A study of ACP-001, a long-acting human growth hormone product, for treatment of Growth Hormone Deficiency in pre-pubertal childre

Conditions: Growth Hormone Deficiency (GHD) in pre-pubertal children
MedDRA version: 17.1Level: PTClassification code 10056438Term: Growth hormone deficiencySystem Organ Class: 10014698 - Endocrine disorders
Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Registration Number: EUCTR2012-002787-27-HU

Lead Sponsor: Ascendis Pharma A/S

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 52

Inclusion Criteria

1. Pre-pubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage I aged:
a. Boys: 3 years = boy’s age = 12 years
b. Girls: 3 years = girl’s age = 11 years;
2. Diagnosis of GHD confirmed by two different GH provocation tests, defined as a peak GH level of = 10 ng/mL, determined by a central laboratory using a validated assay. One well documented historical test (with properly recorded sampling times and results as well as euthyroid status of the patient) performed within 3 months prior to Screening can be accepted to replace one GH stimulation test analyzed by the central laboratory;
3. Bone age (BA) not greater than the chronological age;
4. Impaired height and height velocity defined as:
a. Height (HT) of at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS = - 2.0) according to the CDC standards of 2000
b. Height velocity (HV) of at least 1 SD below the mean HV for chronological age and sex (HV SDS = - 1.0) according to the standards of Prader et al. of 1989, whereas the time between 2 height measurements is not less than 6 months;
5. BMI within ±2 SD of the mean BMI for chronological age and sex according to the 2000 CDC standards;
6. Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age and sex (IGF-I SDS = -1.0) according to the central laboratory reference values;
7. Normal fundoscopy at Screening (without signs/symptoms of intracranial hypertension);
8. Children with multiple hormonal deficiencies must be on stable replacement therapy (stable dose and normal blood hormone levels) for other hypothalamo-pituitary axes for at least 3 months. Thyroid replacement therapy for thyroid hormone deficiency must be instituted at least 6 months (and be stable for at least 3 months) prior to Screening. Temporary adjustment of glucocorticoid replacement therapy, as appropriate, is acceptable;
9. Normal 46 XX karyotype for girls;
10. Written informed consent of the parent or legal guardian of the patient and written assent of the patient (if the patient is able to read, understand, and sign).
Are the trial subjects under 18? yes
Number of subjects for this age range: 52
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Children with a body weight below 11 kg;
2. Children with hematocrit at Screening below 34%;
3. Prior exposure to rhGH or IGF-I therapy;
4. Children with past or present intracranial tumor growth as confirmed
by a sellar MRI scan (with contrast) at Screening (if no MRI was
performed within the last 6 months prior to Screening);
5. Children born small for gestational age (SGA), i.e. birth weight and/or
birth length < -2 SD for gestational age;
6. Malnutrition, defined as:
a. Serum albumin level below the lower limit of normal (LLN) according
to the reference ranges of the central laboratory, and
b. Serum iron below the lower limit of normal (LLN) according to the
reference ranges of the central laboratory, and
c. BMI < -2 SD for age and sex;
7. Children with psychosocial dwarfism;
8. Children with idiopathic short stature;
9. Other causes of short stature such as coeliac disease (confirmed by
anti-transglutaminase antibodies test), hypothyroidism, or rickets;
10. Presence of anti-hGH binding antibodies at Screening;
11. History or presence of malignant disease; any evidence of present
tumor growth;
12. Any clinically significant abnormality likely to affect growth or the
ability to evaluate growth, e.g. chronic diseases like renal insufficiency,
spinal cord irradiation, etc.;
13. Patients with diabetes mellitus or impaired fasting sugar (based on
WHO standards);
14. Chromosomal abnormalities and medical syndromes (Turner
syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome,
Russell-Silver syndrome, SHOX mutations/deletions and skeletal
dysplasias) with the exception of septo-optic dysplasia;
15. Closed epiphyses;
16. Concomitant administration of other treatments that may have an
effect on growth such as anabolic steroids or methylphenidate for
attention deficit hyperactivity disorder (ADHD) with the exception of
hormone replacement therapies (thyroxine, hydrocortisone,
desmopressin);
17. Children requiring glucocorticoid therapy (e.g. asthma) who are
taking a dose of greater than 400 µg/d of inhaled budesonide or
equivalents for longer than 1 month during a calendar year;
18. Major medical conditions and/or presence of contraindication to rhGH treatment;
19. Known or suspected HIV-positive patient;
20. Known hypersensitivity to the components of the study medication;
21. The patient and/or the parent/legal guardian are likely to be
noncompliant in respect to study conduct;
22. Participation in any other trial of an investigational agent within 30
days prior to Screening.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Related Trials