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A Prospective, Single-Arm, 2-Stage, Open-Label, Phase II Trial of Azacitidine in Relapsed and Refractory Multiple Myeloma.

Phase 2
17 Years
Open (Enrolling)
Multiple Myeloma

Thank you

Trial Information

A Prospective, Single-Arm, 2-Stage, Open-Label, Phase II Trial of Azacitidine in Relapsed and Refractory Multiple Myeloma.

Multiple myeloma (MM) is an incurable disease with an annual incidence of 14,000 new cases
in the US alone. Despite initial sensitivity to corticosteroids, chemotherapy and
radiotherapy, relapse is inevitable and there is a median survival of only 2.5 to 3 years.
The use of autologous stem cell transplantation (SCT) has improved the duration of disease
remission for younger patients but still only results in a median survival of 5 – 6 years.

Since the early 1970s, azacitidine has been investigated for the treatment of acute
leukemia. More recently it has been investigated in the treatment of patients with
myelodysplastic syndrome (a pre-leukaemic condition). It has been shown to prolong the time
to development of acute myeloid leukaemia (AML) or death and has now been approved for use
in these patients.

Azacitidine is a cytotoxic drug and is directly toxic to cells, preventing their
reproduction or growth. It is also able to cause cells to undergo the process whereby they
mature into normal cells. The Myeloma Research Group at The Alfred Hospital has looked at
the effect of azacitidine on human myeloma cell lines in the laboratory. Azacitidine was
found to prevent both cell growth and causes cell death. In mouse models with multiple
myeloma azacitidine prolonged their survival.

The primary aim of this study is to determine the effectiveness of azacitidine in treating
patients with multiple myeloma. The other aims of this study are to see whether treating
patients with azacitidine extends the time that their myeloma is under control, to determine
the number of cycles of azacitidine required to first achieve a response and to determine
how safe and tolerable azacitidine is in treating multiple myeloma.

In the first stage a total of 14 people will participate in this project. If in this group
of patients azacitidine is shown to be effective as a treatment against multiple myeloma
then a further 10 patients will be invited to participate.

Inclusion Criteria:

- diagnosis of MM as per IMWG criteria

- age greater than 17 years

- have received at least 2 but no more than 4 prior lines of therapy

- have failed to respond to the most recently administered anti-MM therapy or have
demonstrably progressive disease as defined by accepted standard criteria

- have a life expectancy of at least 3 months

- ECOG performance status < 3

- at registration haematological values within the following limits:

1. absolute neutrophil count (ANC) > 1.0 x 109/L

2. platelet count > 50 x 109/L unsupported

- At registration biochemical values within the following limits

1. Bilirubin < 1.5 x upper limit of normal (ULN) and transaminases < 2 x ULN unless
considered secondary to hepatic myelomatous infiltration

2. Serum creatinine < 0.19mMol/L

- Written informed consent

- Must agree to use adequate contraceptive measures if indicated. Specifically, women
of childbearing potential (WOCBP) may participate provided they meet the following

1. Agree to use at least 2 effective contraceptive methods throughout the study and
for 30 days following the study

2. Have a negative serum pregnancy test within 24 hours of commencing on study

3. Male participants with female partners that are WOCBP must agree to use 2
effective contraceptive methods throughout the study and for 30 days following
the study

Exclusion Criteria:

- Patients with monoclonal gammopathy of undetermined significance (MGUS) or
indolent/smouldering MM

- Known or suspected hypersensitivity to AZA or mannitol

- Patients whose general condition makes them unsuitable for intensive treatment e.g.
significant cardiac or pulmonary disease

- Active infections or other illnesses that precludes chemotherapy administration or
patient compliance

- Active viral infection with known human immunodeficiency virus (HIV) or viral
hepatitis type B or C

- Pregnant or lactating women

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall response rate

Principal Investigator

Andrew Spencer, Assoc. Prof

Investigator Role:

Study Chair


Australia: Department of Health and Ageing Therapeutic Goods Administration

Study ID:




Start Date:

December 2006

Completion Date:

Related Keywords:

  • Multiple Myeloma
  • Myeloma
  • Relapsed
  • Refractory
  • Multiple Myeloma
  • Neoplasms, Plasma Cell