Clinical trial NCT00750126: Allogeneic Hematopoietic Stem Cell Transplantation
Official title: Feasibility Study on Allogeneic Hematopoietic Stem Cell Transplantation Following Fludarabine-Busulfan-ALS-based Reduced-intensity Conditioning in Children With Hematological Malignancy or Solid Tumor Not Responding to Standard Therapy or for Which the Indication for Allograft is Maintained But Cannot be Carried Out Due to the Toxicity Involved in Myeloablative Conditioning.
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Solid Tumors
Hematologic Neoplasms
Keywords: Allograft
Children
malignancy
Reduced Intensity Conditioning
Fludarabin
Busulfex
Thymoglobulin
Lead sponsor: University Hospital, Clermont-Ferrand
Sponsor class: OTHER
First posted: 2008-09-10
Enrollment: 30
Sex: ALL
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To assess the engraftment of hematopoietic stem cells following reduced-intensity conditioning in children presenting with solid tumors or hematological malignancy by evaluating post-transplantation chimerism and hematological reconstitution.

To study acute and/or chronic graft-versus-host reaction (incidence and severity) and immune reconstitution, post-transplantation To study the effectiveness of the protocol on tumor response.

To study overall survival.

Detailed description: Primary criterion

* Hematological reconstitution and chimerism post-transplantation

Secondary criteria

* Adverse effects, graft-related mortality at D90 and at 12 months, incidence and severity of acute and/or chronic GVH reactions
* Analysis of variations in post-transplantation immunological function profiles
* Median duration of response in therapy-responsive patients and median duration of stability in stabilized patients

Eligibility criteria: Inclusion Criteria:

* Patients aged less than 20 years old
* Lansky score \> 60%
* Life expectancy greater than 2 months
* Diagnoses:

  3- Solid tumor or hematological malignancy remaining unresponsive to the reference strategies according to French best practices in pediatrics.

  4- Malignancies for which allografting is the recognized indication but is contraindicated with myeloablative conditioning.
* Usual criteria for allogeneic grafting (pre-graft profile)
* Having a HLA-identical sibling donor for HLA-A, HLA-B and HLA-DR antigens or a HLA mismatch on only one antigen, or having a 10/10 pheno-identical donor, or compatible cord blood.
* Signed informed consent

Exclusion Criteria:

* Patient presenting rapidly-progressive malignancy
* In cases where the potential donor is related, sibling presenting contraindication against hematopoietic stem cell donation
* Unable to sufficiently understand the treatment and its consequences, even after explanation

Primary outcomes:
measure: Primary criterion - Hematological reconstitution and chimerism post-transplantation
timeFrame: post-transplantation
Secondary outcomes:
measure: Secondary criteria - Adverse effects, graft-related mortality at D90 and at 12 months, incidence and severity of acute and/or chronic GVH reactions
timeFrame: post-transplantation
Other outcomes:
Locations:
city: Clermont-Ferrand
country: France
facility: Paillard Catherine
Study officials:
affiliation: University Hospital, Clermont-Ferrand
name: Paillard Catherine, Dr
role: PRINCIPAL_INVESTIGATOR
References: