AnemiaPhase 1NCT01372137
First-in-human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of NOX-H94
A Phase 1 study testing NOX-H94 and Glucose 5% in people with Anemia and Chronic Diseases, run by TME Pharma AG at 1 site in United Kingdom.
Trial summary
This is the first clinical trial with NOX-H94. The purpose of this clinical trial is to identify a safe and efficacious treatment regimen for the clinical development of NOX-H94 in patients with anemia of chronic disease (inflammation). Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Anemia, Chronic Diseases, Inflammation
- What is being tested?
- NOX-H94 and Glucose 5%
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 64 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Jul 2011to Mar 2012
- 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 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Male subjects or female subjects of non-childbearing potential (Groups A to E), male subjects (groups F to H)
- Age 18-65 years
- Healthy as determined by medical history, physical examination, vital signs, 12-lead electrocardiogram, and clinical laboratory parameters
- Males willing to use 2 means of contraceptive methods for at least 2 months after the final examination
Who cannot join
- Anemia predominantly caused by other factors than chronic disease.
- Iron overload or disturbances in utilization of iron.
- Intravenous iron treatment or blood transfusion within 4 weeks prior to screening visit.
- Erythropoietin treatment within 4 weeks prior to screening visit.
- Intake of Intravenous iron, Blood transfusions, Erythropoietin during their trial participation.
- Resting supine pulse rate < 40 or > 100 beats / min.
Show all 28Show fewer
- Resting supine blood pressure:
- Systolic blood pressure < 90 or > 160 mmHg Diastolic blood pressure < 40 or > 100 mmHg.
- History or presence of confirmed orthostatic hypotension defined.
- Positive test of HIV type 1/2 antibodies, HBs antigen, HBc antibodies, HCV antibodies.
- Participation in another clinical trial during the last 3 months before starting this trial.
- Positive test for drugs of abuse.
- Diseases or condition known to interfere with the absorption, distribution, metabolism or excretion of drugs.
- Marked repolarization abnormality.
- Current bronchial asthma, childhood asthma which has been resolved is allowed.
- Definite or suspected history of drug allergy or hypersensitivity or intolerance to PEG
- Regular intake of over 14 units of alcohol per week for women and 21 units for men.
- Not able to abstain from consumption of:
- Caffeine containing beverages or food (tea, coffee, cola, chocolate, etc.)
- Quinine containing beverages or food (bitter lemon, tonic water)
- Grapefruit juice (sweet or sour)
- Poppy seeds containing beverages or food
- Subjects who have donated any blood, plasma or platelets in the month prior to screening
- History of seizures or at risk
- Known or suspected of not being able to comply with the trial protocol and/or clinical unit restrictions.
- History of or presence of clinically significant diseases other than the underlying disease.
- Surgery or trauma with significant blood loss within the last 2 months before administration of study drug.
- 28. History of increased bleeding risk.
Trial locations and contacts
1 site in 1 country.
About this study
This is the first clinical trial with NOX-H94. The purpose of this clinical trial is to identify a safe and efficacious treatment regimen for the clinical development of NOX-H94 in patients with anemia of chronic disease (inflammation).
Read the detailed description
NOX H94 is a pegylated Spiegelmer that specifically binds to human hepcidin, thereby antagonizing its role in hemostasis, and is therefore indicated for use in anemia of inflammation. Human hepcidin has emerged as the central regulatory molecule of systemic iron homeostasis. Hepcidin expression in hepatocytes is regulated by multiple, in particular opposing signals, including systemic iron availability, hepatic iron stores, erythropoietic activity, hypoxia, and inflammatory states. These different signals are integrated transcriptionally. In chronic inflammation, such as occurs in rheumatoid arthritis, chronic kidney disease or cancer, elevated hepcidin levels have been measured and may be a key factor leading to anemia in these patients.
NOX-H94 is therefore indicated for treatment of patients with an anemia of inflammation, which is characterized by increased intracellular iron stores, increased serum ferritin concentrations and reduced sensitivity to treatment with erythropoiesis stimulating agents (ESAs), due to the limited availability of serum iron. Antagonism of hepcidin by NOX-H94 therefore leads to elevated levels of iron and transferrin saturation in the peripheral blood and could supply iron for erythropoiesis thereby correcting the anemia.
