History of Changes for Study: NCT02500316

Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

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Study Record Versions

Version	Submitted Date	Changes
1	July 14, 2015	None (earliest Version on record)
2	August 17, 2016	Study Status
3	May 6, 2018	Contacts/Locations, Study Status, Arms and Interventions, Outcome Measures, Study Design, Conditions and Study Description
4	October 8, 2018	Study Status
5	July 7, 2019	Study Status
6	December 12, 2019	Study Status and Study Identification
7	March 4, 2021	Study Status
8	May 11, 2021	Study Status
9	April 18, 2022	Study Status
10	November 15, 2023	Study Status
11	February 6, 2024	Recruitment Status and Study Status

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	April 18, 2022	November 15, 2023

Study Identification


Unique Protocol ID:	CP-4-004-extension	CP-4-004-extension
Brief Title:	Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children	Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
Official Title:	Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily R-human Growth Hormone (hGH) Therapy in Pre-pubertal Growth Hormone Deficient Children	Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily R-human Growth Hormone (hGH) Therapy in Pre-pubertal Growth Hormone Deficient Children
Secondary IDs:	2011-004553-60 [EudraCT Number]	2011-004553-60 [EudraCT Number]

Study Status


Record Verification:	April 2022	November 2023
Overall Status:	Active, not recruiting	Active, not recruiting
Study Start:	February 2013	February 2013
Primary Completion:	December 2023 [Anticipated]	December 2023 [Anticipated]
Study Completion:	December 2023 [Anticipated]	December 2023 [Anticipated]

First Submitted:	July 12, 2015	July 12, 2015
First Submitted that Met QC Criteria:	July 14, 2015	July 14, 2015
First Posted:	July 16, 2015 [Estimate]	July 16, 2015 [Estimate]

Last Update Submitted that Met QC Criteria:	April 18, 2022	November 15, 2023
Last Update Posted:	April 19, 2022 [Actual]	November 18, 2023 [Actual]

Sponsor/Collaborators


Sponsor:	OPKO Health, Inc.	OPKO Health, Inc.
Responsible Party:	Sponsor	Sponsor
Collaborators:

Oversight


U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring:	Yes	Yes

Study Description


Brief Summary:	Protocol CP-4-004-EXT is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.	Protocol CP-4-004-EXT is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.
Detailed Description:

Conditions


Conditions:	Growth Hormone Deficiency (GHD)	Growth Hormone Deficiency (GHD)
Keywords:	Growth hormone deficiency (GHD) r-hGH Long-acting	Growth hormone deficiency (GHD) r-hGH Long-acting

Study Design


Study Type:	Interventional	Interventional
Primary Purpose:	Treatment	Treatment
Study Phase:	Phase 2	Phase 2
Interventional Study Model:	Single Group Assignment	Single Group Assignment
Number of Arms:	1	1
Masking:	None (Open Label)	None (Open Label)
Allocation:	N/A	N/A
Enrollment:	48 [Actual]	48 [Actual]

Arms and Interventions

Arms	Assigned Interventions
Experimental: MOD-4023 Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).	Drug: MOD-4023 Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).

Outcome Measures


Primary Outcome Measures:
1.	Annual Height Velocity [ Time Frame: 8 years ]	Annual Height Velocity [ Time Frame: 8 years ]
2.	Delta height SDS every 12 months [ Time Frame: 8 years ]	Delta height SDS every 12 months [ Time Frame: 8 years ]
Secondary Outcome Measures:
1.	Absolute IGF-I levels on day 4 after MOD-4023 dosing [ Time Frame: 8 years ]	Absolute IGF-I levels on day 4 after MOD-4023 dosing [ Time Frame: 8 years ]
2.	IGF-I SDS on day 4 after MOD-4023 dosing [ Time Frame: 8 years ]	IGF-I SDS on day 4 after MOD-4023 dosing [ Time Frame: 8 years ]
Other Outcome Measures:
1.	IGFBP-3 levels on day 3 or 4 after MOD-4023 dosing [ Time Frame: 8 years ]	IGFBP-3 levels on day 3 or 4 after MOD-4023 dosing [ Time Frame: 8 years ]

Eligibility


Minimum Age:	3 Years	3 Years
Maximum Age:	11 Years	11 Years
Sex:	All	All
Gender Based:
Accepts Healthy Volunteers:	No	No
Criteria:	Inclusion Criteria: Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study. Exclusion Criteria: Children with past or present intracranial tumor growth as confirmed by an MRI scan (with contrast). History of radiation therapy or chemotherapy. Malnourished children defined as: Serum albumin below the lower limit of normal (LLN) according to the reference ranges of central laboratory; Serum iron below the lower limit of normal (LLN) according to the reference ranges of central laboratory; BMI < -2 Standard Deviation for age and sex; Children with psychosocial dwarfism. Children born small for gestational age (SGA - birth weight and/or birth length < -2 SD for gestational age). Presence of anti-hGH antibodies at screening. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc. Patients with diabetes mellitus. Patients with impaired fasting sugar (based on WHO; fasting blood sugar >110 mg/dl or 6.1 mmol/l) after repeated blood analysis. Chromosomal abnormalities and medical "syndromes" (Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, Russell-Silver Syndrome, short stature homeobox-containing gene (SHOX) mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia. Closed epiphyses. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD), with the exception of hormone replacement therapies (thyroxine, hydrocortisone, desmopressin (DDAVP)) Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 µg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year. Major medical conditions and/or presence of contraindication to r-hGH treatment. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis. Drug, substance, or alcohol abuse. Known hypersensitivity to the components of study medication. Other causes of short stature such as coeliac disease, hypothyroidism and rickets. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct. Participation in any other trial of an investigational agent within 30 days prior to Screening.	Inclusion Criteria: Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study. Exclusion Criteria: Children with past or present intracranial tumor growth as confirmed by an MRI scan (with contrast). History of radiation therapy or chemotherapy. Malnourished children defined as: Serum albumin below the lower limit of normal (LLN) according to the reference ranges of central laboratory; Serum iron below the lower limit of normal (LLN) according to the reference ranges of central laboratory; BMI < -2 Standard Deviation for age and sex; Children with psychosocial dwarfism. Children born small for gestational age (SGA - birth weight and/or birth length < -2 SD for gestational age). Presence of anti-hGH antibodies at screening. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc. Patients with diabetes mellitus. Patients with impaired fasting sugar (based on WHO; fasting blood sugar >110 mg/dl or 6.1 mmol/l) after repeated blood analysis. Chromosomal abnormalities and medical "syndromes" (Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, Russell-Silver Syndrome, short stature homeobox-containing gene (SHOX) mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia. Closed epiphyses. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD), with the exception of hormone replacement therapies (thyroxine, hydrocortisone, desmopressin (DDAVP)) Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 µg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year. Major medical conditions and/or presence of contraindication to r-hGH treatment. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis. Drug, substance, or alcohol abuse. Known hypersensitivity to the components of study medication. Other causes of short stature such as coeliac disease, hypothyroidism and rickets. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct. Participation in any other trial of an investigational agent within 30 days prior to Screening.

Contacts/Locations

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