Minimum effective low dose of antithymocyte globulin in people aged 5-25 years with recent-onset stage 3 type 1 diabetes (MELD-ATG): a phase 2, multicentre, double-blind, randomised, placebo-controlled, adaptive dose-ranging trial

Type 1 diabetes remains an important health-care problem, with no disease-modifying therapies available in people with recent-onset, clinical type 1 diabetes. Adaptive trial designs, allowing faster evaluation of treatment modalities, remain underexplored in this stage of the disease. We aimed to id...

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Vydáno v:The Lancet (British edition) Ročník 406; číslo 10510; s. 1375
Hlavní autoři: Mathieu, Chantal, Wych, Julie, Hendriks, A Emile J, Van Ryckeghem, Lisa, Tree, Timothy, Chmura, Piotr, Möller, Christopher, Casteels, Kristina, Danne, Thomas, Reschke, Felix, Šmigoc Schweiger, Darja, Battelino, Tadej, Johannesen, Jesper, Rami-Merhar, Birgit, Pieber, Thomas, De Block, Christophe, Evans, Mark, Hilbrands, Robert, Bosi, Emanuele, Willemsen, Ruben H, Basu, Supriyo, Pulkkinen, Mari-Anne, Knip, Mikael, Cnop, Miriam, Nitsche, Almut, Schulte, Anke M, Niemöller, Elisabeth, Peakman, Mark, Wilhelm-Benartzi, Charlotte, Gillespie, David, Overbergh, Lut, Mander, Adrian P, Marcovecchio, M Loredana
Médium: Journal Article
Jazyk:angličtina
Vydáno: England 27.09.2025
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ISSN:1474-547X, 1474-547X
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Abstract Type 1 diabetes remains an important health-care problem, with no disease-modifying therapies available in people with recent-onset, clinical type 1 diabetes. Adaptive trial designs, allowing faster evaluation of treatment modalities, remain underexplored in this stage of the disease. We aimed to identify the minimum effective dose of antithymocyte globulin (ATG) in people aged 5-25 years with recent-onset, clinical type 1 diabetes. MELD-ATG was a phase 2, double-blind, randomised, placebo-controlled, multi-arm, adaptive dose-ranging, parallel-cohort trial done in 14 accredited trial centres in eight countries (the UK, Denmark, Germany, Finland, Italy, Belgium, Austria, and Slovenia). Participants aged 5-25 years, diagnosed with clinical, stage 3 type 1 diabetes 3-9 weeks before treatment, with random C-peptide concentrations 0·2 nmol/L or more and at least one diabetes-related autoantibody (GADA, IA-2A, or ZnT8) were randomly assigned by a web-based randomisation system into seven consecutive cohorts receiving placebo, 2·5 mg/kg ATG, 1·5 mg/kg ATG, 0·5 mg/kg ATG, or 0·1 mg/kg ATG. Participants in cohort 1 were randomly assigned 1:1:1:1:1, participants in cohorts 2 and 3 were randomly assigned 1:1:1:1, and participants in cohorts 4-7 were randomly assigned 1:1:1. All cohorts included one placebo group and one 2·5 mg/kg ATG group. The other groups were assigned to ATG doses that were determined based on accruing data and the decision of the dose determining committee. The trial cohorts were stratified by age group (5-9 years, 10-17 years, and 18-25 years) with block sizes varying by cohort. Concealment lists, outlining the treatment allocation, were only available for the pharmacists; participants and study teams were masked to treatment allocation. ATG was administered by an intravenous infusion over 2 consecutive days. The primary outcome was the area under the curve (AUC) of the stimulated C-peptide concentration during a 2-h mixed-meal tolerance test at 12 months measured as ln(AUC C-peptide + 1). Conditional on finding a statistically significant difference at p<0·05 for 2·5 mg/kg ATG versus placebo, the minimum effective dose of ATG was determined. All randomly assigned participants were included in the primary analysis. All participants who received the study drug were included in the safety analysis. The trial was registered at ClinicalTrials.gov (NCT04509791) and is completed. Between Nov 24, 2020, and Dec 13, 2023, 152 people were recruited and screened, 117 of whom were randomly assigned (placebo n=31, 0·1 mg/kg ATG n=6, 0·5 mg/kg ATG n=35, 1·5 mg/kg ATG n=12, and 2·5 mg/kg n=33). 