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| Name | Class |
|---|---|
| University of Oklahoma | OTHER |
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The objective of this study was to evaluate the long-term safety and effectiveness of mecasermin (the study drug) in children with growth failure due to severe Primary insulin-like growth factor-1 deficiency (Primary IGFD).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| mecasermin, injections BID of rhIGF-1 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| mecasermin | Drug | injections BID of rhIGF-1, mecasermin |
|
| Measure | Description | Time Frame |
|---|---|---|
| Annualized Height Velocity Up to 12 Years | Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements. | Baseline (Pre-dose) and up to 12 years |
| Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment | Height measurements were performed using wall-mounted stadiometers for analysis of growth data. | Baseline (Pre-dose) and 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Height Velocity Standard Deviation Score Up to 12 Years | Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height velocity-standard deviation score was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference mean height velocity. Greater height velocity standard deviation score indicates better outcome. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ipsen | Brisbane | California | 94005 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 17192294 | Result | Chernausek SD, Backeljauw PF, Frane J, Kuntze J, Underwood LE; GH Insensitivity Syndrome Collaborative Group. Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrinol Metab. 2007 Mar;92(3):902-10. doi: 10.1210/jc.2006-1610. Epub 2006 Dec 27. |
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Participants who had been treated with mecasermin in previous Genentech-sponsored studies (F0206s, F0375g, F0632g, F0671g), as well as new participants naïve to mecasermin treatment, were enrolled in this study.
This Phase 3, open-label study was conducted in children with short stature due to severe primary insulin like growth factor-1 deficiency at 2 investigative sites in the US in conjunction with sites in 23 other countries (Argentina, Australia, Austria, Bahamas, Brazil, Canada, Egypt, France, Germany, Iran, Israel, Italy, Kuwait, Pakistan, Poland, Russia, Saudi Arabia, Spain, Sweden, Syria, Taiwan, Ukraine, and Yemen).
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| ID | Title | Description |
|---|---|---|
| FG000 | Mecasermin | Participants who were entered from previous studies continued to receive mecasermin 80 to 120 microgram per kilograms (mcg/kg) subcutaneously (SC) twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
All participants who received at least 1 dose of mecasermin treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Mecasermin | Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated. |
| 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 | Annualized Height Velocity Up to 12 Years | Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements. | The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous recombinant human insulin-like growth factor-1 (rhIGF-1) and whose pre-treatment height velocity were available were reported. | Posted | Mean | Standard Deviation | centimeter per year (cm/y) | Baseline (Pre-dose) and up to 12 years |
|
Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Mecasermin | Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA (14.1) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA (14.1) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Ipsen | see email | clinical.trials@ipsen.com |
| ID | Term |
|---|---|
| D046150 | Laron Syndrome |
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| C000604197 | mecasermin |
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| Baseline (Pre-dose) and up to 12 years |
| Height Standard Deviation Score Up to 12 Years | Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height standard deviation score was calculated as height minus reference mean height divided by standard deviation of the reference mean height. A higher height standard deviation score indicates a better outcome. | Baseline (Pre-dose) and up to 12 years |
| Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height | Height measurements were performed using wall-mounted stadiometers for analysis of growth data. | Baseline (Pre-dose) and up to 19 years |
| Poor growth |
|
| Parent/patient decision |
|
| Participant transferred to commercial drug |
|
| Unable to provide study drug |
|
| On-treatment at end of study |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants | No |
|
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
|
| Units | Counts |
|---|---|
| Participants |
|
|
| Primary | Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment | Height measurements were performed using wall-mounted stadiometers for analysis of growth data. | The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous rhIGF-1 were reported. | Posted | Count of Participants | Participants | No | Baseline (Pre-dose) and 1 year |
|
|
|
| Secondary | Height Velocity Standard Deviation Score Up to 12 Years | Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height velocity-standard deviation score was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference mean height velocity. Greater height velocity standard deviation score indicates better outcome. | The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous rhIGF-1 and whose pre-treatment height velocity were available were reported. | Posted | Mean | Standard Deviation | Standard deviation score | Baseline (Pre-dose) and up to 12 years |
|
|
|
