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Trial record 7 of 41 for:    short bowel syndrome | Recruiting, Not yet recruiting, Available Studies

Characterization of the Long-term Safety, Efficacy, and Pharmacodynamics Revestive® in the Management of Short Bowel Syndrome Pediatric Patients (REVE)

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ClinicalTrials.gov Identifier: NCT03562130
Recruitment Status : Not yet recruiting
First Posted : June 19, 2018
Last Update Posted : June 19, 2018
Sponsor:
Collaborator:
Imagine Institute
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Brief Summary:
The purpose of this study is to evaluate if the treatment could maximize intestinal absorption, minimize the inconvenience of diarrhea, and avoid, reduce or eliminate the need for parenteral support (PS) to achieve normal growth, to avoid parenteral nutrition complications and to achieve the best possible quality of life for the patient

Condition or disease Intervention/treatment Phase
Short Bowel Syndrome Drug: Teduglutide Phase 4

Detailed Description:

The short bowel syndrome (SBS) may be defined as a severe malabsorption caused by reduction of intestinal absorptive surface following massive resection of the small intestine. Teduglutide (Revestive®) is an analog of glucagon-like peptide 2 (GLP-2), a naturally occurring hormone that regulates the functional and structural integrity of the cells lining the gastrointestinal tract. The aim of the treatment is to maximize intestinal absorption, minimize the inconvenience of diarrhea, and avoid, reduce or eliminate the need for parenteral support (PS) to achieve the best possible quality of life for the patient. The rationale for the use of Revestive® is based on data obtained, especially in the trial in SBS patients.

Treatment with 0.05 mg/kg/day was safe and well tolerated (no recorded side effects).

Patients remained stable despite substantial reduction in parenteral nutrition (PN) supply as evidenced by stable body weight and height, serum electrolytes, pancreatic enzymes and renal function tests.

Treatment was associated with:

  • Reduced PN volume and calories delivered by 25 and 45% respectively with 20% of patients weaned off PN during the study period
  • Increased Enteral Nutrition (EN) supply in volume and calories by 40 and 62% respectively
  • Increased in plasma citrulline during the treatment period, but decreased after Teduglutide discontinuation The recommended dose of Revestive® in children and adolescents (aged 1 to 17 years) is the same as for adults (0.05 mg/kg body weight once daily).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Monocentric Single-arm Study to Characterize the Long-term Safety, Efficacy, and Pharmacodynamic of GLP-2 Analog (Revestive®) in the Management of Short Bowel Syndrome Pediatric Patients on Home-parenteral Nutrition (HPN)
Estimated Study Start Date : June 2018
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Teduglutide

Arm Intervention/treatment
Experimental: Revestive
Revestive® (teduglutide)is administered in children sub cutaneous injection at 0.05 mg/kg body weight once daily
Drug: Teduglutide
Daily sub cutaneous injection 0,05 mg/kg/day




Primary Outcome Measures :
  1. Decrease in parenteral nutrition: Parenteral Nutrition/Resting Energy Expenditure (PN/REE) [ Time Frame: At week 24 ]
    Evaluate the efficacy of Revestive® treatment


Secondary Outcome Measures :
  1. Ostomy output defined as stool balance testing, urine output and plasma citrulline [ Time Frame: up to week 48 ]
    Evaluate the impact of Revestive on ostomy flow

  2. Change in days per week of Parenteral Nutrition (PN) [ Time Frame: up to week 48 ]
    Quantify the impact of Revestive on the number of perfusion in a week

  3. Change in number of stool per day [ Time Frame: up to week 48 ]
    to evaluate the impact of Revestive on diarrhea

  4. Change in stools consistency (Bristol stool chart) [ Time Frame: up to week 48 ]
    to evaluate the impact of Revestive on diarrhea

  5. Ingesta (calorimetric measure) [ Time Frame: Every 4 weeks up to week 48 ]
    to evaluate the impact of Revestive on Intestinal absorption

  6. Stool weight/24h [ Time Frame: Every 4 weeks up to week 48 ]
    to evaluate the impact of Revestive on Intestinal absorption

  7. Percentage of lipid in stool [ Time Frame: Every 4 weeks up to week 48 ]
    to evaluate the impact of Revestive on Intestinal absorption

  8. Percentage of nitrogen in stool [ Time Frame: Every 4 weeks up to week 48 ]
    to evaluate the impact of Revestive on Intestinal absorption

  9. Percentage of carbohydrate in stool [ Time Frame: Every 4 weeks up to week 48 ]
    to evaluate the impact of Revestive on Intestinal absorption

  10. Percentage of sodium in stool [ Time Frame: Every 4 weeks up to week 48 ]
    to evaluate the impact of Revestive on Intestinal absorption

