Clinical Trial Results:
Pilot trial of recombinant human growth hormone for remyelination in multiple sclerosis
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Summary
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EudraCT number |
2006-006465-16 |
Trial protocol |
DE |
Global end of trial date |
15 Mar 2016
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Results information
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Results version number |
v1(current) |
This version publication date |
04 Jul 2020
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First version publication date |
04 Jul 2020
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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 |
rhGH in MS
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01541605 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
University of Leipzig
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Sponsor organisation address |
Ritterstr. 26, Leipzig, Germany, 04109
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Public contact |
Prof. Dr. F. Then Bergh
, Klinik und Poliklinik für Neurologie
Universität Leipzig
, ThenBerF@medizin.uni-leipzig.de
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Scientific contact |
Prof. Dr. F. Then Bergh
, Klinik und Poliklinik für Neurologie
Universität Leipzig
, ThenBerF@medizin.uni-leipzig.de
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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 |
17 Jan 2017
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
11 Nov 2015
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Global end of trial reached? |
Yes
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Global end of trial date |
15 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 rule out an increase in inflammatory disease activity by s.c. rhGH as detected by monthly contrast-enhanced MRI: Primary outcome measure is the cumulative number of active (new or gadolinium-enhancing) lesions during the 12-week baseline period and the first 12 weeks of the treatment period.
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Protection of trial subjects |
There were two safety assessments earlon on in the trial:
1) Major increase in inflammatory disease activity by s.c. rhGH. An clinical safety assessment will be performed after four patients have completed week 12 of rhGH treatment. The study may be discontinued, if more than 2 out of the first four patients fulfill the following condition:
The cumulative number of new active lesions during the treatment phase exceeds the maximum of 12 and is more than twice the cumulative number of new active lesions during the baseline period.
2) Significant increase in clinical disease activity. A second clinical safety assessment is planned when 15 patients have completed 24 weeks of rhGH treatment. The study may be discontinued if more than 30% of these patients display a clinically significant increase of the relapse rate or neurological disability inconsistent with prior disease history and not explained by other intervening variables.
The patients were also closely watched for serious unexpected adverse drug reactions, serious unexpected interactions with immunomodulatory treatments for MS (interferon-beta, copolymer-1) / not justifiable toxicity
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Background therapy |
- | ||
Evidence for comparator |
Cross-over design; no active comparator. | ||
Actual start date of recruitment |
19 Jun 2009
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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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 |
Germany: 37
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Worldwide total number of subjects |
37
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EEA total number of subjects |
37
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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) |
37
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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 |
- | ||||||||||||||
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Pre-assignment
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Screening details |
Only subjects who met all inclusion criteria, but none of the exclusion criteria specified for the screening phase were enrolled. | ||||||||||||||
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Period 1
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Period 1 title |
Screening
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Is this the baseline period? |
No | ||||||||||||||
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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pre-treatment | ||||||||||||||
Arm description |
- | ||||||||||||||
Arm type |
No intervention | ||||||||||||||
Investigational medicinal product name |
No investigational medicinal product assigned in this arm
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Period 2
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Period 2 title |
Treatment
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Is this the baseline period? |
Yes [1] | ||||||||||||||
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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recombinant growth factor | ||||||||||||||
Arm description |
- | ||||||||||||||
Arm type |
Experimental | ||||||||||||||
Investigational medicinal product name |
Recombinant human growth hormone (rhGH)
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Investigational medicinal product code |
H01AC01
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Other name |
Genotropin®
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Pharmaceutical forms |
Solution for injection
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
Then dosage was induividually adjusted to achieve an IGF-1 concentration in the upper quartile of the age-adjusted normal range.
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| Notes [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period. Justification: Period 1 is the screening period. The screening period had a duration of 12 weeks, including regular visits and assessements. Data from the screening period was used as baseline data for the evaluation of trial endpoints. However, only patients eventually included into the treatment phase are analysed, and therefore baseline data is reported for patients who entered the second period only. |
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| Notes [2] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same. Justification: Period 1 is the screening period. The screening period had a duration of 12 weeks, including regular visits and assessements. Data from the screening period was used as baseline data for the evaluation of trial endpoints. However, only patients eventually included into the treatment phase are analysed, and therefore baseline data is reported for patients who entered the second period only. |
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Baseline characteristics reporting groups
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Reporting group title |
Treatment
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Reporting group description |
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End points reporting groups
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Reporting group title |
pre-treatment
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Reporting group description |
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Reporting group title |
recombinant growth factor
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Reporting group description |
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End point title |
Ratio pre/post of number of active lesions on brain MRI [1] | ||||||||
End point description |
Cumulative number of active (i.e. new or gadolinium-enhancing) lesions on brain MRI performed every four weeks during the 12-week baseline period and the first 12 weeks of the treatment period. That is, numbers of active lesions from week –8, week –4 and baseline will be compared to numbers of active lesions from week 4, week 8 and week 12.
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End point type |
Primary
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End point timeframe |
24 weeks
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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: This is a single arm trial. Reporting of statistical analyses in this database require at least two arms, otherwise an error message occurs. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Change in VEP P100 latencies versus baseline | ||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Pre / Post difference
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Adverse events information
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Timeframe for reporting adverse events |
Adverse events were recorded at every visit from visit 2 (-8 weeks) until the follow-up visit (week 36).
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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 |
18.0?
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Reporting groups
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Reporting group title |
Safety set
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Reporting group description |
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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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 Sep 2008 |
Change in trial product |
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31 Mar 2009 |
Change in an inclusion criterion |
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26 Nov 2009 |
Change in selection criteria |
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01 Jul 2010 |
Safety laboratory values added |
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16 Nov 2010 |
Extension of trial period |
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04 Oct 2011 |
Change in trial flow chart |
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10 Dec 2012 |
Extension of trial period |
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16 Dec 2014 |
Extension of trial period |
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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 | |||
