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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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13 November 2023 |
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Main ID: |
NCT02349906 |
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Date of registration:
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26/01/2015 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
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Scientific title:
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Clinical Phase II Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Paediatric Patients With Non-malignant Diseases |
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Date of first enrolment:
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April 2015 |
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Target sample size:
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106 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT02349906 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Austria
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Czech Republic
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Czechia
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Germany
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Italy
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Poland
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Contacts
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Name:
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Karl-Walter Sykora, MD and Prof |
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Address:
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Telephone:
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Email:
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Affiliation:
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Hannover Medical University |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Non-malignant disease indicated for first myeloablative allogeneic HSCT, including
inborn errors of metabolism, primary immunodeficiencies, haemoglobinopathies and bone
marrow failure syndromes.
2. First allogeneic HSCT.
3. Available matched sibling donor (MSD), matched family donor (MFD) or matched unrelated
donor (MUD). For bone marrow (BM) and peripheral blood (PB) match is defined as at
least 9/10 allele matches after four digit typing in human leucocyte antigen (HLA)-A,
-B, -C, -DRB1 and DQB1 antigens. For umbilical cord blood (UCB) match is defined as at
least 5/6 matches after two digit typing in HLA-A and -B and four digit typing in DRB1
antigens.
Exclusion Criteria:
1. Second or later HSCT.
2. HSCT from mismatched donor (less than 9/10 BM/peripheral blood stem cells (PBSC) or
less than 5/6 matched cord donor).
3. Preterm newborn infants (<37 weeks gestational age) and term newborn infants aged 0 -
27 days at time of registration.
4. Obese paediatric patients with body mass index weight (kg)/[height (m)]² > 30 kg/m².
5. Diagnosis of Fanconi anaemia and other chromosomal breakage disorders,
radiosensitivity disorders (deoxyribonucleic acid (DNA) Ligase 4, Cernunnos- X-ray
repair cross-complementing protein 4 (XRCC4) like factor (XLF), Nijmegen Breakage
Syndrome (NBS)) and Dyskeratosis Congenita.
Age minimum:
28 Days
Age maximum:
17 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Haemoglobinopathies
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Inborn Errors of Metabolism
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Bone Marrow Failure Syndromes
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Primary Immunodeficiencies
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Intervention(s)
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Drug: Busilvex
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Drug: Treosulfan
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Primary Outcome(s)
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Comparative evaluation of freedom from transplant (treatment)-related mortality (TRM), defined as death from any transplant-related cause from the day of first administration of study medication (day -7) until day +100 after HSCT.
[Time Frame: day -7 to day +100]
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Secondary ID(s)
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MC-FludT.16/NM
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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