AnemiaPhase 1/2NCT02079896
Lexaptepid Pegol (NOX-H94) in ESA-hyporesponsive Anemia in Dialysis Patients
A Phase 1/2 study testing Lexaptepid pegol (NOX-H94) in people with Anemia and End Stage Renal Disease, run by TME Pharma AG at 10 sites in 3 countries.
Trial summary
Dialysis patients regularly suffer from anemia which may be caused by various contributing factors, alone or in combination, including blood loss, low erythropoietin and iron sequestration. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Anemia, End Stage Renal Disease
- What is being tested?
- Lexaptepid pegol (NOX-H94)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 33 participants
- Where
- 10 sitesin 3 countries
- Study dates
- May 2014to Nov 2015
- Phase
- Phase 1/2
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- End stage renal disease treated with maintenance hemodialysis.
- Anemia : Hb 7 to 11 g/dL.
- Functional iron deficiency: Transferrin saturation <30%, Ferritin ≥300 ng/mL.
- ESA-hyporesponsiveness with erythropoietin dose ≥12,000 IU/ week.
Who cannot join
- Treatment with darbepoetin or methoxy-polyethyleneglycol-epoetin.
- Uncontrolled / unstable cardiovascular , peripheral arterial or cerebrovascular disease.
- Congestive heart failure: New York Heart Association Class III or IV.
- Unstable angina, myocardial infarction, percutaneous transluminal coronary angioplasty/stents, or coronary artery bypass grafting <3 months prior screening.
- Any other medical conditions requiring a change in treatment within 4 weeks prior to screening or making study participation unadvisable.
- History of clinically relevant hemolysis and/or blood loss.
Show all 10Show fewer
- AST, ALT, or bilirubin ≥2.0 times the upper limit of normal.
- Known bone marrow fibrosis.
- Treatment with i.v. iron <4 weeks prior to screening or during the screening period or change in erythropoietin dose during last month.
- Any acute or chronic infection, viral or bacterial within 4 weeks prior to screening or during the screening period considered as systemic infection.
Trial locations and contacts
10 sites in 3 countries.
10 sites in 3 countries
United Kingdom5 sites
Hospital
Leicester
Hospital
London
King's College London
London
Lister Hospital
Stevenage
Hospital
Swansea, Wales
Germany4 sites
Dialysis Unit
Düsseldorf
University Hospital
Halle
Hospital
Leipzig
Dialysis Unit
Villingen-Schwenningen
About this study
Official title: Safety, PK/PD, and Efficacy of NOX-H94 in Dialysis Patients With ESA-hyporesponsive Anemia: A Randomized, Double Blind, Placebo Controlled Parallel Group Study With a Single Blind Cross-over Group
Dialysis patients regularly suffer from anemia which may be caused by various contributing factors, alone or in combination, including blood loss, low erythropoietin and iron sequestration. In most patients, the anemia is responsive to treatment with erythropoietin or other erythropoiesis stimulating agents (ESA) alone or in combination with intravenous (i.v.) iron. In about 10% of patients however, the anaemia does not respond appropriately to this standard treatment and high to very high doses of ESA and i.v. iron are used to maintain acceptable hemoglobin concentrations. In these patients, hepcidin was identified as a causative factor leading to anemia of chronic disease with functional iron deficiency and ESA-hyporesponsiveness.
The Spiegelmer lexaptepid pegol (NOX-H94) offers a hepcidin-specific approach to the treatment of anemia of chronic disease. The safety and the activity of lexaptepid pegol are supported by data from healthy subjects and patients with multiple myeloma or lymphoma. The present study in dialysis patients with functional iron deficiency and ESA-hyporesponsiveness is conducted to demonstrate the safety of lexaptepid pegol in this population, to investigate its pharmacokinetic (PK) and pharmacodynamic (PD) profiles and its efficacy in increasing haemoglobin (Hb) in dialysis patients.
- Conditions
- Anemia, End Stage Renal Disease
- Being tested
- Lexaptepid pegol (NOX-H94), Placebo
- Keywords
- Dialysis
- Medical subject headings
- Anemia, Kidney Failure, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lexaptepid pegol (NOX-H94)Drug
Also called NOX-H94
anti-hepcidin L-RNA-aptamer (Spiegelmer)
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Single dose cross-over pilot | Experimental | Single dose of lexaptepid pegol (NOX-H94) cross-over with single dose of placebo Receives: Drug: Lexaptepid pegol (NOX-H94); Drug: Placebo |
| Control | Placebo comparator | Twice weekly doses of placebo, 9 total Receives: Drug: Placebo |
| Lexaptepid pegol (NOX-H94) | Experimental | Twice weekly doses of lexaptepid pegol (NOX-H94), 9 total Receives: Drug: Lexaptepid pegol (NOX-H94) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events
Measured: up to 8 weeks
Secondary outcomes (3)
Pharmacokinetics
Peak concentrations, systemic exposure, elimination
Measured: Weeks 1, 2, 3, 4, 5, 6, 8
Pharmacodynamics
Change in serum iron concentrations
Measured: 0 to 48 hours
Efficacy
Change in hemoglobin
Measured: Weeks 1, 2, 3, 4, 5, 6, 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2014 (estimated)
- Study startMay 2014
- Primary completionNov 2015
- Study completionNov 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AnemiaStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- TME Pharma AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kai Riecke, MD, Study director, TME Pharma AG
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02079896
- Sponsor's protocol ID
- SNOXH94C301
- Other IDs
- 2013-003585-14
- Status last verified
- Nov 2015
- First submitted
- 28 Feb 2014
- Last updated
- 24 Nov 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Park EJ, Choi J, Lee KC, Na DH. Emerging PEGylated non-biologic drugs. Expert Opin Emerg Drugs. 2019 Jun;24(2):107-119. doi: 10.1080/14728214.2019.1604684. Epub 2019 Apr 19.
