Clinical Trial Result Information

Protocol number
ML17850

Title of Study
An open-label, multi-center study to investigate safety of once weekly NeoRecormon therapy in patients with refractory anemia.

Sponsor
Roche farmacevtska druzba d.o.o.

Company division
Pharmaceutical

Product name
NeoRecormon

Generic name
epoetin beta

Therapeutic area
Anemia

Clinical study summary
This was an open-label study of the effects of weekly administration of NeoRecormon (30 000 IU s.c.) in patients with refractory anemia resulting from myelodysplastic syndrome.

Study center(s)
6 centers in Slovenia.

Phase of development
II

Objectives
The objective of the study was to assess the efficacy and safety of once weekly administration of 30 000 IU of NeoRecormon in patients with myelodysplastic syndrome and with refractory anemia with or without ringed sideroblasts.

Methodology
Eligible patients were treated with weekly subcutaneous injections of NeoRecormon (30 000 IU in pre-filled syringes), in a hospital setting. The dose was to be increased to 60 000 IU of the hemoglobin level did not increase to more than 10g/L or if blood transfusion was required in the previous week, and was to be halved if the hemoglobin level increased to more than 20g/L after 4 weeks.

Number of patients (planned/analyzed)
Planned: 30; enrolled: 26.

Diagnosis and main criteria for inclusion
Cytologically or histologically confirmed refractory anemia, with or without ringed sideroblasts.

Test product, dose and mode of administration or test procedure
NeoRecormon (epoetin beta) 30 000 IU s.c. weekly.

Duration of treatment
12 weeks

Reference therapy, dose and mode of administration or reference procedure
N/A.

Criteria for evaluation (efficacy, safety)
Efficacy: hemoglobin, hematocrit, serum iron, serum erythropoietin, number of required transfusions. Safety: Adverse events and deaths, vital signs.

Statistical methods
All patients who completed the study were included in the efficacy analysis. Patients who received at least one dose of NeoRecormon, and for whom safety data were available for at least one visit, were included in the safety analysis.

Summary (efficacy, safety, other results)
Efficacy: There was no significant difference in hemoglobin values between baseline and week 12. The majority of patients (17/26) needed a NeoRecormon dose increase to 60 000 IU. No statistical correlation was found between hemoglobin response and exogenous epoetin or serum epoetin level.
Safety: The safety data from this small sample size did not indicate that use of the NeoRecormon 30 000 IU pre-filled syringe was associated with any safety issues. One patient withdrew from treatment due to adverse events considered probably related to treatment (malaise, pins and needles, rustling in the head, nose bleed) after the NeoRecormon dose had been increased to 60 000 IU. One patient died, due to disease progression.

Conclusions
Due to the limitations in study design and the small sample size, NeoRecormon was not shown to have a statistically significant effect on hemoglobin levels. The majority of patients had their dose increased to 60 000 IU, due to poor response. NeoRecormon administered by 30 000 IU pre-filled syringe was found to be well tolerated in patients with refractory anemia.

Date of report
10/28/2005

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