Diabetes Mellitus, Type 1Phase 1RETAINNCT01183468
A Research Trial of Aralast in New Onset Diabetes (RETAIN)
A Phase 1 study testing Aralast NP 45 mg dose, Aralast NP 90 mg dose and Aralast NP 180 mg dose in people with Diabetes Mellitus, Type 1, run by National Institute of Allergy and Infectious Diseases (NIAID) at 15 sites in United States.
Trial summary
The drug Alpha-1 Antitrypsin (AAT, Aralast NP) is being tested in this study as an anti-inflammatory drug (a medication that decreases inflammation, which is part of the body's normal ability to fight infection and respond to injuries) that affects the cells thought to be involved in the development of type 1 diabetes… 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 45 mg dose, Aralast NP 90 mg dose and Aralast NP 180 mg dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 15 sitesUnited States
- Study dates
- Oct 2010to Jul 2013
- Phase
- Phase 1
- 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 8 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with T1DM within the past 100 days (of enrollment)
- 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 > 0.2 pmol/mL following a mixed meal tolerance test (MMTT)
Who cannot join
- Severe active disease (chronic active hepatitis; cardiac, pulmonary disease, hepatic, renal or immunodeficiency)
- History of any bleeding or clotting factor deficiencies, or stroke
- History of vascular disease or significant vascular abnormalities
- Positive serology of exposure to (hepatitis B virus) HBV, HCV (hepatitis C virus), HIV (human immunodeficiency virus) or toxoplasmosis
- Clinically active infection with EBV (Epstein-Barr virus), CMV (cytomegalovirus), 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 T1DM or immunologic status
Show all 14Show fewer
- Prior treatment with Alpha 1-Antitrypsin (Aralast NP, AAT) or hypersensitivity to alpha 1-antitrypsin 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
- IgA (immunoglobulin A) 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
Children's Hospital of Atlanta (Emory University)
Atlanta, Georgia, 30322
Barbara Davis Center (University of Colorado)
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 Children's Hospital
Iowa City, Iowa, 52242
Children's Mercy Hospital
Kansas City, Missouri, 64108
Yale University
New Haven, Connecticut, 06520
Naomi Berrie Diabetes Center (Columbia University)
New York, New York, 10032
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
RADY Children's Hospital (University of California, San Diego)
San Diego, California, 92093
Pacific Northwest Research Institute-University of Washington
Seattle, Washington, 98122
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01655
About this study
Official title: Effect of Intravenous Alpha-1 Antitrypsin on Preserving Beta-cell Function in New-onset Type 1 Diabetes Mellitus (ITN041AI)
The drug Alpha-1 Antitrypsin (AAT, Aralast NP) is being tested in this study as an anti-inflammatory drug (a medication that decreases inflammation, which is part of the body's normal ability to fight infection and respond to injuries) that affects the cells thought to be involved in the development of type 1 diabetes mellitus (T1DM, T1D).
All subjects enrolled in this study have new-onset T1DM (diagnosis of T1DM within 100 days of Visit 0; T1DM diagnosis fulfilling American Diabetes Association standard T1DM criteria). The focus of Part I of this trial (NCT01183468) is pharmacokinetics (PK), pharmacodynamics (PD) and safety. Upon completion of Part I, including a satisfactory safety review, enrollment in Part II (NCT01183455, Phase II Clinical Trial) will begin.
Read the detailed description
Researchers are interested in conducting this study to assess whether Aralast NP (AAT, Alpha-1 Antitrypsin ) will help slow the progression of T1DM.
Part I of this study has two parts:-1a and -1b:
Part 1a (Complete): An open-label, dose-escalation, PK, PD and safety study. Participants receive 12 intravenous (IV) infusions of Aralast NP. Infusions 1 through 6 are administered at 45 mg/kg/wk and infusions 7 through 12 are administered at 90 mg/kg/wk.
Part Ia consists of two groups:
- Subjects aged 16 - 35 years at enrollment with new-onset T1DM
- Subjects aged 8 -15 years at enrollment with new-onset T1DM.
