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

NCT ID: NCT01947907

Last Updated: 2017-01-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 53 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-07-31
• Study Completion Date: 2015-09-30

Brief 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.

Conditions

Growth Hormone Deficiency (GHD)

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ACP-001, dose-level 1

Once weekly subcutaneous injection of ACP-001

Group Type: EXPERIMENTAL

ACP-001

Intervention Type: DRUG

Once weekly subcutaneous injection

ACP-001, dose-level 2

Once weekly subcutaneous injection of ACP-001

Group Type: EXPERIMENTAL

ACP-001

Intervention Type: DRUG

Once weekly subcutaneous injection

ACP-001, dose-level 3

Once weekly subcutaneous injection of ACP-001

Group Type: EXPERIMENTAL

ACP-001

Intervention Type: DRUG

Once weekly subcutaneous injection

Human Growth Hormone

Once daily subcutaneous injection of human Growth Hormone (rhGH)

Group Type: ACTIVE_COMPARATOR

Human Growth Hormone

Intervention Type: DRUG

Once daily subcutaneous injection of human Growth Hormone

Interventions

ACP-001

Once weekly subcutaneous injection

Intervention Type: DRUG

Human Growth Hormone

Once daily subcutaneous injection of human Growth Hormone

Intervention Type: DRUG

Other Intervention Names

Somatropin

Eligibility 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 (standard deviations)
• Baseline IGF-1 (insulin-like growth factor)
• 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

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Ascendis Pharma A/S

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Pierre Chatelain, Prof, MD

Role: PRINCIPAL_INVESTIGATOR

University of Lyon

Michael Beckert, MD

Role: STUDY_DIRECTOR

Ascendis Pharma A/S

Locations

2nd Children City Clinic

Minsk, , Belarus

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

Varna, , Bulgaria

Masaryk´s Hospital

Ústí nad Labem, , Czechia

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

Pécs, , 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

Iași, , Romania

Louis Turcanu Emergency Hospital for Children Timisoara

Timișoara, , Romania

Federal State Budgetary Institution

Moscow, , Russia

St. Petersburg State Pediatric Medical Academy

Saint Petersburg, , Russia

Samara State Medical University

Samara, , Russia

Bashkir State Medical University

Ufa, , Russia

Children's University Hospital

Ljubljana, , Slovenia

Ankara University School of Medicine

Ankara, , Turkey (Türkiye)

Ege Üniversity

Izmir, , Turkey (Türkiye)

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

Odesa, , Ukraine

Countries

Belarus; Bulgaria; Czechia; Egypt; France; Germany; Greece; Hungary; Poland; Romania; Russia; Slovenia; Turkey (Türkiye); Ukraine

References

Chatelain P, Malievskiy O, Radziuk K, Senatorova G, Abdou MO, Vlachopapadopoulou E, Skorodok Y, Peterkova V, Leff JA, Beckert M; TransCon GH Working Group. A Randomized Phase 2 Study of Long-Acting TransCon GH vs Daily GH in Childhood GH Deficiency. J Clin Endocrinol Metab. 2017 May 1;102(5):1673-1682. doi: 10.1210/jc.2016-3776.

Reference Type: DERIVED

PMID: 28201598 (View on PubMed)

Other Identifiers

ACP-001_CT-004

Identifier Type: -

Identifier Source: org_study_id