Crohn's DiseasePhase 2NCT00109473
Trial of Growth Hormone Therapy in Pediatric Crohn's Disease
A Phase 2 study testing growth hormone and cortecosteroid in people with Crohn's Disease, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The purpose of this study is to determine whether taking a growth hormone (GH) drug called somatropin causes the intestine of a person with Crohn's Disease (CD) to heal faster when compared to a person with Crohn's Disease that does not receive growth hormone drug. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Crohn's Disease
- What is being tested?
- growth hormone and cortecosteroid
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2005to Aug 2009
- Phase
- Phase 2
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Ability to provide written informed consent
- Age ≥ 5 years.
- Diagnosis of Crohn's disease (CD) with ileo-colonic involvement as determined by standard clinical, radiological, and pathological criteria.
- Moderate to severely active CD as defined by a PCDAI (Pediatric Crohn's Disease Activity Index) ≥ 30.
- Currently taking Prednisone or Budesonide at starting dose (not tapering)
- May continue stable doses of AZA/6-MP, methotrexate, and/or mesalamine at entry.
- For the 52 week extension, baseline bone age ≤ 12 years for girls and ≤ 13 years for boys.
- For the 52 week extension phase, remission or mild Crohn's disease as determined by a PCDAI < 30.
Who cannot join
- Acute critical illness
- Active neoplasia
- Diabetes mellitus
- History of intracranial lesion and/or neoplasia
- Severe disease requiring hospitalization for treatment
- Current therapy with infliximab as this may independently rapidly reduce clinical disease activity and promote mucosal healing
Show all 12Show fewer
- Use of prednisone or budesonide and in tapering phase
- Family history of colorectal cancer before age 50
- Personal or familial history of familial polyposis syndrome
- Pregnancy (positive pregnancy test) prior to randomization
- Any other condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated
- Participation in another simultaneous medical investigation or trial other than the Pediatric IBD (Inflammatory Bowel Disease) registry
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: A Phase II Randomized Trial of Growth Hormone Therapy in Pediatric Crohn's Disease
The purpose of this study is to determine whether taking a growth hormone (GH) drug called somatropin causes the intestine of a person with Crohn's Disease (CD) to heal faster when compared to a person with Crohn's Disease that does not receive growth hormone drug.
Read the detailed description
The optimal treatment goals in childhood CD include: 1) clinical remission in conjunction with mucosal healing and 2) restoration of normal growth and development. Current therapy in most cases includes induction of remission with corticosteroids followed by maintenance of remission with 6-mercaptopurine (6-MP) or mesalamine. With this approach, the goals of achieving mucosal healing with normalization of growth are not achieved in a significant number of children. GH therapy is now used in several chronic childhood diseases which are complicated by growth failure despite adequate GH secretion. These include chronic renal failure (CRF), juvenile rheumatoid arthritis (JRA), and Turner's syndrome. However, despite a comparable frequency and magnitude of permanent growth failure, the efficacy of GH therapy in this respect has not yet been determined in a controlled trial for CD. Moreover, whether GH therapy may also directly reduce disease activity and promote intestinal healing is not known. This represents a significant clinically unmet need in this patient population. Therefore, new therapeutic approaches are needed to both improve final adult height and enhance intestinal mucosal healing in children with CD.
The primary objective of this study is to determine the effect of growth hormone (GH) therapy upon colon mucosal healing in a 12 week randomized trial in children with Crohn's Disease (CD). Children with active CD will be randomized to GH + prednisone (GP) or prednisone alone (P) for a 12 week period. This study also involves a 52 week extension phase where all participants that meet eligibility will be given the opportunity to take or continue taking growth hormone for 52 weeks.
