A Double blind, third party open, single dose study to explore the safety, pharmacokinetics and pharmacodynamics of pha-794428 in paediatric patients with growth hormone deficiency - N/A

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2005-005593-79-GB
• Date of registration: 21/03/2006
• Prospective Registration: Yes
• Primary sponsor: Pfizer Limited
• Public title: A Double blind, third party open, single dose study to explore the safety, pharmacokinetics and pharmacodynamics of pha-794428 in paediatric patients with growth hormone deficiency - N/A
• Scientific title: A Double blind, third party open, single dose study to explore the safety, pharmacokinetics and pharmacodynamics of pha-794428 in paediatric patients with growth hormone deficiency - N/A
• Date of first enrolment: 02/06/2006
• Target sample size: 32
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2005-005593-79
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: yes Other trial design description: Third party open to study site pharmacist If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Germany, Spain, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male and female children with a minimum age of 6 years.

2. Pre-pubertal as defined by Tanner staging (Tanner JM, 1962).

3. Severe growth hormone deficiency as defined by GRS Guidelines; GH<8 ng/ml on dynamic testing, together with serum IGF-1 level =-1.5 SD. Evidence of severe GHD should be demonstrated by 1 dynamic test if there is supporting pathology, or 2 dynamic tests in the absence of supporting pathology.

4. Growth hormone pre-treatment for at least 6 months prior to the screening visit.
Are the trial subjects under 18? yes
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:
1. PGHD subjects with uncontrolled pituitary tumor growth. Subjects with pituitary tumors must have demonstrated stable neuro-imaging within the last 12 months of the screening visit.

2. Tumors within 3 mm of the optic chiasm. Subjects with pituitary suprasellar disease must be scanned within 3 months of the screening visit.

3. Serum ALT and/or AST = 1.5 times the upper limit of normal range (ULN), or clinically significant hepatic disease.

4. Subjects with diabetes mellitus.

5. Any defined syndrome with short stature

6. Subjects with birth weight and/or birth length = 10th centile.

7. Subjects with body weight below 16 kg.


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

Health Condition(s) or Problem(s) studied

Paediatric Growth Hormone Deficiency
MedDRA version: 8.1 Level: LLT Classification code 10056438

Intervention(s)

Product Name: N/A
Product Code: PHA-794428
Pharmaceutical Form: Powder for injection*
INN or Proposed INN: N/A
CAS Number: N/A
Current Sponsor code: N/A
Other descriptive name: Pegylated polypeptide hormone of human recombinant origin PEG-hGH
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Powder for injection*
Route of administration of the placebo: Subcutaneous use

Product Name: N/A
Product Code: PHA-794428
Pharmaceutical Form: Powder for injection*
INN or Proposed INN: N/A
CAS Number: N/A
Current Sponsor code: N/A
Other descriptive name: Pegylated polypeptide hormone of human recombinant origin PEG-hGH
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Powder for injection*
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Main Objective: To explore the pharmacokinetics and pharmacodynamics of PHA-794428 after single subcutaneous injections in PGHD patients in order to support the development of a PK/PD model in this patient population.

To explore the safety, tolerance and humoral response of PHA-794428 after single subcutaneous injections in PGHD patients.

Primary end point(s): · Safety and tolerance of a single subcutaneous injection of PHA-794428: Monitoring of AE, safety lab, vital signs, physical examination, body temperature, signs of fluid retention (body weight), reactions at the injection site (Draize scoring and Gracely Box scale).

· Pharmacodynamic effects of PHA-794428 on IGF-1.

· PHA-794428 pharmacokinetics (AUCinf, AUClast, Cmax, Tmax, t1/2) after single subcutaneous injection.

Secondary Objective:

Secondary ID(s)

N/A

A6391004