Clinical trial NCT00109473: Trial of Growth Hormone Therapy in Pediatric Crohn's Disease
Official title: A Phase II Randomized Trial of Growth Hormone Therapy in Pediatric Crohn's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Crohn's Disease
Keywords: Pediatric Crohn's disease
Pediatric
Crohn's Disease
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
First posted: 2005-04-29
Enrollment: 22
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Crohn's Disease Histologic Index of Severity (CDHIS)
timeFrame: Baseline and 12 weeks
Secondary outcomes:
measure: Serum IGF-1 (Insulin-like Growth Factor 1)z Score
timeFrame: Baseline, 12 weeks, 24 weeks
measure: IMPACT III Score
timeFrame: Baseline, 12 weeks, 24 weeks
measure: Pediatric Crohn's Disease Activity Index (PCDAI)
timeFrame: Baseline, 12 and 24 weeks
measure: Total Corticosteroid Use
timeFrame: 12 weeks
measure: Crohn's Disease Endoscopic Index of Severity (CDEIS)
timeFrame: Baseline and 12 weeks
measure: Height Velocity
timeFrame: Baseline, week 12, 24 and 48
measure: Fecal Calprotectin
timeFrame: At 24 and 64 weeks
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Lee Denson, M.D.
role: PRINCIPAL_INVESTIGATOR
References: