# Appendix A: Full Trial Register (ClinicalTrials.gov harvest)

*59 core relevant trials across 8 programs. Full machine-readable version: trials_master.csv.*


## ATX-MS-1467  (2 trials)

| NCT | Phase | Status | N | Design | Start | Primary outcome(s) |
|---|---|---|---|---|---|---|
| NCT01097668 | PHASE1 | COMPLETED | 43 | Non Randomized, None mask | 2010-03 | Safety and Tolerability |
| NCT01973491 | PHASE2 | COMPLETED | 37 | — | 2014-02-28 | Change From Baseline in the Average Number of Time Constant 1 (T1) Contrast-enhanced Lesions (CELs) Over On-treatment Scans |

## GAD-alum/Diamyd  (18 trials)

| NCT | Phase | Status | N | Design | Start | Primary outcome(s) |
|---|---|---|---|---|---|---|
| NCT00456027 | PHASE2 | COMPLETED | 160 | — | 2004-12 | The development over time of safety variables, i.e. injection site discomfort, vital signs, laboratory values and AEs/SAEs as well as development of d |
| NCT00435981 | PHASE2 | COMPLETED | 70 | Randomized, Double mask | 2005-01 | To evaluate the efficacy of Diamyd® 20ug versus placebo with respect to preserving residual insulin secretion as measured by C-peptide levels. The eff |
| NCT00723411 | PHASE3 | TERMINATED | 334 | Randomized, Quadruple mask | 2008-07 | Meal stimulated C-peptide (area under the curve) |
| NCT00751842 | PHASE3 | TERMINATED | 331 | Randomized, Quadruple mask | 2008-09 | Meal Stimulated C-peptide (area under the curve) |
| NCT00529399 | PHASE2 | COMPLETED | 145 | Randomized, Triple mask | 2009-02 | The Primary Outcome is the Area Under the Stimulated C-peptide Curve (AUC) at the One Year Visit |
| NCT00837759 | PHASE2 | TERMINATED | 7 | — | 2009-02 | Change in C-peptide |
| NCT01122446 | PHASE2 | COMPLETED | 50 | Randomized, Triple mask | 2009-04 | Adverse Events |
| NCT01129232 | PHASE2 | TERMINATED | 6 | Randomized, Quadruple mask | 2011-01 | Intensity of insulitis in proportion to living, insulin-staining beta cells in pancreatic biopsies // Prevalence of virus infected islets in pancreati |
| NCT01785108 | PHASE2 | COMPLETED | 60 | Randomized, Quadruple mask | 2013-02 | Change in C-peptide (90 minute value and AUC mean 0-120 min) during a Mixed Meal Tolerance Test from baseline to month 6, 15 and 30 |
| NCT02352974 | PHASE1 | COMPLETED | 12 | — | 2015-01 | Number of Subjects With Injection Site Reactions Month 1 // Number of Subjects With Injection Site Reactions Month 2 // Number of Subjects With Inject |
| NCT02002130 | PHASE1 | COMPLETED | 101 | Randomized, Quadruple mask | 2015-01 | Compare the effect of oral GABA or oral GABA/GAD combination administration on pancreatic beta cell function by quantitative C-peptide secretion |
| NCT02387164 | PHASE2 | TERMINATED | 26 | Randomized, Quadruple mask | 2015-03-09 | Type 1 Diabetes Month 24 // Type 1 Diabetes Status Overall |
| NCT02464033 | PHASE2 | COMPLETED | 20 | — | 2015-05 | Number of Patients With Reactions of the Injection Site as an Assessment of the Tolerability // Number of Patients With Reactions of the Injection Sit |
| NCT03345004 | PHASE2 | COMPLETED | 109 | Randomized, Quadruple mask | 2017-12-20 | Change in Stimulated C-peptide During a MMTT |
| NCT04262479 | PHASE2 | COMPLETED | 14 | — | 2020-03-02 | Injection Site Skin Reactions // Occurrence of Adverse Events (AEs) During 5 Months From Baseline. // Occurrence of Adverse Events (AEs) During the St |
| NCT05351879 | PHASE1/PHASE2 | COMPLETED | 6 | — | 2022-05-09 | Number of Clinically Significant Abnormal Results from Physical examinations, including neurological and Vital Signs assessments // Injection site rea |
| NCT05018585 | PHASE3 | ACTIVE_NOT_RECRUITING | 321 | Randomized, Quadruple mask | 2022-05-19 | Beta cell function // Glycemic control |
| NCT05683990 | PHASE2 | ACTIVE_NOT_RECRUITING | 5 | Randomized, None mask | 2024-07-09 | Occurrence of AEs (including Injection site reactions) and SAEs // Number of Clinically Significant Abnormal Results from Physical examinations, inclu |

