Brittle Type 1 Diabetes MellitusPhase 1/2NCT05480657
Safety, Tolerability, and Efficacy of Immunomodulation With AT-1501 in Combination With Transplanted Islet Cells in Adults With Brittle T1D
A Phase 1/2 study testing AT-1501 IV Infusion and Isolated cadaveric islet cells in people with Brittle Type 1 Diabetes Mellitus, run by Eledon Pharmaceuticals at 1 site in United States.
Trial summary
This study will evaluate the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with T1D undergoing an islet cell transplant. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Brittle Type 1 Diabetes Mellitus
- What is being tested?
- AT-1501 IV Infusion and Isolated cadaveric islet cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Sep 2022to Aug 2028
- Phase
- Phase 1/2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and women 18-65 years of age
- A diagnosis of T1D ≥5 years with onset of disease at <40 years of age
- Involvement in appropriate diabetes management in accordance with the standard of care, as directed by an endocrinologist or diabetologist with at least 3 clinical evaluations within the 12 months prior to Screening; using an insulin pump or multiple daily injection (MDI) insulin therapy; and, unable to achieve acceptable metabolic control because of the occurrence of unexplained SHEs
- At least 3 unexplained SHEs not secondary to a missed meal or dosing error, in the 12 months prior to Screening
- Glycosylated hemoglobin (HbA1c) level greater than 7% (53 mmol/mol) and less than 9.5% (80 mmol/mol) inclusive
- Absence of stimulated C peptide (< 0.3 ng/mL) in response to a mixed meal tolerance test (MMTT) measured at 60 and 90 minutes after the start of consumption
- Impaired awareness of hypoglycemia (IAH) as defined by a Clarke Score [Clarke 1995] of 4 or more at the time of Screening, during the Screening period, and within the last 6 months prior to the transplant
Who cannot join
- Any previous solid organ or islet allotransplant
- Body mass index (BMI) >30 kg/m2
- Insulin requirement >1.0 unit/kg/day or <15 units/day
Trial locations and contacts
1 site in 1 country.
About this study
Official title: AT-1501-I206: An Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of Transplanted Islet Cells and AT-1501 Immunomodulation in Adults With Brittle Type 1 Diabetes
This study will evaluate the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with T1D undergoing an islet cell transplant.
Read the detailed description
This study will evaluate the safety, tolerability and efficacy of transplantation of experimental islet cells as a potential treatment for brittle type 1 diabetes mellitus. It will also assess the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with brittle T1D undergoing an islet cell transplant. This is a single arm open-label study and up to 6 participants will be recruited at a single center in the United States.
The objectives include:
- To assess the safety and tolerability of transplanted islet cells and immunomodulation with AT-1501, in combination (AT+) with rabbit anti-thymoglobulin (ATG), etanercept and mycophenolate mofetil (MMF/EC-MPS) in adults with T1D undergoing islet cell transplant.
- To assess the efficacy of transplanted islet cells and immunomodulation with AT-1501 in adults with brittle T1D undergoing islet cell transplant.
The duration of treatment may vary from participant to participant and could be up to 2 years. Participants may receive up to 2 islet cell transplants.
- Conditions
- Brittle Type 1 Diabetes Mellitus
- Being tested
- AT-1501 IV Infusion, Isolated cadaveric islet cells
- Keywords
- AT-1501, Type 1 Diabetes, T1D, Islet Cell Transplant, humanized blocking antibody to CD40L, CD40L inhibitor, monoclonal antibody, Glucose Metabolism Diseases, Diabetes Mellitus, Type 1, Endocrine System Diseases, Graft, Metabolic Diseases, Immune System Diseases, Autoimmune Diseases, Hypoglycemia, Hyperglycemia, Brittle T1D, Brittle Type 1 Diabetes
- Medical subject headings
- Diabetes Mellitus, Type 1, Diabetes Mellitus, Endocrine System Diseases, Metabolic Diseases, Immune System Diseases, Autoimmune Diseases, Hypoglycemia, Hyperglycemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AT-1501 IV InfusionBiological
Investigational study drug
Isolated cadaveric islet cellsBiological
Infusion of human cadaveric islet cells into the portal vein
| Study group | Role | What they receive |
|---|---|---|
| Single Arm AT-1501 | Experimental | Single-arm, open-label trial Receives: Biological: AT-1501 IV Infusion; Biological: Isolated cadaveric islet cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety - Adverse Events (AE) and Adverse Events of Special Interest (AEoSI)
Incidence of adverse events
Measured: Accessed from date of transplant through Day 364 post final transplant for approximately 2 years
Efficacy - Insulin independence
The proportion of participants that become insulin independent at Days 75 and 365 post-first, and final transplant
Measured: Date of transplant through Day 364 post- final transplant
Secondary outcomes (4)
Efficacy - HbA1c
Proportion of participants with HbA1c <7.0% (53 mmol/mol) and free of serious hypoglycemic events (SHEs) from Day 28 to Day 364 post first and final transplant.