PubMed 30957581
Study at a glance
From the registry record, last updated by the sponsor on 24 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, PK/PD, and Efficacy of NOX-H94 in Dialysis Patients With ESA-hyporesponsive Anemia: A Randomized, Double Blind, Placebo Controlled Parallel Group Study With a Single Blind Cross-over Group
- ClinicalTrials.gov ID
- NCT02079896
- Acronym / sponsor ID
- SNOXH94C301
- Conditions
- Anemia, End Stage Renal Disease
- Keywords
- Dialysis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- TME Pharma AGIndustry (pharma, biotech, devices)
- Study start
- May 2014
- Primary completion
- Nov 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 6 Mar 2014 (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.
Dialysis patients regularly suffer from anemia which may be caused by various contributing factors, alone or in combination, including blood loss, low erythropoietin and iron sequestration. In most patients, the anemia is responsive to treatment with erythropoietin or other erythropoiesis stimulating agents (ESA) alone or in combination with intravenous (i.v.) iron. In about 10% of patients however, the anaemia does not respond appropriately to this standard treatment and high to very high doses of ESA and i.v. iron are used to maintain acceptable hemoglobin concentrations. In these patients, hepcidin was identified as a causative factor leading to anemia of chronic disease with functional iron deficiency and ESA-hyporesponsiveness.
The Spiegelmer lexaptepid pegol (NOX-H94) offers a hepcidin-specific approach to the treatment of anemia of chronic disease. The safety and the activity of lexaptepid pegol are supported by data from healthy subjects and patients with multiple myeloma or lymphoma. The present study in dialysis patients with functional iron deficiency and ESA-hyporesponsiveness is conducted to demonstrate the safety of lexaptepid pegol in this population, to investigate its pharmacokinetic (PK) and pharmacodynamic (PD) profiles and its efficacy in increasing haemoglobin (Hb) in dialysis patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Single dose cross-over pilot | Experimental | Drug: Lexaptepid pegol (NOX-H94), Drug: PlaceboSingle dose of lexaptepid pegol (NOX-H94) cross-over with single dose of placebo |
| Control | Placebo comparator | Drug: PlaceboTwice weekly doses of placebo, 9 total |
| Lexaptepid pegol (NOX-H94) | Experimental | Drug: Lexaptepid pegol (NOX-H94)Twice weekly doses of lexaptepid pegol (NOX-H94), 9 total |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- End stage renal disease treated with maintenance hemodialysis.
- Anemia : Hb 7 to 11 g/dL.
- Functional iron deficiency: Transferrin saturation <30%, Ferritin ≥300 ng/mL.
- ESA-hyporesponsiveness with erythropoietin dose ≥12,000 IU/ week.
Key exclusion criteria (10 listed)
- Treatment with darbepoetin or methoxy-polyethyleneglycol-epoetin.
- Uncontrolled / unstable cardiovascular , peripheral arterial or cerebrovascular disease.
- Congestive heart failure: New York Heart Association Class III or IV.
- Unstable angina, myocardial infarction, percutaneous transluminal coronary angioplasty/stents, or coronary artery bypass grafting <3 months prior screening.
- Any other medical conditions requiring a change in treatment within 4 weeks prior to screening or making study participation unadvisable.
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 |
|---|---|---|
| Lexaptepid pegol (NOX-H94)Also: NOX-H94anti-hepcidin L-RNA-aptamer (Spiegelmer) | Drug | Lexaptepid pegol (NOX-H94), Single dose cross-over pilot |
| Placebo | Drug | Control, Single dose cross-over pilot |
Primary outcome
Number of adverse events
Time frame: up to 8 weeks
3 secondary outcomes
Pharmacokinetics
Time frame: Weeks 1, 2, 3, 4, 5, 6, 8
Pharmacodynamics
Time frame: 0 to 48 hours
Efficacy
Time frame: Weeks 1, 2, 3, 4, 5, 6, 8
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 24 Nov 2015.
- Derived from this study: PubMed 30957581Park EJ, Choi J, Lee KC, Na DH. Emerging PEGylated non-biologic drugs. Expert Opin Emerg Drugs. 2019 Jun;24(2):107-119. doi: 10.1080/14728214.2019.1604684. Epub 2019 Apr 19.
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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.
Kai Riecke, MD study director
TME Pharma AG
10 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Lexaptepid pegol (NOX-H94) in people with Anemia and End Stage Renal Disease, run by TME Pharma AG at 10 sites in 3 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 4 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 24 Nov 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United Kingdom, Germany, Italy. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 adverse events (time frame: up to 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02079896, which also lists 1 linked publication.
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 24 Nov 2015.