Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells

Phase 1

• Conditions: Aplastic Anemia; Acute Leukemias; Childhood Solid Tumor
• Interventions: Other: Transplantation of CD3/CD19 depleted stem cells

• Registration Number: NCT01919866
• Lead Sponsor: University Children's Hospital Tuebingen

Brief Summary

Feasibility and toxicity of haploidentical transplantation of CD3/CD19 depleted stem cells in combination with a toxicity reduced conditioning regimen or with standard conditioning regimens according to underlying disease.

Detailed Description

Not available

Study Timeline

• Study Start: 2004-03
• Study First Submitted: 2013-08-07
• Study First Submitted QC: 2013-08-08
• Study First Posted: 2013-08-09
• Status Verified: 2014-10
• Last Update Submitted: 2014-10-07
• Last Update Posted: 2014-10-08
• Primary Completion: 2014-12
• Study Completion: 2015-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 120

Inclusion Criteria

• patients with primary refractory or relapsed disease (including relapse after previous SCT and active disease)
• AML in complete remission of refractory
• MDS RAEB-t/secondary AML
• ALL
• CML
• Non-Hodgkin Lymphoma / Hodgkin Lymphoma
• non malignant diseases (aplastic anemia, thalassemia, SCID)
• relapsed Neuroblastoma
• relapsed soft tissue sarcoma (Rhabdomyosarcoma, Ewing sarcoma, PNET
• soft tissue sarcoma with primary bone metastases or bone marrow
• in whom no matched donor was available,

Exclusion Criteria

• < 6 months after previous HSCT
• active cerebral seizure conditions
• massive progression of leukemias or solid tumours before planned trp.
• left ventricular ejection fraction <25%
• creatinine clearance <40ml/min before conditioning
• respiratory insufficiency with oxygen demand or DLCO <30%
• Bilirubin >4mg/dl, GOT/GPT >400
• severe infection (HIV, Aspergillosis)
• pregnancy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Transplantation of CD3/CD19 depleted stem cells	Transplantation of CD3/CD19 depleted stem cells	Patients receive a minimum dosage of 10x10E6/kg bodyweight CD3/CD19 depleted CD34+ stem cells. Maximum dosage of residual T cells will be 1x10E5/kg BW

Primary Outcome Measures

Name	Time	Method
Engraftment rate and transplant related mortality	365 days	engraftment rate will be evaluated by leukocyte counts in peripheral blood . Definition of engraftment: \>500 neutrophiles/µl blood. Transplant related mortality will be evaluated at day 100 and day 365 post transplant

Secondary Outcome Measures

Name	Time	Method
Graft versus Host disease and speed of immune reconstitution	365 days	Incidence of acute and chronic GvHD will be evaluated according to Glucksberg criteria. Immune recovery will be evaluated by flow cytometry. Percentages and numbers of T, B and NK cells will be measured weekly until day 100, afterwards on day 180 and 365.

Trial Locations

Locations (2)

University Children's Hospital; 🇩🇪 Tuebingen, Germany

Medizinische Hochschule Zentrum für Kinderheilkunde; 🇩🇪 Hannover, Germany