- Conditions
- Anemia, Chronic Diseases, Inflammation
- Being tested
- NOX-H94, Glucose 5%
- Keywords
- NOX-H94, Hepcidin, Anemia, Inflammation
- Medical subject headings
- Anemia, Chronic Disease, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NOX-H94Drug
Also called lexaptepid pegol
Dosage form: NOX-H94 25 mg (oligonucleotide basis) Solution for Injection Strength: 14.6 mg NOX-H94 / mL Dose: 0.3 - 4.8 mg/kg single dose Route: IV infusion over 15 minutes / SC administration
Glucose 5%Other
Placebo
| Study group | Role | What they receive |
|---|---|---|
| NOX-H94 | Experimental | Group A: single 15 minutes IV infusion of 0.3 mg/kg NOX-H94 Group B: single 15 minutes IV infusion of 0.6 mg/kg NOX-H94 Group C: single 15 minutes IV infusion of 1.2 mg/kg NOX-H94 Group D: single 15 minutes IV infusion of 2.4 mg/kg NOX-H94 Group E: single 15 minutes IV infusion of 4.8 mg/kg NOX-H94 Group F: single / repeated SC injection of NOX-H94 over a treatment period of 2 weeks Group G: multiple doses of NOX-H94 as a 15 minutes IV infusion over a treatment period of 2 weeks Group H: multiple doses of NOX-H94 as a 15 minutes IV infusion over a treatment period of 2 weeks Receives: Drug: NOX-H94 |
| Glucose 5% | Placebo comparator | Group A to Group H get NOX-H94 or Placebo Receives: Other: Glucose 5% |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
Measured: 0 to 90 days
Secondary outcomes (1)
drug plasma concentrations
Measured: 0 to 29 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2011 (estimated)
- Study startJul 2011
- Primary completionMar 2012
- Study completionMar 2012
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- TME Pharma AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Riecke Kai, MD, Study director, TME Pharma AG
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01372137
- Sponsor's protocol ID
- SNOXH94C001
- Other IDs
- 2011-000705-46
- Status last verified
- Jun 2014
- First submitted
- 8 Jun 2011
- Last updated
- 25 Jan 2016
- 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 25 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- First-in-human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of NOX-H94
- ClinicalTrials.gov ID
- NCT01372137
- Acronym / sponsor ID
- SNOXH94C001
- Conditions
- Anemia, Chronic Diseases, Inflammation
- Keywords
- NOX-H94, Hepcidin, Anemia, Inflammation
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- TME Pharma AGIndustry (pharma, biotech, devices)
- Study start
- Jul 2011
- Primary completion
- Mar 2012 (actual)
- Study completion
- Mar 2012 (actual)
- First posted
- 13 Jun 2011 (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.
This is the first clinical trial with NOX-H94. The purpose of this clinical trial is to identify a safe and efficacious treatment regimen for the clinical development of NOX-H94 in patients with anemia of chronic disease (inflammation).
Detailed description
NOX H94 is a pegylated Spiegelmer that specifically binds to human hepcidin, thereby antagonizing its role in hemostasis, and is therefore indicated for use in anemia of inflammation. Human hepcidin has emerged as the central regulatory molecule of systemic iron homeostasis. Hepcidin expression in hepatocytes is regulated by multiple, in particular opposing signals, including systemic iron availability, hepatic iron stores, erythropoietic activity, hypoxia, and inflammatory states. These different signals are integrated transcriptionally. In chronic inflammation, such as occurs in rheumatoid arthritis, chronic kidney disease or cancer, elevated hepcidin levels have been measured and may be a key factor leading to anemia in these patients.
NOX-H94 is therefore indicated for treatment of patients with an anemia of inflammation, which is characterized by increased intracellular iron stores, increased serum ferritin concentrations and reduced sensitivity to treatment with erythropoiesis stimulating agents (ESAs), due to the limited availability of serum iron. Antagonism of hepcidin by NOX-H94 therefore leads to elevated levels of iron and transferrin saturation in the peripheral blood and could supply iron for erythropoiesis thereby correcting the anemia.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NOX-H94 | Experimental | Drug: NOX-H94Group A: single 15 minutes IV infusion of 0.3 mg/kg NOX-H94 Group B: single 15 minutes IV infusion of 0.6 mg/kg NOX-H94 Group C: single 15 minutes IV infusion of 1.2 mg/kg NOX-H94 Group D: single 15 minutes IV infusion of 2.4 mg/kg NOX-H94 Group E: single 15 minutes IV infusion of 4.8 mg/kg NOX-H94 Group F: single / repeated SC injection of NOX-H94 over a treatment period of 2 weeks Group G: multiple doses of NOX-H94 as a 15 minutes IV infusion over a treatment period of 2 weeks Group H: multiple doses of NOX-H94 as a 15 minutes IV infusion over a treatment period of 2 weeks |
| Glucose 5% | Placebo comparator | Other: Glucose 5%Group A to Group H get NOX-H94 or Placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Male subjects or female subjects of non-childbearing potential (Groups A to E), male subjects (groups F to H)
- Age 18-65 years
- Healthy as determined by medical history, physical examination, vital signs, 12-lead electrocardiogram, and clinical laboratory parameters
- Males willing to use 2 means of contraceptive methods for at least 2 months after the final examination
Key exclusion criteria (28 listed)
- Anemia predominantly caused by other factors than chronic disease.
- Iron overload or disturbances in utilization of iron.
- Intravenous iron treatment or blood transfusion within 4 weeks prior to screening visit.
- Erythropoietin treatment within 4 weeks prior to screening visit.
- Intake of Intravenous iron, Blood transfusions, Erythropoietin during their trial participation.
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 |
|---|---|---|
| NOX-H94Also: lexaptepid pegolDosage form: NOX-H94 25 mg (oligonucleotide basis) Solution for Injection Strength: 14.6 mg NOX-H94 / mL Dose: 0.3 - 4.8 mg/kg single dose Route: IV infusion over 15 minutes / SC administration | Drug | NOX-H94 |
| Glucose 5%Placebo | Other | Glucose 5% |
Primary outcome
Incidence of adverse events
Time frame: 0 to 90 days
1 secondary outcome
drug plasma concentrations
Time frame: 0 to 29 days
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 25 Jan 2016.
- 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.
Riecke Kai, MD study director
TME Pharma AG
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NOX-H94 and Glucose 5% in people with Anemia and Chronic Diseases, run by TME Pharma AG at 1 site in United Kingdom.
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 28 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 25 Jan 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in 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?
Incidence of adverse events (time frame: 0 to 90 days). 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 NCT01372137, 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 25 Jan 2016.