## KAN-101  (3 trials)

| NCT | Phase | Status | N | Design | Start | Primary outcome(s) |
|---|---|---|---|---|---|---|
| NCT04248855 | PHASE1 | COMPLETED | 41 | Randomized, Double mask | 2020-01-21 | Incidence and Severity of Treatment-emergent Adverse Events (TEAEs) |
| NCT05574010 | PHASE1/PHASE2 | TERMINATED | 128 | Randomized, Triple mask | 2022-11-15 | Incidence and Severity of TEAEs as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) in Part A // Change in Pre- and Post-Gluten Chal |
| NCT06001177 | PHASE2 | COMPLETED | 55 | Randomized, Triple mask | 2023-12-13 | Changes From Baseline in Villous Height to Crypt Depth (Vh:Cd) as Assessed by Esophagogastroduodenoscopy With Biopsy After 2-week Gluten Challenge (GC |

## Nexvax2  (4 trials)

| NCT | Phase | Status | N | Design | Start | Primary outcome(s) |
|---|---|---|---|---|---|---|
| NCT00879749 | PHASE1 | COMPLETED | 34 | Randomized, Quadruple mask | 2009-04 | — |
| NCT02528799 | PHASE1 | COMPLETED | 38 | Randomized, Quadruple mask | 2015-08 | Incidence of toxicity and safety of Nexvax2 according to the "Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0" |
| NCT03543540 | PHASE1 | COMPLETED | 14 | Randomized, Quadruple mask | 2018-05-01 | Safety of Nexvax 2 administered subcutaneously (SQ) // Evaluate bioavailability of the constituents of Nexvax2 after SQ versus intradermal (ID) admini |
| NCT03644069 | PHASE2 | UNKNOWN | 146 | Randomized, Quadruple mask | 2018-08-06 | Efficacy of Nexvax2 compared to placebo in reducing Celiac Disease (CeD) associated GI symptoms. |

## Proinsulin peptide  (3 trials)

| NCT | Phase | Status | N | Design | Start | Primary outcome(s) |
|---|---|---|---|---|---|---|
| NCT01536431 | PHASE1/PHASE2 | COMPLETED | 27 | Randomized, Quadruple mask | 2012-01 | Safety |
| NCT02620332 | PHASE1 | COMPLETED | 27 | Randomized, Quadruple mask | 2015-10-20 | Assessment of MultiPepT1De safety profile |
| NCT02837094 | PHASE1 | COMPLETED | 6 | — | 2016-09-29 | To examine the risk of C19A3 GNP administration in terms of general safety and induction of hypersensitivity. |

## TAK-101/TIMP-GLIA  (3 trials)

| NCT | Phase | Status | N | Design | Start | Primary outcome(s) |
|---|---|---|---|---|---|---|
| NCT03486990 | PHASE1 | COMPLETED | 23 | Non Randomized, None mask | 2018-01-23 | Number of Participants Reporting One or More Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) // Number of Participants Wit |
| NCT03738475 | PHASE2 | COMPLETED | 34 | Randomized, Quadruple mask | 2018-11-11 | Change From Baseline in Interferon-Gamma Spot Forming Units (IFN-gamma SFUs) in a Gliadin-specific Enzyme-linked Immunospot (ELISpot) at Day 20 |
| NCT04530123 | PHASE2 | COMPLETED | 102 | Randomized, Triple mask | 2022-06-23 | Change From Baseline in Interferon-gamma Spot Forming Units (IFN-γ SFUs) in Human Leukocyte Antigens Density Quotient (HLA-DQ2.5-positive) Participant |

## TPM502/Topas  (1 trials)

| NCT | Phase | Status | N | Design | Start | Primary outcome(s) |
|---|---|---|---|---|---|---|
| NCT05660109 | PHASE2 | COMPLETED | 28 | Randomized, Quadruple mask | 2022-12-12 | Incidence, severity, causality, and outcomes of treatment-emergent adverse events |