54 (46%) of 117 participants were male and 63 (54%) were female. Participants were mainly European. The 0·1 mg/kg dose and the 1·5 mg/kg dose were progressively dropped from the study. At 12 months, the mean ln(AUC C-peptide + 1) was 0·411 nmol/L per min (SD 0·032) in the placebo group and 0·535 nmol/L per min (0·032) in the 2·5 mg/kg ATG group. The mean difference in the ln(AUC C-peptide + 1) between 2·5 mg/kg ATG and placebo was 0·124 nmol/L per min (95% CI 0·043-0·205; p=0·0028). At 12 months, the mean ln(AUC C-peptide + 1) in the 0·5 mg/kg ATG group, the remaining middle dose, was 0·513 nmol/L per min (SD 0·032), with a mean baseline-adjusted difference from placebo of 0·102 nmol/L per min (95% CI 0·021-0·183; p=0·014). Cytokine release syndrome occurred in 11 (33%) of 33 participants in the 2·5 mg/kg ATG group, eight (24%) of 34 in the 0·5mg/kg ATG group, and no participants in the placebo group. Serum sickness occurred in 27 (82%) participants in the 2·5 mg/kg ATG group, 11 (32%) in the 0·5 mg/kg ATG group, and no participants in the placebo group. There were no deaths related to adverse events. In young people with recent-onset, clinical type 1 diabetes, 2·5 mg/kg and 0·5 mg/kg ATG reduced loss of β-cell function, showing the potential of an affordable, repurposed agent, ATG, in a low and safe dose, as a disease-modifying agent in this population. The European Union's Innovative Medicines Initiative 2 Joint Undertaking INNODIA.
AbstractList Type 1 diabetes remains an important health-care problem, with no disease-modifying therapies available in people with recent-onset, clinical type 1 diabetes. Adaptive trial designs, allowing faster evaluation of treatment modalities, remain underexplored in this stage of the disease. We aimed to identify the minimum effective dose of antithymocyte globulin (ATG) in people aged 5-25 years with recent-onset, clinical type 1 diabetes.BACKGROUNDType 1 diabetes remains an important health-care problem, with no disease-modifying therapies available in people with recent-onset, clinical type 1 diabetes. Adaptive trial designs, allowing faster evaluation of treatment modalities, remain underexplored in this stage of the disease. We aimed to identify the minimum effective dose of antithymocyte globulin (ATG) in people aged 5-25 years with recent-onset, clinical type 1 diabetes.MELD-ATG was a phase 2, double-blind, randomised, placebo-controlled, multi-arm, adaptive dose-ranging, parallel-cohort trial done in 14 accredited trial centres in eight countries (the UK, Denmark, Germany, Finland, Italy, Belgium, Austria, and Slovenia). Participants aged 5-25 years, diagnosed with clinical, stage 3 type 1 diabetes 3-9 weeks before treatment, with random C-peptide concentrations 0·2 nmol/L or more and at least one diabetes-related autoantibody (GADA, IA-2A, or ZnT8) were randomly assigned by a web-based randomisation system into seven consecutive cohorts receiving placebo, 2·5 mg/kg ATG, 1·5 mg/kg ATG, 0·5 mg/kg ATG, or 0·1 mg/kg ATG. Participants in cohort 1 were randomly assigned 1:1:1:1:1, participants in cohorts 2 and 3 were randomly assigned 1:1:1:1, and participants in cohorts 4-7 were randomly assigned 1:1:1. All cohorts included one placebo group and one 2·5 mg/kg ATG group. The other groups were assigned to ATG doses that were determined based on accruing data and the decision of the dose determining committee. The trial cohorts were stratified by age group (5-9 years, 10-17 years, and 18-25 years) with block sizes varying by cohort. Concealment lists, outlining the treatment allocation, were only available for the pharmacists; participants and study teams were masked to treatment allocation. ATG was administered by an intravenous infusion over 2 consecutive days. The primary outcome was the area under the curve (AUC) of the stimulated C-peptide concentration during a 2-h mixed-meal tolerance test at 12 months measured as ln(AUC C-peptide + 1). Conditional on finding a statistically significant difference at p<0·05 for 2·5 mg/kg ATG versus placebo, the minimum effective dose of ATG was determined. All randomly assigned participants were included in the primary analysis. All participants who received the study drug were included in the safety analysis. The trial was registered at ClinicalTrials.gov (NCT04509791) and is completed.METHODSMELD-ATG was a phase 2, double-blind, randomised, placebo-controlled, multi-arm, adaptive dose-ranging, parallel-cohort trial done in 14 accredited trial centres in eight countries (the UK, Denmark, Germany, Finland, Italy, Belgium, Austria, and Slovenia). Participants aged 5-25 years, diagnosed with clinical, stage 3 type 1 diabetes 3-9 weeks before treatment, with random C-peptide concentrations 0·2 nmol/L or more and at least one diabetes-related autoantibody (GADA, IA-2A, or ZnT8) were randomly assigned by a web-based randomisation system into seven consecutive cohorts receiving placebo, 2·5 mg/kg ATG, 1·5 mg/kg ATG, 0·5 mg/kg ATG, or 0·1 mg/kg ATG. Participants in cohort 1 were randomly assigned 1:1:1:1:1, participants in cohorts 2 and 3 were randomly assigned 1:1:1:1, and participants in cohorts 4-7 were randomly assigned 1:1:1. All cohorts included one placebo group and one 2·5 mg/kg ATG group. The other groups were assigned to ATG doses that were determined based on accruing data and the decision of the dose determining committee. The trial cohorts were stratified by age group (5-9 years, 10-17 years, and 18-25 years) with block sizes varying by cohort. Concealment lists, outlining the treatment allocation, were only available for the pharmacists; participants and study teams were masked to treatment allocation. ATG was administered by an intravenous infusion over 2 consecutive days. The primary outcome was the area under the curve (AUC) of the stimulated C-peptide concentration during a 2-h mixed-meal tolerance test at 12 months measured as ln(AUC C-peptide + 1). Conditional on finding a statistically significant difference at p<0·05 for 2·5 mg/kg ATG versus placebo, the minimum effective dose of ATG was determined. All randomly assigned participants were included in the primary analysis. All participants who received the study drug were included in the safety analysis. The trial was registered at ClinicalTrials.gov (NCT04509791) and is completed.Between Nov 24, 2020, and Dec 13, 2023, 152 people were recruited and screened, 117 of whom were randomly assigned (placebo n=31, 0·1 mg/kg ATG n=6, 0·5 mg/kg ATG n=35, 1·5 mg/kg ATG n=12, and 2·5 mg/kg n=33). 54 (46%) of 117 participants were male and 63 (54%) were female. Participants were mainly European. The 0·1 mg/kg dose and the 1·5 mg/kg dose were progressively dropped from the study. At 12 months, the mean ln(AUC C-peptide + 1) was 0·411 nmol/L per min (SD 0·032) in the placebo group and 0·535 nmol/L per min (0·032) in the 2·5 mg/kg ATG group. The mean difference in the ln(AUC C-peptide + 1) between 2·5 mg/kg ATG and placebo was 0·124 nmol/L per min (95% CI 0·043-0·205; p=0·0028). At 12 months, the mean ln(AUC C-peptide + 1) in the 0·5 mg/kg ATG group, the remaining middle dose, was 0·513 nmol/L per min (SD 0·032), with a mean baseline-adjusted difference from placebo of 0·102 nmol/L per min (95% CI 0·021-0·183; p=0·014). Cytokine release syndrome occurred in 11 (33%) of 33 participants in the 2·5 mg/kg ATG group, eight (24%) of 34 in the 0·5mg/kg ATG group, and no participants in the placebo group. Serum sickness occurred in 27 (82%) participants in the 2·5 mg/kg ATG group, 11 (32%) in the 0·5 mg/kg ATG group, and no participants in the placebo group. There were no deaths related to adverse events.FINDINGSBetween Nov 24, 2020, and Dec 13, 2023, 152 people were recruited and screened, 117 of whom were randomly assigned (placebo n=31, 0·1 mg/kg ATG n=6, 0·5 mg/kg ATG n=35, 1·5 mg/kg ATG n=12, and 2·5 mg/kg n=33). 