Measured: Date of transplant through Day 364 post-final transplant
Efficacy - Graft failure
Date of transplant through Day 364 post final transplant
Measured: Proportion of participants with graft failure at Day 364 post final transplant
Efficacy - Durability of insulin independence
The proportion of participants that become insulin independent at Day 364 post-transplant
Measured: Date of transplant through Day 364 post final transplant
Efficacy - Durability of insulin independence - long term
The proportion of participants that become insulin independent at year 2 and year 3
Measured: 2 and 3 years after discontinuation of AT- 1501
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2022
- Study start30 Sep 2022
- Primary completionAug 2024 (estimated)
- Study completionAug 2028 (estimated)
Why it stopped: Study withdrawn
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Brittle 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
- Not applicable
- Intervention model
- Single group
- Model description
- Single-arm, open-label trial
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eledon Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jeff Bornstein, MD, Study director, Eledon Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05480657
- Sponsor's protocol ID
- AT-1501-I206
- Status last verified
- Jan 2023
- First submitted
- 27 Jul 2022
- Last updated
- 18 Jan 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- AT-1501-I206: An Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of Transplanted Islet Cells and AT-1501 Immunomodulation in Adults With Brittle Type 1 Diabetes
- ClinicalTrials.gov ID
- NCT05480657
- Acronym / sponsor ID
- AT-1501-I206
- Conditions
- Brittle Type 1 Diabetes Mellitus
- Keywords
- AT-1501, Type 1 Diabetes, T1D, Islet Cell Transplant, humanized blocking antibody to CD40L, CD40L inhibitor, monoclonal antibody, Glucose Metabolism Diseases
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Eledon PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2022 (actual)
- Primary completion
- Aug 2024 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 29 Jul 2022 (actual)
- 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.
This study will evaluate the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with T1D undergoing an islet cell transplant.
Detailed description
This study will evaluate the safety, tolerability and efficacy of transplantation of experimental islet cells as a potential treatment for brittle type 1 diabetes mellitus. It will also assess the safety, tolerability and efficacy of AT-1501 in an immunomodulation regimen in adult patients with brittle T1D undergoing an islet cell transplant. This is a single arm open-label study and up to 6 participants will be recruited at a single center in the United States.
The objectives include:
- To assess the safety and tolerability of transplanted islet cells and immunomodulation with AT-1501, in combination (AT+) with rabbit anti-thymoglobulin (ATG), etanercept and mycophenolate mofetil (MMF/EC-MPS) in adults with T1D undergoing islet cell transplant.
- To assess the efficacy of transplanted islet cells and immunomodulation with AT-1501 in adults with brittle T1D undergoing islet cell transplant.
The duration of treatment may vary from participant to participant and could be up to 2 years. Participants may receive up to 2 islet cell transplants.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single-arm, open-label trial
| Arm | Type | Receives |
|---|---|---|
| Single Arm AT-1501 | Experimental | Biological: AT-1501 IV Infusion, Biological: Isolated cadaveric islet cellsSingle-arm, open-label trial |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Men and women 18-65 years of age
- A diagnosis of T1D ≥5 years with onset of disease at <40 years of age
- Involvement in appropriate diabetes management in accordance with the standard of care, as directed by an endocrinologist or diabetologist with at least 3 clinical evaluations within the 12 months prior to Screening; using an insulin pump or multiple daily injection (MDI) insulin therapy; and, unable to achieve acceptable metabolic control because of the occurrence of unexplained SHEs
- At least 3 unexplained SHEs not secondary to a missed meal or dosing error, in the 12 months prior to Screening
- Glycosylated hemoglobin (HbA1c) level greater than 7% (53 mmol/mol) and less than 9.5% (80 mmol/mol) inclusive
Key exclusion criteria (3 listed)
- Any previous solid organ or islet allotransplant
- Body mass index (BMI) >30 kg/m2
- Insulin requirement >1.0 unit/kg/day or <15 units/day
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 |
|---|---|---|
| AT-1501 IV InfusionInvestigational study drug | Biological | Single Arm AT-1501 |
| Isolated cadaveric islet cellsInfusion of human cadaveric islet cells into the portal vein | Biological | Single Arm AT-1501 |
Primary outcome
Safety - Adverse Events (AE) and Adverse Events of Special Interest (AEoSI)
Time frame: Accessed from date of transplant through Day 364 post final transplant for approximately 2 years
Incidence of adverse events
Efficacy - Insulin independence
Time frame: Date of transplant through Day 364 post- final transplant
The proportion of participants that become insulin independent at Days 75 and 365 post-first, and final transplant
4 secondary outcomes
Efficacy - HbA1c
Time frame: Date of transplant through Day 364 post-final transplant
Efficacy - Graft failure
Time frame: Proportion of participants with graft failure at Day 364 post final transplant
Efficacy - Durability of insulin independence
Time frame: Date of transplant through Day 364 post final transplant
Efficacy - Durability of insulin independence - long term
Time frame: 2 and 3 years after discontinuation of AT- 1501
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jan 2023.
- 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.
Jeff Bornstein, MD study director
Eledon Pharmaceuticals
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 AT-1501 IV Infusion and Isolated cadaveric islet cells in people with Brittle Type 1 Diabetes Mellitus, run by Eledon Pharmaceuticals 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 3 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 18 Jan 2023. 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 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?
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?
Safety - Adverse Events (AE) and Adverse Events of Special Interest (AEoSI) (time frame: Accessed from date of transplant through Day 364 post final transplant for approximately 2 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Jan 2023.