## Tzield/teplizumab  (25 trials)

| NCT | Phase | Status | N | Design | Start | Primary outcome(s) |
|---|---|---|---|---|---|---|
| NCT00806572 | PHASE2 | TERMINATED | 10 | Randomized, None mask | 2002-05 | 4-hour C-peptide AUC |
| NCT00129259 | PHASE2 | COMPLETED | 83 | Randomized, Single mask | 2005-09 | Change in Mean C-peptide Area Under the Curve (AUC) Response to a Mixed Meal Tolerance Test (MMTT) |
| NCT00239720 | PHASE2 | TERMINATED | 4 | Randomized, Double mask | 2006-03-16 | Proportion of Participants Who Received at Least Two Cycles of Treatment and Who Showed Predefined Levels of Improvement in Primary Efficacy Parameter |
| NCT00378508 | PHASE2 | COMPLETED | 63 | Randomized, Triple mask | 2006-09 | C-peptide Area Under the Curve (AUC) Response to a Mixed Meal Tolerance Test (MMTT) at 12 Months // C-peptide Area Under the Curve (AUC) Response to a |
| NCT00385697 | PHASE2/PHASE3 | COMPLETED | 554 | Randomized, Triple mask | 2006-10 | Number of Subjects in Segment 2 With Both a Total Daily Insulin Dose of Less Than 0.5 U/kg/Day and Hemoglobin A1c (HbA1c) Level of Less Than 6.5%. //  |
| NCT00920582 | PHASE3 | TERMINATED | 254 | Randomized, Triple mask | 2009-09 | Proportion of Subjects With Both a Total Daily Insulin Dose of Less Than 0.5 U/kg/Day and Hemoglobin A1c (HbA1c) Level of Less Than 6.5%. // Mean Chan |
| NCT00954915 | PHASE1/PHASE2 | TERMINATED | 1 | — | 2009-12 | Adverse Events (AE) |
| NCT01189422 | PHASE1 | TERMINATED | 1 | Randomized, Quadruple mask | 2010-08 | Dose regimen |
| NCT01030861 | PHASE2 | COMPLETED | 76 | Randomized, Quadruple mask | 2010-08 | Rate of New Diabetes Per Year |
| NCT03751007 | PHASE1/PHASE2 | COMPLETED | 45 | Randomized, Quadruple mask | 2018-10-24 | Incidence of Treatment-emergent Adverse Events (TEAE) |
| NCT03875729 | PHASE3 | COMPLETED | 328 | Randomized, Quadruple mask | 2019-04-05 | Change in C-peptide ln(AUC+1) Standardized by Duration of the Mixed Meal Tolerance Test (MMTT) |
| NCT04270942 | PHASE2 | COMPLETED | 6 | — | 2020-02-26 | Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Adverse Events of Special Interest (TEAESIs) and Treatment-e |
| NCT04598893 | nan | ACTIVE_NOT_RECRUITING | 188 | — | 2020-10-26 | Incidence of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs), including infections and malignancies |
| NCT05757713 | PHASE4 | ACTIVE_NOT_RECRUITING | 20 | — | 2023-07-25 | Treatment-emergent adverse events (TEAEs), adverse events of special interest (AESIs), TEAEs leading to withdrawal, and serious adverse events (SAEs) |
| NCT06338553 | EARLY_PHASE1 | RECRUITING | 24 | Randomized, Quadruple mask | 2024-06-12 | Investigate the impact of GLP-1Ra on postprandial glycemia in a pilot study // Study the impact of GLP-1Ra on the disposition index (DI) in a pilot st |
| NCT06481904 | nan | RECRUITING | 200 | — | 2024-09-27 | Number of adverse events of special interests (AESI) // Number of serious adverse events (SAE) // Number of adverse events (AE) in mothers, fetuses, a |
| NCT06892002 | nan | COMPLETED | 110 | — | 2025-02-11 | Participant demographic characteristics at teplizumab initiation // Participants' family history of T1D and autoimmune diseases // Presence of T1D sus |
| NCT06791291 | PHASE2 | RECRUITING | 10 | Randomized, None mask | 2025-07-25 | Number of participants with Stage 3 Type 1 Diabetes based on American Diabetes Association criteria // Change from baseline in area under the curve (A |
| NCT07088068 | PHASE3 | RECRUITING | 723 | Randomized, Quadruple mask | 2025-08-06 | For United States (US) and non-European Union (EU) countries: Glycated hemoglobin (HbA1c) change from baseline // For US and non-EU countries: Total n |
| NCT07260110 | nan | RECRUITING | 550 | — | 2025-10-31 | Change in participant and caregiver-reported outcomes from survey responses: ease of diabetes management questions // Change in participant and caregi |
| NCT07457580 | nan | RECRUITING | 60 | — | 2026-03-16 | Participant demographics at teplizumab initiation // Participants' family history of T1D and autoimmune diseases // Presence of T1D susceptibility gen |
| NCT07360080 | nan | RECRUITING | 1000 | — | 2026-03-19 | Time from teplizumab infusion start to the onset of Stage 3 T1D |
| NCT07216391 | PHASE2 | NOT_YET_RECRUITING | 60 | Randomized, None mask | 2026-07-30 | Change in DPTRS at six months |
| NCT07610213 | PHASE1 | NOT_YET_RECRUITING | 60 | Randomized, Single mask | 2027-01-01 | Change in Stimulated C-peptide AUC |
| NCT00008801 | PHASE1/PHASE2 | UNKNOWN | — | Non Randomized, None mask | — | — |