Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
This study has been terminated.
(Unacceptable frequency of hypoglycemia observed at and above 200 ug/kg/day)
Sponsor:
Ipsen
Information provided by:
Ipsen
ClinicalTrials.gov Identifier:
NCT00330668
First received: May 26, 2006
Last updated: May 31, 2011
Last verified: May 2011
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Results First Received: February 28, 2011
| Study Type: | Interventional |
|---|---|
| Study Design: | Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment |
| Condition: |
Growth Disorders |
| Intervention: |
Drug: rh IGF-1 (mecasermin) |
Participant Flow
Recruitment Details
| Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations |
|---|
|
Study Initiation Date: 21 OCT 2005. Study completion Date: 19 JAN 2010. 44 investigators enrolled 114 subjects. Patients who completed a prior Tercica study, MS301 (NCT00125164). |
Pre-Assignment Details
| Significant events and approaches for the overall study following participant enrollment, but prior to group assignment |
|---|
| Patients initially received either mecasermin 80 or 120 µg/kg twice daily. Patients who received 80 µg/kg then switched to receive 120 µg/kg. During the once daily period, all patients were first switched to receive mecasermin 160 µg/kg, followed by individual dose-escalation to a targeted maximum dose of 240 µg/kg. |
Reporting Groups
| Description | |
|---|---|
| rhIGF-1 Injection | All patients entering study began rhIGF-1 twice daily treatment (range from 40 ug/kg to 120 ug/kg). Following protocol Amendment 2, all patients first received 160 ug/kg once daily followed by individual dose-escalation to 240 ug/kg once daily. |
Participant Flow: Overall Study
| rhIGF-1 Injection | |
|---|---|
| STARTED | 114 |
| COMPLETED | 0 |
| NOT COMPLETED | 114 |
| Adverse Event | 1 |
| Lost to Follow-up | 9 |
| Non-Compliance | 5 |
| Withdrawal by Subject | 20 |
| Sponsor's decision | 76 |
| Lack of Efficacy | 1 |
| Use of exclusionary medication | 1 |
| Patient started Lupron | 1 |
Baseline Characteristics
Reporting Groups
| Description | |
|---|---|
| rhIGF-1 Injection | All patients entering study began rhIGF-1 twice daily treatment (range from 40 ug/kg to 120 ug/kg). Following protocol Amendment 2, all patients first received 160 ug/kg once daily followed by individual dose-escalation to 240 ug/kg once daily. |
Baseline Measures
| rhIGF-1 Injection | |
|---|---|
|
Number of Participants
[units: participants] |
114 |
|
Age
[units: years] Mean ± Standard Deviation |
8.5 ± 2.4 |
|
Gender
[units: participants] |
|
| Female | 32 |
| Male | 82 |
|
Race/Ethnicity, Customized
[units: Participants] |
|
| Hispanic | 6 |
| White | 100 |
| Black | 1 |
| Asian | 4 |
| Native Hawaiian | 1 |
| Other | 2 |
|
Region of Enrollment
[units: participants] |
|
| United States | 114 |
|
Height Standard Deviation (SD) Score
[1] [units: SDĀ score] Mean ± Standard Deviation |
-2.4 ± 0.7 |
|
IGF-1 Standard Deviation (SD) Score
[1] [units: SDĀ score] Mean ± Standard Deviation |
-0.9 ± 1.4 |
|
Maximum Stimulated Growth Hormone
[2] [units: ng/mL] Mean ± Standard Deviation |
19.2 ± 12.9 |
|
Weight
[units: Kg] Mean ± Standard Deviation |
21.8 ± 6.2 |
| [1] | Please note that SD is a standard term used in growth studies and represents Standard Deviations calculated as the patient value minus the mean divided by the standard deviation. Standard Deviation Scores vary depending on the age and sex of the child. |
|---|---|
| [2] | Growth Hormone Stimulation Test results expressed in ng/mL. |
Outcome Measures
| 1. Primary: | Height Velocity in Modified Intent-to-Treat Population (ITT Patients Randomized to 120 Mcg/kg Twice Daily) [ Time Frame: after one year of treatment ] |
| 2. Secondary: | Height Velocities During Subsequent Years of rh IGF-1 Treatment [ Time Frame: after 2, 3 and 5 years of treatment ] |
| 3. Secondary: | Height Velocity Standard Deviation (SD) Score [ Time Frame: during the course of the study ] |
| 4. Secondary: | Height SD Score [ Time Frame: during the course of the study ] |
More Information
Certain Agreements:
Limitations and Caveats
Results Point of Contact:
No publications provided
| Principal Investigators are NOT employed by the organization sponsoring the study. | ||||||
| There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. | ||||||
The agreement is:
|
Limitations and Caveats
| Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data |
|---|
| The study was terminated by the sponsor on identification of an unacceptable frequency of hypoglycemia which was reported at doses of ≥200 mcg/kg once daily. Only safety results are presented as efficacy data were not analyzed according to protocol. |
Results Point of Contact:
Name/Title: Sr Vice President, Clinical Development and Medical Affairs
Organization: Ipsen, US
phone: clinical.trials@ipsen.com
e-mail: clinical.trials@ipsen.com
Organization: Ipsen, US
phone: clinical.trials@ipsen.com
e-mail: clinical.trials@ipsen.com
No publications provided
| Responsible Party: | Senior Vice President, Clinical Development and Medical Affairs, Ipsen |
| ClinicalTrials.gov Identifier: | NCT00330668 History of Changes |
| Other Study ID Numbers: | MS306 |
| Study First Received: | May 26, 2006 |
| Results First Received: | February 28, 2011 |
| Last Updated: | May 31, 2011 |
| Health Authority: | United States: Food and Drug Administration |