Combination Chemotherapy Followed by Stem Cell Transplant in High-risk Neuroblastoma Patients (NB2004-HR)

University of Cologne

Status and phase: Completed; Phase 3

Conditions: Neuroblastoma

Treatments: Drug: Cycles N8, N5 and N6; Drug: Cycles N5 and N6

Study type: Interventional

Funder types: Other

Identifiers

NCT03042429

UCologne

Details and patient eligibility

About

Improvement of event free survival of high-risk neuroblastoma patients by introduction of two additional topotecan containing chemotherapy cycles into the multimodal standard treatment (induction chemotherapy, myeloablative therapy, radiation, surgery as indicated, and consolidation therapy).

Full description

Experimental intervention (6 weeks + duration of the control intervention):

2 x N8 cycle (topotecan, cyclophosphamide, and etoposide) followed by standard arm treatment (i.e., control intervention)

Control intervention (total duration 70-76 weeks):

3 x N5 cycle (cisplatin, etoposide, and vindesine) 3 x N6 cycle (vincristine, dacarbacine, ifosfamide, and doxorubicine), myeloablative chemotherapy with autologous stem cell transplantation (melphalan, carboplatin, etoposide) 9 x retinoic acid cycles (6 months, 3 months break, 3 months) supportive care (PCP/fungal prophylaxis, transfusions, antibiotics, G-CSF)

Enrollment: 360 patients

Sex: All

Ages: 6 months to 21 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• neuroblastoma
• stage 4 neuroblastoma and age ≥1 - 21 years or MYCN amplification and age ≥6 months - 21 years
• informed consent obtained

Exclusion criteria

• Participation in other trials
• Pregnancy, lactation, or insufficient contraception for girls in childbearing age,
• Any concomitant non-protocol anticancer therapy,
• Incomplete initial staging.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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360 participants in 2 patient groups

experimental arm

Experimental group

Description:

Drug: Cycles N8, N5, and N6 Drug: topotecan, cyclophosphamide, and etoposide (N8 cycle) followed by Drug: cisplatin, etoposide, and vindesine (N5 cycle) and Drug: vincristine, dacarbacine, ifosfamide, and doxorubicine (N6 cycle) followed by myeloablative chemotherapy with autologous stem cell transplantation (melphalan, carboplatin, etoposide) and by 9 x retinoic acid cycles (6 months, 3 months break, 3 months)

Treatment:

Drug: Cycles N8, N5 and N6

standard arm

Active Comparator group

Description:

Drug: Cycles N5 and N6 Drug: cisplatin, etoposide, and vindesine (N5 cycle) and Drug: vincristine, dacarbacine, ifosfamide, and doxorubicine (N6 cycle) followed by myeloablative chemotherapy with autologous stem cell transplantation (melphalan, carboplatin, etoposide) and by 9 x retinoic acid cycles (6 months, 3 months break, 3 months)

Treatment:

Drug: Cycles N5 and N6