Part 1b (study terminated prior to subject enrollment): An open-label, dose-escalation PK, PD and safety study in which participants receive 12 infusions of Aralast NP. Infusions 1 through 6 are administered at 90 mg/kg/wk and infusions 7 through 12 are administered at 180 mg/kg/wk. Part Ib consists of two groups:
- Subjects aged 16 - 35 years at enrollment with new-onset T1DM
- Subjects 8 - 15 years at enrollment with new-onset T1DM.
Participants in Part Ib do not roll over into Part II (Refer to NCT01183455).
- Conditions
- Diabetes Mellitus, Type 1
- Being tested
- Aralast NP 45 mg dose, Aralast NP 90 mg dose, Aralast NP 180 mg dose
- Keywords
- Diabetes Mellitus, Type 1 (new-onset), Diabetes Mellitus, Insulin-Dependent (new-onset), new-onset T1DM, new-onset T1D, Diabetes Mellitus, Juvenile-Onset, Diabetes, Autoimmune, Aralast NP, 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 NP 45 mg doseBiological
Also called Alpha 1-Antitrypsin, AAT
- 45 mg/kg/week
Aralast NP 90 mg doseBiological
Also called Alpha 1-Antitrypsin, AAT
- 90 mg/kg/week
Aralast NP 180 mg doseBiological
Also called Alpha-1 Antitrypsin, AAT
- 180 mg/kg/week
| Study group | Role | What they receive |
|---|---|---|
| Part 1a(Aralast NP)-Subjects Aged 16-35 Yrs | Experimental | Subjects aged 16-35 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by intravenous (IV) infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions. Receives: Biological: Aralast NP 45 mg dose; Biological: Aralast NP 90 mg dose |
| Part 1a (Aralast NP)-Subjects 8-15 Yrs | Experimental | Subjects aged 8-15 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions. Receives: Biological: Aralast NP 45 mg dose; Biological: Aralast NP 90 mg dose |
| Part 1b (Aralast NP)--Subjects Aged 18-35 Yrs | Experimental | Aralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions. Receives: Biological: Aralast NP 90 mg dose; Biological: Aralast NP 180 mg dose |
| Part 1b (Aralast NP)-Subjects 8-17 Yrs | Experimental | Aralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions. Receives: Biological: Aralast NP 90 mg dose; Biological: Aralast NP 180 mg dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
C-peptide 2-hour AUC in Response to a Mixed-meal Tolerance Test at Week 52
No results for the primary outcome measure are available since the study was terminated prior to reaching the outcome measure time frame of 52 weeks.
Measured: Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2010 (estimated)
- Study startOct 2010
- Primary completionJul 2013
- Study completionJul 2013
Why it stopped: Due to lack of mechanistic signal and competing industry studies
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 Diabetes Mellitus, Type 1Studies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- Immune Tolerance Network (ITN), Juvenile Diabetes Research Foundation
- Responsible party
- Sponsor
- Investigators
- Gordon Weir, MD, Study chair, Joslin Diabetes Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01183468
- Sponsor's protocol ID
- DAIT ITN041AI Part 1
- Acronym
- RETAIN
- Status last verified
- May 2018
- First submitted
- 16 Aug 2010
- Last updated
- 13 Jun 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Participant level data and additional relevant materials are available to the public in TrialShare, the Immune Tolerance Network (ITN) Clinical Trials Research Portal.