- Conditions
- Crohn's Disease
- Being tested
- growth hormone, cortecosteroid
- Keywords
- Pediatric Crohn's disease, Pediatric, Crohn's Disease
- Medical subject headings
- Crohn Disease, Pediatric Crohn's disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
growth hormoneDrug
Also called Nutropin AQ
Nutropin AQ 0.075mg/kg/day subcutaneously daily
cortecosteroidDrug
Also called Prednisone, Entocort
As prescribed by the referring gastroenterologist
| Study group | Role | What they receive |
|---|---|---|
| Growth Hormone plus cortecosteroid | Experimental | Growth Hormone (nutropin AQ 0.075 mg/kg/day subcutaneously daily) Receives: Drug: growth hormone |
| Cortecosteroids alone | Active comparator | Cortecosteroid therapy as prescribed by the referring gastroenterologist Receives: Drug: cortecosteroid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Crohn's Disease Histologic Index of Severity (CDHIS)
The CDHIS was developed and validated in order to determine the effect of therapies upon histologic disease activity in Crohn's Disease. It has been used to assess mucosal healing in response to infliximab and 6-MP/AZA.It contains eight items which reflect epithelial injury, mucosal inflammation, and the extent of involvement. Scores range from 0-16, with patients with moderate to severely active CD typically having scores of 6-12. It was computed by a GI pathologist. The higher the score indicates worsening of disease, the lowest score is 0 and highest possible is 16
Measured: Baseline and 12 weeks
Secondary outcomes (7)
Serum IGF-1 (Insulin-like Growth Factor 1)z Score
Elevated serum IGF-1 levels have been implicated in the development of colorectal cancer, both in the general population and in patients with an excess of growth hormone production. The serum IGF-1 levels were monitored to maintain them in the physiologic range during growth hormone therapy to reduce the risk of tumorigenesis.
The levels are reported as a z score, a statistical way of standardizing data. The standard deviation is the unit of measurement of the z-score. Each z score corresponds to a point in a normal distribution, describing how much a point deviates from a mean.
Measured: Baseline, 12 weeks, 24 weeks
IMPACT III Score
Health-related quality of life (QOL)was assessed using the IMPACT 111 questionnnaire. It is a self-administered 35 item questionnaire which typically takes 10-15 minutes to complete. Scores range from 0-350, with higher scores reflecting better perceived quality of life.
Measured: Baseline, 12 weeks, 24 weeks
Pediatric Crohn's Disease Activity Index (PCDAI)
The PCDAI is a previously validated measure of clinical disease activity for children with CD. It contains three self-report items which reflect patient abdominal pain, diarrhea, and general well being; three laboratory values; height and weight velocity; and three physical examination parameters reflecting abdominal tenderness, perirectal disease, and extra-intestinal manifestations. Scores may range from 0-100. Remission is defined as 0-10, mild disease as 10-30, and moderate to severe disease as greater than 30.
Measured: Baseline, 12 and 24 weeks
Total Corticosteroid Use
The total corticosteroid use over 12 weeks between groups, using the unpaired t test.
Measured: 12 weeks
Crohn's Disease Endoscopic Index of Severity (CDEIS)
Measure of mucosal disease at baseline and week 12 obtained during colonoscopy. The CDEIS score generally ranges from 0-30. A higher score indicates more severe mucosal inflammation.
Measured: Baseline and 12 weeks
Height Velocity
Height velocity was computed every 12 weeks up to week 64 and then yearly during the Maintenance study. Since 40 to 80% of children with Crohn's disease have significant growth failure at diagnosis, height velocity is used to track for changes in height.
It is calculated by measuring height at two points of time and then dividing the change by the amount of time.
Measured: Baseline, week 12, 24 and 48
Fecal Calprotectin
Fecal calprotectin is a previously validated stool marker of intestinal inflammation in Crohn's Disease.
Measured: At 24 and 64 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Apr 2005 (estimated)
- Study startApr 2005
- Primary completionJul 2009
- Study completionAug 2009
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 Crohn's DiseaseStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Sponsor
- Investigators
- Lee Denson, M.D., Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00109473
- Sponsor's protocol ID
- CCHMC IRB #: 04-12-06
- Other IDs
- IND # 71,344
- Status last verified
- Oct 2020
- First submitted
- 28 Apr 2005
- Last updated
- 30 Oct 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Randomized Trial of Growth Hormone Therapy in Pediatric Crohn's Disease
- ClinicalTrials.gov ID
- NCT00109473
- Acronym / sponsor ID
- CCHMC IRB #: 04-12-06
- Conditions
- Crohn's Disease
- Keywords
- Pediatric Crohn's disease, Pediatric, Crohn's Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- Apr 2005
- Primary completion
- Jul 2009 (actual)
- Study completion
- Aug 2009 (actual)
- First posted
- 29 Apr 2005 (estimated)
- Results
- Posted 16 Sep 2011
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 taking a growth hormone (GH) drug called somatropin causes the intestine of a person with Crohn's Disease (CD) to heal faster when compared to a person with Crohn's Disease that does not receive growth hormone drug.
