Study to Evaluate Imetelstat (GRN163L) in Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS).
- Conditions
- Myelodysplastic syndrome (MDS)MedDRA version: 20.0Level: HLTClassification code 10028536Term: Myelodysplastic syndromesSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Cancer [C04]
- Registration Number
- EUCTR2015-002874-19-PL
- Lead Sponsor
- Geron Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 170
1. Man or woman =18 years of age (or the legal age of consent in the jurisdiction the study is taking place);
2. Criterion modified per Amendment 1.
2.1. Criterion modified per Amendment 2.
2.2. Criterion modified per Amendment 3
2. 3 In Part 1, diagnosis of MDS according to WHO criteria confirmed by bone marrow aspirate and biopsy within 12 weeks prior to C1D1. A local laboratory report from this diagnostic bone marrow aspirate and biopsy must be reviewed and approved by the sponsor (Attachment 1). In Part 2, diagnosis of MDS according to WHO criteria confirmed by bone marrow aspirate and biopsy within 12 weeks prior to Randomization (Attachment 1). A sample of the baseline bone marrow aspirate and biopsy must be submitted to the Independent Central Pathology Reviewer for diagnostic confirmation. Central laboratory review is required to confirm diagnosis prior to Randomization.
3. IPSS low or intermediate-1 risk MDS (Attachment 3);
4. Criterion modified per Amendment 1.
4.1. RBC transfusion dependent, defined as requiring at least 4 RBC units transfused over an 8-week period during the 16 weeks prior to C1D1 (Part 1) or Randomization (Part 2) (defined in Section 3.1); pre-transfusion Hb should be =9.0 g/dL to count towards the 4 units total;
5. Has MDS that is relapsed/refractory to ESA treatment; as defined by meeting any one of the criteria below:
5.1. Received at least 8 weeks of treatment with a minimum weekly dose of epoetin alfa 40,000 U, epoetin beta 30,000 U or darbepoetin alfa 150 mcg (or equivalent agent/dose), without having achieved a Hb rise =1.5 g/dL or decreased RBC transfusion requirement by at least 4 units over 8 weeks
5.2.1 Transfusion dependence or reduction in Hb by =1.5 g/dL after hematologic improvement from at least 8 weeks of treatment with therapies outlined in inclusion criteria 5.1, in the absence of another explanation.
5.4. Endogenous serum EPO level >500 mU/mL;
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More details see protocol
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 120
1. Subject has known allergies, hypersensitivity, or intolerance to imetelstat or its excipients (refer to the IB );
2.1 Subject has received an experimental or investigational drug or used an invasive investigational medical device within 30 days prior to C1D1 (Part 1) or Randomization (Part 2) (defined in Section 3.1) or is currently enrolled in an investigational study;
3. Prior treatment with imetelstat;
4. Criterion modified per Amendment 2. 4.1 Have received corticosteroids >30 mg/day prednisone or equivalent, or growth factor treatment within 4 weeks prior to C1D1 (Part 1) or Randomization (Part 2);
5. Criterion modified per Amendment 2. 5.1 Prior treatment with a hypomethylating agent (eg, azacitidine, decitabine);
5.2 Criterion 5.2 replaced by criterion 5.2.1 per Amendment 5.
5.2.1 Prior treatment with lenalidomide, thalidomide, or other thalidomide analogues;
5.3 Criterion 5.3 replaced by criterion 5.3.1 per Amendment 5.
5.3.1 Has received an ESA or any anti-MDS therapy, chemotherapy, immunomodulatory, or immunosuppressive therapy within 4 weeks prior to C1D1 (Part 1) or Randomization (Part 2) (8 weeks for long-acting ESAs);
6. Prior history of hematopoietic stem cell transplant;
7. Anemia attributed to factors other than MDS (including hemolysis, chronic renal failure, hepatitis, gastrointestinal bleeding);
8. Major surgery within 4 weeks prior to C1D1 (Part 1) or Randomization (Part 2)(excluding the placement of vascular access and other minor surgical procedures);
9. Diagnosed or treated for malignancy other than MDS, except:
9.1. Malignancy treated with curative intent and with no known active disease present for =3 years before C1D1 (Part 1) or Randomization (Part 2)
9.2. Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease
9.3. Adequately treated cervical carcinoma in situ without evidence of disease;
10. Clinically significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of C1D1 (Part 1) or Randomization (Part 2), or any Class 3 (moderate) or Class 4 (severe) cardiac disease as defined by the New York Heart Association Functional Classification;
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More details see protocol
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method