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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00064974




Registration number
NCT00064974
Ethics application status
Date submitted
16/07/2003
Date registered
17/07/2003
Date last updated
4/04/2013

Titles & IDs
Public title
Efficacy and Safety Study of CC-5013 Monotherapy in Subjects With Myelodysplastic Syndromes
Scientific title
A Multicenter, Single-Arm, Open-Label Study of the Efficacy and Safety of CC-5013 Monotherapy in Subjects With Myelodysplastic Syndromes
Secondary ID [1] 0 0
CC-5013-MDS-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - CC-5013

Experimental: CC-5013 - CC-5013 10 mg (two 5 mg capsules) daily on days 1-28 every 28 days (28 day cycles)


Treatment: Drugs: CC-5013
CC-5013 10 mg (two 5 mg capsules) daily on days 1-28 every 28 days (28 day cycles)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
RBC Transfusion Independence
Timepoint [1] 0 0
Secondary outcome [1] 0 0
= 50% decrease in RBC transfusion requirement
Timepoint [1] 0 0
Secondary outcome [2] 0 0
Platelet Response
Timepoint [2] 0 0
Secondary outcome [3] 0 0
Neutrophil Response
Timepoint [3] 0 0
Secondary outcome [4] 0 0
Bone marrow Response
Timepoint [4] 0 0
Secondary outcome [5] 0 0
Duration of Response
Timepoint [5] 0 0
Secondary outcome [6] 0 0
Hemoglobin concentration
Timepoint [6] 0 0
Secondary outcome [7] 0 0
Number of Participants with Adverse Event
Timepoint [7] 0 0

Eligibility
Key inclusion criteria
- Must understand and voluntarily sign an informed consent form.

- Age = 18 years at the time of signing the informed consent form.

- Must be able to adhere to the study visit schedule and other protocol requirements.

- Diagnosis of low - or intermediate-1-risk IPSS (Appendix III) MDS without an
abnormality of chromosome 5 involving a deletion between bands q31 and q33.

- Red blood cell (RBC) transfusion-dependent anemia defined as having received = to 2
units of RBCs within 8 weeks of the first day of study drug treatment.

- Eastern Cooperative Oncology Group (ECOG) (Appendix IV) performance status score of 0,
1, or 2.

- Women of childbearing potential (WCBP) must have a negative serum or urine pregnancy
test within 7 days of starting study drug.

- Sexually active WCBP must agree to use adequate contraceptive methods (oral,
injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine
device; barrier contraceptive with spermicide; or vasectomized partner) while on study
drug.

- WCBP must agree to have pregnancy tests every 4 weeks while on study drug.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Pregnant or lactating females.

- Prior therapy with lenalidomide.

- An abnormality of chromosome 5 involving a deletion between bands q31 and q33.

- Lab Abnormality: Absolute neutrophil count (ANC) <500 cells/mm3 (0.5 x 109/L)

- Lab Abnormality: Platelet count <50,000/mm3 (50 x 109/L)

- Lab Abnormality: Serum creatinine >2.5 mg/dL (221 mmol/L)

- Lab Abnormality: Serum glutamic oxaloacetic transaminase/Aspartate transaminase
(SGOT/AST) or Serum glutamic pyruvic transaminase/Alanine transaminase (SGPT/ALT) >3.0
x upper limit of normal (ULN)

- Lab Abnormality: Serum total bilirubin >2.0 mg/dL (34 mmol/L)

- Prior = grade 3 National Cancer Institute (NCI) Common Toxicity Criteria (CTC)
(Appendix VI) allergic reaction/hypersensitivity to thalidomide.

- Prior = grade 3 NCI CTC (Appendix VI) rash or any desquamation (blistering) while
taking thalidomide.

- Clinically significant anemia due to factors such as iron, B12 or folate deficiencies,
autoimmune or hereditary hemolysis or gastrointestinal bleeding

- If a marrow aspirate is not evaluable for storage iron, transferrin saturation must be
> 20 % and serum ferritin not less than 50 ng/mL.

