Safety and Pharmacology of NOX-H94 in dialysis patients with ESA resistant anaemia

Phase 1

• Conditions: Erythropoiesis stimulating agents (ESA) hyporesponsive anaemia in dialysis patients; MedDRA version: 20.0Level: LLTClassification code 10058123Term: Renal anaemiaSystem Organ Class: 10005329 - Blood and lymphatic system disorders; MedDRA version: 20.0Level: SOCClassification code 10005329Term: Blood and lymphatic system disordersSystem Organ Class: 10005329 - Blood and lymphatic system disorders; MedDRA version: 20.0Level: SOCClassification code 10038359Term: Renal and urinary disordersSystem Organ Class: 10038359 - Renal and urinary disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2013-003585-14-IT
• Lead Sponsor: OXXON PHARMA AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2015-11-16
• Study Start: 2021-01-19
• Last Update Posted: 24 August 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

1. Written informed consent
2. Male or female of non-childbearing potential
3. Age 18 to 85 years
4. End stage renal disease treated with maintenance haemodialysis since =3 months, 3 times weekly
5. Anaemia : Hb 7 to 11 g/dL
6. Functional iron deficiency: Transferrin saturation <30%, Ferritin =300 ng/mL.
7. ESA hyporesponsiveness with erythropoietin dose =12,000 IU/ week stable for =4 weeks
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 22
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

1. Inability to personally provide written informed consent or to understand and collaborate throughout the study.
2. Inability or unwillingness to comply with study restrictions.
3. Participation in any clinical research study evaluating an investigational drug or therapy within 30 days from screening visit or prior enrolment in this study.
4. Pregnancy or lactation.
5. Treatment with darbepoetin or methoxy-polyethylenglycol-epoetin
6. Uncontrolled cardiovascular disease, unstable peripheral arterial or cerebrovascular disease, uncontrolled hypertension (systolic blood pressure >180 mm Hg or diastolic blood pressure >100 mm Hg).
7. Congestive heart failure: New York Heart Association Class III or IV.
8. Unstable angina, myocardial infarction, percutaneous transluminal coronary angioplasty/stents, or coronary artery bypass grafting <3 months prior screening.
9. Any other medical conditions requiring a change in treatment within 4 weeks prior to screening or making study participation unadvisable (at
the discretion of the investigator).
10. History of clinically relevant haemolysis and/or blood loss.
11. AST, ALT, or bilirubin =2.0 times the upper limit of normal.
12. Known bone marrow fibrosis.
13. Treatment with i.v. iron <4 weeks prior to screening or during the screening period or change in erythropoietin dose during last month.
14. Any acute or chronic infection, viral or bacterial within 4 weeks prior to screening or during the screening period that, according to investigator's judgement, is considered as systemic infection.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety and tolerability of single and repeated doses of NOX-H94 in dialysis patients;Secondary Objective: ¿ To study the PK with a focus on plasma accumulation and elimination, the PD, and the efficacy of NOX-H94 in dialysis patients with functional iron deficiency<br>¿ To obtain first data on efficacy of NOX-H94 with regard to the response of Hb;Primary end point(s): Incidence of adverse events, either spontaneously reported or resulting from safety and tolerability.;Timepoint(s) of evaluation of this end point: Safety parameters will be evaluated after IMP administration throughout the treatment and follow up periods.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): ¿ PK of NOX-H94 after single and repeat i.v. doses of IMP in dialysis patients;<br>¿ PD of NOX-H94 after single i.v. dose of NOX H94 evaluated through<br>serum iron concentration changes<br>¿ PD and efficacy of NOX-H94 after repeated doses of NOX-H94<br>evaluated through changes in serum ferritin, soluble transferrin receptors (sTfR), interleukin-6, reticulocyte haemoglobin content (CHr), mean corpuscular haemoglobin (MCH), Hb and reticulocytes.;Timepoint(s) of evaluation of this end point: PK endpoints assessed at the following timepoints:<br>Group 1: Day 8, 9, 10, 12, 15, 22 and 36<br>Groups 2/3: Day 1, 3, 8, 15, 22, 29, 36, 43 and 57<br>Pharmacodynamics and efficacy endpoints:<br>Group 1: Day 1, 2, 8, 9, 10, 12, 15 and 22<br>Groups 2/3: Day 1, 8, 15, 22, 29, 36 and 43