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Completed

NCT Number: NCT01196715

Comparison Study of Standard Care Against Combination of Growth Factors Agents for Low-risk Myelodysplastic Syndromes

REGIME is comparing two treatments, with Darbepoetin Alpha (DA) and Filgrastim (Granulocyte Colony Stimulating Factor, G-CSF), to the standard treatment for Myelodysplastic Syndrome (MDS).

After giving Informed Consent patients will undergo a number of tests to confirm eligibility. Once eligibility is confirmed patients will be randomly assigned to one of the three treatments group: A: Darbepoetin Alpha (DA), B: Darbepoetin Alpha and Filgrastim (DA+G-CSF), C: Blood transfusion only. Patients will be required to attend the clinic once a month for 24 weeks. After 24 weeks if a patient has reacted favorably to the treatment they may continue on the treatment regime up to 52 weeks. After week 24 all patients will be required to attend the clinic twice more, at week 36 and 52.

Patients will be followed for a further 5 years to record loss of response, transformation to Acute Myeloid Leukaemia and/or Refractory Anemia with Excess Blasts and death.

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Key information

Conditions

Myelodysplastic Syndrome Bone Marrow Diseases Disease Attributes Disease Progression Hematologic Diseases Hemic and Lymphatic Diseases Myelodysplastic Syndromes Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Birmingham Cancer Research UK Clinical Trial Unit, Birmingham, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Males and females aged over 18 years, (no upper age limit)
  • ECOG performance status 0-2
  • Life expectancy more than 6 months
  • A confirmed diagnosis of MDS - WHO type:
  • refractory anaemia (RA)
  • hypoplastic RA ineligible for/or failed immunosuppressive therapy (ALG, cyclosporine)
  • refractory anaemia with ring sideroblasts (RARS)
  • refractory cytopenia with multilineage dysplasia
  • myelodysplastic syndrome unclassifiable
  • IPSS low or Int-1, but with BM blasts less than 5%
  • A haemoglobin concentration of less than 10g/dl and/or red cell transfusion dependence
  • Able to understand the implications of participation in the Trial and give written informed consent.

Exclusion criteria

  • MDS with bone marrow blasts greater or equal than 5%
  • Myelodysplastic syndrome associated with del(5q)(q31-33) syndrome
  • Chronic myelomonocytic leukaemia (monocytes greater than1.0x109/l)
  • Therapy-related MDS
  • Splenomegaly, with spleen greater or equal than 5 cm from left costal margin
  • Platelets less than 30x109/l
  • Uncorrected haematinic deficiency. Patient deplete to iron, B12 and folate according to local lab ranges
  • Women who are pregnant or lactating.
  • Females of childbearing potential and all males must be willing to use an effective method of contraception (hormonal or barrier method of birth control; abstinence) for the duration of the study and for up to 3 months after the last dose of study medication. Note: Subjects are not considered of child bearing potential if they are surgically sterile (they have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are postmenopausal
  • Females of childbearing potential must have a negative pregnancy test prior to starting the study.
  • Uncontrolled hypertension, previous venous thromboembolism, or uncontrolled cardiac or pulmonary disease
  • Previous serious adverse events to the study medications or its components
  • Patients who have had previous therapy with ESAs ± G-CSF within 4 weeks of study entry
  • Patients currently receiving experimental therapy, e.g. with thalidomide, or who are participating in another CTIMP.
  • Medical or psychiatric illness, which makes the patient unsuitable or unable to give informed consent.
  • Patients with malignancy requiring active treatment (except hormonal therapy).
  • Patients with a history of seizures

Treatment and study plan

Darbepoetin alpha

Drug

Aranesp 500 mcg vials once every 2 weeks.

Other names: Aranesp

Filgrastim

Drug

300 mcg vials twice a week, 3-4 days apart

Other names: Neupogen

Blood Red Cell Transfusion

Procedure

Red cell transfusion support to achieve a predicted post-transfusion haemoglobin of 11.0 to 12.0 g/dl at a quantity and frequency such that the minimum haemoglobin is never below 8.0 g/dl or such that the patient is never excessively symptomatic, according to local transfusion guidelines/policy.

Other names: Blood transfusion.

Primary outcomes

  1. Quality of Life - To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: weeks 0

    To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  2. Haemoglobin response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 0

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  3. Quality of Life - To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: weeks 12

    To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  4. Quality of Life - To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: weeks 24

    To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  5. Quality of Life - To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: weeks 36

    To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  6. Quality of Life - To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: weeks 52

    To compare the Quality of Life of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  7. Haemoglobine response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 4

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  8. Haemoglobine response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 8

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  9. Haemoglobine response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 12

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  10. Haemoglobine response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 16

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  11. Haemoglobine response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 20

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  12. Haemoglobine response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 24

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  13. Haemoglobine response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 36

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

  14. Haemoglobine response - To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

    Time frame: week 52

    To compare the haemoglobin response of low risk Myelodysplastic Syndrome (MDS) patients randomised to receive prolonged treatment with DA alone, DA with G-CSF or best supportive care alone

Secondary outcomes

  1. Utility of prognostic factor and predictive factor assessment

    Time frame: every week

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

  2. Utility of prognostic factor and predictive factor assessment

    Time frame: week 4

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

  3. Utility of prognostic factor and predictive factor assessment

    Time frame: week 8

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

  4. Utility of prognostic factor and predictive factor assessment

    Time frame: week 12

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

  5. Utility of prognostic factor and predictive factor assessment

    Time frame: week 16

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

  6. Utility of prognostic factor and predictive factor assessment

    Time frame: week 20

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

  7. Utility of prognostic factor and predictive factor assessment

    Time frame: week 24

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

  8. Utility of prognostic factor and predictive factor assessment

    Time frame: week 36

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

  9. Utility of prognostic factor and predictive factor assessment

    Time frame: week 52

    To assess the utility of prognostic factor and predictive factor assessment, in particular against the predictive model proposed by Hellstrom-Lindberg.

Sponsors and collaborators

Lead sponsor

Barts & The London NHS Trust

Other

Collaborators

  • Amgen
  • Cancer Research UK

Registry information

Official study title

REGIME: A Randomised Controlled Trial of Prolonged Treatment With Darbepoetin Alpha, With or Without Recombinant Human Granulocyte Colony Stimulating Factor, Versus Best Supportive Care in Patients With Low-risk Myelodysplastic Syndromes (MDS).

Acronym: REGIME

Important dates

Study start
2010
Primary completion
2015
Study completion
2015
First posted
Sep 8, 2010
Registry last updated
Feb 6, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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