Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2019-001095-11 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This study is intended to assess the effects of once daily dosing of recombinant human insulin-like growth factor (rhIGF-1) in increasing height velocity.
Growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in growth hormone (GH) action. In this protocol, primary IGFD is defined as short stature (<-2 standard deviations [SDs] below the mean for age and gender), and abnormal serum IGF-1 (<-2 SDs below the mean for age and gender).
The trial is an open-label, concentration-controlled trial conducted at up to 20 centers throughout the United States.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rhIGF-1 QD | Experimental | Subjects received subcutaneous injection (SC) injections of rhIGF-1 once a day. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rhIGF-1 (mecasermin) for a period of 86 weeks | Drug | Once a day rhIGF-1 injections |
|
| Measure | Description | Time Frame |
|---|---|---|
| Height Velocity From Pretreatment (Week 0) to Week 34 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 34 heights were imputed using the last height SD score carried forward. | Pretreatment to Week 34 |
| Height Velocity From Week 34 to 86 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 86 heights were imputed using the last height SD score carried forward. | Week 34 to 86 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Height SD Score From Pretreatment to Week 34 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. The SD score is calculated as the subject value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child. |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Ipsen Study Director, M.D. | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ipsen | Brisbane | California | 94005 | United States |
The screening period consisted of two-staged clinic visits for up to 6 weeks, followed by an open-label treatment period of 86 weeks.
This was a open-label, multi-center and single-arm study conducted at 12 investigational sites (11 active) between 12 January 2005 and 14 January 2009. A total of 45 subjects were enrolled in this study.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | rhIGF-1 QD | During the treatment phase (Day 1 to Week 86), subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 microgram per kilogram (mcg/kg) once daily (QD) starting on Day 1 (Visit 3). From Week 2 (Visit 4) subsequent dose adjustments were made in order to achieve the target serum IGF-1 concentration for the subject's age and sex. The maximum dose in any circumstance was 240 mcg/kg/day. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
The Intent-to-treat (ITT) population included all treated subjects.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | rhIGF-1 QD | During the treatment phase (Day 1 to Week 86), subjects received SC injections of rhIGF-1 at an initial dose of 60 mcg/kg QD starting on Day 1 (Visit 3). From Week 2 (Visit 4) subsequent dose adjustments were made in order to achieve the target serum IGF-1 concentration for the subject's age and sex. The maximum dose in any circumstance was 240 mcg/kg/day. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Height Velocity From Pretreatment (Week 0) to Week 34 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 34 heights were imputed using the last height SD score carried forward. | The ITT population included all treated subjects. | Posted | Mean | Standard Deviation | centimeters per year (cm/yr) | Pretreatment to Week 34 |
|
Treatment emergent adverse events were collected from Day 1 to Week 86 (approximately 21 months).
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | rhIGF-1 QD | During the treatment phase (Day 1 to Week 86), subjects received SC injections of rhIGF-1 at an initial dose of 60 mcg/kg QD starting on Day 1 (Visit 3). From Week 2 (Visit 4) subsequent dose adjustments were made in order to achieve the target serum IGF-1 concentration for the subject's age and sex. The maximum dose in any circumstance was 240 mcg/kg/day. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastroenteritis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal Pain | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Ipsen | see email | clinical.trials@ipsen.com |
| ID | Term |
|---|---|
| D006130 | Growth Disorders |
| ID | Term |
|---|---|
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C000604197 | mecasermin |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Pretreatment and Week 34 |
| Change in Height SD Score From Pretreatment to Week 86 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Subjects were repositioned between each measurement. The SD score is calculated as the patient value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child. | Pretreatment and Week 86 |
| Change in Bone Age From Pretreatment to Week 86 Minus Change in Chronological Age | Plain X-rays of the left hand and wrist were exposed for bone age appraisal. The films were sent to a central facility for standardized evaluation. | Pretreatment to Week 86 |
| Percent Change in Serum Concentration of IGFBP-1, IGFBP-2 and IGFBP-3 From Pretreatment to Week 86 | Growth factor panels for measuring IGFBP-1, IGFBP-2 and IGFBP-3 were evaluated from screening and at each study visit up to Week 86. Inter-quartile range (Q1-Q3) is 10th to 90th percentile. | Pretreatment and Week 86 |
