Trial · Interventional

TerminatedPhase 3Results postedRegistry data

Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency

NCTNCT00330668Other idMS306Other id2019-000844-81Other id2019

Open at ClinicalTrials.govAsk about this trial

This trial was stopped early.

Reason given at the registry: Unacceptable frequency of hypoglycemia observed at and above 200 ug/kg/day

Results were posted for this trial.

Overview

Status: Terminated
Phase: Phase 3
Enrollment: 114 participants (actual)
Sponsor: Ipsen · Industry
Design: Allocation not applicable · Open label (no masking)
Countries: 1 country
France
Start: Nov 2005
Primary completion: Feb 2010
Completion: Mar 2010
First posted: 29 May 2006 (estimated)
Last update posted: 14 Aug 2020
Conditions: Growth Disorders

Timeline

1 trial · 1 stopped early · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

2005200620072008200920102011

Ph 3
Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
NCT00330668
Nov 2005 to Mar 2010
Terminated
Terminated

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

rh IGF-1 (mecasermin)Drug

Arms1

Experimental
All rhIGF-1 Subjects
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.

Primary outcomes1

Outcomes the sponsor listed as primary.

Height Velocity During BID Dosing Period
Time frame · At Years 1, 2 and 3 in BID dosing period.
Registry description
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 \[2-3 years\]).

Secondary outcomes(4)

Mean Change From Baseline in Height Standard Deviation (SD) Score During BID Dosing Period
Time frame · At baseline and Years 1, 2 and 3 in BID dosing period.
Registry description
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 \[2-3 years\]).
Mean Change From Baseline in Body Mass Index (BMI) SD Score During BID Dosing Period
Time frame · At baseline and Years 1, 2 and 3 in BID dosing period.
Registry description
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 \[2-3 years\]).
Mean Change From Baseline in Bone Age During BID Dosing Period
Time frame · At baseline and Years 1, 2 and 3 in BID dosing period.
Registry description
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 \[2-3 years\]).
Mean Change From Baseline in Predicted Adult Height During BID Dosing Period
Time frame · At baseline and Years 1, 2 and 3 in BID dosing period.
Registry description
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 \[2-3 years\]).

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· At Years 1, 2 and 3 in BID dosing period.

Height Velocity During BID Dosing Period

Group	Value (cm/year)	Spread
120 μg/kg rhIGF-1 BID (MITT Population)	7.7	1.5

Secondary and other outcome results(4)

Secondary outcomeMean· At baseline and Years 1, 2 and 3 in BID dosing period.

Mean Change From Baseline in Height Standard Deviation (SD) Score During BID Dosing Period

Group	Value (SD score/year)	Spread
120 μg/kg rhIGF-1 BID (MITT Population)	0.5	0.3

Secondary outcomeMean· 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

Group	Value (SD score/year)	Spread
120 μg/kg rhIGF-1 BID (MITT Population)	0.3	0.5

Secondary outcomeMean· At baseline and Years 1, 2 and 3 in BID dosing period.

Mean Change From Baseline in Bone Age During BID Dosing Period

Group	Value (Years)	Spread
120 μg/kg rhIGF-1 BID (MITT Population)	1.2	0.5

Secondary outcomeMean· At baseline and Years 1, 2 and 3 in BID dosing period.

Mean Change From Baseline in Predicted Adult Height During BID Dosing Period

Group	Value (cm)	Spread
120 μg/kg rhIGF-1 BID (MITT Population)	2.7	2.0

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
All rhIGF-1 SubjectsAll subjects entering MS306 began recombinant human insulin-like growth factor-1 (rhIGF-1) 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 μg/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 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.	0 / 114	6 / 114	107 / 114

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: All
Age: from 4 Years up to 15 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

Inclusion Criteria: * Parents or legally authorized representatives must give signed informed consent before any trial related activities are conducted * Where required, assent of the subject will be appropriately documented prior to any study related activities * Completion of assessments at Visit 9 (Month 120 of Study MS301 \[NCT00125164\]) Exclusion Criteria: * Incomplete participation in MS301 (NCT00125164) * Known or suspected allergy to the trial product (mecasermin, recombinant human IGF-1 injection) or its formulation * Development or presence of a chronic condition except as approved by the Medical Monitor * Pregnancy * Any social or medical condition that, in the opinion of the investigator, would be detrimental to either the subject or the study

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