Clinical Trial Results:
A Phase 2 open-label, dose-escalation study to evaluate the safety, pharmacokinetics, immunogeniticy and pharmacodynamics/efficacy of EDI200, an EDA-A1 replacement protein, administered to male infants with X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).
Summary
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EudraCT number |
2012-003561-17 |
Trial protocol |
DE GB IT FR |
Global end of trial date |
17 Mar 2016
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Results information
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Results version number |
v1(current) |
This version publication date |
27 Aug 2016
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First version publication date |
27 Aug 2016
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Other versions |
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Summary report(s) |
_Edimer ECP-002 Abbrv. CSR Final_31May2016_Signed wAtt |
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 |
ECP-002
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01775462 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Edimer Pharmaceuticals, Inc.
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Sponsor organisation address |
55 Cambridge Parkway, Suite 102W, Cambridge, United States, MA 02142
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Public contact |
Clinical Trials Information, Edimer Pharmaceuticals, Inc., +1 6177584305, ramsey@edimerpharma.com
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Scientific contact |
Clinical Trials Information, Edimer Pharmaceuticals, Inc., +1 6177584305, ramsey@edimerpharma.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 |
28 Sep 2015
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
28 Sep 2015
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Global end of trial reached? |
Yes
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Global end of trial date |
17 Mar 2016
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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 safety, pharmacokinetics and immunogenicity of EDI200 administered to XLHED-affected neonates
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Protection of trial subjects |
Patients were always under close monitoring in the hospital by specific qualified and trained paediattic trial staff (Investigators and study nurses).
All possible steps were taken to prevent the patients from any stress, pain or discomfort.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
03 Jun 2013
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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 |
United Kingdom: 2
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Country: Number of subjects enrolled |
France: 1
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Country: Number of subjects enrolled |
Germany: 4
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Country: Number of subjects enrolled |
United States: 3
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Worldwide total number of subjects |
10
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EEA total number of subjects |
7
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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) |
10
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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) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
- | ||||||||||||
Pre-assignment
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Screening details |
Male neonates documented by genetic diagnosis to carry an EDA mutation associated with XLHED, and their male siblings | ||||||||||||
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 |
Non-randomised - controlled
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Blinding used |
Not blinded | ||||||||||||
Arms
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Arm title
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Treatment | ||||||||||||
Arm description |
- | ||||||||||||
Arm type |
Experimental | ||||||||||||
Investigational medicinal product name |
EDI200
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
3/10/20 mg/kg (5 doses total) mg/kg milligram(s)/kilogram per day
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Notes [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left. Justification: 2 Subjects were enrolled in cohort 3 till study completion [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left. Justification: Dose was adjusted after 5 patients completed cohort 2 [3] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left. Justification: Dose was adjusted after 3 patients completed cohort 1 |
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End points reporting groups
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Reporting group title |
Treatment
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Reporting group description |
- |
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End point title |
Clinical endpoints [1] | ||||||
End point description |
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End point type |
Primary
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End point timeframe |
overall
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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: Proof of concept study to be compared with a parallel natural history study |
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No statistical analyses for this end point |
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Adverse events information [1]
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Timeframe for reporting adverse events |
16 Sep 2013 - 28 Sep 2015
Time from first patient in till last patient out visit
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Assessment type |
Systematic | ||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||
Dictionary version |
10.0
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Frequency threshold for reporting non-serious adverse events: 0% | |||
Notes [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported. Justification: All serious advers events can be found in the Appendix 16.2.7 of the CSR |
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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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24 Jun 2013 |
Protocol Version 3 (see CSR 9.8 page 15)) |
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04 Dec 2014 |
Protocol AMD 5 (See CSR 9.8 page 16) |
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11 Feb 2015 |
Protocol AMD 6 (See CSR 9.9 page 15) |
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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 |