In this study, plerixafor, the study drug, will be given in addition to GCSF to see if it works the same way in children as in adults.
ID
Source
Brief title
Condition
- Other condition
Synonym
Health condition
solide tumoren
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary efficacy endpoint will be the difference between the 2 treatment
arms in the proportion of patients achieving at least a doubling of peripheral
blood CD34+ count from the morning of the day preceding the first apheresis day
to the morning prior to apheresis.
Secondary outcome
• Number of days of apheresis required to reach >=2 × 106 CD34+ cells/kg
• CD34+ yield for each apheresis
• Total CD34+ yield
• Percentage of patients proceeding to transplant
• Percentage of patients successfully engrafting
• Percentage of patients with durable engraftment at 3, 6, 12 and 24 months
post-transplant
Background summary
The purpose of this study is to find out if the drug, plerixafor, is safe and
effective in children.
Plerixafor is a drug that has already been tested and approved for use in
adults. It is used to help collect blood stem cells for transplantation (after
chemotherapy) in patients with cancer. Blood stem cells are the original cells
from which all of the other blood cells develop. They are made in the bone
marrow (the spongy structure in the centre of large bones).
Study objective
In this study, plerixafor, the study drug, will be given in addition to GCSF to
see if it works the same way in children as in adults.
Study design
This is a open-label, comparative, randomised trial
Intervention
Group 1: standard therapy with GCSF and plerixafor
Group 2: standard therapy with GCSF
In a ratio of 2:1
Study burden and risks
The side effects listed below are seen in adults taking part in clinical trials
of plerixafor. It is likely that similar side effects may be seen in children
but we cannot be certain.
Very common side effects seen in more than 1 in 10 patients:
Diarrhoea, nausea (feeling sick), injection site redness or irritation
Common side effects seen in between 1 and 10 of every 100 patients:
Headache, dizziness, feeling tired or unwell, difficulty in sleeping,
flatulence, constipation, indigestion, vomiting, stomach symptoms such as pain,
swelling or discomfort, dry mouth, numbness around the mouth, sweating,
generalized redness of the skin, joint pains, pains in muscles and bones.
Uncommon side effects seen in between 1 and 10 of every 1,000 patients:
Allergic reactions such as skin rash, swelling around the eyes, shortness of
breath and loss of consciousness.
Kampenringweg 45E
GOUDA 2803PE
NL
Kampenringweg 45E
GOUDA 2803PE
NL
Listed location countries
Age
Inclusion criteria
1. Age 1 to < 18 years
2. Ewing*s sarcoma, soft tissue sarcoma, lymphoma, neuroblastoma, or other malignancy including brain tumours (excluding any form of leukaemia) requiring treatment with high dose chemotherapy and autologous transplant as rescue therapy
3. Eligible for autologous transplantation
4. Recovered from all acute significant toxic effects of prior chemotherapy
5. Adequate performance status
- for patients >=16 years of age, defined as Karnofsky score >60
- for patients <16 years of age, defined as Lansky score > 60
6. Absolute neutrophil count >0.75 × 10P9/L
7. Platelet count > 50 × 10P9/L
8. Calculated creatinine clearance (using the Schwartz method):
- during study Stage 2, >;60 mL/min/1.73mP2
9. Liver functions < 3 × upper limit of normal
10. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent
Exclusion criteria
1. Any form of leukaemia
2. A co-morbid condition, such as ventricular arrhythmias, which, in the view of the Investigator, renders the patient at high-risk from treatment complications
3. Previous stem cell transplantation
4. Patients with persistent high percentage marrow involvement prior to mobilisation will be prohibited.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2010-019340-40-NL |
| ClinicalTrials.gov | NCT01288573 |
| CCMO | NL47333.078.13 |