  11. Number of adverse events [ Time Frame: At week 48 ]
    to evaluate the long term safety of Revestive

  12. Change in body weight [ Time Frame: At baseline, then at 6 and 12 months ]
  13. Change in heart rate [ Time Frame: At baseline, then at 6 and 12 months ]
  14. Change in blood pressure [ Time Frame: At baseline, then at 6 and 12 months ]
  15. Endogenous GLP-2 rates (antibody ELISA) [ Time Frame: up to week 48 ]
    to evaluate the response rate of Revestive



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Being aged from 2 to 18 years old included ;
  • Presenting less than 80 cm of residual small intestine with or without the terminal ileum, ileocecal valve and right colon or having less than 120 cm in case of Short Bowel Syndrome (SBS) caused by Hirschsprung disease;
  • Being stable on PN support (inability to significantly reduce PN intake for the last six months before inclusion) ;
  • Being dependent on PN for at least 2 years and enterally fed (oral or tube feeding) ;
  • Having a normal colonoscopy in the 12 months before screening for children with maintained colon (=SBS type 2 or 3) older than 12 years ;
  • Having signed the Informed consent form (or parents or legal representative for minor patients).

Exclusion Criteria:

  • Having a major gastrointestinal surgical intervention like serial transverse enteroplasty or any other bowel lengthening procedure performed within 6 months of screening ;
  • Having a clinically significant untreated intestinal obstruction or active stenosis ;
  • Having an unstable absorption due to cystic fibrosis or known DNA abnormalities ;
  • Presenting a radiographic or manometric evidence of pseudo-obstruction or severe known dysmotility syndrome, including persistent, severe gastroschisis-related motility disorders ;
  • Having an unstable cardiac disease, congenital heart disease or cyanotic disease, with the exception of patients who had undergone ventricular or atrial septal defect repair ;
  • Having a history of cancer or clinically significant lymphoproliferative disease; excepted resected cutaneous basal or squamous cell carcinoma, or in situ non-aggressive and surgically resected cancer ;
  • Having participated in a clinical study using an experimental drug within 1 month or an experimental antibody treatment within 3 months prior to screening, or concurrent participation in any clinical study using an experimental drug that would affect the safety of teduglutide ;
  • Having already used native GLP-2 and glucagon-like peptide-1 analog or human growth hormone within 3 months prior to screening ;
  • Having already used oral or IV glutamine, octreotide, or dipeptidyl peptidase IV (DPP-IV) inhibitors within 3 months prior to screening ;
  • Having an active Crohn's disease which has been treated with biological therapy within the 6 months prior to screening ;
  • Having an intestinal polyposis;
  • Being, for female patient, both lactating and breast-feeding or having a positive pregnancy test during the screening period;
  • Refusing the follow the protocol requirements in terms of birth control ;
  • Being unable to follow the study procedures for any reason: psychological, geographical…
  • Hypersensitivity to the active substance or to any of the excipients listed in section 6.1 of Summary of Product Characteristics (SPC), or trace residues of tetracycline.
  • Active or suspected malignancy.
  • Patients with a history of malignancies in the gastrointestinal tract including the hepatobiliary system within the last five years.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03562130


Contacts
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Contact: Olivier GOULET, MD, PhD + 33 1 44 49 25 16 olivier.goulet@aphp.fr
Contact: Prissile BAKOUBOULA, PhD +33 1 71 19 64 94 prissile.bakouboula@aphp.fr

Locations
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France
Hôpital Necker - Enfants malades Not yet recruiting
Paris, France, 75015
Contact: Olivier GOULET, MD, PhD    + 33 1 44 49 25 16    olivier.goulet@aphp.fr   
Contact: Cécile LAMBE, MD    + 33 1 44 49 25 60    cecile.lambe@aphp.fr   
Sub-Investigator: Cécile TALBOTEC, MD         
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Imagine Institute
Investigators
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Principal Investigator: Olivier GOULET, MD, PhD Assistance Publique - Hôpitaux de Paris

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Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT03562130     History of Changes
Other Study ID Numbers: P171002J
2017-001405-32 ( EudraCT Number )
First Posted: June 19, 2018    Key Record Dates
Last Update Posted: June 19, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Teduglutide
Short Bowel Syndrome
Parenteral nutrition
Intestinal failure

Additional relevant MeSH terms:
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Syndrome
Short Bowel Syndrome
Malabsorption Syndromes
Intestinal Diseases
Disease
Pathologic Processes
Gastrointestinal Diseases
Digestive System Diseases
Postoperative Complications
Teduglutide
Gastrointestinal Agents
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs