We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
ClinicalTrials.gov Menu

Trial of Growth Hormone Therapy in Pediatric Crohn's Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00109473
Recruitment Status : Completed
First Posted : April 29, 2005
Results First Posted : September 16, 2011
Last Update Posted : October 30, 2020
Genentech, Inc.
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Tracking Information
First Submitted Date  ICMJE April 28, 2005
First Posted Date  ICMJE April 29, 2005
Results First Submitted Date  ICMJE June 24, 2011
Results First Posted Date  ICMJE September 16, 2011
Last Update Posted Date October 30, 2020
Study Start Date  ICMJE April 2005
Actual Primary Completion Date July 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 10, 2011)
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
Original Primary Outcome Measures  ICMJE
 (submitted: June 23, 2005)
Crohn's Disease Histologic Index of Severity
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 27, 2020)
  • Serum IGF-1 (Insulin-like Growth Factor 1)z Score [ Time Frame: Baseline, 12 weeks, 24 weeks ]
    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.
  • IMPACT III Score [ Time Frame: Baseline, 12 weeks, 24 weeks ]
    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.
  • Pediatric Crohn's Disease Activity Index (PCDAI) [ Time Frame: Baseline, 12 and 24 weeks ]
    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.
  • Total Corticosteroid Use [ Time Frame: 12 weeks ]
    The total corticosteroid use over 12 weeks between groups, using the unpaired t test.
  • Crohn's Disease Endoscopic Index of Severity (CDEIS) [ Time Frame: Baseline and 12 weeks ]
    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.
  • Height Velocity [ Time Frame: Baseline, week 12, 24 and 48 ]
    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.
  • Fecal Calprotectin [ Time Frame: At 24 and 64 weeks ]
    Fecal calprotectin is a previously validated stool marker of intestinal inflammation in Crohn's Disease.
Original Secondary Outcome Measures  ICMJE
 (submitted: June 23, 2005)
  • Serum IGF-1
  • Colon crypt epithelial cell (CEC) proliferation labeling index
  • Pediatric Crohn's disease Activity Index
  • Total corticosteroid use
  • Intra-abdominal fat
  • Height velocity
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Trial of Growth Hormone Therapy in Pediatric Crohn's Disease
Official Title  ICMJE A Phase II Randomized Trial of Growth Hormone Therapy in Pediatric Crohn's Disease
Brief 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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Crohn's Disease
Intervention  ICMJE
  • Drug: growth hormone
    Nutropin AQ 0.075mg/kg/day subcutaneously daily
    Other Name: Nutropin AQ
  • Drug: cortecosteroid
    As prescribed by the referring gastroenterologist
    Other Name: Prednisone, Entocort
Study Arms  ICMJE
  • Experimental: Growth Hormone plus cortecosteroid
    Growth Hormone (nutropin AQ 0.075 mg/kg/day subcutaneously daily)
    Intervention: Drug: growth hormone
  • Active Comparator: Cortecosteroids alone
    Cortecosteroid therapy as prescribed by the referring gastroenterologist
    Intervention: Drug: cortecosteroid
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: October 7, 2009)
Original Enrollment  ICMJE
 (submitted: June 23, 2005)
Actual Study Completion Date  ICMJE August 2009
Actual Primary Completion Date July 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00109473
Other Study ID Numbers  ICMJE CCHMC IRB #: 04-12-06
IND # 71,344 ( Other Identifier: FDA )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Children's Hospital Medical Center, Cincinnati
Original Responsible Party Not Provided
Current Study Sponsor  ICMJE Children's Hospital Medical Center, Cincinnati
Original Study Sponsor  ICMJE Denson, Lee, M.D.
Collaborators  ICMJE Genentech, Inc.
Investigators  ICMJE
Principal Investigator: Lee Denson, M.D. Children's Hospital Medical Center, Cincinnati
PRS Account Children's Hospital Medical Center, Cincinnati
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP