Type 1 Diabetes MellitusPhase 1/2NCT03895996

Safety, Tolerability and Potential Efficacy of AVT001 in Patients With Type 1 Diabetes

A Phase 1/2 study testing AVT001 in people with Type 1 Diabetes Mellitus, run by Avotres Inc. at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This is a double-blind, randomized , placebo-controlled study to evaluate the safety and tolerability of AVT001, and to assess AVT001 as a potential treatment for type 1 diabetes (T1D). The trial will involve approximately 24 new-onset T1D subjects. Read more about this study

Who is it for?: Ages 16 years and older
Conditions the study is for: Type 1 Diabetes Mellitus
What is being tested?: AVT001
Type of treatment: Injection or infusion
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 25 participants
Where: 1 siteUnited States
Study dates: 20 Jun 2019to 19 Dec 2023
Phase: Phase 1/2
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 16 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Diagnosis of type 1 diabetes, within 12 months of first dosing, confirmed by positive lab result for one or more of the following types of autoantibodies:
Glutamic acid decarboxylase (GAD65)
Insulinoma associated protein 2 (IA-2, also known as ICA-512)
Zinc transporter 8 (ZnT8).
Age 16 or older and able to provide informed consent/assent.
If a participant is female with reproductive potential, willing to avoid pregnancy through the duration of the trial.
Signed and dated written informed consent/assent.

Who cannot join

Poorly controlled diabetes despite insulin therapy, who in the opinion of the investigator would not be a good candidate for participation in a clinical trial
Screening hemoglobin <10.0 g/dL; leukocytes <3,000/uL; neutrophils <1,500/uL; lymphocytes <800/uL; platelets <100,000/uL
Screening Urine Albumin Excretion > 300mg/gmCr
Screening eGFR < 60 mL/min/1.73m2
Screening ALT or AST > 1.5x upper limit of normal (ULN)
Screening bilirubin > 2.0 mg / dL, or > 3.0 mg / dL for participants with Gilbert's Syndrome

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Current use of immunosuppressive or immunomodulatory therapies, including pharmacologic doses of systemic steroids. However, topical steroidal creams and inhaled steroids without large systemic absorption are allowed.
Coincident medical condition likely to require immunosuppressive or immunomodulatory therapies.
Coincident medical condition likely to limit short term (5 year) life expectancy (malignancy, symptomatic coronary artery disease, recent stroke)
Prior radiation therapy, immunotherapy (within 1 year of screening), or chemotherapy
Serologic evidence of current HIV-1 or HIV-2 infection
Serologic evidence of hepatitis C infection
Serologic evidence of acute or chronic active hepatitis B as measured by Core Ab positive and / or Surface Antibody antigen positive
Subjects with other autoimmune conditions (except compensated or treated autoimmune thyroid, celiac, alopecia, or vitiligo diseases)
Women who are pregnant (pregnancy testing during screening), breastfeeding, or planning pregnancy during the study period
Inadequate venous access to support leukapheresis
Any condition that in the opinion of the investigator(s) would preclude the subject from participating in a clinical trial.
Abnormal screening ECG that in the opinion of the investigator or sponsor would pose a safety risk.

Trial locations and contacts

1 site in 1 country.

United States1 site

Joslin Diabetes Center, Harvard Medical School
Boston, Massachusetts, 02215

Other trials in United States

About this study

Official title: A Phase 1 / 2 Double-Blind, Randomized, Placebo Controlled Study of Safety, Tolerability and Potential Efficacy of AVOTRES Cell-Based Therapy (AVT001) in Patients With Type 1 Diabetes

Conditions: Type 1 Diabetes Mellitus
Being tested: AVT001, Placebo
Medical subject headings: Diabetes Mellitus, Type 1

What participants receive

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

AVT001Drug
autologous dendritic cell therapy
PlaceboOther
matched placebo

Study groups in NCT03895996
Study group	Role	What they receive
AVT001 (Treatment)	Experimental	Infusion of AVT001 (treatment) Receives: Drug: AVT001
Matched placebo	Placebo comparator	Infusion of AVT001-matched placebo Receives: Other: Placebo

What is being measured

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

Number of Participants With Treatment-emergent Adverse Events (TEAE)
Treatment-emergent AEs (TEAEs) are defined as any AE that started on or after the first dose of study medication through 30 days following the last dose.
Measured: At the Primary Analysis (when all the patients have completed their Day 150 visit)
Number of Participants and Severity of Local i.v.-Site Reactions,
Number of Participants and severity of local intravenous site reactions after receiving the three doses are reported.
Measured: 5 months post first dose
Changes From Baseline of Creatinine
Safety/tolerability outcomes - creatinine
Measured: 5 months post first dose
Changes From Baseline of Aspartate Aminotransferase
Safety/tolerability outcomes - Aspartate Aminotransferase
Measured: 5 months post first dose
Changes From Baseline of Alanine Aminotransferase
Safety/tolerability outcomes - Alanine Aminotransferase
Measured: 5 months post first dose
Changes From Baseline of Total Bilirubin
Safety/tolerability outcomes - Total Bilirubin
Measured: 5 months post first dose

Secondary outcomes (3)

Assessment of the HLA-E-restricted CD8+ T Cell Regulatory Activity ("Potency Assay")
CD8+ T cell Inhibition Assay is used to determine whether AVT001 corrects the defect of the dysfunctional Q/E CD8+ Treg pathway in T1D patients. More specifically, this assay detects the specific recognition between the TCR on patients' Q/E CD8+Treg cells and the "common target structure", the HLA-E/Hsp60sp complex, expressed on the surface of the artificially established target cells.
The % inhibition measures the function of down-regulation by Q/E CD8+ Tregs via comparing the % of inhibition of TH1 cells versus TB1 cells.
By assessing the % inhibition of the TH1 cells of the patient's CD8+ T cells, the CD8+ T cell Inhibition Assay detects the specific recognition of the common target structure (HLA-E/Hsp60sp) on TH1 cells by the TCR on the patient's T cells to be tested.
A negative value means the measured Q/E CD8+ Tregs completely lose its inhibition function, and the TH1 cells cultured with it happened to grow faster than the corresponding TB1 cells as its control.
Measured: 5 months post first dose
Changes From Baseline in the Area Under the Curve (AUC) of the Stimulated C-peptide Levels Over a 4-hour Mixed Meal Tolerance Test (MMTT)
The area under the stimulated C-peptide curve (AUC) over the first 4-hour period of a mixed meal glucose tolerance test is calculated using the trapezoidal rule that is a weighted sum of the C-peptide values over the 240 minutes. The AUC is normalized by dividing it with 240 mins, therefore, its unit is nmol/L. Missing C-peptide levels at any given timepoint is not imputed. In the calculation of the AUC when C-peptide levels are missing, a line is drawn from the last timepoint with a non-missing C-peptide to the next timepoint with non-missing C-peptide.
Measured: 5 months post first dose
Changes From Baseline in HbA1c
HbA1c is a blood test that is used to monitor blood glucose control in people with diabetes.
HbA1c is short for glycated haemoglobin. The test is also sometimes called haemoglobin A1c.
Measured: 5 months post first dose

Timeline

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

20 Jun 201919 Dec 2023

First posted29 Mar 2019
Study start20 Jun 2019
Primary completion17 May 2022
Study completion19 Dec 2023

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 Type 1 Diabetes MellitusStudies 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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 25 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03895996
Sponsor's protocol ID: AVT001-T1D-01
Status last verified: Feb 2025
First submitted: 15 Mar 2019
Last updated: 5 Mar 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Gaglia JL, Daley HL, Bryant NK, Ritz J, Dong T, Skyler JS, Jiang H. Novel Autologous Dendritic Cell Therapy AVT001 for Type 1 Diabetes. NEJM Evid. 2024 Jul;3(7):EVIDoa2300238. doi: 10.1056/EVIDoa2300238. Epub 2024 Jun 25.
PubMed 38916421

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Type 1 Diabetes Mellitus trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1 / 2 Double-Blind, Randomized, Placebo Controlled Study of Safety, Tolerability and Potential Efficacy of AVOTRES Cell-Based Therapy (AVT001) in Patients With Type 1 Diabetes
ClinicalTrials.gov ID: NCT03895996
Acronym / sponsor ID: AVT001-T1D-01
Conditions: Type 1 Diabetes Mellitus
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 25 participants (actual)
Lead sponsor: Avotres Inc.Industry (pharma, biotech, devices)
Study start: 20 Jun 2019 (actual)
Primary completion: 17 May 2022 (actual)
Study completion: 19 Dec 2023 (actual)
First posted: 29 Mar 2019 (actual)
Results: Posted 5 Mar 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
AVT001 (Treatment)	Experimental	Drug: AVT001Infusion of AVT001 (treatment)
Matched placebo	Placebo comparator	Other: PlaceboInfusion of AVT001-matched placebo

Who may qualify

Ages: 16 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (7 listed)

Diagnosis of type 1 diabetes, within 12 months of first dosing, confirmed by positive lab result for one or more of the following types of autoantibodies:
Age 16 or older and able to provide informed consent/assent.
If a participant is female with reproductive potential, willing to avoid pregnancy through the duration of the trial.
Signed and dated written informed consent/assent.

Key exclusion criteria (18 listed)

Poorly controlled diabetes despite insulin therapy, who in the opinion of the investigator would not be a good candidate for participation in a clinical trial
Screening hemoglobin <10.0 g/dL; leukocytes <3,000/uL; neutrophils <1,500/uL; lymphocytes <800/uL; platelets <100,000/uL
Screening Urine Albumin Excretion > 300mg/gmCr
Screening eGFR < 60 mL/min/1.73m2
Screening ALT or AST > 1.5x upper limit of normal (ULN)

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
AVT001autologous dendritic cell therapy	Drug	AVT001 (Treatment)
Placebomatched placebo	Other	Matched placebo

Primary outcome

Number of Participants With Treatment-emergent Adverse Events (TEAE)
Time frame: At the Primary Analysis (when all the patients have completed their Day 150 visit)
Treatment-emergent AEs (TEAEs) are defined as any AE that started on or after the first dose of study medication through 30 days following the last dose.
Number of Participants and Severity of Local i.v.-Site Reactions,
Time frame: 5 months post first dose
Number of Participants and severity of local intravenous site reactions after receiving the three doses are reported.
Changes From Baseline of Creatinine
Time frame: 5 months post first dose
Safety/tolerability outcomes - creatinine
Changes From Baseline of Aspartate Aminotransferase
Time frame: 5 months post first dose
Safety/tolerability outcomes - Aspartate Aminotransferase
Changes From Baseline of Alanine Aminotransferase
Time frame: 5 months post first dose
Safety/tolerability outcomes - Alanine Aminotransferase
Changes From Baseline of Total Bilirubin
Time frame: 5 months post first dose
Safety/tolerability outcomes - Total Bilirubin

3 secondary outcomes

Assessment of the HLA-E-restricted CD8+ T Cell Regulatory Activity ("Potency Assay")
Time frame: 5 months post first dose
Changes From Baseline in the Area Under the Curve (AUC) of the Stimulated C-peptide Levels Over a 4-hour Mixed Meal Tolerance Test (MMTT)
Time frame: 5 months post first dose
Changes From Baseline in HbA1c
Time frame: 5 months post first dose

Results and safety

Results posted to the registry on 5 Mar 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
AVT001 (Treatment)	16	16
Matched Placebo	9	8

Primary outcome: Number of Participants With Treatment-emergent Adverse Events (TEAE)

Time frame: At the Primary Analysis (when all the patients have completed their Day 150 visit). Number. Unit: participants

Measure	AVT001 (Treatment) n = 16	Matched Placebo n = 9
Participants with Any TEAE	9	6
Neutrophil count decreased	4	1
Hyponatraemia	3	1
Cough	3	0
White blood cell count decreased	2	1
Ear pain	2	0
Lymphocyte count decreased	2	0
Dizziness	1	2

Population analysed: The safety (SAF) population is defined as all subjects who receive at least one dose of study medication.

Primary outcome: Number of Participants and Severity of Local i.v.-Site Reactions,

Time frame: 5 months post first dose. Number. Unit: Number of patients

Measure	AVT001 (Treatment) n = 16	Matched Placebo n = 9
Value	0	0

Population analysed: The safety (SAF) population is defined as all subjects who receive at least one dose of study medication.

Primary outcome: Changes From Baseline of Creatinine

Time frame: 5 months post first dose. Mean (Standard Deviation). Unit: umol/L

Measure	AVT001 (Treatment) n = 16	Matched Placebo n = 9
Value	1.9338 (7.51638)	0.4911 (5.48705)

Population analysed: The safety (SAF) population is defined as all subjects who receive at least one dose of study medication.

Primary outcome: Changes From Baseline of Aspartate Aminotransferase

Time frame: 5 months post first dose. Mean (Standard Deviation). Unit: U/L

Measure	AVT001 (Treatment) n = 16	Matched Placebo n = 9
Value	-1.9 (7.76)	2.2 (6.10)

Population analysed: The safety (SAF) population is defined as all subjects who receive at least one dose of study medication.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: All anticipated and unanticipated adverse events occured during the study (2 years), including both Treatment Emergent Adverse Events (TEAEs) and Post-Treatment Adverse Events (PTAEs), that exceed a 5% frequency threshold within any arm of the clinical study.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
AVT001 (Treatment)	0 of 16 (0%)	16 of 16 (100%)	0 of 16
Matched Placebo	0 of 9 (0%)	9 of 9 (100%)	0 of 9

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 38916421Gaglia JL, Daley HL, Bryant NK, Ritz J, Dong T, Skyler JS, Jiang H. Novel Autologous Dendritic Cell Therapy AVT001 for Type 1 Diabetes. NEJM Evid. 2024 Jul;3(7):EVIDoa2300238. doi: 10.1056/EVIDoa2300238. Epub 2024 Jun 25.
Avotres Inc. in collaboration with Joslin Diabetes Center, a Harvard Medical School affiliate, have reported one year data of a phase I/II clinical trial of AVT001 in type 1 diabetes.Link provided by the sponsor.
Individual participant dataSharing planned: No

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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.

Jason Gaglia, MD principal investigator

Joslin Diabetes Center, Harvard Medical School

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing AVT001 in people with Type 1 Diabetes Mellitus, run by Avotres Inc. 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 7 inclusion and 18 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 5 Mar 2025. 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. 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 combined 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.

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?

Yes. Results are posted in the registry (first posted 5 Mar 2025). 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?

Number of Participants With Treatment-emergent Adverse Events (TEAE) (time frame: At the Primary Analysis (when all the patients have completed their Day 150 visit)). The study lists 6 primary outcomes in all. 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 NCT03895996, 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 5 Mar 2025.