Safety, Tolerability and Potential Efficacy of AVT001 in Patients With Type 1 Diabetes
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|ClinicalTrials.gov Identifier: NCT03895996|
Recruitment Status : Active, not recruiting
First Posted : March 29, 2019
Last Update Posted : December 12, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Type 1 Diabetes Mellitus||Drug: AVT001 Other: Placebo||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Actual Study Start Date :||June 20, 2019|
|Actual Primary Completion Date :||May 17, 2022|
|Estimated Study Completion Date :||December 2023|
Experimental: AVT001 (Treatment)
Infusion of AVT001 (treatment)
autologous dendritic cell therapy
Placebo Comparator: Matched placebo
Infusion of AVT001-matched placebo
- The incidence of treatment-emergent adverse events [ Time Frame: 5 months post first dose ]Safety/tolerability outcomes
- Changes from baseline of clinical parameters on CBC/differential, chemistry panel [ Time Frame: 5 months post first dose ]Safety/tolerability outcomes - the clinical parameters tested include creatinine, AST, ALT, and total bilirubin
- The incidence and severity of local i.v.-site reactions, [ Time Frame: 5 months post first dose ]Safety/tolerability outcomes
- Assessment of the HLA-E-restricted CD8+ T cell regulatory activity ("potency assay") [ Time Frame: 5 months post first dose ]Efficacy outcomes - "potency assay" measures the activity of CD8+ T regulatory cells
- 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 ]Efficacy outcomes
- Changes from baseline in HbA1c [ Time Frame: 5 months post first dose ]Efficacy outcomes
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|Ages Eligible for Study:||16 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
Key Inclusion Criteria:
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.
Key Exclusion Criteria:
- 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
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03895996
|United States, Massachusetts|
|Joslin Diabetes Center, Harvard Medical School|
|Boston, Massachusetts, United States, 02215|
|Principal Investigator:||Jason Gaglia, MD||Joslin Diabetes Center, Harvard Medical School|
|Responsible Party:||Avotres Inc.|
|Other Study ID Numbers:||
|First Posted:||March 29, 2019 Key Record Dates|
|Last Update Posted:||December 12, 2022|
|Last Verified:||December 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Endocrine System Diseases
Immune System Diseases