Haploidentical Stem Cell Transplantation and IL-15 NK Cel... | NCT01337544

Studies
Haploidentical Stem Cell Transplantation and IL-15 NK Cell Infusion for Paediatric Refractory Solid Tumours

NCT01337544

Sponsor

Hospital Infantil Universitario Niño Jesús, Madrid, Spain

Status

Terminated

Last Update Posted

Nov 11, 2013Estimated

Enrollment

6Actual

Phase

Phase 1Phase 2

Conditions

Childhood Solid Tumor

Interventions

HAPLOIDENTICAL IL-15 STIMULATED NK CELLS

Countries

Spain

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2013

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

The parents of two patients enrolled who died, presented a claim against the hospital and the study was halted.

Expanded Access Info

Start Date

Jan 2011

Primary Completion Date

Nov 2012Actual

Completion Date

Nov 2012Actual

First Submitted Date

Mar 25, 2011

First Submission Date that Met QC Criteria

Apr 18, 2011

First Posted Date

Apr 19, 2011Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 8, 2013

Last Update Posted Date

Nov 11, 2013Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

Antonio Perez-Martinez, Servicio de Hemato-Oncología Pediátrica, Hospital Infantil Universitario Niño Jesús, Madrid, SpainPrincipal Investigator

Lead Sponsor

Hospital Infantil Universitario Niño Jesús, Madrid, SpainOTHER

Collaborators

Name	Class
SPANISH HEALTH RESEARCH FUND (FIS)	UNKNOWN

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

The investigators propose a new antitumor cell therapy for treating childhood refractory solid tumours. The aim of this study is explore the graft versus tumour effect mediated by allogenic natural killer cells (NKs). NK cell alloreactivity can be predicted by donor killer immunoglobulin-like receptors (KIRs) and human leukocyte antigen (HLA) class I alleles mismatch. Cells without an inhibitory HLA ligand may trigger natural killer cell activation and elimination of those target cells. Reduced risk of relapsed has been described in malignant cancer after haploidentical stem cell transplantation when HLA ligands against the inhibitory KIRs present in the donor were absent in the recipient (KIR-HLA receptor-ligand mismatch). NK alloreactivity could also be obtained by Natural Killer Receptor (NCR), Toll-Like-Receptors (TLRs) and NKG2D receptor stimulation mediated by cytokines or tumour cell lines.

This will be an open, non randomized, Phase I/II clinical trial, with a double objective: therapeutic exploratory. The investigators aim at studying safety and efficacy of haploidentical stem cell transplantation for the treatment of these malignancies with no cure known. Patients will receive an haploidentical stem cell transplantation, followed by IL-15 stimulated NK cells infusion one month after transplantation. Efficacy of the procedure will be evaluated with up-to-date radiological techniques, molecular studies and functional assays.

Detailed Description

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

6Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
IL-15 STIMULATED NK CELLS	Experimental		Other: HAPLOIDENTICAL IL-15 STIMULATED NK CELLS

Interventions

Name	Type	Description	Arm Group Labels	Other Names
HAPLOIDENTICAL IL-15 STIMULATED NK CELLS	Other	ONE MEGADOSE 30 DAYS AFTER TRANSPLANTATION (DOSE WILL DEPEND ON PATIENT BODY WEIGHT)	IL-15 STIMULATED NK CELLS

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of patients with adverse events according to NCI-CTC v3.0 CRITERIA as a measure of safety and tolerability		Up To 1 Year After Transplantation

Secondary Outcomes

Measure	Description	Time Frame
Objective Response Rate According RECIST V1.1		Up To One Year After Transplantation

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Age 6 months to 22 years.
Histological solid tumor confirmation.
Measurable solid tumor by image or molecular techniques.
Solid tumors that have failed to at least 2 chemotherapy protocols.
Suitable haploidentical donor available.
Lansky score > 60%.

Exclusion Criteria:

Serum bilirubin > 3 mg/dl
GFR < 40 ml/min/1.73 mw
Cardiac left ventricular ejection fraction < 40%
HIV+
Pregnant
Unfavorable psycho-social report.
Antecedent of abandonment treatment.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Months

Maximum Age

22 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
ANTONIO PEREZ-MARTINEZ, MD, PhD	HOSPITAL UNIVERSITARIO NINO JESUS	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital Infantil Universitario Niño Jesus		Madrid	Madrid	28009	Spain

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
19215002	Background	Perez-Martinez A, Leung W, Munoz E, Iyengar R, Ramirez M, Vicario JL, Lassaletta A, Sevilla J, Gonzalez-Vicent M, Madero L, Diaz-Perez MA. KIR-HLA receptor-ligand mismatch associated with a graft-versus-tumor effect in haploidentical stem cell transplantation for pediatric metastatic solid tumors. Pediatr Blood Cancer. 2009 Jul;53(1):120-4. doi: 10.1002/pbc.21955.

ID	Term
D001691	Biological Therapy
D013812	Therapeutics