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

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2005-005593-79-GB

Enrollment

Registered

2006-03-21

Start date

2006-06-02

Completion date

Unknown

Last updated

2012-03-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

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 h

Eligibility

Sex/Gender

All

Inclusion 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=65 years) F.1.3.1 Number of subjects for this age range

Exclusion criteria

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.

Design outcomes

Primary

Measure	Time frame
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.;Secondary Objective: ;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.	—

Measure

Time frame

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.;Secondary Objective: ;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.

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Countries

Germany, Spain, United Kingdom

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026