1 diabetes mellitus (T1DM, T1D). This study, known as RETAIN, was planned as a two-part trial to investigate the effect of Aralast NP on preserving beta cell function and to determine if the intervention would slow the progression of type 1 diabetes.

Part I of this trial (NCT 01183468) was an open-label, safety and dose level study consisting of two groups. After completion of Part I, including a satisfactory safety review, enrollment in Part II was to begin. Part II was designed as a two-arm, double-blind, placebo-controlled clinical trial, and participants were to be randomly assigned to either the Aralast NP treatment or placebo group.

Read the detailed description

T1D is an autoimmune disease. This means that your immune system (the part of your body that helps fight infections) mistakenly attacks the cells that produce insulin (beta cells in the pancreas). As beta cells are destroyed by your immune cells, your ability to produce insulin is decreased. Insulin helps keep blood glucose levels normal.

Individuals with T1D who have the ability to produce some of their own insulin (even though they still need to take insulin) may be able to achieve better glucose control than people who produce no insulin at all. Better glucose control has been shown to reduce the long-term complications of diabetes. Previous research has shown that giving medicines to affect the immune system soon after type 1 diabetes is diagnosed may stop, delay or decrease the destruction of beta cells, resulting in better glucose control.

In mouse models of disease, alpha-1 proteinase inhibitors have been shown to reverse new-onset diabetes and induce a state of self-tolerance. The RETAIN clinical trial was intended to investigate the effect of Aralast NP on preserving beta cell function and slowing the progression of T1D.

Being tested
Aralast NP, Placebo
Keywords
Diabetes Mellitus, Type 1 (new-onset), T1DM (new-onset), T1D (new-onset), Diabetes, Autoimmune, Alpha1-Proteinase Inhibitor, Alpha-1 Antitrypsin, AAT
Medical subject headings
Diabetes Mellitus, Type 1, Diabetes Mellitus, alpha 1-Antitrypsin Deficiency

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Aralast NPDrug

    Also called Alpha 1-Proteinase Inhibitor Human, Alpha,-antitrypsin, AAT

    Participants will receive IV infusions of Aralast NP (90mg/kg) once a week for 12 weeks.

  • PlaceboDrug

    Also called Placebo for Aralast NP

    Participants will receive IV infusions of placebo once a week for 12 weeks.

Study groups in NCT01183455
Study groupRoleWhat they receive
Aralast NPExperimentalParticipants will receive Aralast NP (90mg/kg) intravenously once a week for 12 weeks.
Receives: Drug: Aralast NP
PlaceboPlacebo comparatorParticipants will receive placebo intravenously once a week for 12 weeks.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Mixed-Meal Tolerance Test (MMTT)-Stimulated 2-hour C-peptide Area Under the Curve (AUC)

    Measured: Week 52

Secondary outcomes (9)

  1. MMTT-Stimulated Peak and 4-hour C-peptide AUC

    Measured: Weeks 52 and 104

  2. MMTT-Stimulated 2-hour C-Peptide AUC Assessed Over Time

    Measured: Weeks 0, 14, 26, 52, and 104

  3. Insulin Use in Units Per Kilogram Body Weight Per Day

    Measured: Weeks 52 and 104

  4. Hypoglycemic Events Occurring from Randomization to End of Trial

    Measured: Throughout the Study

  5. Glycosylated Hemoglobin (HbA1c) Levels

    Measured: Weeks 52 and 104

  6. Emergence of Anti-Alpha 1-Antitrypsin (AAT) Antibodies

    Measured: Throughout the Study

  7. Frequency and Severity of All Adverse Events (AEs)

    Measured: Throughout the study

  8. Changes in D-dimer Levels Indicating Alteration in Coagulant/Anticoagulant Balance

    Measured: Throughout the study

  9. Pharmacokinetic Parameters of Aralast NP

    Measured: Throughout the study

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Aug 2010 (estimated)
  2. Study startOct 2010
  3. Primary completionAug 2014
  4. Study completionAug 2014

Why it stopped: Sponsor decision-lack of mechanistic signal and competing industry studies

Resources

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
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01183455
Sponsor's protocol ID
DAIT ITN041AI Part II
Acronym
RETAIN
Status last verified
Dec 2014
First submitted
16 Aug 2010
Last updated
31 Dec 2014
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Aralast NP in people with Diabetes Mellitus, Type 1, run by National Institute of Allergy and Infectious Diseases (NIAID) at 15 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 14 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: withdrawn, as of the sponsor's last update on 31 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 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. Some participants in this study may receive a placebo. 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 was withdrawn before enrolling participants, so there are no results.

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?

Mixed-Meal Tolerance Test (MMTT)-Stimulated 2-hour C-peptide Area Under the Curve (AUC) (time frame: Week 52). 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 NCT01183455.

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 31 Dec 2014.

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