Effects of butyrate enemas on postoperative intestinal motility disorders in Hirschsprung’s disease

2025-523094-42-00 Protocol RCAPHM18_0018 Therapeutic confirmatory (Phase III) Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 22 sites · Protocol RCAPHM18_0018

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)
Status Authorised, recruitment pending
Participants planned 58
Countries 1
Sites 22

Hirschsprung's disease

Assess the short-term efficacy of preoperative butyrate enemas at reducing the time to recovery of bowel function after curative surgery in HD patients.

Key facts

Sponsor
Assistance Publique Hopitaux De Marseille
Participant type
Pediatric, Patients
Age range
0-17 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Decision date (initial)
2025-12-16
Transition trial
No
Low-intervention
No
Rare-disease indication
Yes
Vulnerable population
Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Prophylaxis

Assess the short-term efficacy of preoperative butyrate enemas at reducing the time to recovery of bowel function after curative surgery in HD patients.

Secondary objectives 5

  1. Incidence of postoperative functional intestinal obstructive symptoms
  2. Evaluation of stool consistency
  3. Measurement of total intestinal transit time after surgery
  4. Incidence and grading of postoperative Hirschsprung-associated enterocolitis (HAEC)
  5. Measurement of postoperative intestinal inflammatory response

Conditions and MedDRA coding

Hirschsprung's disease

VersionLevelCodeTermSystem organ class
20.0 PT 10010539 Congenital megacolon 100000004850

Regulatory references

Plan to share IPD
No
EU CT numberTitleSponsor
2023-509697-29-00 Effects of butyrate enemas on postoperative intestinal motility disorders in Hirschsprung’s disease Assistance Publique Hopitaux De Marseille

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

  1. Newborn with a diagnosis of Hirschsprung’s disease the 2 first months of life managed successfully with colonic decompressions/irrigations before curative surgery
  2. Born at or after 35 weeks of gestation (37 weeks of amenorrhea)
  3. Short-segment Hirschsprung’s disease limited to the rectum and/or sigmoid colon diagnosed on rectal biopsy with established pathological criteria (absence of ganglionic cells +/- hypertrophic extrinsic nerve fibres)
  4. Uncomplicated form (without enterocolitis as defined by the HAEC score and/or diverting colostomy)
  5. Curative surgery and follow-up in one of the included centres
  6. With consent of the 2 parents or legal(s) representative(s)
  7. Absence of severe or lethal associated malformations
  8. Affiliation with the French social security system

Exclusion criteria 8

  1. Long segment Hirschsprung’s disease prior to the junction between the left colon and the sigmoid colon
  2. Hirschsprung’s disease not managed successfully with colonic decompressions/irrigations and requiring a diverting colostomy before the curative surgery
  3. Hirschsprung-associated enterocolitis occurring before the randomization
  4. Severe or lethal associated malformation, including Down syndrome
  5. Intestinal associated malformations (intestinal atresia, gastroschisis, omphalocele, intestinal malrotation and volvulus)
  6. Any pathological condition that can modify intestinal motility or intestinal transit time (cystic fibrosis, hypothyroidism)
  7. Refusal of parent(s) or legal representative(s)
  8. Neonates under tutorship or guardianship

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Time to recovery of bowel function after the curative surgery

Secondary endpoints 5

  1. Assessmenof postoperative functional intestinal obstructive symptoms
  2. Stool consistency evaluated using the validated ‘Bristol’ stool form scale modified for children
  3. Red carmine total intestinal transit time measured
  4. HAEC episode(s) defined by the HAEC score (Pastor et al., 2009; Frykman et al., 2017) and grading of HAEC (Gosain et al, 2017).
  5. Fecal calprotectin measurement

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sodium butyrate 1,1 g/L sol prep

PRD12050062 · Product

Active substance
Sodium Butyrate
Pharmaceutical form
ENEMA
Route of administration
OTHER USE
Max daily dose
10 Other
Max total dose
10 Other
Max treatment duration
4 Week(s)
Authorisation status
Not Authorised
MA holder
APHM
Paediatric formulation
No
Orphan designation
No

Auxiliary 1

-

V04CX · Product

Pharmaceutical form
PHF00006MIG
Route of administration
ORAL USE
Max daily dose
250 mg milligram(s)
Max total dose
250 mg milligram(s)
Max treatment duration
1 Month(s)
Authorisation status
Authorised
ATC code
V04CX — OTHER DIAGNOSTIC AGENTS
Marketing authorisation
-
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Marseille

9 Total trials 4 Recruiting 1 Ended
Academic / Non-commercial
Sponsor organisation
Assistance Publique Hopitaux De Marseille
Address
80 Rue Brochier
City
Marseille
Postcode
13005
Country
France

