Clinical trial NCT00884000: A Study of Zomacton in Children With Growth Hormone Deficiency
Official title: A Randomised, Open-label, Parallel-group, Multi-centre Trial to Compare the Efficacy and Safety for 12 Months of Zomacton to Genotropin in Children With Idiopathic Growth Hormone Deficiency
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Growth Hormone Deficiency
Lead sponsor: Ferring Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2009-04-20
Enrollment: 165
Sex: ALL
Minimum age (years): 3
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This trial is set up to compare Zomacton to Genotropin for the treatment of growth hormone deficiency in children. The children will be treated for 1 year. Half of the patients will be treated with Genotropin and half with Zomacton. During this time they will be dosed every day by themselves or their parents at home in the evening. There will be 138 patients in the trial from age 3 to age 11. The patients cannot have been treated before with growth hormone and the patients must have a proven growth hormone deficiency, this will be shown by a specific test that will be performed before the trial in the local clinic and once during the trial. During the time of the treatment the patients will come to visit the clinic every 3 months. At these visits their heights will be measured, blood samples will be taken, physical examinations will be performed and questions about their health will be asked. At 2 times in the trial they will have a hand x-ray taken to measure the bone age. At the end of the trial the patients will stop the treatment and continue on one of the marketed products available to treat growth hormone deficiency.


Eligibility criteria: Inclusion Criteria:

1. Signed informed consent
2. Children aged ≤3yrs old and not above 10 yrs for girls or 11 yrs for boys
3. Idiopathic growth hormone deficiency confirmed during the pre-screening period by a standard GH stimulation test (defined as peak level of \<10ng/ml pr lower if so required by the country specific board(s)
4. Height SDS \<-2 SD of ref value for CA
5. Height velocity SDSCA ≤ 0 SD of ref value for at lease 6 months prior to pre-screening
6. Height recorded for at least 6 months but not more than 18 months of pre-screening
7. The difference between CA-BA≥ 1
8. A positive locally performed GH stimulation test (defined as a peak plasma level of\<9ng/ml or lower if so required by the country specific board(s)) prior to the pre-screening

Exclusion Criteria:

1. BA above 9 yrs for girls and 10 yrs for boys
2. Puberty Tanner stage \>1
3. Weight \<12 Kg at screening
4. Any prior treatment with GH
5. Closed epiphysis
6. Any diagnosed or suspected syndrome (e.g. Silver -Russell, Turner's or seckel syndrome) which possibly could affect growth
7. Any other diagnosed or suspected endocrine or metabolic disorder
8. Any diagnosed or suspected sever chronic disease
9. Clinical signs of dysmorphic features, malformations or mental retardations
10. Growth failure due to other disorders
11. Previous or present use of drugs that could interfere with GH treatment (e.g. steroids)
12. Diagnosed malignant disease
13. Any abnormal CS lab results that requires further investigation
14. Receipt of an investigational drug within the last 28 days preceding screening or longer if considered possible to influence the outcome of the current trial
15. Any knowledge of hypersensitivity to somatropin or any of the excipients of Zomacton or Genotropin

