Mucopolysaccharidosis IPhase 2/3NCT00748969
Clinical Trial of Growth Hormone in MPS I, II, and VI
A Phase 2/3 study testing Somatropin (DNA origin) in people with Mucopolysaccharidosis I and Mucopolysaccharidosis II, run by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- What is being tested?
- Somatropin (DNA origin)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2008to Sep 2013
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Show all 23Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota
Minneapolis, Minnesota, 55455
About this study
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
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.
Read the 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.
- Being tested
- Somatropin (DNA origin)
- Keywords
- MPS I, MPS II, MPS VI, growth hormone, short stature
- Medical subject headings
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI, Sudden Infant Death, Dwarfism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Somatropin (DNA origin)Drug
Also called Nutropin AQ
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.
| Study group | Role | What they receive |
|---|---|---|
| Growth hormone treatmen | Experimental | Growth hormone treatment arm. Somatropin (DNA origin) Receives: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Growth Velocity From Baseline to End of Study Year 1.
Measured: 12 months
Secondary outcomes (1)
Safety: Number Drug Related SAEs
Measured: 1 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2008 (estimated)
- Study startNov 2008
- Primary completionSep 2013
- Study completionSep 2013
Why it stopped: Insufficient recruitment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Mucopolysaccharidosis IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lynda E Polgreen, M.D., Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00748969
- Sponsor's protocol ID
- 0808M43681
- Status last verified
- Nov 2014
- First submitted
- 8 Sep 2008
- Last updated
- 5 Sep 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00748969
- Acronym / sponsor ID
- 0808M43681
- Keywords
- MPS I, MPS II, MPS VI, growth hormone, short stature
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Insufficient recruitment.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterUniversity, hospital or other
- Study start
- Nov 2008
- Primary completion
- Sep 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 9 Sep 2008 (estimated)
- Results
- Posted 24 Nov 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Growth hormone treatmen | Experimental | Drug: Somatropin (DNA origin)Growth hormone treatment arm. 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. |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (23 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Somatropin (DNA origin)Also: Nutropin AQThe 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. | Drug | Growth hormone treatmen |
Primary outcome
Change in Growth Velocity From Baseline to End of Study Year 1.
Time frame: 12 months
1 secondary outcome
Safety: Number Drug Related SAEs
Time frame: 1 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Mucopolysaccharidosis I
Active, not recruitingObservational study
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Active, not recruitingPhase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Mucopolysaccharidosis Disorders, Hurler Syndrome
RecruitingObservational study
Cardiac Structure and Function in MPS
MPS I, MPS IV A
Active, not recruitingPhase 1/2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
RecruitingPhase 2/3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
MPS IIIA, Sanfilippo Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lynda E Polgreen, M.D. principal investigator
University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Somatropin (DNA origin) in people with Mucopolysaccharidosis I and Mucopolysaccharidosis II, run by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Nov 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Growth Velocity From Baseline to End of Study Year 1. (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Sep 2018.