| Percent Change in Serum Concentration of ALS From Pretreatment to Week 86 | Growth factor panels for measuring ALS were evaluated from screening and at each study visit up to Week 86. Inter-quartile range (Q1-Q3) is 10th to 90th percentile. | Pretreatment and Week 86 |
| Other |
|
| Lost to Follow-up |
|
| years |
|
| Age, Customized | Count of Participants | Participants | No |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | participants |
|
| Body Mass Index Standard Deviation (SD) Score | Mean | Standard Deviation | SDs |
|
| Bone Age Imputed | Mean | Standard Deviation | years |
|
| Height for Age SD Score | Mean | Standard Deviation | SDs |
|
| IGFBP-3 SD Score | Mean | Standard Deviation | SDs |
|
| IGF-1 SD Score | Mean | Standard Deviation | SDs |
|
| Maximum Stimulated GH | Mean | Standard Deviation | nanogram per milliliter |
|
| Weight for Age SD Score | Mean | Standard Deviation | SDs |
|
|
|
| Primary | Height Velocity From Week 34 to 86 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 86 heights were imputed using the last height SD score carried forward. | The ITT population included all treated subjects. Only subjects in the ITT population who continued past Week 34 were included in the analysis. | Posted | Mean | Standard Deviation | cm/yr | Week 34 to 86 |
|
|
|
| Secondary | Change in Height SD Score From Pretreatment to Week 34 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. The SD score is calculated as the subject value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child. | The ITT population included all treated subjects. | Posted | Mean | Standard Deviation | SDs | Pretreatment and Week 34 |
|
|
|
| Secondary | Change in Height SD Score From Pretreatment to Week 86 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Subjects were repositioned between each measurement. The SD score is calculated as the patient value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child. | The ITT population included all treated subjects. Only subjects who had both pretreatment and Week 86 measurements were included in the analysis. | Posted | Mean | Standard Deviation | SDs | Pretreatment and Week 86 |
|
|
|
| Secondary | Change in Bone Age From Pretreatment to Week 86 Minus Change in Chronological Age | Plain X-rays of the left hand and wrist were exposed for bone age appraisal. The films were sent to a central facility for standardized evaluation. | The ITT population included all treated subjects. Only subjects who had both pretreatment and Week 86 measurements were included in the analysis. | Posted | Mean | Standard Deviation | years | Pretreatment to Week 86 |
|
|
|
| Secondary | Percent Change in Serum Concentration of IGFBP-1, IGFBP-2 and IGFBP-3 From Pretreatment to Week 86 | Growth factor panels for measuring IGFBP-1, IGFBP-2 and IGFBP-3 were evaluated from screening and at each study visit up to Week 86. Inter-quartile range (Q1-Q3) is 10th to 90th percentile. | The ITT population included all treated subjects. Only subjects who had both pretreatment and Week 86 measurements were included in the analysis. | Posted | Median | Inter-Quartile Range | percent change | Pretreatment and Week 86 |
|
|
|
| Secondary | Percent Change in Serum Concentration of ALS From Pretreatment to Week 86 | Growth factor panels for measuring ALS were evaluated from screening and at each study visit up to Week 86. Inter-quartile range (Q1-Q3) is 10th to 90th percentile. | The ITT population included all treated subjects. Only subjects who had both pretreatment and Week 86 measurements were included in the analysis. | Posted | Median | Inter-Quartile Range | percent change | Pretreatment and Week 86 |
|
|
|
| Post-Hoc | Increase in Height Velocity From Pretreatment to Week 34 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 34 heights were imputed using the last height SD score carried forward. | Completer population included all subjects who remained on study to Week 86. Subjects who completed Week 34 were included in the analysis. | Posted | Mean | Standard Deviation | cm/yr | Pretreatment to Week 34 |
|
|
|
| Post-Hoc | Increase in Height Velocity From Pretreatment to Week 86 | Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 86 heights were imputed using the last height SD score carried forward. | Completer population included all subjects who remained on study to Week 86. | Posted | Mean | Standard Deviation | cm/yr | Pretreatment to Week 86 |
|
|
|
| 0 |
| 45 |
| 2 |
| 45 |
| 41 |
| 45 |
| Arnold-Chiari Malformation | Congenital, familial and genetic disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Syringomyelia | Nervous system disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Stomach Discomfort | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Injection Site Bruising | General disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Gastroenteritis Viral | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Otitis Media | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Viral Infection | Infections and infestations | MedDRA (11.0) | Non-systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Pharyngolaryngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Dermatitis Contact | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (11.0) | Non-systematic Assessment |
|
Not provided
| Title | Measurements |
|---|---|
|