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mbilical cord blood transplantation in high-risk hematological patients using stemregenin-1 expanded hematopoietic stem cells.<br>A feasibility study focussing on engraftment and hematopoietic recovery.

Phase 2
Conditions
high-risk hematological patients
10018849
Registration Number
NL-OMON43292
Lead Sponsor
Erasmus MC, Universitair Medisch Centrum Rotterdam
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Pending
Sex
Not specified
Target Recruitment
10
Inclusion Criteria

- Age 18-70 years inclusive
- Diagnosis of poor-risk hematological malignancy or (V)SAA relapsing after or failing immunosuppressive therapy and meeting the criteria for a MUD allo SCT
- Lacking a sufficiently matched volunteer unrelated donor or lacking such a donor within the required time period of <= 2 months in case of urgently needed alloSCT
- Availability of 1 (>=5/6) matched UCB graft with a nuclear cell count 2.7 x 107/kg (see paragraph 8.2).
- Availability of an back-up autograft, harvested and frozen earlier in the course of treatment, (harvest according to local aphersis policies)
- WHO performance status 0-2
- Written informed consent

Exclusion Criteria

• Bilirubin and/or transaminases > 2.5 x normal value
• Creatinine clearance < 40 ml/min
• Cardiac dysfunction as defined by:
-Reduced left ventricular function with an ejection fraction < 45% as measured by MUGA scan or echocardiogram (another method for measuring cardiac function is acceptable)
- Unstable angina
- Unstable cardiac arrhythmias
• Pulmonary function test with VC, FEV1 and/ or DCO < 50%
• Active, uncontrolled infection
• History of high dose (>= 8 Gy) total body irradiation
• Pregnant or lactating females
• HIV positivity

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
<p>Feasibility as defined by, and to be achieved in >= 80% of (evaluable) patients:<br /><br>1. SR-1 mediated expansion, resulting in > 20-fold expansion of CD34+ cells, and<br /><br>2. effective hematopoietic (neutrophils > 0.5 x 109/L) engraftment within 30<br /><br>days upon transplantation </p><br>
Secondary Outcome Measures
NameTimeMethod
<p>NA</p><br>
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