Hurler SyndromeNCT00286689
Effects of Growth Hormone in Chronically Ill Children
A study testing Growth Hormone, Whole body Protein turnover and DEXA scan in people with Hurler Syndrome and Cerebral Palsy, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
The specific aims for this study are - 1. To determine the effect of GH on height, height velocity, body weight and lean body mass. This specific aim tests the hypothesis that GH significantly improves height, height velocity, weight, weight velocity and lean body mass in chronically ill children who have grown poorly… Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis
- What is being tested?
- Growth Hormone, Whole body Protein turnover and DEXA scan
- Type of treatment
- Surgery or procedure
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Feb 2006to 3 Feb 2006
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Ages 3-17 years
- Tanner stages 1-3
- Each child must have received adequate nutritional therapy supplied by aggressive oral supplementation, gastrostomy tube, or TPN for at least 1 year prior to enrollment.
- All children will have been referred for continued poor growth and will be less than the 10th percentile for height compared to age and gender normal values.
- low IGF-1 level at the time of enrollment (measured in the Endocrine clinic).
- Chronic illness to be included are Hurler Syndrome (MPS-1) with short stature and muscle wasting, cerebral palsy with muscle wasting, juvenile rheumatoid arthritis with muscle wasting and short stature, Crohn's disease and HIV infection.
Who cannot join
- previous diagnosis with diabetes, chronic fevers (temp > 101.5) or chronic bacterial infection.
- substantial change in steroid dosing, or having a formerly steroid negative patient start long-term-steroids (anticipated use greater that 7 days
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Medical Center of Dallas
Dallas, Texas, 75390
About this study
The specific aims for this study are -
- To determine the effect of GH on height, height velocity, body weight and lean body mass. This specific aim tests the hypothesis that GH significantly improves height, height velocity, weight, weight velocity and lean body mass in chronically ill children who have grown poorly despite adequate nutritional rehabilitation.
- To determine the effect of GH on whole body protein turnover (WBPT), IGF-1 levels and on cytokines. This specific aim tests the hypothesis that chronically ill children have increased catabolism, caused by high levels of circulating cytokines and low levels of IGF-1, and that these abnormalities improve with GH treatment.
- Evaluation of bone mineral density and bone turnover. This specific aim tests the hypothesis that bone density is low in chronically ill children secondary to increased osteoclast activity correlating with elevated cytokine levels.
We hypothesize that the anabolic effects of growth hormone (GH) will improve the height and weight of chronically ill children who have failed to grow despite receiving adequate nutrition via gastrostomy tube or oral supplementation.
Read the detailed description
We will test our hypotheses by using a pilot study, in which we will recruit 18 chronically ill children from our clinical practice. We will obtain medical records for each patient 12 months prior to starting the study. Those patients without pre-study medical records will be studied for 12 months prior to starting GH. If we can obtain 6 months of prior medical records, then the patients will be studied for 6 months before starting GH. Anything less than 6 months will be studied for the full 12 months prior to starting GH. Patients will receive treatment with GH (0.3 mg/kg/wk) for 12 months and their growth will be compared to the year before treatment. All subjects will be followed every three months for the entire study. We will measure height and weight using a standardized stadiometer and scale, respectively, every three months during the study. From these measurements we will calculate height and weight velocity and height and weight Z score. Lean body mass (LBM) will be measured by DEXA every six months. Utilizing the stable isotope 1-[13C] leucine, we will measure whole body protein turnover (WBPT). Measurements of WBPT will be correlated with LBM and changes in height and weight velocity. This data will be compared to that from age matched normal children (archival data maintained by the PI). We will measure IGF-1 and the cytokines TNF-α, IL-6 and IL 10 at baseline and very six months. We will evaluate GH effects on these levels.
