Study to Evaluate Imetelstat (JNJ-63935937) in Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS).

• Register: EUCTR
• Last refreshed on: 7 December 2015
• Main ID: EUCTR2015-002874-19-ES
• Date of registration: 05/10/2015
• Prospective Registration: Yes
• Primary sponsor: Janssen-Cilag International NV
• Public title: Study to Evaluate Imetelstat (JNJ-63935937) in Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS).
• Scientific title: A Study to Evaluate Imetelstat (JNJ-63935937) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment
• Date of first enrolment: 24/11/2015
• Target sample size: 200
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-002874-19
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1

Countries of recruitment

Belgium, Brazil, France, Germany, Italy, Korea, Republic of, Mexico, Netherlands, Russian Federation, Spain, Switzerland, United Kingdom, United States

Contacts

• Name: Global Clinical Operations Spain
• Affiliation: Janssen-Cilag S.A.

• Name: Global Clinical Operations Spain
• Affiliation: Janssen-Cilag S.A.

Key inclusion & exclusion criteria

Inclusion criteria:
- Man or woman greater than or equal to (>=) 18 years of age
- Diagnosis of myelodysplastic syndrome (MDS) according to WHO criteria or French-American-British (FAB) classification confirmed by bone marrow aspirate and biopsy within 12 weeks prior to Study Entry. A local laboratory report from this diagnostic bone marrow aspirate and biopsy must be reviewed and approved by the sponsor
- International Prognostic Scoring System (IPSS) low Risk or intermediate-1 risk MDS
- Red blood cell (RBC) transfusion dependent, defined as requiring 4 units RBC over 8 weeks during the 12 weeks prior to Study Entry; pre-transfusion hemoglobin (Hb) should be less than equal to 9.0 gram per deciliter (g/dL) to count towards the 4 units total
- Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2
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 60
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 140

Exclusion criteria:
- Participant has known allergies, hypersensitivity, or intolerance to imetelstat or its excipients
- Participant has received an investigational drug or used an invasive investigational medicaldevice within 30 days prior to Study Entry or is currently enrolled in an investigational study
- Prior treatment with imetelstat
- Have received any chemotherapy, immunomodulatory or immunosuppressive therapy, corticosteroids greater than 30 milligram per day prednisone or equivalent, or growth factor treatment within 28 days prior to study entry
- Have received other treatments for MDS within 4 weeks prior to Study Entry


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Cancer [C04]

Myelodysplastic syndrome (MDS)
MedDRA version: 18.0 Level: HLT Classification code 10028536 Term: Myelodysplastic syndromes System Organ Class: 100000004851

Intervention(s)

Product Name: Imetelstat sodium
Product Code: JNJ-63935937
Pharmaceutical Form: Lyophilisate for solution for infusion
INN or Proposed INN: Imetelstat sodium
CAS Number: 1007380-31-5
Current Sponsor code: JNJ-63935937
Other descriptive name: IMETELSTAT SODIUM
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 210-

Primary Outcome(s)

Main Objective: Part 1: To evaluate the efficacy and safety of imetelstat in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment.

Part 2: To compare the efficacy, in terms of RBC TI, of imetelstat to placebo in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment.

Timepoint(s) of evaluation of this end point: 8 weeks

Secondary Objective: - To assess the safety of imetelstat in subjects with MDS
- To assess the time to RBC TI and duration of RBC TI
- To assess the rate of hematologic improvement
- To assess the rates of CR or PR
- To assess OS
- To assess time to progression to AML
- To assess the rate and amount of supportive care, including transfusions and myeloid growth factors
- To evaluate the pharmacokinetics and immunogenicity of imetelstat in subjects with MDS
- To assess the effect of imetelstat treatment on patient reported outcomes (PROs)
- To assess the effect of treatment on medical resource utilization

Primary end point(s): Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 8 week period.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1/ up to follow-up (30 days posttreatment [approximately 2 years])
2-12/ up to 2 years after enrollment of the last participant
13-14/ During treatment (approximately 2 years)
15-16/ up to 2 years after enrollment of the last participant

Secondary end point(s): 1/ Number of Participants with Adverse Events (AEs)
2/ Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 24 week period
3/ Time to the 8-week RBC transfusion independence (TI)
4/ Duration of RBC TI
5/ Percentage of Participants with hematologic improvement
6/ Percentage of Participants with Complete remission (CR) or Partial remission (PR) as Per International Working Group (IWG) Response CriteriaI 2006
7/ Overall survival
8/ Time to Progression to Acute Myeloid Leukemia
9/ Percentage of Participants with Transfusion
10/ Amount of Transfusions
11/ Percentage of Participants receiving any myeloid growth factors
12/ Change from baseline in Functional Assessment of Cancer Therapy -Anemia-Related Effects (FACT-An) Score and EuroQol-EQ-5D-5L ESA (EQ-5D-5L) Score
13/ Maximum Observed Plasma Concentration (Cmax)
14/ Area under the drug concentrationplasma time curve from time zero to last measurable concentration (AUC0-t)
15/ Percentage of Participants with antibodies to imetelstat
16/ Medical resource utilization data

Secondary ID(s)

63935937MDS3001

Source(s) of Monetary Support

Janssen Research & Development, LLC