Scientific contact point

Organisation
Assistance Publique Hopitaux De Marseille
Contact name
Pediatric surgeon

Public contact point

Organisation
Assistance Publique Hopitaux De Marseille
Contact name
Pediatric surgeon

Locations

1 EU/EEA country · 22 investigational sites

By country

CountryMS statusPlanned subjectsSites
France Authorised, recruitment pending 58 22
Rest of world — 0 —

Investigational sites

France

22 sites · Authorised, recruitment pending
Centre Hospitalier Universitaire De Lille
Pediatric surgery, Avenue Eugene Avinee, 59037, Lille Cedex
Centre Hospitalier Regional D'Angers
Pediatric surgery, pôle FME, 4 Rue Larrey, 49100, Angers
Centre Hospitalier Universitaire De Saint Etienne
Chirurgie pédiatrique, St Priest En Jarez, 25 Boulevard Pasteur, St Etienne Cedex 2
Centre Hospitalier Universitaire De Nantes
Pediatric Surgery, 5 Allee De L Ile Gloriette, Cs 69301, Nantes Cedex 1
Assistance Publique Hopitaux De Paris
Chirurgie viscérale et thoracique pédiatriques, 48 Boulevard Serurier, 75019, Paris
Centre Hospitalier Et Universitaire De Limoges
chirurgie viscérale pédiatrique, 2 Avenue Martin Luther King, 87000, Limoges
Centre Hospitalier Universitaire De Montpellier
Chirurgie Infantile Viscérale, Urologique, 191 Avenue Du Doyen Gaston Giraud, 34295, Montpellier Cedex 5
Fondation Lenval Nice
chirurgie visérale, 57 Avenue De La Californie, 06200, Nice
Centre Hospitalier Universitaire Rouen
Pediatric surgery, 1 Rue De Germont, 76000, Rouen
CHRU De Nancy
Chirurgie Viscérale Infantile, 29 Avenue Du Mal De Lattre De Tassigny, 54000, Nancy
Centre Hospitalier Regional De Marseille
Chirurgie infantile, 264 Rue Saint Pierre, 13005, Marseille
Centre Hospitalier Universitaire Reims
Chirurgie pédiatrique, 45 Rue Cognacq Jay, 51100, Reims
Centre Hospitalier Universitaire De Rennes
Chirurgie pédiatrique, 16 Boulevard De Bulgarie, Bp 90349, Rennes
Assistance Publique Hopitaux De Paris
Digestive and Urologic pediatric surgery, 149 Rue De Sevres, 75015, Paris
Centre Hospitalier Universitaire De Toulouse
chirurgie pédiatrique viscérale, 1 Avenue Irene Joliot Curie, 31059, Toulouse Cedex 9
Centre Hospitalier Regional Et Universitaire De Brest
chirurgie pédiatrique, 2 Avenue Marechal Foch, 29200, Brest
Hospices Civils De Lyon
Chirurgie pédiatrique uro-viscerale, thoracique et transplantation, 59 Boulevard Pinel, 69500, Bron
Centre Hospitalier Universitaire De Caen Normandie
Chirurgie Pédiatrique, Avenue De La Cote De Nacre, 14000, Caen
Les Hopitaux Universitaires De Strasbourg
Pediatric surgery, 1 Avenue Moliere, Bp 49, Strasbourg Cedex 2
Assistance Publique Hopitaux De Paris
Chirurgie viscérale pédiatrique et néonatale, 26 Avenue Du Docteur Arnold Netter, 75012, Paris
Centre Hospitalier Universitaire Grenoble Alpes
Chirurgie pediatrique viscérale, Boulevard De La Chantourne, Cs 10217, Grenoble Cedex 9
Centre Hospitalier Universitaire De Poitiers
Chirurgie Pédiatrique, 2 Rue De La Miletrie, 86000, Poitiers

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 7 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

TypeTitleVersion
Protocol (for publication) D1_ Protocol EU CT 2025-523094-42-00 BUTY-HIRSCH_TC 1.3
Protocol (for publication) D1_Protocol_2025-523094-42-00_BUTY-HIRSCH 1.3
Recruitment arrangements (for publication) K1_Recruitment arrangements 1
Subject information and informed consent form (for publication) L1_SIS and ICF Parents 1.2
Subject information and informed consent form (for publication) L1_SIS and ICF Parents_TC 1.2
Synopsis of the protocol (for publication) D1_Protocol synopsis FR_2025-523094-42-00_BUTY-HIRSCH 1.3
Synopsis of the protocol (for publication) D1_Protocol synopsis FR_2025-523094-42-00_BUTY-HIRSCH_TC 1.3

Application history

1 submissions — initial application plus substantial / non-substantial modifications

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2025-09-08 France Acceptable
2025-12-05
 2025-12-16