MOD-4023 (Long-Lasting Human Growth Hormone (hGH)) Study in Growth Hormone Deficient Adults (GHDA)

NCT01225666 | Completed Phase 2 DMC

• 2 other identifiers: CP-4-0032010-019374-32
• Study Type: interventional
• Target: 52
• Locations: 5 countries
• Sites: 12

Brief Summary

This study aims to assess the safety, tolerability and Pharmacokinetics/ Pharmacodynamics (PK/PD) profile of three doses of MOD-4023 on a weekly regime and one dose on an every-other-week regime administered for a period of 4 weeks in Growth Hormone Deficient Adult (GHDA) patients who previously were on a stable r-hGH treatment. An additional extension period of 16 weeks once-weekly administration of MOD-4023 aims to confirm the dose selection for future trials.

Conditions

Adult Growth Hormone Deficiency

Keywords

MOD-4023; Growth Hormone Deficient Adults; Phase II

Outcome Measures

Primary Outcomes (2)

• 1. Safety and Tolerability

  Adverse events (AE's), vital signs, electrocardiogram (ECG), laboratory tests, local reaction

  Following 4 weeks of MOD-4023 treatment

• Maintenance of normal Insulin-like-Growth-Factor-1 (IGF-1)levels in GHDA

  The primary efficacy endpoint will be the mean time interval of IGF-I levels that lay within ±1.5 SDS after the last dose administration during stage I (4w treatment) expressed in hours.

  Following 4 weeks MOD-4023 treatment

Secondary Outcomes (3)

• Change of IGF-I levels over time expressed in absolute and SDS values

  Following 4 and 16 weeks of MOD-4023 treatment

• Change of IGFBP-3 over time expressed in absolute values

  Following 4 and 16 weeks of MOD-4023 treatment

• Number of IGF-1 normalized patients in stage II

  Following 16 weeks of MOD-4023 treatment

Study Arms (4)

Weekly low dose

EXPERIMENTAL

MOD-4023

Drug: MOD-4023

Weekly middle dose

EXPERIMENTAL

MOD-4023

Drug: MOD-4023

Weekly high dose

EXPERIMENTAL

MOD-4023

Drug: MOD-4023

Every-other week dose

EXPERIMENTAL

MOD-4023

Drug: MOD-4023

Interventions

MOD-4023 DRUG

liquid solution for subcutaneous injection - 30% of the cumulative weekly dose achieved with daily rhGH, Once weekly

Weekly low dose

Eligibility Criteria

• Age: 23 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Genders Eligible for Study: Both
• Ages Eligible for Study: Males - 23 to 60 years, Females - 23 to 50 years.
• GHDA subjects as defined in the Consensus guidelines for the diagnosis and treatment of adults with GH deficiency II (2007).
• Patients using hormonal replacement therapy(s) for deficiencies of other hypothalamo-pituitary axes must be on an optimized and stable treatment regimen (hormone levels within normal ranges on screening) for at least three months prior to screening:
• Temporary adjustment of glucocorticoid replacement therapy, as appropriate, is acceptable.
• Peripheral thyroid hormones (FT4, FT3) within the normal range.
• Fertile females must agree to use appropriate contraceptive methods
• Growth Hormone (GH) replacement therapy for more than 6 months with registered GH product.
• The IGF-I level at screening within -1.5 to +1.5 SDS of the age and sex normal ranges according to the central laboratory measurements.
• Body Mass Index (BMI, kg/m2) of 22.0 to 35.0 kg/m2, both inclusive
• Confirmed to be negative for anti r-hGH antibodies at the time of screening.
• Willing and able to provide written informed consent prior to performing any study procedures.

You may not qualify if:

• Females who are pregnant or breast-feeding
• Evidence of growth of pituitary adenoma or other intracranial tumor within the last 12 months (confirmed by computer tomography (CT) or magnetic resonance imaging (MRI) scan (with contrast) within 3 months prior to study entry or at screening).
• History of malignancy other than i) cranial irradiation (for cranial tumor or leukemia) causing GHD or ii) fully treated basal cell carcinoma
• Signs of intracranial hypertension at screening
• Heart insufficiency, NYHA class greater than 2
• History of impaired glucose tolerance, insulin resistance or overt diabetes mellitus defined according to the American Diabetes Association (ADA) Criteria
• Impaired liver function defined as elevation of liver enzymes \>2 x upper limit of normal
• Impaired kidney function defined as increased serum creatinine levels \>1.5 x upper limit of normal
• Active acromegaly in the last 18 months and less than 6 months of active r- hGH replacement therapy
• Active Carpal tunnel syndrome
• Prader-Willi syndrome
• Active Cushing's syndrome within the last 12 months
• Systemic corticosteroids other than in replacement doses within the 3 months before study entry (temporary adjustment of glucocorticoids, as appropriate, is acceptable)
• Anabolic steroids other than gonadal steroid replacement therapy within 2 months before study entry
• History of non-compliance with medications, un-cooperativeness or drug abuse

Sponsors & Collaborators

• OPKO Health, Inc.: lead

Study Sites (15)

Internal Clinic in University Hospital St. Anna

Brno, 656 91, Czechia

Location

University Hospital, 2nd Department of Internal Medicine

Hradec Králové, 500 05, Czechia

Location

State Health Center, 2nd department of internal medicine

Budapest, 1062, Hungary

Location

Semmelweis University, 2nd Clinic of Internal Medicine

Budapest, 1088, Hungary

Location

Petz Aladár County Teaching Hospital, Department of Endocrinology, Metabolism and Diabetology

Győr, 9023, Hungary

Location

University of Pécs, Medical School, 1st Department of Internal Medicine

Pécs, 7624, Hungary

Location

Szeged University, 1st Internal Medicine Clinic, Endocrinology

Szeged, 6720, Hungary

Location

Hetényi Géza Hospital and Out-Patient Clinic, 1st Department of Internal Medicine

Szolnok, 5004, Hungary

Location

Endocrinology and Metabolism Service, Hadassah-Hebrew University Medical Center

Jerusalem, 91120, Israel

Location

Institute of Endocrinology, Tel Aviv-Sourasky Medical Center

Tel Aviv, 64239, Israel

Location

Clinical Center of Serbia, Institute for endocrinology, diabetes and metabolism disease

Belgrade, 11000, Serbia

Location

. Department of Internal Medicine V, University Hospital Ruzinov

Bratislava, 82104, Slovakia

Location

Slovak Health University, Division of Endocrinology

Bratislava, 83307, Slovakia

Location

National Institute of Endocrinology and Diabetology

Ľubochňa, 034 91, Slovakia

Location

University Medical Centre Ljubljana, Department of Endocrinology, Diabetes and Metabolic Diseases

Ljubljana, 1525, Slovenia

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

MOD-4023

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 19, 2010
• First Posted: October 21, 2010
• Study Start: August 1, 2010
• Primary Completion: July 1, 2011
• Study Completion: April 1, 2012
• Last Updated: October 8, 2019

Record last verified: 2019-09

Locations

IL (2); CZ (2); SL (1); HU (6); SE (1)