Back to resultsA Two-part Single- and Repeat-dose Study of CSJ137 in Anemic Chronic Hemodialysis Patients
Primary Purpose
Anemia, Iron-Deficiency
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
CSJ137
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Anemia, Iron-Deficiency focused on measuring Anemia
Eligibility Criteria
Inclusion Criteria:
- Hemodialysis-dependent for at least 2 months prior to screening.
- Receiving hemodialysis at least 2 times per week
- Receiving erythropoietin (EPO) therapy.
- Hemoglobin (Hgb) ≥ 8.5 and < 11.5 g/dL at screening.
- Ferritin >500 ng/mL and ≤ 2000 ng/mL at screening.
- TSAT ≤ 50% at a minimum of one time point during the 90 days prior to baseline.
Exclusion Criteria:
- Known diagnosis of hemochromatosis, bone marrow malignancy, lymphatic malignancy or myelodysplastic syndrome.
- History of dialysis AV fistula thrombosis within 2 months prior to screening, or 2 or more episodes of AV fistula thrombosis within 6 months prior to screening.
- Liver disease/dysfunction (Child-Pugh score ≥ 6), prior liver transplant, heart failure (NYHA Class III or IV); gastrointestinal bleeding.
- A positive Hepatitis B surface antigen test result. Patients with Hepatitis C Virus (HCV) infection may be included if all other liver function eligibility criteria are met.
- ALT, AST or bilirubin ≥ 1.5x ULN within 4 weeks prior to baseline.
- Uncontrolled renal osteodystrophy
- Conditions predisposing to an increased risk of serious infection, such as an indwelling vascular catheter (central venous line or non-tunneled/acute hemodialysis catheter) or active infection requiring antibiotic therapy at any time during the 2 weeks prior to screening. Tunneled hemodialysis catheters, and other "permanent" catheters are permitted.
- Blood transfusion administered within 4 weeks prior to baseline.
- Patients who received CSJ137 dose in the past.
Other protocol-defined inclusion/exclusion criteria may apply.
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
CSJ137
Placebo
Arm Description
In Part 1 up to 48 subjects will receive a single dose of CSJ137. In Part 2, up to 40 patients will be randomized to one of 2 arms with equal allocation: one CSJ137 dose arm from Part 1 and a placebo arm. Each patient in Part 2 will receive up to 2 doses (repeat dose).
In Part 2, up to 40 patients will be randomized to one of 2 arms with equal allocation: one CSJ137 dose arm from Part 1 and a placebo arm. Each patient in Part 2 will receive up to 2 doses (repeat dose).
Outcomes
Primary Outcome Measures
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
safety and tolerability following administration of CSJ137
Minimum active dose of CSJ137 determined by assessment of levels of hemoglobin in blood, without evidence of liver dysfunction or other safety concerns.
to determine the minimum dose of CSJ137 that is active for treatment
Secondary Outcome Measures
Peak concentration (Cmax) of CSJ137 in serum
to assess the concentration of CSJ137 in the body over time
Area under the serum concentration versus time curve (AUC)
to assess the concentration of CSJ137 in the body over time
Full Information
NCT ID
NCT02570854
First Posted
September 29, 2015
Last Updated
October 7, 2021
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02570854
Brief Title
A Two-part Single- and Repeat-dose Study of CSJ137 in Anemic Chronic Hemodialysis Patients
Official Title
A First-in-human, Two-part (Open Label, and Randomized/Double Blind/Placebo Controlled), Single- and Repeat-dose Study of CSJ137 in Erythropoietin-treated Chronic Hemodialysis Patients With Functional Iron-deficiency Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
October 2021
Overall Recruitment Status
Completed
Study Start Date
September 22, 2015 (Actual)
Primary Completion Date
May 13, 2020 (Actual)
Study Completion Date
May 13, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The study will assess the safety, tolerability and efficacy of CSJ137 in chronic hemodialysis patients. It is hypothesized that treatment with CSJ137 may improve the level of hemoglobin in patients on chronic hemodialysis with iron-restricted anemia while reducing the need for dosing with erythropoietin and intravenous iron in these patients.
Detailed Description
This posting discloses information about Part 1 of CCSJ137X2201. Another part, Part 2, of this trial was planned for conduct subsequent to the initiation of Part 1 and would have a different design than Part 1. However, due to internal strategic non-safety related decision, Part 2 is not going to be conducted and the trial will be terminated after Part 1.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Anemia, Iron-Deficiency
Keywords
Anemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
40 (Actual)
8. Arms, Groups, and Interventions
Arm Title
CSJ137
Arm Type
Experimental
Arm Description
In Part 1 up to 48 subjects will receive a single dose of CSJ137. In Part 2, up to 40 patients will be randomized to one of 2 arms with equal allocation: one CSJ137 dose arm from Part 1 and a placebo arm. Each patient in Part 2 will receive up to 2 doses (repeat dose).
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
In Part 2, up to 40 patients will be randomized to one of 2 arms with equal allocation: one CSJ137 dose arm from Part 1 and a placebo arm. Each patient in Part 2 will receive up to 2 doses (repeat dose).
