Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells

Aplastic Anemia Clinical Trial

Official title:

Haploidentical Stem Cell Transplantation With CD3/CD19 Depleted Stem Cells in Pediatric Patients With Refractory Hematological and Oncological Diseases

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01919866
• Other study ID #: 1075/01 PEI
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: August 7, 2013
• Last updated: October 7, 2014
• Start date: March 2004
• Est. completion date: December 2015

Study information

• Verified date: October 2014
• Source: University Children’s Hospital Tuebingen
• Contact: Peter Lang, MD
• Phone: +4970712981386
• Email: peter.lang[@]med.uni-tuebingen.de
• Is FDA regulated: No
• Health authority: Germany: Paul-Ehrlich-Institut
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 120
• Est. completion date: December 2015
• Est. primary completion date: December 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 25 Years

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Acute Leukemias
• Anemia, Aplastic
• Aplastic Anemia
• Childhood Solid Tumor

Intervention

Other:
• Transplantation of CD3/CD19 depleted stem cells

Locations

Country	Name	City	State
Austria	University Children's Hospital	Graz
Germany	University Children's Hospital	Halle
Germany	Medizinische Hochschule Zentrum für Kinderheilkunde	Hannover
Germany	University Children's Hospital	Jena
Germany	University Children's Hospital	Tuebingen

Sponsors (1)

Lead Sponsor	Collaborator
University Children’s Hospital Tuebingen

Countries where clinical trial is conducted

Austria,  Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Engraftment rate and transplant related mortality	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	365 days	Yes
Secondary	Graft versus Host disease and speed of immune reconstitution	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.	365 days	Yes