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 13 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Intravenous Alpha-1 Antitrypsin on Preserving Beta-cell Function in New-onset Type 1 Diabetes Mellitus (ITN041AI)
- ClinicalTrials.gov ID
- NCT01183468
- Acronym / sponsor ID
- RETAIN / DAIT ITN041AI Part 1
- Conditions
- Diabetes Mellitus, Type 1
- Keywords
- Diabetes Mellitus, Type 1 (new-onset), Diabetes Mellitus, Insulin-Dependent (new-onset), new-onset T1DM, new-onset T1D, Diabetes Mellitus, Juvenile-Onset, Diabetes, Autoimmune, Aralast NP, Alpha-1 Antitrypsin
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to lack of mechanistic signal and competing industry studies
- Enrolment
- 17 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Collaborators
- Immune Tolerance Network (ITN), Juvenile Diabetes Research Foundation
- Study start
- Oct 2010
- Primary completion
- Jul 2013 (actual)
- Study completion
- Jul 2013 (actual)
- First posted
- 17 Aug 2010 (estimated)
- Results
- Posted 16 Jan 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The drug Alpha-1 Antitrypsin (AAT, Aralast NP) is being tested in this study as an anti-inflammatory drug (a medication that decreases inflammation, which is part of the body's normal ability to fight infection and respond to injuries) that affects the cells thought to be involved in the development of type 1 diabetes mellitus (T1DM, T1D).
All subjects enrolled in this study have new-onset T1DM (diagnosis of T1DM within 100 days of Visit 0; T1DM diagnosis fulfilling American Diabetes Association standard T1DM criteria). The focus of Part I of this trial (NCT01183468) is pharmacokinetics (PK), pharmacodynamics (PD) and safety. Upon completion of Part I, including a satisfactory safety review, enrollment in Part II (NCT01183455, Phase II Clinical Trial) will begin.
Detailed description
Researchers are interested in conducting this study to assess whether Aralast NP (AAT, Alpha-1 Antitrypsin ) will help slow the progression of T1DM.
Part I of this study has two parts:-1a and -1b:
Part 1a (Complete): An open-label, dose-escalation, PK, PD and safety study. Participants receive 12 intravenous (IV) infusions of Aralast NP. Infusions 1 through 6 are administered at 45 mg/kg/wk and infusions 7 through 12 are administered at 90 mg/kg/wk.
Part Ia consists of two groups:
- Subjects aged 16 - 35 years at enrollment with new-onset T1DM
- Subjects aged 8 -15 years at enrollment with new-onset T1DM.
Part 1b (study terminated prior to subject enrollment): An open-label, dose-escalation PK, PD and safety study in which participants receive 12 infusions of Aralast NP. Infusions 1 through 6 are administered at 90 mg/kg/wk and infusions 7 through 12 are administered at 180 mg/kg/wk. Part Ib consists of two groups:
- Subjects aged 16 - 35 years at enrollment with new-onset T1DM
- Subjects 8 - 15 years at enrollment with new-onset T1DM.
Participants in Part Ib do not roll over into Part II (Refer to NCT01183455).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part 1a(Aralast NP)-Subjects Aged 16-35 Yrs | Experimental | Biological: Aralast NP 45 mg dose, Biological: Aralast NP 90 mg doseSubjects aged 16-35 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by intravenous (IV) infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions. |
| Part 1a (Aralast NP)-Subjects 8-15 Yrs | Experimental | Biological: Aralast NP 45 mg dose, Biological: Aralast NP 90 mg doseSubjects aged 8-15 years at enrollment with new-onset type 1 diabetes mellitus (T1DM) received Aralast NP 45 mg/kg by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants underwent a minimum 3-week washout period. After the washout period, each participant proceeded to a high dose of Aralast NP 90 mg/kg by IV infusion for the next 6 weeks, for a total of 12 infusions. |
| Part 1b (Aralast NP)--Subjects Aged 18-35 Yrs | Experimental | Biological: Aralast NP 90 mg dose, Biological: Aralast NP 180 mg doseAralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions. |
