Clinical Trial Results:
Phase IIa study to characterize the effects of the Spiegelmer® NOX-H94 on anemia of chronic disease in patients with cancer
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Summary
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EudraCT number |
2012-001525-27 |
Trial protocol |
AT BG |
Global end of trial date |
30 Dec 2013
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Results information
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Results version number |
v1(current) |
This version publication date |
03 Feb 2016
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First version publication date |
30 Jul 2015
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
SNOXH94C201
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01691040 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
NOXXON Pharma AG
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Sponsor organisation address |
Max-Dohrn Strasse 8-10, Berlin, Germany, 10589
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Public contact |
Clinical Trial Disclosure Desk NOXXON, NOXXON Pharma AG, clinicaltrialdisclosuredesk@noxxon.com
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Scientific contact |
Clinical Trial Disclosure Desk NOXXON, NOXXON Pharma AG, clinicaltrialdisclosuredesk@noxxon.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
29 Apr 2014
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
30 Dec 2013
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Global end of trial reached? |
Yes
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Global end of trial date |
30 Dec 2013
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To assess the response rate of anemia in patients with cancer to treatment with NOX-H94.
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Protection of trial subjects |
This clinical study was written, conducted and reported in accordance with the protocol, ICH GCP Guidelines, and applicable local regulations, including the European Directive 2001/20/EC, 2005/28/EC, and 2003/63/EC and relevant national and local legislations, and with the ethical principles that have their origin in the Declaration of Helsinki. Only subjects that met all the study inclusion and none of the exclusion criteria were randomized. Study drug administrations were performed by qualified and trained study personnel. Patient who received treatment were closely followed by means of adverse event reporting and vital signs. In the event of a study related adverse event, patient would have been monitored to determine the outcome. The clinical course of the AE were followed up according to accepted standards of medical practice, even after the end of the period of observation, until a satisfactory explanation is found or the Investigator considers it medically justifiable to terminate follow-up.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
25 Sep 2012
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Romania: 7
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Country: Number of subjects enrolled |
Bulgaria: 5
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Worldwide total number of subjects |
12
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EEA total number of subjects |
12
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
6
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From 65 to 84 years |
6
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||
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Pre-assignment
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Screening details |
A total of 33 patients were screened and thereof 21 were screen failure. After a screening period of maximum 28 days patients started to be treated. | ||||||||||
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Period 1
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Period 1 title |
Overall trial (overall period)
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
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Arms
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Arm title
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Lexaptepid | ||||||||||
Arm description |
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Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
Lexaptepid Pegol
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Investigational medicinal product code |
NOX-H94
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Other name |
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Intravenous use
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Dosage and administration details |
Patients were treated twice weekly with intravenous (i.v.) doses of 1.2 mg/kg Lexaptepid Pegol administered by slow injection over 1 minute. Nine doses were administered over a treatment period of 4 weeks.
Formulation was a preservative-free, sterile solution in an aqueous citrate buffer containing sucrose.
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Baseline characteristics reporting groups
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Reporting group title |
Overall trial
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Reporting group description |
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End points reporting groups
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Reporting group title |
Lexaptepid
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Reporting group description |
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End point title |
Response Rate [1] | ||||||||
End point description |
Treatment responders were defined by Hb increase ≥1 g/dL OR reticulocyte index (RI) normalization (≥1%) at any time point until 1 week after the end of treatment AND absence of all of the following treatment failure criteria until 1 week after the end of treatment:
• Erythrocyte transfusion, ESA or i.v. iron
• Hb drop by ≥1 g/dL
• Treatment interruption due to adverse events (AEs)
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End point type |
Primary
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End point timeframe |
Any time point until 1 week after the end of treatment.
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: The primary efficacy endpoint was assessed descriptively only, no statistical analysis has been performed. For the primary endpoint only "responder: yes / no" was assessed according to the end point description above and counted. |
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| No statistical analyses for this end point | |||||||||
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Adverse events information
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Timeframe for reporting adverse events |
Baseline (Day 1) until end of follow up (Day 85)
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
15.1
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Reporting groups
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Reporting group title |
Lexaptepid
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Reporting group description |
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| Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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18 Feb 2013 |
AT/BG: Amendment 1:
The study population and few inclusion/exclusion criteria were modified:
- The study population was enlarged to anemic patients with solid tumors. This change required also an update of the study title.
- The cut-off value for TSAT was increased to 50% in accordance to the new definition of functional iron deficiency from the NCCN Guideline 1.2013.
- The criterion on previous treatment with systemic anti-cancer therapy was deleted.
The following exclusion criteria were added and protocol sections modified:
- A known or suspected chronic bleeding.
- Patients with tumor with gastro-intestinal involvement, without a negative test for fecal occult blood. The test was added to the screening procedures in this kind of patients.
- Selection and withdrawal of patients (Section 7.1): The possibility to re-screen patients after an appropriate interval was introduced, in case the patient did not meet the selection criteria at the first screening visit.
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27 May 2013 |
RO: Amendment 1:
- The study population and few inclusion/exclusion criteria were modified:
- The study population was enlarged to anemic patients with solid tumors. This change required also an update of the study title.
- The cut-off value for TSAT was increased to 20% in accordance to the new definition of functional iron deficiency from the NCCN Guideline 1.2013.
- The criterion on previous treatment with systemic anti-cancer therapy was deleted.
The following exclusion criteria were added and protocol sections modified:
- A known or suspected chronic bleeding.
- Patients with tumor with gastro-intestinal involvement, without a negative test for fecal occult blood. The test was added to the screening procedures in this kind of patients.
- Selection and withdrawal of patients (Section 7.1): The possibility to re-screen patients after an appropriate interval was introduced, in case the patient did not meet the selection criteria at the first screening visit.
Exclusion of patients with tumor of the gastrointestinal tract and of the liver (metastatic or primary); for this reason the fecal blood test was not required anymore. |
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Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