54 (46%) of 117 participants were male and 63 (54%) were female. Participants were mainly European. The 0·1 mg/kg dose and the 1·5 mg/kg dose were progressively dropped from the study. At 12 months, the mean ln(AUC C-peptide + 1) was 0·411 nmol/L per min (SD 0·032) in the placebo group and 0·535 nmol/L per min (0·032) in the 2·5 mg/kg ATG group. The mean difference in the ln(AUC C-peptide + 1) between 2·5 mg/kg ATG and placebo was 0·124 nmol/L per min (95% CI 0·043-0·205; p=0·0028). At 12 months, the mean ln(AUC C-peptide + 1) in the 0·5 mg/kg ATG group, the remaining middle dose, was 0·513 nmol/L per min (SD 0·032), with a mean baseline-adjusted difference from placebo of 0·102 nmol/L per min (95% CI 0·021-0·183; p=0·014). Cytokine release syndrome occurred in 11 (33%) of 33 participants in the 2·5 mg/kg ATG group, eight (24%) of 34 in the 0·5mg/kg ATG group, and no participants in the placebo group. Serum sickness occurred in 27 (82%) participants in the 2·5 mg/kg ATG group, 11 (32%) in the 0·5 mg/kg ATG group, and no participants in the placebo group. There were no deaths related to adverse events.In young people with recent-onset, clinical type 1 diabetes, 2·5 mg/kg and 0·5 mg/kg ATG reduced loss of β-cell function, showing the potential of an affordable, repurposed agent, ATG, in a low and safe dose, as a disease-modifying agent in this population.INTERPRETATIONIn young people with recent-onset, clinical type 1 diabetes, 2·5 mg/kg and 0·5 mg/kg ATG reduced loss of β-cell function, showing the potential of an affordable, repurposed agent, ATG, in a low and safe dose, as a disease-modifying agent in this population.The European Union's Innovative Medicines Initiative 2 Joint Undertaking INNODIA.FUNDINGThe European Union's Innovative Medicines Initiative 2 Joint Undertaking INNODIA.
Type 1 diabetes remains an important health-care problem, with no disease-modifying therapies available in people with recent-onset, clinical type 1 diabetes. Adaptive trial designs, allowing faster evaluation of treatment modalities, remain underexplored in this stage of the disease. We aimed to identify the minimum effective dose of antithymocyte globulin (ATG) in people aged 5-25 years with recent-onset, clinical type 1 diabetes. MELD-ATG was a phase 2, double-blind, randomised, placebo-controlled, multi-arm, adaptive dose-ranging, parallel-cohort trial done in 14 accredited trial centres in eight countries (the UK, Denmark, Germany, Finland, Italy, Belgium, Austria, and Slovenia). Participants aged 5-25 years, diagnosed with clinical, stage 3 type 1 diabetes 3-9 weeks before treatment, with random C-peptide concentrations 0·2 nmol/L or more and at least one diabetes-related autoantibody (GADA, IA-2A, or ZnT8) were randomly assigned by a web-based randomisation system into seven consecutive cohorts receiving placebo, 2·5 mg/kg ATG, 1·5 mg/kg ATG, 0·5 mg/kg ATG, or 0·1 mg/kg ATG. Participants in cohort 1 were randomly assigned 1:1:1:1:1, participants in cohorts 2 and 3 were randomly assigned 1:1:1:1, and participants in cohorts 4-7 were randomly assigned 1:1:1. All cohorts included one placebo group and one 2·5 mg/kg ATG group. The other groups were assigned to ATG doses that were determined based on accruing data and the decision of the dose determining committee. The trial cohorts were stratified by age group (5-9 years, 10-17 years, and 18-25 years) with block sizes varying by cohort. Concealment lists, outlining the treatment allocation, were only available for the pharmacists; participants and study teams were masked to treatment allocation. ATG was administered by an intravenous infusion over 2 consecutive