Clinical Trial Results:
An Open Label Follow-up Study of Patients Who Participated in Clinical Study B9R-HL-GDDV
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Summary
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EudraCT number |
2007-000469-39 |
Trial protocol |
FI |
Global end of trial date |
21 Sep 2007
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Results information
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Results version number |
v1(current) |
This version publication date |
23 Aug 2019
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First version publication date |
23 Aug 2019
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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 |
B9R-HL-GDGN
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
11890: Trial ID | ||
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Sponsors
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Sponsor organisation name |
Eli Lilly and Company
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Sponsor organisation address |
Lilly Corporate Center, Indianapolis, IN, United States, 46285
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Public contact |
Available Mon - Fri 9 AM - 5 PM EST, Eli Lilly and Company, 1 877-CTLilly,
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Scientific contact |
Available Mon - Fri 9 AM - 5 PM EST, Eli Lilly and Company, 1 877-285-4559,
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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? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
21 Sep 2007
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
21 Sep 2007
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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 |
The primary objective of this study is to obtain adult height measurement on all 20 subjects with Prader Willi Syndrome (PWS) who participated in the one-year trial (B9R-HL-GDDV) of GH treatment in 1997 – 1999. In patients who have achieved their final height, the result will be compared to the predicted adult heights from the previous study (before starting GH and at one year).
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Protection of trial subjects |
This study was conducted in accordance with International Conference on Harmonization (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
04 May 2007
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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 |
Finland: 20
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Worldwide total number of subjects |
20
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EEA total number of subjects |
20
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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) |
20
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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 |
All participants who had received Growth Hormone (GH) therapy in study B9R-HL-GDDV and completed that study were eligible to participate in the B9R-HL-GDGN study. | ||||||||||
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Pre-assignment
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Screening details |
No text entered. | ||||||||||
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Period 1
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Period 1 title |
Overall (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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All Participants | ||||||||||
Arm description |
All participants who were diagnosed with Prader-Willi syndrome as a child and received recombinant human growth hormone (rhGH) treatment in study B9R-HL-GDDV. B9R-HL-GDGN is a non-drug, non-interventional study. | ||||||||||
Arm type |
Standard Care | ||||||||||
Investigational medicinal product name |
Recombinant Human Growth Hormone (rhGH)
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Injection
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Routes of administration |
Subcutaneous use
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Dosage and administration details |
B9R-HL-GDGN was a non-drug interventional study. Recombinant growth hormone therapy was implemented according to normal clinical practice.
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Baseline characteristics reporting groups
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Reporting group title |
Overall
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Reporting group description |
All participants who agreed to participate in the 10-year follow-up study B9R-HL-GDGN. | ||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
All Participants
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Reporting group description |
All participants who were diagnosed with Prader-Willi syndrome as a child and received recombinant human growth hormone (rhGH) treatment in study B9R-HL-GDDV. B9R-HL-GDGN is a non-drug, non-interventional study. | ||
Subject analysis set title |
Male participants
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Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
Male participants.
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Subject analysis set title |
Female Participants
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Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
Female participants.
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End point title |
Mean Adult Height Measurement [1] | ||||||||||||
End point description |
Mean adult height measurement of participants who achieved their final height.
Analysis Population Description: All participants who agreed to participate in this 10-year follow-up study and achieved final height.
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End point type |
Primary
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End point timeframe |
10 Year Follow-up Visit
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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 study, there are no comparison groups for statistical analysis. |
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Expected Adult Height Standard Deviation Score (SDS) Compared to Achieved Adult Height SDS | ||||||||||||
End point description |
Standard Deviation Score reports the number of standard deviations from the mean for age and sex for an individual measurement (normal range: -2 to +2 SDS). Height SDS is derived by subtracting the population mean from individual's height value and then dividing that difference by the population standard deviation. Greater height SDS values indicates greater height.
Analysis Population Description: All participants who agreed to participate in the 10-year follow-up study and achieved adult height.
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End point type |
Secondary
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End point timeframe |
10 Year Follow-up Visit
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Body Composition (Fat vs. Lean Body Mass [LBM]) Assessed by Dual-energy X-ray Absorptiometry (DEXA) | ||||||||||||
End point description |
Body composition (lean body mass and fat mass) was assessed using dual energy x-ray absorptiometry (DEXA).
Analysis Population Description: All participants who a participated in this 10-year follow-up study.
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End point type |
Secondary
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End point timeframe |
10 Year Follow-up Visit
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Health Related Quality of Life (HRQol) 16D | ||||||||||||||||||||||||||||||||||||||||||
End point description |
The HRQoL 16D is a generic self-assessment measure of health-related dimensions: mobility, vision, hearing, breathing, sleeping, eating, elimination, speech, mental function, discomfort and symptoms, school and hobbies, friends, physical appearance, depression, distress, and vitality. Within each dimension are 5 ordinal levels (1-5) by which that attribute is distinguished. The participant selects the level within in each dimension that best describes his/her current health status. The higher number indicates better health. Calculation of the final score by algorithm has a maximum score of 1.0. The 16D score -representing overall health and quality of life with a range from 0 (worst possible) to 1 (best possible) -is calculated by combining previously determined dimension importance ratings and desirability values with the participants own assessment of each dimension.
Population Analysis Description: All participants who agreed to participate in this 10-year follow-up study.
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End point type |
Secondary
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End point timeframe |
10 Year Follow-up Visit
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||
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End point title |
Total Weight | ||||||||||||
End point description |
Analysis Population Description: All participants who agreed to participate in this 10-year follow-up study.
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End point type |
Secondary
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End point timeframe |
10 Year Follow-up Visit
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Sleep Polygraphy | ||||||||||||
End point description |
Sleep polygraphy was performed at the 1- year follow-up visit using a portable monitoring device (PM). Results of the sleep polygraphy were graded as normal, slightly abnormal, and markedly abnormal based on the rate of occurrence of apnea/hypopnea and other findings, including sleep position and reactions to apnea events.
Analysis Population Description:All participants who agreed to participate in this 10-year follow-up study and had evaluable polygraphy.
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End point type |
Secondary
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End point timeframe |
10 Year Follow-up Visit
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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 |
Entire study.
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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 |
10.0
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Reporting groups
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Reporting group title |
Overall
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Reporting group description |
All participants who a participated in 10-year follow-up study. | ||||||||||
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| Frequency threshold for reporting non-serious adverse events: 0% | |||||||||||
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| 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: There were no adverse events for this trial. |
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
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 | |||