- Use of hematopoietic growth factors within 7 days of the first day of study drug
treatment.

- Chronic use (>2 weeks) of greater than physiologic doses of a corticosteroid agent
(dose equivalent to >10 mg/day of prednisone) within 28 days of the first day of study
drug treatment.

- Use of experimental or standard drugs (i.e. chemotherapeutic, immunosuppressive, and
cytoprotective agents) for the treatment of MDS within 28 days of the first day of
study drug treatment.

- Prior history of malignancy other than MDS (except basal cell or squamous cell
carcinoma or carcinoma in situ of the cervix or breast) unless the subject has been
free of disease for greater than or equal to 3 years.

- Use of any other experimental therapy within 28 days of the first day of study drug
treatment.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/06/2003
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/02/2007
Sample size
Target
Accrual to date
Final
215
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - SA Pathology Haematology - Adelaide
Recruitment hospital [2] 0 0
Princess Alexandra Hospital - Haematology - Brisbane
Recruitment hospital [3] 0 0
Royal Prince Alfred Hospital - Institute of Haematology - Camperdown
Recruitment hospital [4] 0 0
Peter McCallum Cancer Institute - Directorate of Cancer Medecine - East Melbourne
Recruitment hospital [5] 0 0
Frankston Hospital-peninsula Health - Oncology Day Unit - Frankston
Recruitment hospital [6] 0 0
The Alfred Hospital - malignant haematology & stem cell transplantation - Melbourne
Recruitment hospital [7] 0 0
Calvary Mater Newcastle - Haematology - Waratah
Recruitment hospital [8] 0 0
Border Medical Oncology - Wodonga
Recruitment hospital [9] 0 0
Wollongong Hospital - Haematology - Wollongong
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
4102 - Brisbane
Recruitment postcode(s) [3] 0 0
2050 - Camperdown
Recruitment postcode(s) [4] 0 0
3002 - East Melbourne
Recruitment postcode(s) [5] 0 0
3199 - Frankston
Recruitment postcode(s) [6] 0 0
3004 - Melbourne
Recruitment postcode(s) [7] 0 0
2298 - Waratah
Recruitment postcode(s) [8] 0 0
3690 - Wodonga
Recruitment postcode(s) [9] 0 0
2500 - Wollongong
Recruitment outside Australia
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United States of America
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Arizona
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California
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Oregon
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Pennsylvania
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Texas
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Washington
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Gent
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Yvoir
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Hradec Kralove
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Prague
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Aalborg
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Ulm
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Padova
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Piacenza
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Reggio Emilia
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Roma
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Torino
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Amsterdam
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Santander
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Goteborg
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Stockholm
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Genève
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Zurich
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Bournemouth
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Leeds
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London
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Newcastle Upon Tyne
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Nottingham
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Plymouth
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Sheffield
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Surrey
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United Kingdom
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Wolverhampton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Celgene Corporation
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study is a multi-center, single-arm, open-label study of oral CC-5013 monotherapy
administered at a dose of 10 mg daily on Days 1-21 every 28 days (28-day cycles) to red blood
cell (RBC) transfusion-dependent subjects with low- or intermediate-1-risk MDS who do not
have a del (5q31-33) cytogenetic abnormality. Screening procedures will take place within 28
days of first day of study drug treatment. Subjects will receive study drug (CC-5013) in
28-day cycles for up to 6 cycles, or until bone marrow disease progression or
progression/relapse following erythroid hematologic improvement (Appendix I) is documented.
Study visits will occur every cycle (every 28 days) and laboratory monitoring to assess
hematological parameters will occur every 14 days. Safety and efficacy assessments to be
performed during the study are outlined in the Schedule of Study Assessments.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00064974
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Robert Knight, MD
Address 0 0
Celgene Corporation
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00064974