Detailed description
The optimal treatment goals in childhood CD include: 1) clinical remission in conjunction with mucosal healing and 2) restoration of normal growth and development. Current therapy in most cases includes induction of remission with corticosteroids followed by maintenance of remission with 6-mercaptopurine (6-MP) or mesalamine. With this approach, the goals of achieving mucosal healing with normalization of growth are not achieved in a significant number of children. GH therapy is now used in several chronic childhood diseases which are complicated by growth failure despite adequate GH secretion. These include chronic renal failure (CRF), juvenile rheumatoid arthritis (JRA), and Turner's syndrome. However, despite a comparable frequency and magnitude of permanent growth failure, the efficacy of GH therapy in this respect has not yet been determined in a controlled trial for CD. Moreover, whether GH therapy may also directly reduce disease activity and promote intestinal healing is not known. This represents a significant clinically unmet need in this patient population. Therefore, new therapeutic approaches are needed to both improve final adult height and enhance intestinal mucosal healing in children with CD.
The primary objective of this study is to determine the effect of growth hormone (GH) therapy upon colon mucosal healing in a 12 week randomized trial in children with Crohn's Disease (CD). Children with active CD will be randomized to GH + prednisone (GP) or prednisone alone (P) for a 12 week period. This study also involves a 52 week extension phase where all participants that meet eligibility will be given the opportunity to take or continue taking growth hormone for 52 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Growth Hormone plus cortecosteroid | Experimental | Drug: growth hormoneGrowth Hormone (nutropin AQ 0.075 mg/kg/day subcutaneously daily) |
| Cortecosteroids alone | Active comparator | Drug: cortecosteroidCortecosteroid therapy as prescribed by the referring gastroenterologist |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Ability to provide written informed consent
- Age ≥ 5 years.
- Diagnosis of Crohn's disease (CD) with ileo-colonic involvement as determined by standard clinical, radiological, and pathological criteria.
- Moderate to severely active CD as defined by a PCDAI (Pediatric Crohn's Disease Activity Index) ≥ 30.
- Currently taking Prednisone or Budesonide at starting dose (not tapering)
Key exclusion criteria (12 listed)
- Acute critical illness
- Active neoplasia
- Diabetes mellitus
- History of intracranial lesion and/or neoplasia
- Severe disease requiring hospitalization for treatment
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 hormoneAlso: Nutropin AQNutropin AQ 0.075mg/kg/day subcutaneously daily | Drug | Growth Hormone plus cortecosteroid |
| cortecosteroidAlso: Prednisone, EntocortAs prescribed by the referring gastroenterologist | Drug | Cortecosteroids alone |
Primary outcome
Crohn's Disease Histologic Index of Severity (CDHIS)
Time frame: Baseline and 12 weeks
The CDHIS was developed and validated in order to determine the effect of therapies upon histologic disease activity in Crohn's Disease. It has been used to assess mucosal healing in response to infliximab and 6-MP/AZA.It contains eight items which reflect epithelial injury, mucosal inflammation, and the extent of involvement. Scores range from 0-16, with patients with moderate to severely active CD typically having scores of 6-12. It was computed by a GI pathologist. The higher the score indicates worsening of disease, the lowest score is 0 and highest possible is 16
7 secondary outcomes
Serum IGF-1 (Insulin-like Growth Factor 1)z Score
Time frame: Baseline, 12 weeks, 24 weeks
IMPACT III Score
Time frame: Baseline, 12 weeks, 24 weeks
Pediatric Crohn's Disease Activity Index (PCDAI)
Time frame: Baseline, 12 and 24 weeks
Total Corticosteroid Use
Time frame: 12 weeks
Crohn's Disease Endoscopic Index of Severity (CDEIS)
Time frame: Baseline and 12 weeks
Height Velocity
Time frame: Baseline, week 12, 24 and 48
Fecal Calprotectin
Time frame: At 24 and 64 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Lee Denson, M.D. principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing growth hormone and cortecosteroid in people with Crohn's Disease, run by Children's Hospital Medical Center, Cincinnati 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 8 inclusion and 12 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: finished, as of the sponsor's last update on 30 Oct 2020. 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 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.
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 16 Sep 2011). 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?
Crohn's Disease Histologic Index of Severity (CDHIS) (time frame: Baseline and 12 weeks). 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 30 Oct 2020.