days. The primary outcome was the area under the curve (AUC) of the stimulated C-peptide concentration during a 2-h mixed-meal tolerance test at 12 months measured as ln(AUC C-peptide + 1). Conditional on finding a statistically significant difference at p<0·05 for 2·5 mg/kg ATG versus placebo, the minimum effective dose of ATG was determined. All randomly assigned participants were included in the primary analysis. All participants who received the study drug were included in the safety analysis. The trial was registered at ClinicalTrials.gov (NCT04509791) and is completed. Between Nov 24, 2020, and Dec 13, 2023, 152 people were recruited and screened, 117 of whom were randomly assigned (placebo n=31, 0·1 mg/kg ATG n=6, 0·5 mg/kg ATG n=35, 1·5 mg/kg ATG n=12, and 2·5 mg/kg n=33). 54 (46%) of 117 participants were male and 63 (54%) were female. Participants were mainly European. The 0·1 mg/kg dose and the 1·5 mg/kg dose were progressively dropped from the study. At 12 months, the mean ln(AUC C-peptide + 1) was 0·411 nmol/L per min (SD 0·032) in the placebo group and 0·535 nmol/L per min (0·032) in the 2·5 mg/kg ATG group. The mean difference in the ln(AUC C-peptide + 1) between 2·5 mg/kg ATG and placebo was 0·124 nmol/L per min (95% CI 0·043-0·205; p=0·0028). At 12 months, the mean ln(AUC C-peptide + 1) in the 0·5 mg/kg ATG group, the remaining middle dose, was 0·513 nmol/L per min (SD 0·032), with a mean baseline-adjusted difference from placebo of 0·102 nmol/L per min (95% CI 0·021-0·183; p=0·014). Cytokine release syndrome occurred in 11 (33%) of 33 participants in the 2·5 mg/kg ATG group, eight (24%) of 34 in the 0·5mg/kg ATG group, and no participants in the placebo group. Serum sickness occurred in 27 (82%) participants in the 2·5 mg/kg ATG group, 11 (32%) in the 0·5 mg/kg ATG group, and no participants in the placebo group. There were no deaths related to adverse events. In young people with recent-onset, clinical type 1 diabetes, 2·5 mg/kg and 0·5 mg/kg ATG reduced loss of β-cell function, showing the potential of an affordable, repurposed agent, ATG, in a low and safe dose, as a disease-modifying agent in this population. The European Union's Innovative Medicines Initiative 2 Joint Undertaking INNODIA.
Author Evans, Mark
Johannesen, Jesper
De Block, Christophe
Gillespie, David
Hendriks, A Emile J
Overbergh, Lut
Möller, Christopher
Pulkkinen, Mari-Anne
Mander, Adrian P
Wilhelm-Benartzi, Charlotte
Casteels, Kristina
Wych, Julie
Battelino, Tadej
Cnop, Miriam
Marcovecchio, M Loredana
Niemöller, Elisabeth
Pieber, Thomas
Chmura, Piotr
Peakman, Mark
Basu, Supriyo
Tree, Timothy
Rami-Merhar, Birgit
Willemsen, Ruben H
Hilbrands, Robert
Knip, Mikael
Nitsche, Almut
Danne, Thomas
Reschke, Felix
Šmigoc Schweiger, Darja
Van Ryckeghem, Lisa
Schulte, Anke M
Mathieu, Chantal
Bosi, Emanuele
Author_xml – sequence: 1
  givenname: Chantal
  surname: Mathieu
  fullname: Mathieu, Chantal
  email: chantal.mathieu@uzleuven.be
  organization: Department of Endocrinology, UZ Gasthuisberg, UZ Leuven, Leuven, Belgium; Department of Chronic Diseases and Metabolism, KU Leuven, Leuven, Belgium. Electronic address: chantal.mathieu@uzleuven.be
– sequence: 2
  givenname: Julie
  surname: Wych
  fullname: Wych, Julie
  organization: Centre for Trials Research College of Biomedical and Life Sciences, Cardiff University, Cardiff, UK
– sequence: 3
  givenname: A Emile J
  surname: Hendriks
  fullname: Hendriks, A Emile J
  organization: Department of Paediatrics, University of Cambridge, Cambridge, UK; Department of Paediatric Diabetes and Endocrinology, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK
– sequence: 4
  givenname: Lisa
  surname: Van Ryckeghem
  fullname: Van Ryckeghem, Lisa
  organization: Department of Endocrinology, UZ Gasthuisberg, UZ Leuven, Leuven, Belgium
– sequence: 5
  givenname: Timothy
  surname: Tree
  fullname: Tree, Timothy
  organization: Department of Immunobiology, School of Immunology and Microbial Sciences, King's College London, London, UK
– sequence: 6
  givenname: Piotr
  surname: Chmura
  fullname: Chmura, Piotr
  organization: Novo Nordisk Foundation Center for Protein Research, University of Copenhagen, Copenhagen, Denmark
– sequence: 7
  givenname: Christopher
  surname: Möller
  fullname: Möller, Christopher
  organization: Novo Nordisk Foundation Center for Protein Research, University of Copenhagen, Copenhagen, Denmark
– sequence: 8
  givenname: Kristina
  surname: Casteels
  fullname: Casteels, Kristina
  organization: Department of Paediatrics, UZ Gasthuisberg, UZ Leuven, Leuven, Belgium
– sequence: 9
  givenname: Thomas
  surname: Danne
  fullname: Danne, Thomas
  organization: Center for Paediatric Endocrinology, Diabetology, and Clinical Research, Auf Der Bult Children's Hospital, Hannover, Germany
– sequence: 10
  givenname: Felix
  surname: Reschke
  fullname: Reschke, Felix
  organization: Center for Paediatric Endocrinology, Diabetology, and Clinical Research, Auf Der Bult Children's Hospital, Hannover, Germany
– sequence: 11
  givenname: Darja
  surname: Šmigoc Schweiger
  fullname: Šmigoc Schweiger, Darja
  organization: Department of Endocrinology, Diabetes, and Metabolic Diseases, University Children's Hospital, University Medical Center Ljubljana, Ljubljana, Slovenia; Faculty of Medicine, University of Ljubljana, Ljubljana, Slovenia
– sequence: 12
  givenname: Tadej
  surname: Battelino
  fullname: Battelino, Tadej
  organization: Department of Endocrinology, Diabetes, and Metabolic Diseases, University Children's Hospital, University Medical Center Ljubljana, Ljubljana, Slovenia; Faculty of Medicine, University of Ljubljana, Ljubljana, Slovenia
– sequence: 13
  givenname: Jesper
  surname: Johannesen
  fullname: Johannesen, Jesper
  organization: Department of Clinical Medicine, University of Copenhagen, Copenhagen, Denmark; Copenhagen University Hospital Herlev, Department of Paediatrics, Copenhagen, Denmark; Steno Diabetes Center, Copenhagen, Denmark
– sequence: 14
  givenname: Birgit
  surname: Rami-Merhar
  fullname: Rami-Merhar, Birgit
  organization: Department of Paediatric and Adolescent Medicine, Division of Paediatric Pulmonology, Allergology, and Endocrinology, Comprehensive Centre for Paediatrics, Medical University of Vienna, Vienna, Austria
– sequence: 15
  givenname: Thomas
  surname: Pieber
  fullname: Pieber, Thomas
  organization: Division of Endocrinology and Diabetology, Department of Internal Medicine, Medical University of Graz, Graz, Austria
– sequence: 16
  givenname: Christophe
  surname: De Block
  fullname: De Block, Christophe
  organization: Department of Endocrinology-Diabetology-Metabolism, Antwerp University Hospital and University of Antwerp, Antwerp, Belgium
– sequence: 17
  givenname: Mark
  surname: Evans
  fullname: Evans, Mark
  organization: Institute of Metabolic Science and Department of Medicine, University of Cambridge, Cambridge, UK
– sequence: 18
  givenname: Robert
  surname: Hilbrands
  fullname: Hilbrands, Robert
  organization: Academic Hospital and Diabetes Research Centre, Vrije Universiteit Brussels, Brussels, Belgium
– sequence: 19
  givenname: Emanuele
  surname: Bosi
  fullname: Bosi, Emanuele
  organization: Diabetes Research Institute San Raffaele Hospital and San Raffaele Vita Salute University, Milan, Italy
– sequence: 20
  givenname: Ruben H
  surname: Willemsen
  fullname: Willemsen, Ruben H
  organization: Department of Paediatric Diabetes and Endocrinology, The Royal London Children's hospital, Barts Health NHS Trust, London, UK
– sequence: 21
  givenname: Supriyo
  surname: Basu
  fullname: Basu, Supriyo
  organization: Department of Paediatric Endocrinology and Diabetes, Oxford University Hospitals NHS Foundation Trust, Oxford, UK
– sequence: 22
  givenname: Mari-Anne
  surname: Pulkkinen