- Conditions
- Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections
- Being tested
- Growth Hormone, Whole body Protein turnover, DEXA scan
- Medical subject headings
- Mucopolysaccharidosis I, Cerebral Palsy, Arthritis, Juvenile, Crohn Disease, HIV Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Growth HormoneDrug
Whole body Protein turnoverProcedure or surgery
DEXA scanProcedure or surgery
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2006 (estimated)
- Study start3 Feb 2006
- Primary completion3 Feb 2006
- Study completion3 Feb 2006
Why it stopped: Record Owner no longer with the institution
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hurler SyndromeStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Investigators
- Dana S Hardin, MD, Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00286689
- Sponsor's protocol ID
- 0403-239
- Status last verified
- Dec 2018
- First submitted
- 1 Feb 2006
- Last updated
- 14 Dec 2018
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Growth Hormone in Chronically Ill Children
- ClinicalTrials.gov ID
- NCT00286689
- Acronym / sponsor ID
- 0403-239
- Conditions
- Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Record Owner no longer with the institution
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Study start
- 3 Feb 2006 (actual)
- Primary completion
- 3 Feb 2006 (actual)
- Study completion
- 3 Feb 2006 (actual)
- First posted
- 3 Feb 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The specific aims for this study are -
- To determine the effect of GH on height, height velocity, body weight and lean body mass. This specific aim tests the hypothesis that GH significantly improves height, height velocity, weight, weight velocity and lean body mass in chronically ill children who have grown poorly despite adequate nutritional rehabilitation.
- To determine the effect of GH on whole body protein turnover (WBPT), IGF-1 levels and on cytokines. This specific aim tests the hypothesis that chronically ill children have increased catabolism, caused by high levels of circulating cytokines and low levels of IGF-1, and that these abnormalities improve with GH treatment.
- Evaluation of bone mineral density and bone turnover. This specific aim tests the hypothesis that bone density is low in chronically ill children secondary to increased osteoclast activity correlating with elevated cytokine levels.
We hypothesize that the anabolic effects of growth hormone (GH) will improve the height and weight of chronically ill children who have failed to grow despite receiving adequate nutrition via gastrostomy tube or oral supplementation.
Detailed description
We will test our hypotheses by using a pilot study, in which we will recruit 18 chronically ill children from our clinical practice. We will obtain medical records for each patient 12 months prior to starting the study. Those patients without pre-study medical records will be studied for 12 months prior to starting GH. If we can obtain 6 months of prior medical records, then the patients will be studied for 6 months before starting GH. Anything less than 6 months will be studied for the full 12 months prior to starting GH. Patients will receive treatment with GH (0.3 mg/kg/wk) for 12 months and their growth will be compared to the year before treatment. All subjects will be followed every three months for the entire study. We will measure height and weight using a standardized stadiometer and scale, respectively, every three months during the study. From these measurements we will calculate height and weight velocity and height and weight Z score. Lean body mass (LBM) will be measured by DEXA every six months. Utilizing the stable isotope 1-[13C] leucine, we will measure whole body protein turnover (WBPT). Measurements of WBPT will be correlated with LBM and changes in height and weight velocity. This data will be compared to that from age matched normal children (archival data maintained by the PI). We will measure IGF-1 and the cytokines TNF-α, IL-6 and IL 10 at baseline and very six months. We will evaluate GH effects on these levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Ages 3-17 years
- Tanner stages 1-3
- Each child must have received adequate nutritional therapy supplied by aggressive oral supplementation, gastrostomy tube, or TPN for at least 1 year prior to enrollment.
- All children will have been referred for continued poor growth and will be less than the 10th percentile for height compared to age and gender normal values.
- low IGF-1 level at the time of enrollment (measured in the Endocrine clinic).
Key exclusion criteria (2 listed)
- previous diagnosis with diabetes, chronic fevers (temp > 101.5) or chronic bacterial infection.
- substantial change in steroid dosing, or having a formerly steroid negative patient start long-term-steroids (anticipated use greater that 7 days
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 |
|---|---|---|
| Growth Hormone | Drug | – |
| Whole body Protein turnover | Procedure or surgery | – |
| DEXA scan | Procedure or surgery | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Dec 2018.
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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
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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.
Dana S Hardin, MD principal investigator
University of Texas Southwestern Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Growth Hormone, Whole body Protein turnover and DEXA scan in people with Hurler Syndrome and Cerebral Palsy, run by University of Texas Southwestern 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 2 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: withdrawn, as of the sponsor's last update on 14 Dec 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.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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.
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 14 Dec 2018.