E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064848 |
E.1.2 | Term | Chronic kidney disease |
E.1.2 | System Organ Class | 100000004857 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
- To evaluate the efficacy and safety of episodic treatment of iron deficiency anemia (IDA) with ferumoxytol.
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E.2.2 | Secondary objectives of the trial |
- To assess the pattern of recurrence of IDA. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects who have completed participation in study FER CKD 251 or FER CKD-252 within the past 4 weeks
2. Female subjects of childbearing potential who are sexually active must be on an effective method of birth control and agree to remain on birth control until completion of the study
3. Subject and/or legal guardian is capable of understanding and complying with the protocol requirements and is available for the duration of the study
4. Subject and/or legal guardian has been informed of the investigational nature of this study and has given voluntary written informed consent, and, if appropriate, child/adolescent has provided ‘assent’ and Health Insurance Portability and Accountability Act (HIPAA) or patient protection authorization in accordance with institutional, local, and national personal health data protection guidelines |
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E.4 | Principal exclusion criteria |
1. Experienced a serious adverse event (SAE) related to intravenous (IV) iron therapy in study FER CKD 251 or FER-CKD-252
2. Hemoglobin ≤7 g/dL
3. Major surgery or invasive intervention within 4 weeks prior to Screening or during the Screening Period
4. Development of an active malignancy during participation in study FER CKD 251 or FER-CKD-252 or prior to enrollment in this study (except nonmelanoma skin cancer or carcinoma in situ that is excisable)
5. Received an investigational agent during participation in study FER CKD 251 or FER-CKD-252 or prior to enrollment in this study, other than as specified by the study protocols
6. Received a non-study-specified IV iron product during or after participation in study FER-CKD-251 or FER-CKD-252
7. Receiving oral iron therapy at the time of screening´
8. Femal subjects who are pregnant or intend to become pregnant or have a positive serum or urine pregnancy test.
9. Development of any other clinically significant medication or psychiatric desease or condition or subject responsibility that, in the Investigator's opinion, may interfere with a subject's (and/or legal guardian's) ability to adhere to the protocol, interfere with assessment of the investigational product, or serve as a contraindication to the subject's participation in the study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
PRIMARY ENDPOINT:
• Mean change in hemoglobin from Baseline to Week 5 following the first course of ferumoxytol
ADDITIONAL EFFICACY ENDPOINTS:
• Mean change in hemoglobin from Baseline to Week 7 following the first course of ferumoxytol
• Proportion of subjects with an increase in hemoglobin ≥1.0 g/dL during the period from Baseline to Week 5 and Week 7 following each course of ferumoxytol
• Proportion of subjects with an increase in hemoglobin to ≥12.0 g/dL during the period from Baseline to Week 5 and Week 7 following each course of ferumoxytol
• Mean change in TSAT from Baseline to Week 5 and Week 7 following the first course of ferumoxytol
• Proportion of subjects requiring initiation of ESA or >20% increase in dose during the study
• Proportion of subjects receiving blood transfusions during the study
• Mean change in other markers of iron stores (eg, serum ferritin and serum iron) from Baseline to Week 5 and Week 7 following the first course of ferumoxytol
• Frequency of treatment with ferumoxytol
Time to retreatment with ferumoxytol or other anemie treatment.
Additional analyses of efficacy endpoints will also be performed based on age cohort, CKD status (stage of CKD, dialysis modality, transplant status), ESA use, and treatment course (first vs subsequent courses of treatment).
SAFETY ENDPOINTS
• Adverse events of special interest (AESI) (hypotension and hypersensitivity)
• SAEs
• Severe AEs
• Cardiovascular AEs (myocardial infarction, heart failure, moderate to severe hypertension, and hospitalization due to any cardiovascular cause)
• AEs leading to study drug discontinuation
• All AEs, vital signs (blood pressure, heart rate, respiration rate) and body temperature, and routine laboratory parameters (hematology, chemistry and iron panel) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Proportion of subjects with an increase in hemoglobin ≥1.0 g/dL during the period from Baseline; Proportion of subjects with an increase in hemoglobin to ≥12.0 g/dL during the period from Baseline; Mean change in TSAT from Baseline; Time to an increase in hemoglobin of ≥1.0 g/dL from Baseline; Proportion of subjects requiring initiation of ESA or >20% increase in dose during the study; Proportion of subjects receiving blood transfusions during the study; Mean change in other markers of iron stores (eg, serum ferritin and serum iron) from Baseline; Frequency of treatment with ferumoxytol
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Week 5 and 7 post initial dose |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 28 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Bulgaria |
France |
Germany |
Hungary |
Italy |
Lithuania |
Mexico |
Peru |
Poland |
Romania |
Russian Federation |
Spain |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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last visit of the last subject undergoing the trial |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |