A Phase 3 interventional study of rh IGF-1 (mecasermin) in Growth Disorders, sponsored by Ipsen. Terminated at 1 site in France. Open to participants aged 4 Years to 15 Years. Per ClinicalTrials.gov, last updated 2020-08-14.
Sponsored by Ipsen · Phase 3, Interventional, and Treatment
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and adolescents treated for primary IGF-1 deficiency (IGFD). The secondary objective is to use the data collected to learn more about the relationship of IGF-1 exposure to the promotion of normal growth and pubertal development.
Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action. In this protocol, subjects that have completed one year of mecasermin treatment on Tercica protocol MS301 (NCT00125164) will be allowed to enroll in this extension study. All subjects were planned to receive treatment.
This is a Phase IIIb open-label, multi-center, parallel dose, extension study conducted in approximately 40 centers across the United States.
227 studies on the registry are indexed under Growth Disorders; 25 are open to participants now.
This study's enrollment of 114 is below the median of 413 across 143 interventional studies indexed under Growth Disorders.
Browse Growth Disorders studies →Ipsen is the lead sponsor of 289 studies on the registry; 17 are open to participants now.
Of its 26 completed or terminated interventional studies of FDA-regulated products, 22 (85%) have results posted.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All subjects entering MS306 began recombinant human insulin-like growth factor-1 (rhIGF-1) twice a day (BID) treatment. Each subject treated in MS301 had an MS306 starting dose that was based on their dose at the completion of MS301 (i.e. subcutaneous injections of rhIGF-1 at 40, 80, or 120 micrograms \[μg\]/ kilogram \[kg\] BID). MS301 untreated control subjects were randomised in MS306 in a 1:1 ratio to a dose of either 80 or 120 μg/kg rhIGF-1 BID. Following Protocol Amendment 1, all subjects received either 80 or 120 μg/kg rhIGF-1 BID until the implementation of Protocol Amendment 2. Following Protocol Amendment 2, all subjects were first switched to receive subcutaneous injections of 160 μg/kg rhIGF-1 once a day (QD), followed by individual dose-escalation first to 200 μg/kg rhIGF-1 QD and subsequently to a targeted maximum dose of 240 μg/kg rhIGF-1 QD. Subjects were treated QD until the early termination of the study.
Drug: rh IGF-1 (mecasermin)
Patients from untreated arm for prior study MS301 (NCT00125164) were randomized to a dose of either 80 or 120 mcg/kg twice daily. For patients receiving active treatment in previous study MS 301 (NCT00125164), they started on a dose of 80 or 120 mcg/kg twice daily based on the dose reached at end of the previous study. Following a protocol amendment in May 2009, all patients were switched to once daily doses of 160 µg/kg, escalated to a targeted maximum dose of 240 µg/kg.
Also known as: Increlex
Height Velocity During BID Dosing Period
Height was measured standing, without shoes, as the average of 3 measurements by the same observer identical technique with a Harpenden or other wall-mounted stadiometer at baseline and each study visit up to 3 years. Height velocity (during any interval of time (annualised) is computed as (height on date 2 - height on date 1)/(age on date 2 - age on date 1) where height is expressed as centimetres so that height velocity is expressed as centimetres per year (cm/yr). Height velocity is presented for subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
Time frame: At Years 1, 2 and 3 in BID dosing period.
Mean Change From Baseline in Height Standard Deviation (SD) Score During BID Dosing Period
Height was measured standing, without shoes, as the average of 3 measurements by the same observer using identical technique with a Harpenden or other wall-mounted stadiometer at baseline and each study visit up to 3 years. Height SD score was determined using the National Center for Health Statistics 2000 data as provided by the Center for Disease Control. The SD score was 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. Mean change from baseline in height SD score is presented for all subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
Time frame: At baseline and Years 1, 2 and 3 in BID dosing period.
Mean Change From Baseline in Body Mass Index (BMI) SD Score During BID Dosing Period
BMI SD score was calculated using the National Center for Health Statistics 2000 data as provided by the Center for Disease Control. The SD score was 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. Mean change from baseline in BMI SD score is presented for all subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
Time frame: At baseline and Years 1, 2 and 3 in BID dosing period.
Mean Change From Baseline in Bone Age During BID Dosing Period
Radiographs of the left hand and wrist were taken on an approximately annual basis for determination of bone (skeletal) age. The films were sent to a central facility for standardised evaluation. Mean change from baseline in bone age is presented for all subjects completing each year of BID treatment (i.e. Year1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
Time frame: At baseline and Years 1, 2 and 3 in BID dosing period.
Mean Change From Baseline in Predicted Adult Height During BID Dosing Period
Predicted adult heights were estimated using the Roche-Wainer-Theissen method which takes into account changes in age, height and bone age. Mean change from baseline in predicted adult height is presented for all subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
Time frame: At baseline and Years 1, 2 and 3 in BID dosing period.