| Part 1b (Aralast NP)-Subjects 8-17 Yrs | Experimental | Biological: Aralast NP 90 mg dose, Biological: Aralast NP 180 mg doseAralast NP 90 mg/kg/wk by IV infusion once a week for 6 weeks. Following the Week 6 infusion, participants undergo a minimum 3-wk washout period than then, each participant proceeds to high dose Aralast NP 180 mg/kg/wk by IV infusion for the next 6 weeks, for a total of 12 infusions. |
Who may qualify
- Ages
- 8 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Diagnosed with T1DM within the past 100 days (of enrollment)
- 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 > 0.2 pmol/mL following a mixed meal tolerance test (MMTT)
Key exclusion criteria (14 listed)
- Severe active disease (chronic active hepatitis; cardiac, pulmonary disease, hepatic, renal or immunodeficiency)
- History of any bleeding or clotting factor deficiencies, or stroke
- History of vascular disease or significant vascular abnormalities
- Positive serology of exposure to (hepatitis B virus) HBV, HCV (hepatitis C virus), HIV (human immunodeficiency virus) or toxoplasmosis
- Clinically active infection with EBV (Epstein-Barr virus), CMV (cytomegalovirus), or tuberculosis(TB)
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 |
|---|---|---|
| Aralast NP 45 mg doseAlso: Alpha 1-Antitrypsin, AAT- 45 mg/kg/week | Biological | Part 1a (Aralast NP)-Subjects 8-15 Yrs, Part 1a(Aralast NP)-Subjects Aged 16-35 Yrs |
| Aralast NP 90 mg doseAlso: Alpha 1-Antitrypsin, AAT- 90 mg/kg/week | Biological | Part 1a (Aralast NP)-Subjects 8-15 Yrs, Part 1a(Aralast NP)-Subjects Aged 16-35 Yrs, Part 1b (Aralast NP)--Subjects Aged 18-35 Yrs, Part 1b (Aralast NP)-Subjects 8-17 Yrs |
| Aralast NP 180 mg doseAlso: Alpha-1 Antitrypsin, AAT- 180 mg/kg/week | Biological | Part 1b (Aralast NP)--Subjects Aged 18-35 Yrs, Part 1b (Aralast NP)-Subjects 8-17 Yrs |
Primary outcome
C-peptide 2-hour AUC in Response to a Mixed-meal Tolerance Test at Week 52
Time frame: Week 52
No results for the primary outcome measure are available since the study was terminated prior to reaching the outcome measure time frame of 52 weeks.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
- National Institute of Allergy and Infectious Diseases (NIAID) websiteLink provided by the sponsor.
- Immune Tolerance Network (ITN) websiteLink provided by the sponsor.
- Juvenile Diabetes Research Foundation (JDRF)Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Participant level data and additional relevant materials are available to the public in TrialShare, the Immune Tolerance Network (ITN) Clinical Trials Research Portal.
Related trials for Diabetes Mellitus, Type 1
Not yet recruitingPhase 3
A Study to Evaluate the Effectiveness and Safety of the Drug AZD6234 for Managing Weight in Adults Who Are Obese or Overweight. These Participants May or May Not Also Have Type 2 Diabetes
Obesity, Overweight
RecruitingPhase 1
Pharmacokinetics, Safety, and Immunogenicity Comparison of Bmab1700 and Opdivo® as Adjuvant Monotherapy in Participants With Melanoma
Melanoma
Active, not recruitingPhase 2
A Study of Botensilimab and Balstilimab for the Treatment of Colorectal Cancer
Metastatic Colorectal Cancer
Not yet recruiting
Inter-Arm Differences in Continuous Glucose Monitor Readings in Adults With Diabetes
Continuous Glucose Monitoring, Type 1 Diabetes
RecruitingObservational study
Estimated Risk of Preeclampsia in the First Trimester and Its Association With Subclinical Atherosclerosis and Cardiovascular Risk in Women With Type 1 Diabetes
Type 1 Diabetes, Preeclampsia
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.
Gordon Weir, MD study chair
Joslin Diabetes Center
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Aralast NP 45 mg dose, Aralast NP 90 mg dose and Aralast NP 180 mg dose 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: stopped early, as of the sponsor's last update on 13 Jun 2018. 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. 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?
Yes. Results are posted in the registry (first posted 16 Jan 2015). 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?
C-peptide 2-hour AUC in Response to a Mixed-meal Tolerance Test at Week 52 (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 NCT01183468, 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 13 Jun 2018.