Intervention Type
Biological
Intervention Name(s)
CSJ137
Intervention Description
Starting dose is 0.010 mg/kg and dose escalation will proceed with semi-log increase steps to a maximum dose level 10 mg/kg. Subjects receive the treatment via up to 30 minutes intravenous infusion.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Subjects will be dosed with a matching placebo (vehicle control) via up to 30 minutes intravenous infusion.
Primary Outcome Measure Information:
Title
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Description
safety and tolerability following administration of CSJ137
Time Frame
baseline through 115 days after CSJ137 is administered
Title
Minimum active dose of CSJ137 determined by assessment of levels of hemoglobin in blood, without evidence of liver dysfunction or other safety concerns.
Description
to determine the minimum dose of CSJ137 that is active for treatment
Time Frame
Hemoglobin response at 28 days post-dose
Secondary Outcome Measure Information:
Title
Peak concentration (Cmax) of CSJ137 in serum
Description
to assess the concentration of CSJ137 in the body over time
Time Frame
before CSJ137 is administered, then 0.5 hours and 6 hours after CSJ137 is administered on Day 1. Also 1, 2, 3, 5, 12, 19, 28, and 84 days after CSJ137 is administered
Title
Area under the serum concentration versus time curve (AUC)
Description
to assess the concentration of CSJ137 in the body over time
Time Frame
before CSJ137 is administered, then 0.5 hours and 6 hours after CSJ137 is administered on Day 1. Also 1, 2, 3, 5, 12, 19, 28, and 84 days after CSJ137 is administered
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Hemodialysis-dependent for at least 2 months prior to screening.
Receiving hemodialysis at least 2 times per week
Receiving erythropoietin (EPO) therapy.
Hemoglobin (Hgb) ≥ 8.5 and < 11.5 g/dL at screening.
Ferritin >500 ng/mL and ≤ 2000 ng/mL at screening.
TSAT ≤ 50% at a minimum of one time point during the 90 days prior to baseline.
Exclusion Criteria:
Known diagnosis of hemochromatosis, bone marrow malignancy, lymphatic malignancy or myelodysplastic syndrome.
History of dialysis AV fistula thrombosis within 2 months prior to screening, or 2 or more episodes of AV fistula thrombosis within 6 months prior to screening.
Liver disease/dysfunction (Child-Pugh score ≥ 6), prior liver transplant, heart failure (NYHA Class III or IV); gastrointestinal bleeding.
A positive Hepatitis B surface antigen test result. Patients with Hepatitis C Virus (HCV) infection may be included if all other liver function eligibility criteria are met.
ALT, AST or bilirubin ≥ 1.5x ULN within 4 weeks prior to baseline.
Uncontrolled renal osteodystrophy
Conditions predisposing to an increased risk of serious infection, such as an indwelling vascular catheter (central venous line or non-tunneled/acute hemodialysis catheter) or active infection requiring antibiotic therapy at any time during the 2 weeks prior to screening. Tunneled hemodialysis catheters, and other "permanent" catheters are permitted.
Blood transfusion administered within 4 weeks prior to baseline.
Patients who received CSJ137 dose in the past.
Other protocol-defined inclusion/exclusion criteria may apply.
Facility Information:
Facility Name
Novartis Investigative Site
City
San Diego
State/Province
California
ZIP/Postal Code
91942
Country
United States
Facility Name
Novartis Investigative Site
City
Lakewood
State/Province
Colorado
ZIP/Postal Code
80228
Country
United States
Facility Name
Novartis Investigative Site
City
Orlando
State/Province
Florida
ZIP/Postal Code
32809
Country
United States
Facility Name
Novartis Investigative Site
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
Novartis Investigative Site
City
Chattanooga
State/Province
Tennessee
ZIP/Postal Code
37404
Country
United States
Facility Name
Novartis Investigative Site
City
Praha
ZIP/Postal Code
12808
Country
Czechia
Facility Name
Novartis Investigative Site
City
Ashkelon
ZIP/Postal Code
78278
Country
Israel
Facility Name
Novartis Investigative Site
City
Hadera
ZIP/Postal Code
38100
Country
Israel
Facility Name
Novartis Investigative Site
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Facility Name
Novartis Investigative Site
City
Petach Tikva
ZIP/Postal Code
49100
Country
Israel
Facility Name
Novartis Investigative Site
City
Ramat Gan
Country
Israel
Facility Name
Novartis Investigative Site
City
Tel Aviv
ZIP/Postal Code
62439
Country
Israel
Facility Name
Novartis Investigative Site
City
London
ZIP/Postal Code
SW17 0QT
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Undecided
IPD Sharing Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Links:
URL
http://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=17809
Description
Results for CCSJ137X2201 from the Novartis Clinical Trials Website
URL
https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=712
Description
A Plain Language Trial Summary is available on novartisclinicaltrials.com
Learn more about this trial
A Two-part Single- and Repeat-dose Study of CSJ137 in Anemic Chronic Hemodialysis Patients
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