Primary outcomes:
measure: Height Velocity
timeFrame: 12 months
Secondary outcomes:
measure: Height SDS
timeFrame: 12 months
measure: Height velocity SDS
timeFrame: 12 months
measure: Change in IGF-1 and IGFBP-3
timeFrame: 12 months
measure: BA (Bone Age)
timeFrame: 12 months
measure: Anti-hGH AB
timeFrame: 12 months
measure: AE and tolerability
timeFrame: 12 months
measure: CS Changes in safety lab, physical examination and vital signs
timeFrame: 12 months
Other outcomes:
Locations:
city: Budapest
country: Hungary
facility: Semmelweiss University, 1st dept of paediatrics
city: Budapest
country: Hungary
facility: Szent Janos Kh Budai
city: Gyor
country: Hungary
facility: Petz Country Teaching Hospital
city: Szeged
country: Hungary
facility: University of Szeged
city: Szombathely
country: Hungary
facility: Markusovszkty Teaching Hospital
city: Chennai
country: India
facility: Associates in Clinical Endocrinology Education and Research (ACEER)
city: Hyderabad
country: India
facility: Apollo Hospitals
city: Indore
country: India
facility: TOTALL Diabetes Hormone Institute
city: Mumbai
country: India
facility: KEM Hospital
city: Mumbai
country: India
facility: Prince Aly Khan Hospital
city: Nasik
country: India
facility: Endocare Clinic
city: Pune
country: India
facility: Jehangir Clinical Development Centre Pvt. Ltd., Jehangir Hospital
city: Trivandrum
country: India
facility: Health & Research Centre
city: Afula
country: Israel
facility: Haemek Medical Center
city: Beer Sheva
country: Israel
facility: Soroka University Medical Center
city: Nahariya
country: Israel
facility: Western Galilee Medical Center
city: Petach Tikva
country: Israel
facility: Schneider Children's Medical Center of Israel
city: Ramat Gan
country: Israel
facility: Edmond and Lily Safra Children's hospital - The Chaim Sheba Medical Center
city: Rehovot
country: Israel
facility: Kaplan Medical Center
city: Tel Aviv
country: Israel
facility: Dana Children's Hospital - Tel-Aviv Sourasky Medical Center
city: Bydgoszcz
country: Poland
facility: Wojewódzki Szpital Dziecięcy im. J.Brudzińskiego w Bydgoszczy
city: Gdansk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne, Gdańsk
city: Krakow
country: Poland
facility: Uniwersytecki Szpital Dziecięcy w Krakowie
city: Szczecin
country: Poland
facility: SPSK nr.1 im. Prof. T. Sokołowskiego PAM Szczecin
city: Bucuresti
country: Romania
facility: Societatea Civila Medicala "Dr. Paveliu"
city: Craiova
country: Romania
facility: Spitalul Clinic Municipal "Filantropia"
city: Iasi
country: Romania
facility: Spitalul Clinic Judetean de Urgenta "Sf. Spiridon"
city: Targu Mures
country: Romania
facility: Spitalul Clinic Judetean Mures
city: Timisoara
country: Romania
facility: Paediatric Endocrinology/Medicali's SRL
city: Timisoara
country: Romania
facility: Spitalul Clinic pentru Copii Louis Turcanu
city: Kazan
country: Russian Federation
facility: State Educational Institution of Higher Professional Education "Kazan State Medical University of ROSZDRAV
city: Moscow
country: Russian Federation
facility: Federal State Institution
city: Moscow
country: Russian Federation
facility: Russian Medical Academy of Post-graduate Education
city: Saint Petersburg
country: Russian Federation
facility: Saint-Petersburg State Health Care Institution
city: Saratov
country: Russian Federation
facility: State Educational Institution for Higher Professional Education
city: Tomsk
country: Russian Federation
facility: State Educational Institution of Higher Professional Education "Siberian State Medical University of ROSZDRAV
city: Donetsk
country: Ukraine
facility: Donetsk Regional Clinical Children's Hospital
city: Ivano-Frankivsk
country: Ukraine
facility: Ivano-Frankivsk Regional Children Clinical Hospital
city: Kharkiv
country: Ukraine
facility: Kharkiv Regional Clinical Children's Hospital
city: Kiev
country: Ukraine
facility: Institute of Endocrinology and Metabolism
city: Kiev
country: Ukraine
facility: Ukrainian Children's Specialized Clinical Hospital
city: Odessa
country: Ukraine
facility: Odessa National Medical University - (Located at Odessa Region Children's Clinical Hospital)
city: Zaporizhya
country: Ukraine
facility: Zaporizhzhya Regional Paediatric Hospital
Study officials:
affiliation: Ferring Pharmaceuticals
name: Clinical Development Support
role: STUDY_DIRECTOR
References: