Clinical trial NCT02570854: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Anemia, Iron-Deficiency
Keywords: Anemia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2015-10-07
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
timeFrame: baseline through 115 days after CSJ137 is administered
measure: Minimum active dose of CSJ137 determined by assessment of levels of hemoglobin in blood, without evidence of liver dysfunction or other safety concerns.
timeFrame: Hemoglobin response at 28 days post-dose
Secondary outcomes:
measure: Peak concentration (Cmax) of CSJ137 in serum
timeFrame: 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
measure: Area under the serum concentration versus time curve (AUC)
timeFrame: 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
Other outcomes:
Locations:
city: San Diego
country: United States
facility: Novartis Investigative Site
state: California
city: Lakewood
country: United States
facility: Novartis Investigative Site
state: Colorado
city: Orlando
country: United States
facility: Novartis Investigative Site
state: Florida
city: Minneapolis
country: United States
facility: Novartis Investigative Site
state: Minnesota
city: Chattanooga
country: United States
facility: Novartis Investigative Site
state: Tennessee
city: Praha
country: Czechia
facility: Novartis Investigative Site
city: Ashkelon
country: Israel
facility: Novartis Investigative Site
city: Hadera
country: Israel
facility: Novartis Investigative Site
city: Jerusalem
country: Israel
facility: Novartis Investigative Site
city: Petach Tikva
country: Israel
facility: Novartis Investigative Site
city: Ramat Gan
country: Israel
facility: Novartis Investigative Site
city: Tel Aviv
country: Israel
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
References: