Clinical Trial of Growth Hormone in MPS I, II, and VI

NCT00748969 | Terminated Phase 2 Results

• 1 other identifier: 0808M43681
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether growth hormone is a safe and effective treatment for short stature in children with Mucopolysaccharidosis type I, II, and VI.

Conditions

Mucopolysaccharidosis I; Mucopolysaccharidosis II; Mucopolysaccharidosis VI

Keywords

MPS I; MPS II; MPS VI; growth hormone; short stature

Outcome Measures

Primary Outcomes (1)

• Change in Growth Velocity From Baseline to End of Study Year 1.

  12 months

Secondary Outcomes (1)

• Safety: Number Drug Related SAEs

  1 months

Study Arms (2)

Growth hormone treatmen

EXPERIMENTAL

Growth hormone treatment arm. Somatropin (DNA origin)

Drug: Somatropin (DNA origin)

No growth hormone treatment in year 1

NO INTERVENTION

No growth hormone treatment in year 1; option for treatment in year 2 open-label period.

Interventions

Somatropin (DNA origin) DRUG

The study starting dose of Nutropin AQ® will be 0.48 mg/kg/week divided into daily SC injections. Nutropin AQ® will be administered by either the subject or, if unable to demonstrate competency in this, then by the guardian. To decrease the risk of increased intracranial hypertension, the dose in the first month of treatment will be decreased by 50% (0.24 mg/kg/week), and then increased to 0.48 mg/kg/week if tolerated well after 1 month.

Also known as: Nutropin AQ

Growth hormone treatmen

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• A parent or legally authorized representative must provide written informed consent and comply with study assessments for the full duration of the study.
• Chronologic age ≥ 5 years and bone age ≤12 years
• Diagnosis of MPS I, II, or VI
• Height ≤ -2 SDS for age and gender
• Ability to travel to study center for evaluations.
• Ability of the participant to cooperate with study procedures, to notify a guardian of symptoms, and provide assent for participation in the study.

You may not qualify if:

• History of treatment with hGH
• Untreated pituitary deficiency
• Pregnancy (positive urine pregnancy test) prior to enrollment in the study
• Participation in another simultaneous medical intervention trial
• Patients with closed epiphysis
• Active neoplasm
• Orthopedic procedure of the femur within the last 6 months.
• Known or suspected allergy to trial product or related products.
• Structural lesion on brain MRI resulting in brain compression
• Any other social or medical condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study.
• Obstructive sleep apnea without BiPAP or tonsillectomy/adenoidectomy treatment.
• CNS shunt.
• Abnormal cardiac function based on echocardiogram within 6 months prior to enrollment :
• Ejection fraction less than 50%
• Left ventricular chamber size greater than or less than 2 standard deviations of normal for body surface area

Sponsors & Collaborators

• Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center: lead

Study Sites (1)

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Mucopolysaccharidosis I; Mucopolysaccharidosis II; Mucopolysaccharidosis VI; Sudden Infant Death; Dwarfism

Interventions

Human Growth Hormone; Growth Hormone

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System; Death, Sudden; Death; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Infant Death; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Dr. Lynda Polgreen
• Organization: LA Biomed at Harbor-UCLA Medical Center

lpolgreen@labiomed.org

310-222-1972

Study Officials

• Lynda E Polgreen, M.D.

  University of Minnesota

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

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

Study Record Dates

• First Submitted: September 8, 2008
• First Posted: September 9, 2008
• Study Start: November 1, 2008
• Primary Completion: September 1, 2013
• Study Completion: September 1, 2013
• Last Updated: September 5, 2018
• Results First Posted: November 24, 2014

Record last verified: 2014-11

Locations

US (1)