All subjects that completed study MS301 (NCT00125164) were eligible to enter this Phase 3b, open-label, extension study in prepubertal and pubertal male and female subjects with growth failure associated with primary insulin-like growth factor-1 deficiency (IGFD). This study (MS306) was terminated early by the sponsor on 01 December 2009.
| Milestone | All rhIGF-1 Subjects |
|---|---|
| Started | 114 |
| Completed | 78 |
| Not completed | 36 |
| Withdrew: Adverse event | 1 |
| Withdrew: Lost to follow-up | 7 |
| Withdrew: Non-compliance | 5 |
| Withdrew: Withdrawal by subject | 19 |
| Withdrew: Sponsor's decision | 1 |
| Withdrew: Other | 3 |
| Milestone | All rhIGF-1 Subjects |
|---|---|
| Started | 78 |
| Completed | 0 |
| Not completed | 78 |
| Withdrew: Sponsor decision to terminate study | 74 |
| Withdrew: Other | 1 |
| Withdrew: Withdrawal by subject | 1 |
| Withdrew: Lost to follow-up | 2 |
Height was measured standing, without shoes, as the average of 3 measurements by the same observer identical technique with a Harpenden or other wall-mounted stadiometer at baseline and each study visit up to 3 years. Height velocity (during any interval of time (annualised) is computed as (height on date 2 - height on date 1)/(age on date 2 - age on date 1) where height is expressed as centimetres so that height velocity is expressed as centimetres per year (cm/yr). Height velocity is presented for subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
| cm/year | 120 μg/kg rhIGF-1 BID (MITT Population) |
|---|---|
| Year 1 | 7.7 ± 1.5 |
| Year 2 | 6.1 ± 1.4 |
| Year 3 | 5.9 ± 1.1 |
Height was measured standing, without shoes, as the average of 3 measurements by the same observer using identical technique with a Harpenden or other wall-mounted stadiometer at baseline and each study visit up to 3 years. Height SD score was determined using the National Center for Health Statistics 2000 data as provided by the Center for Disease Control. The SD score was 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. Mean change from baseline in height SD score is presented for all subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
| SD score/year | 120 μg/kg rhIGF-1 BID (MITT Population) |
|---|---|
| Year 1 | 0.5 ± 0.3 |
| Year 2 | 0.7 ± 0.4 |
| Year 3 | 0.9 ± 0.6 |
BMI SD score was calculated using the National Center for Health Statistics 2000 data as provided by the Center for Disease Control. The SD score was 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. Mean change from baseline in BMI SD score is presented for all subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
| SD score/year | 120 μg/kg rhIGF-1 BID (MITT Population) |
|---|---|
| Year 1 | 0.3 ± 0.5 |
| Year 2 | 0.2 ± 0.5 |
| Year 3 | 0.4 ± 0.5 |
Radiographs of the left hand and wrist were taken on an approximately annual basis for determination of bone (skeletal) age. The films were sent to a central facility for standardised evaluation. Mean change from baseline in bone age is presented for all subjects completing each year of BID treatment (i.e. Year1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
| Years | 120 μg/kg rhIGF-1 BID (MITT Population) |
|---|---|
| Year 1 | 1.2 ± 0.5 |
| Year 2 | 2.3 ± 0.6 |
| Year 3 | 3.4 ± 0.7 |
Predicted adult heights were estimated using the Roche-Wainer-Theissen method which takes into account changes in age, height and bone age. Mean change from baseline in predicted adult height is presented for all subjects completing each year of BID treatment (i.e. Year 1 \[0-1 years\], Year 2 \[1-2 years\], Year 3 \[2-3 years\]).
| cm | 120 μg/kg rhIGF-1 BID (MITT Population) |
|---|---|
| Year 1 | 2.7 ± 2.0 |
| Year 2 | 3.9 ± 3.2 |
| Year 3 | 3.7 ± 3.4 |
Collected over Adverse events were collected for the overall study (including both the BID and QD dosing periods) from Day 1 until early termination of the study (up to 4 years, 3 months).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| All rhIGF-1 Subjects | 0/114 (0%) | 6/114 (5.3%) | 107/114 (93.9%) |
| Event | All rhIGF-1 Subjects |
|---|---|
| AppendicitisInfections and infestations | 1/114 |
| CholelithiasisHepatobiliary disorders | 1/114 |
| Forearm FractureInjury, poisoning and procedural complications | 1/114 |
| HypoglycaemiaMetabolism and nutrition disorders | 1/114 |
| Otitis ExternaInfections and infestations | 1/114 |
| PneumoniaInfections and infestations | 1/114 |
| Event | All rhIGF-1 Subjects |
|---|---|
| HypoglycaemiaMetabolism and nutrition disorders | 46/114 |
| Upper Respiratory Tract InfectionInfections and infestations | 38/114 |
| HeadacheNervous system disorders | 35/114 |
| PyrexiaGeneral disorders | 28/114 |
| Pharyngitis StreptococcalInfections and infestations | 23/114 |
| VomitingGastrointestinal disorders | 23/114 |
| InfluenzaInfections and infestations | 22/114 |
| NasopharyngitisInfections and infestations | 22/114 |
| CoughRespiratory, thoracic and mediastinal disorders | 21/114 |
| Abdominal Pain UpperGastrointestinal disorders | 20/114 |
| Age, Continuous(years) | All rhIGF-1 Subjects |
|---|---|
| Mean | 8.5 ± 2.4 |
| Sex: Female, Male(Participants) | All rhIGF-1 Subjects |
|---|---|
| Female | 32 |
| Male | 82 |
| Race/Ethnicity, Customized(Participants) | All rhIGF-1 Subjects |
|---|---|
| Hispanic | 6 |
| White | 100 |
| Black | 1 |
| Asian | 4 |
| Native Hawaiian | 1 |
| Other | 2 |
The record gives a start of Nov 2005, a primary completion of Feb 2010 and a completion of Mar 2010. It does not say whether these dates are actual or estimated.
Results were first posted in Jun 2011, 1 year 4 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Aug 2020. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Mecasermin