Anemia, Iron-DeficiencyPhase 1NCT02570854
A Two-part Single- and Repeat-dose Study of CSJ137 in Anemic Chronic Hemodialysis Patients
A Phase 1 study testing CSJ137 in people with Anemia, Iron-Deficiency, run by Novartis Pharmaceuticals at 13 sites in 4 countries.
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Anemia, Iron-Deficiency
- What is being tested?
- CSJ137
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 13 sitesin 4 countries
- Study dates
- 22 Sep 2015to 13 May 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 10Show fewer
- 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.
Trial locations and contacts
13 sites in 4 countries.
13 sites in 4 countries
Israel6 sites
Novartis Investigative Site
Ashkelon, 78278
Novartis Investigative Site
Hadera, 38100
Novartis Investigative Site
Jerusalem, 91120
Novartis Investigative Site
Petah Tikva, 49100
Novartis Investigative Site
Ramat Gan
Novartis Investigative Site
Tel Aviv, 62439
United States5 sites
Novartis Investigative Site
Chattanooga, Tennessee, 37404
Novartis Investigative Site
Lakewood, Colorado, 80228
Novartis Investigative Site
Minneapolis, Minnesota, 55404
Novartis Investigative Site
Orlando, Florida, 32809
Novartis Investigative Site
San Diego, California, 91942
About this study
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
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.
Read the 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.
- Conditions
- Anemia, Iron-Deficiency
- Being tested
- CSJ137, Placebo
- Keywords
- Anemia
- Medical subject headings
- Anemia, Iron-Deficiency, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CSJ137Biological
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.
PlaceboDrug
Subjects will be dosed with a matching placebo (vehicle control) via up to 30 minutes intravenous infusion.
| Study group | Role | What they receive |
|---|---|---|
| CSJ137 | Experimental | 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). Receives: Biological: CSJ137 |
| Placebo | Placebo comparator | 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). Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
safety and tolerability following administration of CSJ137
Measured: baseline through 115 days after CSJ137 is administered
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
Measured: Hemoglobin response at 28 days post-dose
Secondary outcomes (2)
Peak concentration (Cmax) of CSJ137 in serum
to assess the concentration of CSJ137 in the body over time
Measured: 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
Area under the serum concentration versus time curve (AUC)
to assess the concentration of CSJ137 in the body over time
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Oct 2015 (estimated)
- Study start22 Sep 2015
- Primary completion13 May 2020
- Study completion13 May 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, Iron-DeficiencyStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02570854
- Sponsor's protocol ID
- CCSJ137X2201
- Other IDs
- 2017-002926-19, 2017
- Status last verified
- Oct 2021
- First submitted
- 29 Sep 2015
- Last updated
- 12 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: 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
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02570854
- Acronym / sponsor ID
- CCSJ137X2201
- Conditions
- Anemia, Iron-Deficiency
- Keywords
- Anemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 22 Sep 2015 (actual)
- Primary completion
- 13 May 2020 (actual)
- Study completion
- 13 May 2020 (actual)
- First posted
- 7 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CSJ137 | Experimental | Biological: CSJ137In 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). |
| Placebo | Placebo comparator | Drug: PlaceboIn 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). |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CSJ137Starting 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. | Biological | CSJ137 |
| PlaceboSubjects will be dosed with a matching placebo (vehicle control) via up to 30 minutes intravenous infusion. | Drug | Placebo |
Primary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: baseline through 115 days after CSJ137 is administered
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.
Time frame: Hemoglobin response at 28 days post-dose
to determine the minimum dose of CSJ137 that is active for treatment
2 secondary outcomes
Peak concentration (Cmax) of CSJ137 in serum
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
Area under the serum concentration versus time curve (AUC)
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2021.
- Results for CCSJ137X2201 from the Novartis Clinical Trials WebsiteLink provided by the sponsor.
- A Plain Language Trial Summary is available on novartisclinicaltrials.comLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
13 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CSJ137 in people with Anemia, Iron-Deficiency, run by Novartis Pharmaceuticals at 13 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Israel, United States, Czechia, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 (time frame: baseline through 115 days after CSJ137 is administered). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Oct 2021.