E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
A disease associated with long-standing inflammatory disease, which can lead to the buildup of protein (amyloid A) inside the kidney and can cause kidney failure. |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10002022 |
E.1.2 | Term | Amyloidosis |
E.1.2 | System Organ Class | 10021428 - Immune system disorders |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this open-label extension (OLE) study is to provide access to Kiacta
(eprodisate disodium) for those patients who have completed the pivotal, randomized,
placebo-controlled Phase 3 Study CL-503012. |
|
E.2.2 | Secondary objectives of the trial |
The secondary objective is to collect data on Kiacta safety and effectiveness in real-life
situations. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patient has completed Study CL-503012, and has undergone all study assessments that
could affect the primary endpoint of Study CL-503012
- Patient is at least 18 years of age
- Patient has a negative pregnancy test, if a female patient of childbearing potential
- Patient is willing to use effective birth control method. Women of childbearing potential
should continue to use effective birth control method as follows:
- Oral contraception with an additional barrier method (since the investigational
product may impair effectiveness of oral contraception)
- Double-barrier method (diaphragm with spermicidal gel or condom with
contraceptive foam)
- Transdermal or long-acting injected contraceptive (e.g., depot
medroxyprogesterone acetate [Depo-Provera®])
- Intrauterine device or implantable contraceptive
- Partner who is surgically sterile (vasectomy)
- Total abstinence
Nonsurgically sterile men should use double-barrier method as described above during
intercourse for the duration of the treatment with Kiacta.
- Patient has provided written informed consent to participate in this OLE study |
|
E.4 | Principal exclusion criteria |
- Patient has a CrCl ≤15 mL/min (as estimated by the Cockcroft-Gault formula prior to
entering into the program
- Patient has liver enzyme values (aspartate aminotransferase, alanine aminotransferase, or
alkaline phosphatase) >5 times the upper limit of normal and/or total bilirubin >50%
higher than the upper limit of normal
- Patient progressed to ESRD during Study CL-503012. In Study CL-503012 ESRD was
defined as one of the following:
i. Presence of Stage 5 chronic kidney disease, i.e., established kidney failure
with an eGFR <15 mL/min, obtained from 2 distinct SCr measurements
separated by 2 to 4 weeks (Visit X and Visit X plus 2 to 4 weeks) or
ii. Initiation of permanent renal replacement therapy, defined as a need for
chronic dialysis (duration of 3 months or more) or transplantation
- Patient was noncompliant while in Study CL-503012
- Patient prematurely withdrawn from Study CL-503012
- Patient has a clinically significant disease that could compromise patient’s safety
- Patient suffers from active alcohol and/or drug abuse
- Patient is pregnant or lactating, or patient is a woman of childbearing potential and is
unwilling to use a clinically approved form of contraception during the program
(continuous use of oral or long-acting injected contraceptive and/or use of an intra-uterine
device or implantable contraceptive, and/or use of a barrier method of birth control)
- Patient was on other investigational drug(s) given within 30 days prior to entry into this
program or during this program (with the exception of study drug taken in previous
CL-50312 program |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Collection of data on Kiacta safety and effectiveness in real-life situations |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | Yes |
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 | Yes |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 6 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
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 |
Egypt |
Lithuania |
Peru |
Poland |
Russian Federation |
Tunisia |
Ukraine |
United Kingdom |
|
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
|
Last visit of the last subject |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |