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Study to Evaluate Imetelstat (JNJ-63935937) in Subjects With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2017 by Janssen Research & Development, LLC
Sponsor:
Information provided by (Responsible Party):
Janssen Research & Development, LLC
ClinicalTrials.gov Identifier:
NCT02598661
First received: October 27, 2015
Last updated: April 6, 2017
Last verified: April 2017
  Purpose
The purpose of this study is to evaluate the efficacy and safety of imetelstat in transfusion dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment.

Condition Intervention Phase
Myelodysplastic Syndromes
Drug: Imetelstat
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Investigator
Primary Purpose: Treatment
Official 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

Resource links provided by NLM:


Further study details as provided by Janssen Research & Development, LLC:

Primary Outcome Measures:
  • Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 8 week period [ Time Frame: 8 weeks ]

Secondary Outcome Measures:
  • Number of Participants with Adverse Events (AEs) [ Time Frame: up to follow-up (30 days post-treatment [approximately 2 years]) ]
  • Percentage of participants without any red blood cell (RBC) transfusion during any consecutive 24 week period [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Time to the 8-week RBC transfusion independence (TI) [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Duration of RBC TI [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Percentage of Participants with hematologic improvement [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Percentage of Participants with Complete remission (CR) or Partial remission (PR) as Per International Working Group (IWG) Response Criteria 2006 [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Overall survival [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Time to Progression to Acute Myeloid Leukemia [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Percentage of Participants with Transfusion [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Amount of Transfusions [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Percentage of Participants receiving any myeloid growth factors [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Maximum Observed Plasma Concentration (Cmax) [ Time Frame: During treatment (approximately 2 years) ]
  • Area under the drug concentration-plasma time curve from time zero to last measurable concentration (AUC0-t) [ Time Frame: During treatment (approximately 2 years) ]
  • Percentage of Participants with antibodies to imetelstat [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Medical resource utilization data [ Time Frame: up to 2 years after enrollment of the last participant ]
  • Assessment of Functional Assessment of Cancer Therapy- Anemia-Related Effects (FACT-An) [ Time Frame: up to 2 years after enrollment of the last participant ]
    The Functional Assessment of Cancer Therapy Anemia (FACT-An), is included in order to provide an assessment of the subject's functional status, well-being, and symptoms over time.

  • Assessment of EuroQol 5 Dimension Questionnaire (EQ-5D-5L) [ Time Frame: up to 2 years after enrollment of the last participant ]
    The EQ-5D-5L is a generic measure of health status. EQ-5D-5L is a 5 item questionnaire that assesses 5 domains including mobility, self-care, usual activities, pain/discomfort and anxiety/depression plus a visual analog scale rating "health today" with anchors ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).

  • Assessment of Quality of Life in Myelodysplasia Scale (QUALMS) [ Time Frame: up to 2 years after enrollment of the last participant ]
    The QUALMS is a 38-item measure that assesses health-related quality of life for patients with MDS. Thirty-three items are used to calculate the total score, as well as the 14 item physical burden (QUALMS-P), 3-item benefit-finding (QUALMS-BF), and 11-item emotional burden (QUALMS-E) subscales.

  • Assessment of Participant Global Impression of Change (PGIC) [ Time Frame: up to 2 years after enrollment of the last participant ]
    The Participant Global Impression of Change (PGIC) is a single-item questionnaire designed to provide an overall assessment of treatment from the participant's perspective since the start of the study. It is measured on a 7-point scale, where 1=very much improved and 7=very much worse. A participant is considered a responder if they have a response of "very much improved" or "much improved".


Estimated Enrollment: 200
Actual Study Start Date: November 24, 2015
Estimated Study Completion Date: May 20, 2020
Estimated Primary Completion Date: April 17, 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Part 1: Imetelstat
Imetelstat will be administered at a starting dose of 7.5 milligram per kilogram (mg/kg) given intravenously every 4 weeks, until disease progression, unacceptable toxicity, or withdrawal of consent, or lack of response.
Drug: Imetelstat
Imetelstat will be administered at a starting dose of 7.5 milligram per kilogram (mg/kg) given intravenously every 4 weeks, until disease progression, unacceptable toxicity, or withdrawal of consent, or lack of response.
Experimental: Part 2: Imetelstat
Imetelstat will be administered at a starting dose of 7.5 milligram per kilogram (mg/kg) given intravenously every 4 weeks, until disease progression, unacceptable toxicity, or withdrawal of consent, or lack of response.
Drug: Imetelstat
Imetelstat will be administered at a starting dose of 7.5 milligram per kilogram (mg/kg) given intravenously every 4 weeks, until disease progression, unacceptable toxicity, or withdrawal of consent, or lack of response.
Placebo Comparator: Part 2: Placebo
Matching Placebo to Imetelstat will be administered.
Drug: Placebo
Matching Placebo to Imetelstat will be administered.

Detailed Description:
This is a Phase 2/3, multicenter study of imetelstat that consists of 2 parts. Part 1 is an open‐label, single-arm design to assess the efficacy and safety of imetelstat. Up to 30 participants will be enrolled and followed for safety, hematologic improvement and reduction in transfusion requirement. Part 2 of the study will be initiated if data from Part 1 are supportive of a satisfactory benefit/risk profile. Part 2 is a double‐blind, randomized design to compare the efficacy of imetelstat with placebo. Approximately 170 participants will be randomized in a 2:1 ratio to receive either imetelstat or placebo, respectively. Each part of the study will consist of 3 phases: a Screening phase (up to 28 days); a treatment phase; and a post-treatment follow-up phase which will continue until death, lost to follow-up, withdrawal of consent, or the End of the Study (whichever occurs first). The End of the Study is defined as 2 years after the study entry of the last participant or anytime the sponsor terminates the study, whichever comes first.
  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Man or woman greater than or equal to (>=) 18 years of age
  • Diagnosis of myelodysplastic syndrome (MDS) according to World Health Organization (WHO) criteria 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 at least 4 RBC units transfused over an 8-week period during the 16 weeks prior to Study Entry; pre-transfusion hemoglobin (Hb) should be less than or 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

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 medical device 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02598661

Contacts
Contact: Use link at the bottom of the page to see if you qualify for an enrolling site (see list). If you still have questions: JNJ.CT@sylogent.com

  Show 85 Study Locations
Sponsors and Collaborators
Janssen Research & Development, LLC
Investigators
Study Director: Janssen Research & Development, LLC Clinical Trial Janssen Research & Development, LLC
  More Information

Additional Information:
Responsible Party: Janssen Research & Development, LLC
ClinicalTrials.gov Identifier: NCT02598661     History of Changes
Other Study ID Numbers: CR107947
63935937MDS3001 ( Other Identifier: Janssen Research & Development, LLC )
2015-002874-19 ( EudraCT Number )
Study First Received: October 27, 2015
Last Updated: April 6, 2017

Studies a U.S. FDA-regulated Drug Product: Yes

Keywords provided by Janssen Research & Development, LLC:
Myelodysplastic Syndromes
Imetelstat

Additional relevant MeSH terms:
Syndrome
Myelodysplastic Syndromes
Preleukemia
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Hematinics
Niacinamide
Vitamin B Complex
Vitamins
Micronutrients
Growth Substances
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 24, 2017