AN EXPLORATORY, OPEN, TWO-ARM, MATCH-CONTROL, RANDOMISED AND STRATIFIED PILOT PHASE II STUDY ASSESSING THE PREVENTION OF THE ADVERSE EFFECTS OF LONG-TERM HIGH-DOSE GLUCOCORTICOID THERAPY BY GROWTH HORMONE (NutropinAqTM) ADMINISTERED BY SUB-CUTANEOUS ROUTE IN CHILDREN WITH CHRONIC DISEASE

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2004-001164-41-SE
• Date of registration: 29/09/2004
• Prospective Registration: Yes
• Primary sponsor: Beaufour Ipsen Pharma
• Public title: AN EXPLORATORY, OPEN, TWO-ARM, MATCH-CONTROL, RANDOMISED AND STRATIFIED PILOT PHASE II STUDY ASSESSING THE PREVENTION OF THE ADVERSE EFFECTS OF LONG-TERM HIGH-DOSE GLUCOCORTICOID THERAPY BY GROWTH HORMONE (NutropinAqTM) ADMINISTERED BY SUB-CUTANEOUS ROUTE IN CHILDREN WITH CHRONIC DISEASE
• Scientific title: AN EXPLORATORY, OPEN, TWO-ARM, MATCH-CONTROL, RANDOMISED AND STRATIFIED PILOT PHASE II STUDY ASSESSING THE PREVENTION OF THE ADVERSE EFFECTS OF LONG-TERM HIGH-DOSE GLUCOCORTICOID THERAPY BY GROWTH HORMONE (NutropinAqTM) ADMINISTERED BY SUB-CUTANEOUS ROUTE IN CHILDREN WITH CHRONIC DISEASE
• Date of first enrolment: 25/11/2004
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-001164-41
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: yes Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Sweden

Key inclusion & exclusion criteria

Inclusion criteria:
Patients MUST satisfy all of the following inclusion criteria before they will be allowed to participate in the study:
a) The patient (if applicable) and his/her parents must give written (personally signed and dated) informed consent before completing any study-related procedure, which means any assessment or evaluation that would not have formed part of the normal medical care of the patient,
b) Male or female child aged between 4 years and bone age of 12 years for girls and bone age of 14 years for boys,
c) Child with chronic disease condition that, based on clinical experience, requires long-term (3 months or more) and high-dose (2 mg/kg/day or more hydrocortisone-equivalent) glucocorticoid treatment: neurologic disorders (Duchene or Beckers), nephrotic syndrome, juvenile rhumatoid arthritis (JRA) or inflammatory bowel disease (IBD),
d) Naïve patient with respect to previous glucocorticoid treatment (history of less than 2-week treatment at any time and no previous glucocorticoid treatment during the past 2 months before study entry),
e) No investigational / commercial agents or therapies other than NutropinAqTM may be administered to the patient with the intent to treat the adverse consequences of glucocorticoid treatment in the timeframe of the study.

Are the trial subjects under 18?
Number of subjects for this age range:
F.1.2 Adults (18-64 years)
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years)
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
Patients presenting with any of the following MUST NOT be included in the study:
a) History of hypersensitivity to any growth hormone or drugs with a similar chemical structure,
b) History of hypersensitivity to any of the NutropinAqTM excipients (phenol, polysorbate 20, sodium citrate or citric acid)
c) Treatment with any other investigational drug within the last 30 days before study entry,
d) Likely to require treatment during the study with drugs that are not permitted by the study protocol (see Section 8.7),
e) Abnormal baseline findings considered by the investigator to indicate conditions that might affect the study results, and in particular fasting glucose > 7 mmol/L, HbA1c > 5.3% or HOMA IR > 4.8,
f) Child with a disease or syndrome affecting growth, osteoporosis, diabetes mellitus, neoplasm or multiple accidental traumas,
g) Child with an acute critical illness or hospitalised in intensive care unit,
h) Patient likely to be non-compliant with the protocol and the follow-up,
i) Any mental condition rendering the subject unable to understand the nature, scope and possible consequences of the study, and/or evidence of an unco operative attitude, unless the disease under study is such that the subject presents with such symptoms and the consent of a legal guardian or carer must be sought (providing the latter is permitted by the local legislation).
j) Any other condition that, in the opinion of the investigator, might increase the risk to the subject or decrease the chance of obtaining satisfactory data needed to achieve the objective(s) of the study.
Under no circumstances will subjects be enrolled more than once.



Age minimum:
Age maximum:
Gender:
Female:
Male:

Health Condition(s) or Problem(s) studied

Adverse effects of the long-term high-dose glucocorticoid administration in naïve children with chronic disease.

Intervention(s)

Trade Name: NutropinAq
Product Name: NutropinAq
Pharmaceutical Form: Solution for injection

Primary Outcome(s)

Main Objective: The primary objective of the study is to determine whether the administration of growth hormone will result in the prevention/reduction of adverse effects induced by long-term high-dose glucocorticoid therapy when administered concomitantly in children with chronic disease, evaluated by assessing the bone function, the growth and the glucidic and lipidic metabolisms.

Primary end point(s): As this clinical trial is an exploratory pilot Phase II study, no primaryendpoints are defined.
The following efficacy variables will be assessed; whole body measurements, bone measurements, physical examination measurements, muscle measurements, calcium measurements, lipid metabolism measurements, quality of life measurements and hormonal functions.

The following safety variables will be assessed; carbohydrate metabolism, Haematologyl, biochemistry, cardiographic measurements, adverse events collection and control of disease.

Pharmacokinetic and Pharmacodynamic variables are not applicable.

Secondary Objective: The secondary objective of the study is to assess the safety profile of patients suffering from chronic disease that requires long-term high-dose glucocorticoid therapy and receiving a daily sub-cutaneous injection of NutropinAqTM during at least 3 months and up to 12 months.

Secondary ID(s)

2-55-58035-002