| Secondary | Height Standard Deviation Score Up to 12 Years | Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height standard deviation score was calculated as height minus reference mean height divided by standard deviation of the reference mean height. A higher height standard deviation score indicates a better outcome. | The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous rhIGF-1 were reported. | Posted | Mean | Standard Deviation | Standard deviation score | Baseline (Pre-dose) and up to 12 years |
|
|
|
| Secondary | Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height | Height measurements were performed using wall-mounted stadiometers for analysis of growth data. | The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous rhIGF-1 and who attained near adult height were reported. | Posted | Mean | Standard Deviation | cm | Baseline (Pre-dose) and up to 19 years |
|
|
|
| 0 |
| 92 |
| 18 |
| 92 |
| 73 |
| 92 |
| Infectious pleural effusion | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Radius fracture | Injury, poisoning and procedural complications | MedDRA (14.1) | Systematic Assessment |
|
| Appendicitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Haematemesis | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Benign intracranial hypertension | Nervous system disorders | MedDRA (14.1) | Systematic Assessment |
|
| Epiphysiolysis | Musculoskeletal and connective tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Dilatation ventricular | Cardiac disorders | MedDRA (14.1) | Systematic Assessment |
|
| Grand mal convulsion | Nervous system disorders | MedDRA (14.1) | Systematic Assessment |
|
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA (14.1) | Systematic Assessment |
|
| Febrile convulsion | Nervous system disorders | MedDRA (14.1) | Systematic Assessment |
|
| Skull fracture | Injury, poisoning and procedural complications | MedDRA (14.1) | Systematic Assessment |
|
| Nephrolithiasis | Renal and urinary disorders | MedDRA (14.1) | Systematic Assessment |
|
| Hypoglycaemic seizure | Metabolism and nutrition disorders | MedDRA (14.1) | Systematic Assessment |
|
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Hypoglycaemic seizure | Metabolism and nutrition disorders | MedDRA (14.1) | Systematic Assessment |
|
| Injection site hypertrophy | General disorders | MedDRA (14.1) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (14.1) | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA (14.1) | Systematic Assessment |
|
| Injection site haematoma | General disorders | MedDRA (14.1) | Systematic Assessment |
|
| Hypertrophy | General disorders | MedDRA (14.1) | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA (14.1) | Systematic Assessment |
|
| Chest pain | General disorders | MedDRA (14.1) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Ear infection | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Pharyngitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Respiratory tract infection | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Tonsillitis | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Varicella | Infections and infestations | MedDRA (14.1) | Systematic Assessment |
|
| Snoring | Respiratory, thoracic and mediastinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Adenoidal hypertrophy | Respiratory, thoracic and mediastinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Dental caries | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Tooth crowding | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA (14.1) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (14.1) | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA (14.1) | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Skin hypertrophy | Skin and subcutaneous tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (14.1) | Systematic Assessment |
|
| Conductive deafness | Ear and labyrinth disorders | MedDRA (14.1) | Systematic Assessment |
|
| Middle ear effusion | Ear and labyrinth disorders | MedDRA (14.1) | Systematic Assessment |
|
| Deafness | Ear and labyrinth disorders | MedDRA (14.1) | Systematic Assessment |
|
| Ear pain | Ear and labyrinth disorders | MedDRA (14.1) | Systematic Assessment |
|
| Ear discomfort | Ear and labyrinth disorders | MedDRA (14.1) | Systematic Assessment |
|
| Cardiac murmur | Investigations | MedDRA (14.1) | Systematic Assessment |
|
| Thymus enlargement | Blood and lymphatic system disorders | MedDRA (14.1) | Systematic Assessment |
|
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA (14.1) | Systematic Assessment |
|
| Papilloedema | Eye disorders | MedDRA (14.1) | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA (14.1) | Systematic Assessment |
|
| Gynaecomastia | Reproductive system and breast disorders | MedDRA (14.1) | Systematic Assessment |
|
| Ear tube insertion | Surgical and medical procedures | MedDRA (14.1) | Systematic Assessment |
|
| Dental operation | Surgical and medical procedures | MedDRA (14.1) | Systematic Assessment |
|
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| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D004700 | Endocrine System Diseases |
|
| >= 2 years |
|
|
| >= 3 years |
|
|
| >= 4 years |
|
|
| >= 5 years |
|
|
| >= 6 years |
|
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| >= 7 years |
|
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| >= 8 years |
|
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| >= 9 years |
|
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| >= 10 years |
|
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| >= 11 years |
|
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| >= 12 years |
|
|
|
| >= 2 years |
|
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| >= 3 years |
|
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| >= 4 years |
|
|
| >= 5 years |
|
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| >= 6 years |
|
|
| >= 7 years |
|
|
| >= 8 years |
|
|
| >= 9 years |
|
|
| >= 10 years |
|
|
| >= 11 years |
|
|
| >= 12 years |
|
|