- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving 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.
Studieoversikt
Status
Intervensjon / Behandling
Studietype
Registrering (Forventet)
Fase
- Fase 2
- Fase 1
Kontakter og plasseringer
Studiesteder
-
-
Pennsylvania
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Pittsburgh, Pennsylvania, Forente stater, 15213
- UPMC
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
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.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Trippel
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: iDC recipients
iDC cells modified by in vitro engineering.
|
|
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Aktiv komparator: Control DC recipients
DC cells which have not been modified.
|
|
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Placebo komparator: Placebo recipients
Saline injections.
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
The incidence of treatment-emergent adverse events.
Tidsramme: Month 12
|
Month 12
|
Sekundære resultatmål
Resultatmål |
Tidsramme |
|---|---|
|
2-hour area under the curve (AUC) average of C-peptide at 12 months after completion of administration of assigned therapy (Protocol Month 15).
Tidsramme: 12 Months
|
12 Months
|
Samarbeidspartnere og etterforskere
Sponsor
Studierekorddatoer
Studer hoveddatoer
Studiestart
Primær fullføring (Forventet)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Anslag)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Anslag)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- DV-0100-100
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