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

Primary Purpose

Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI

Status

Terminated

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

Somatropin (DNA origin)

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring MPS I, MPS II, MPS VI, growth hormone, short stature

Eligibility Criteria

5 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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
Left ventricular wall thickness greater than or less than 2 standard deviations of normal for body surface area
More than mild to moderate aortic insufficiency with abdominal aortic run-off
More than mild to moderate mitral insufficiency with pulmonary hypertension
Abnormal pulmonary function based on pulmonary function tests within 6 months prior to enrollment:
abnormal FVC < 80% of predicted for age, gender, and height
abnormal FEV1 < 80% predicted for age, gender, and height
abnormal FEV1/FVC
abnormal oxygen saturation

Sites / Locations

University of Minnesota

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Growth hormone treatmen

No growth hormone treatment in year 1

Arm Description

Growth hormone treatment arm. Somatropin (DNA origin)

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Safety: Number Drug Related SAEs

Full Information

NCT ID

NCT00748969

First Posted

September 8, 2008

Last Updated

August 6, 2018

Sponsor

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

1. Study Identification

Unique Protocol Identification Number

NCT00748969

Brief Title

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

Official Title

Phase II/III, Randomized, Clinical Trial of the Effects of Nutropin AQ® on Growth and Bone Metabolism in Children With MPS I, II, and VI and Short Stature

Study Type

Interventional

2. Study Status

Record Verification Date

November 2014

Overall Recruitment Status

Terminated

Why Stopped

Insufficient recruitment.

Study Start Date

November 2008 (undefined)

Primary Completion Date

September 2013 (Actual)

Study Completion Date

September 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

4. Oversight

Data Monitoring Committee

5. Study Description

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.

Detailed Description

Although children with MPS I, II, and VI who are treated with Hematopoietic Cell Transplantation (HCT) and/or enzyme replacement therapy (ERT) are living into adulthood with good cognitive development, their quality of life is significantly impacted by their skeletal abnormalities (i.e., kyphosis, scoliosis, and genu valgum), contractures, and severe short stature. Here at the University of Minnesota we have seen some promising clinical outcomes in children with MPS IH whom we have treated with human growth hormone (hGH). There are currently no reports in the literature of the impact of treating children with MPS and short stature, with hGH on their growth velocity or characteristic skeletal abnormalities. This study will advance the care of these children by providing data in this yet unexplored area of pediatric medicine with the goal of improving the quality of life for these children by improving height, mobility, and neuropsychological functioning. This is a Phase II/III randomized, single-center, 12 month clinical trial of growth hormone in male and female participants with MPS I, II, or VI, followed by 12 months open label. Participants with height ≤ -2 SDS for age and gender will be randomized for the first 12 months 1:1 to treatment or no treatment. At the conclusion of the 12 months, all subjects will be offered an additional 12 months of treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

2 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Growth hormone treatmen

Arm Type

Experimental

Arm Description

Growth hormone treatment arm. Somatropin (DNA origin)

Arm Title

No growth hormone treatment in year 1

Arm Type

No Intervention

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Somatropin (DNA origin)

Other Intervention Name(s)

Nutropin AQ

Intervention Description

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.

Primary Outcome Measure Information:

Title

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

Time Frame

12 months

Secondary Outcome Measure Information:

Title

Safety: Number Drug Related SAEs

Time Frame

1 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Maximum Age & Unit of Time

17 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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 Left ventricular wall thickness greater than or less than 2 standard deviations of normal for body surface area More than mild to moderate aortic insufficiency with abdominal aortic run-off More than mild to moderate mitral insufficiency with pulmonary hypertension Abnormal pulmonary function based on pulmonary function tests within 6 months prior to enrollment: abnormal FVC < 80% of predicted for age, gender, and height abnormal FEV1 < 80% predicted for age, gender, and height abnormal FEV1/FVC abnormal oxygen saturation

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Lynda E Polgreen, M.D.

Organizational Affiliation

University of Minnesota

Official's Role

Principal Investigator

Facility Information:

Facility Name

University of Minnesota

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

12. IPD Sharing Statement

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

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