Safety, PK/PD and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)

Growth Hormone Deficiency (GHD) Clinical Trial

Official title:

A Multicenter, Phase 2, Randomized, Open Label, Active-controlled, Parallel-group Study Investigating the Safety, Tolerability, and Efficacy of Different Dose Levels of ACP-001 Administered Once Weekly Versus Standard Daily rhGH Replacement Therapy in Pre-pubertal Children With Growth Hormone Deficiency (GHD)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01947907
• Other study ID #: ACP-001_CT-004
• Status: Completed
• Phase: Phase 2
• First received: June 3, 2013
• Last updated: March 9, 2016
• Start date: July 2013
• Est. completion date: September 2015

Study information

• Verified date: March 2016
• Source: Ascendis Pharma A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Federal Institute for Drugs and Medical DevicesHungary: Ministry of Health, Social and Family AffairsCzech Republic: State Institute for Drug Control
• Study type: Interventional

Clinical Trial Summary

A six month study of ACP-001, a long-acting growth hormone product, versus standard human growth hormone therapy. ACP-001 will be given once-a-week, standard human growth hormone (hGH) will be given on a daily basis. The primary aim is to demonstrate safety, pharmacokinetics and pharmacodynamics over a period of six months. A secondary objective is the comparison of height velocity (HV) of the ACP-001 treated groups to the daily hGH treatment group.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 55
• Est. completion date: September 2015
• Est. primary completion date: July 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Years to 12 Years

Eligibility

Inclusion Criteria:

• Prepubertal children, Tanner stage 1

• Diagnosis of GHD, confirmed by two stimulation tests

• Bone age not greater than chronological age

• Impaired height and height velocity

• BMI within +/- 2 SD

• Baseline IGF-I

• Normal fundoscopy

• Stable hormonal replacement therapy (other than hGH)

• Written Informed Consent

Exclusion Criteria:

• Prior exposure to rhGH or IGF-I

• Past or present intracranial tumor; history or presence of malignant disease

• Small for gestational age (SGA)

• Malnutrition

• Psychosocial dwarfism

• Coeliac disease

• Anti-hGH antibodies

• Diabetes mellitus

• Chromosomal abnormalities (e.g. Turner syndrome, SHOX)

• Closed epiphyses

• Known or suspected HIV infection

Study Design

Allocation: Randomized, Endpoint Classification: Pharmacokinetics/Dynamics Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency (GHD)

Intervention

Drug:
• ACP-001
Once weekly subcutaneous injection
• Human Growth Hormone
Once daily subcutaneous injection of human Growth Hormone

Locations

Country	Name	City	State
Belarus	2nd Children City Clinic	Minsk
Bulgaria	University Multiprofile Hospital for Active Treatment (UMHAT) "Sv. Marina"	Varna
Czech Republic	Masaryk´s Hospital	Ústí nad Labem
Egypt	El Shatby University Hospital	Alexandria
Egypt	Ain Shams University Hospital	Cairo
Egypt	Cairo University Hospital	Cairo
Egypt	El Mansoura University Hospital	El Mansoura
France	Hôpital des enfants Pellegrin	Bordeaux
France	Hôpital Jeanne de Flandre	Lille
France	Hôpital Femme-Mère-Enfant	Lyon
Germany	University Hospital Leipzig	Leipzig
Germany	University Children's Hospital Magdeburg	Magdeburg
Greece	Children's Hospital of Athens "P. A. Kyriakou"	Athens
Hungary	Buda Children`s Hospital	Budapest
Hungary	Heim Pal Children`s Hospital	Budapest
Hungary	University of Pecs	Pecs
Hungary	University of Szeged	Szeged
Poland	University Medical Hospital	Katowice
Poland	Medical University of Lublin	Lublin
Poland	Regional Hospital N°2 Rzeszow	Rzeszów
Poland	Children's Memorial Health Institute Warsaw	Warsaw
Romania	Emergency Clinical Hospital Cluj-Napoca	Cluj- Napoca
Romania	St. Spiridon County Clinic Emergency Hospital	Iasi
Romania	Louis Turcanu Emergency Hospital for Children Timisoara	Timisoara
Russian Federation	Federal State Budgetary Institution	Moscow
Russian Federation	Samara State Medical University	Samara
Russian Federation	St. Petersburg State Pediatric Medical Academy	St. Petersburg
Russian Federation	Bashkir State Medical University	Ufa
Slovenia	Children`s University Hospital	Ljubljana
Turkey	Ankara University School of Medicine	Ankara
Turkey	Ege Üniversity	Izmir
Ukraine	Donetsk Regional Children Clinical Hospital	Donetsk
Ukraine	Kharkiv National Medical University	Kharkiv
Ukraine	Institute of Endocrinology and Metabolism	Kiev
Ukraine	Ukrainian Children Specialized Clinical Hospital	Kiev
Ukraine	Odessa National Medical University	Odessa

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma A/S

Countries where clinical trial is conducted

Belarus,  Bulgaria,  Czech Republic,  Egypt,  France,  Germany,  Greece,  Hungary,  Poland,  Romania,  Russian Federation,  Slovenia,  Turkey,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of ACP-001 on different dosing levels	Number of adverse events	26 weeks	Yes
Primary	Pharmacokinetics of ACP-001	Pharmacokinetic assessment of ACP-001 by AUC	26 weeks	No
Primary	IGF-I response	IGF-I levels and change in IGF-I levels over a period of 26 weeks	26 weeks	Yes
Primary	Local tolerability of ACP-001	Number of injection site reactions	26 weeks	Yes
Primary	Safety of ACP-001 on different dosing levels	Incidence of anti-hGH antibodies	26 weeks	Yes
Secondary	Height velocity	Height velocity over a period of 26 weeks (annualized)	26 weeks	No