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

Condition(s) targeted: Growth Hormone Deficiency (GHD)

Intervention: ACP-001 (Drug); Human Growth Hormone (Drug)

Phase: Phase 2

Status: Active, not recruiting

Sponsored by: Ascendis Pharma A/S

Official(s) and/or principal investigator(s):
Pierre Chatelain, Prof, MD, Principal Investigator, Affiliation: University of Lyon
Michael Beckert, MD, Study Director, Affiliation: Ascendis Pharma A/S

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.

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)

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

Primary outcome:

Safety of ACP-001 on different dosing levels

Pharmacokinetics of ACP-001

IGF-I response

Local tolerability of ACP-001

Safety of ACP-001 on different dosing levels

Secondary outcome: Height velocity

Minimum age: 3 Years. Maximum age: 12 Years. Gender(s): Both.

Criteria:

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

2nd Children City Clinic, Minsk, Belarus

University Multiprofile Hospital for Active Treatment (UMHAT) "Sv. Marina", Varna, Bulgaria

Masaryk´s Hospital, Ústí nad Labem, Czech Republic

El Shatby University Hospital, Alexandria, Egypt

Ain Shams University Hospital, Cairo, Egypt

Cairo University Hospital, Cairo, Egypt

El Mansoura University Hospital, El Mansoura, Egypt

Hôpital des enfants Pellegrin, Bordeaux, France

Hôpital Jeanne de Flandre, Lille, France

Hôpital Femme-Mère-Enfant, Lyon, France

University Hospital Leipzig, Leipzig, Germany

University Children's Hospital Magdeburg, Magdeburg, Germany

Children's Hospital of Athens "P. A. Kyriakou", Athens, Greece

Buda Children`s Hospital, Budapest, Hungary

Heim Pal Children`s Hospital, Budapest, Hungary

University of Pecs, Pecs, Hungary

University of Szeged, Szeged, Hungary

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, Poland

Emergency Clinical Hospital Cluj-Napoca, Cluj- Napoca, Romania

St. Spiridon County Clinic Emergency Hospital, Iasi, Romania

Louis Turcanu Emergency Hospital for Children Timisoara, Timisoara, Romania

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, Russian Federation

Children`s University Hospital, Ljubljana, Slovenia

Ankara University School of Medicine, Ankara, Turkey

Ege Üniversity, Izmir, Turkey

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, Ukraine

Starting date: July 2013
Last updated: January 13, 2015