  fullname: Pulkkinen, Mari-Anne
  organization: New Children's Hospital, Paediatric Research Centre, Helsinki University Hospital and Faculty of Medicine, University of Helsinki, Helsinki, Finland
– sequence: 23
  givenname: Mikael
  surname: Knip
  fullname: Knip, Mikael
  organization: Research Program for Clinical and Molecular Metabolism, University of Helsinki, Helsinki, Finland
– sequence: 24
  givenname: Miriam
  surname: Cnop
  fullname: Cnop, Miriam
  organization: ULB Center for Diabetes Research, Université Libre de Bruxelles, Brussels, Belgium; Division of Endocrinology, ULB Erasmus Hospital, Brussels University Hospital, Université Libre de Bruxelles, Brussels, Belgium
– sequence: 25
  givenname: Almut
  surname: Nitsche
  fullname: Nitsche, Almut
  organization: Diabetes Cardiovascular Metabolic Development, Research and Development, Sanofi-Deutschland, Frankfurt, Germany
– sequence: 26
  givenname: Anke M
  surname: Schulte
  fullname: Schulte, Anke M
  organization: Diabetes Cardiovascular Metabolic Development, Research and Development, Sanofi-Deutschland, Frankfurt, Germany
– sequence: 27
  givenname: Elisabeth
  surname: Niemöller
  fullname: Niemöller, Elisabeth
  organization: Diabetes Cardiovascular Metabolic Development, Research and Development, Sanofi-Deutschland, Frankfurt, Germany
– sequence: 28
  givenname: Mark
  surname: Peakman
  fullname: Peakman, Mark
  organization: Department of Immunobiology, School of Immunology and Microbial Sciences, King's College London, London, UK; Sanofi Research and Development, Paris, France
– sequence: 29
  givenname: Charlotte
  surname: Wilhelm-Benartzi
  fullname: Wilhelm-Benartzi, Charlotte
  organization: Centre for Trials Research College of Biomedical and Life Sciences, Cardiff University, Cardiff, UK
– sequence: 30
  givenname: David
  surname: Gillespie
  fullname: Gillespie, David
  organization: Centre for Trials Research College of Biomedical and Life Sciences, Cardiff University, Cardiff, UK
– sequence: 31
  givenname: Lut
  surname: Overbergh
  fullname: Overbergh, Lut
  organization: Department of Chronic Diseases and Metabolism, KU Leuven, Leuven, Belgium
– sequence: 32
  givenname: Adrian P
  surname: Mander
  fullname: Mander, Adrian P
  organization: Centre for Trials Research College of Biomedical and Life Sciences, Cardiff University, Cardiff, UK
– sequence: 33
  givenname: M Loredana
  surname: Marcovecchio
  fullname: Marcovecchio, M Loredana
  organization: Department of Paediatrics, University of Cambridge, Cambridge, UK; Department of Paediatric Diabetes and Endocrinology, Cambridge University Hospitals NHS Foundation Trust, Cambridge, UK
BackLink https://www.ncbi.nlm.nih.gov/pubmed/40976248$$D View this record in MEDLINE/PubMed
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CitedBy_id crossref_primary_10_1016_S0140_6736_25_01438_2
ContentType Journal Article
Contributor Evans, Mark
Bonifacio, Ezio
Morgan, Noel G
Dayan, Colin M
Agiostratidou, Gina
Wicker, Linda
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Snippet Type 1 diabetes remains an important health-care problem, with no disease-modifying therapies available in people with recent-onset, clinical type 1 diabetes....
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StartPage 1375
SubjectTerms Adolescent
Adult
Antilymphocyte Serum - administration & dosage
Antilymphocyte Serum - adverse effects
Child
Child, Preschool
Diabetes Mellitus, Type 1 - drug therapy
Dose-Response Relationship, Drug
Double-Blind Method
Female
Humans
Immunosuppressive Agents - administration & dosage
Male
Treatment Outcome
Young Adult
Title Minimum effective low dose of antithymocyte globulin in people aged 5-25 years with recent-onset stage 3 type 1 diabetes (MELD-ATG): a phase 2, multicentre, double-blind, randomised, placebo-controlled, adaptive dose-ranging trial
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