- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT01947569
Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy
Autologous Co-stimulation-impaired Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy: A Sequential Open Label Phase IB Safety Assessment/ Randomized, Double-blind Phase IIA Efficacy Trial to Maintain and Improve Functional Beta Cell Mass in New Onset Disease T1DM Patients
Phase IB will evaluate the safety of autologous, ex vivo-engineered, co-stimulation impaired dendtritic cells to maintain and improve functional residual beta cell mass in new onset Type I Diabetes Mellitus (T1DM) patients. Efficacy measures will be collected and summarized.
Phase IIA will evaluate the safety and efficacy of 3 randomized treatment groups in new onset T1DM patients to assess if the antisense DNA-treated co-stimulation-impaired immunoregulatory dendritic cells (iDC) will safely preserve and/or increase B-cell mass resulting in improvement and/or normalization of blood glucose levels and glycated hemoglobin A1c.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Tipo de estudo
Inscrição (Antecipado)
Estágio
- Fase 2
- Fase 1
Contactos e Locais
Locais de estudo
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Pennsylvania
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Pittsburgh, Pennsylvania, Estados Unidos, 15213
- UPMC
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
Inclusion Criteria:(both open label phase IB safety and Phase IIA study):
- Patients with new onset T1DM (>18 years of age for the phase IB and then >16 (first 10 subjects), >12 years of age for the second 10 subjects, > 8 years for the next 10 subjects and, finally, >8 years of age for the remainder of the phase IIA patients) within 6 months of diabetes mellitus diagnosis.
- Evidence of decreased β-cell function as measured by C-peptide and blood glucose levels consistent with impaired glucose tolerance.
- Evidence of at least one high-risk HLA haplotype.
- Evidence of at least one diabetes-related autoantibody (e.g. IA-2, GAD, ZnT8) ,
- Adequate immune competence as assessed by immunoreactivity to alloantigens in mixed leukocyte culture and reactivity to viral antigens (CEF Pool Assay) in vitro.
- Normal hematologic, liver and kidney function.
- Female participants of childbearing potential in this study must agree to use an effective form of birth control during study participation. Reliable and effective forms of birth control include: true abstinence, intrauterine device (IUD), hormonal-based contraception, double-barrier contraception [condom or occlusive cap (diaphragm or cervical cap) with spermicide], or surgical sterilization (vasectomy for male partner, tubal ligation or hysterectomy). Sexually active male participants must agree to use an effective form of birth control such as condoms.
Exclusion Criteria:(both open label phase IB safety and Phase IIA study):
- Enrollment or history of enrollment in a drug, or biologic therapy study sponsored by TrialNet.
- A significant history or current evidence of cardiac disease, uncontrolled hypertension, serious arrhythmias.
- Evidence of active infection requiring antibiotic therapy.
- History of other concurrent significant medical diseases.
- Pregnant or lactating women.
- Patients requiring chronic systemic corticosteroids.
- Any other immune disorder including but not limited to other autoimmune diseases, HIV, HBV, HCV, HPV, HSV positivity.
- Impaired renal function with a creatinine level > 1.5.
- Administration of the following therapies while patients are undergoing treatment on this protocol: i) radiation therapy; ii) chemotherapy; iii) corticosteroids (except when administered in life-threatening circumstances); iv) other particle or cell-based therapies; v) other biologic therapies; vi) other therapies aimed at modulating the immune system; vii) other endocrine-related therapies, hormone replacement (other than thyroxine and contraceptive), glucoregulation.
- A hemaglobinopathy known to interfere with the ability to accurately determine HbA1c.
- No prior radiation therapy, immunotherapy, or chemotherapy.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Triplo
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
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Experimental: iDC recipients
iDC cells modified by in vitro engineering.
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Comparador Ativo: Control DC recipients
DC cells which have not been modified.
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Comparador de Placebo: Placebo recipients
Saline injections.
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Prazo |
|---|---|
|
The incidence of treatment-emergent adverse events.
Prazo: Month 12
|
Month 12
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Medidas de resultados secundários
Medida de resultado |
Prazo |
|---|---|
|
2-hour area under the curve (AUC) average of C-peptide at 12 months after completion of administration of assigned therapy (Protocol Month 15).
Prazo: 12 Months
|
12 Months
|
Colaboradores e Investigadores
Patrocinador
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo
Conclusão Primária (Antecipado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Estimativa)
Atualizações de registro de estudo
Última Atualização Postada (Estimativa)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- DV-0100-100
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