A Two-part Single- and Repeat-dose Study of CSJ137 in Anemic Chronic Hemodialysis Patients

Anemia, Iron-Deficiency Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02570854
• Other study ID #: CCSJ137X2201
• Secondary ID: 2017-002926-19
• Status: Completed
• Phase: Phase 1
• Start date: September 22, 2015
• Est. completion date: May 13, 2020

Study information

• Verified date: October 2021
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: May 13, 2020
• Est. primary completion date: May 13, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Hemodialysis-dependent for at least 2 months prior to screening.

2. Receiving hemodialysis at least 2 times per week

3. Receiving erythropoietin (EPO) therapy.

4. Hemoglobin (Hgb) = 8.5 and < 11.5 g/dL at screening.

5. Ferritin >500 ng/mL and = 2000 ng/mL at screening.

6. TSAT = 50% at a minimum of one time point during the 90 days prior to baseline.

Exclusion Criteria:

1. Known diagnosis of hemochromatosis, bone marrow malignancy, lymphatic malignancy or myelodysplastic syndrome.

2. 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.

3. Liver disease/dysfunction (Child-Pugh score = 6), prior liver transplant, heart failure (NYHA Class III or IV); gastrointestinal bleeding.

4. 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.

5. ALT, AST or bilirubin = 1.5x ULN within 4 weeks prior to baseline.

6. Uncontrolled renal osteodystrophy

7. 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.

8. Blood transfusion administered within 4 weeks prior to baseline.

9. Patients who received CSJ137 dose in the past. Other protocol-defined inclusion/exclusion criteria may apply.

Related Conditions & MeSH terms

• Anemia
• Anemia, Iron-Deficiency

Intervention

Biological:
• CSJ137
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.

Drug:
• Placebo
Subjects will be dosed with a matching placebo (vehicle control) via up to 30 minutes intravenous infusion.

Locations

Country	Name	City	State
Czechia	Novartis Investigative Site	Praha
Israel	Novartis Investigative Site	Ashkelon
Israel	Novartis Investigative Site	Hadera
Israel	Novartis Investigative Site	Jerusalem
Israel	Novartis Investigative Site	Petach Tikva
Israel	Novartis Investigative Site	Ramat Gan
Israel	Novartis Investigative Site	Tel Aviv
United Kingdom	Novartis Investigative Site	London
United States	Novartis Investigative Site	Chattanooga	Tennessee
United States	Novartis Investigative Site	Lakewood	Colorado
United States	Novartis Investigative Site	Minneapolis	Minnesota
United States	Novartis Investigative Site	Orlando	Florida
United States	Novartis Investigative Site	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Czechia,  Israel,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	safety and tolerability following administration of CSJ137	baseline through 115 days after CSJ137 is administered
Primary	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	Hemoglobin response at 28 days post-dose
Secondary	Peak concentration (Cmax) of CSJ137 in serum	to assess the concentration of CSJ137 in the body over time	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
Secondary	Area under the serum concentration versus time curve (AUC)	to assess the concentration of CSJ137 in the body over time	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

External Links

•   A Plain Language Trial Summary is available on novartisclinicaltrials.com
•   Results for CCSJ137X2201 from the Novartis Clinical Trials Website