NCT04170192

Brief Summary

Very early onset inflammatory bowel disease (VEO-IBD) is a special subtype of children's inflammatory bowel disease (IBD). VEO-IBD is mostly caused by single-gene defects and can be cured by allo-hematopoietic stem cell transplantation ( HSCT). Umbilical Cord Blood Transplantation (UCBT) is less reported in these patients.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
50

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Dec 2019

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 12, 2019

Completed
8 days until next milestone

First Posted

Study publicly available on registry

November 20, 2019

Completed
11 days until next milestone

Study Start

First participant enrolled

December 1, 2019

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2022

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2023

Completed
Last Updated

February 17, 2020

Status Verified

February 1, 2020

Enrollment Period

2.9 years

First QC Date

November 12, 2019

Last Update Submit

February 13, 2020

Conditions

Inflammatory Bowel Diseases

Keywords

Umbilical Cord Blood Stem Cell TransplantationInflammatory Bowel DiseaseInterleukin 10 Receptor

Outcome Measures

Primary Outcomes (1)

  • Overall survival rate

    Overall survival is defined as the survival status of patients by the end of 3 years after the transplanting, coded as 1 for dead and 0 for survive.

    3 years after transplantation

Secondary Outcomes (2)

  • Disease free survival rate

    3 years after transplantation

  • Successful engraftment

    3 years after transplantation

Other Outcomes (4)

  • The occurrence of adverse events (AE)

    3 years after transplantation

  • The occurrence of graft-versus-host disease (GVHD)

    3 years after transplantation

  • Transplant-related mortality

    3 years after transplantation

  • +1 more other outcomes

Study Arms (1)

UCBT-IBD-Case

Very early onset IBD patients who underwent Cord Blood Stem Cell Transplantation.

Procedure: Cord Blood Stem Cell Transplantation

Interventions

Cord Blood Stem Cell TransplantationPROCEDURE

Unrelated cord blood stem cell selection; Reduced intensity conditioning regime; GVHD prevention; Infection prevention.

UCBT-IBD-Case

Eligibility Criteria

Age1 Month - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Patients with very early-onset inflammatory bowel disease with interleukin-10 receptor gene deficiency through gastroenterology, pathology and gene diagnosis institutions to improve enteroscopy, histopathology and gene detection.

You may qualify if:

  • Diagnosis: All selected cases will be diagnosed as very early-onset inflammatory bowel disease with interleukin-10 receptor gene deficiency through gastroenterology, pathology and gene diagnosis institutions to improve enteroscopy, histopathology and gene detection.
  • The patients have no HLA matched sibling donor.
  • All organs function normally and meet the following test criteria:
  • Liver function ALT, AST \< 10 times normal value upper limit, TBIL \< 5 times normal value upper limit.
  • Renal function BUN and Cr \< 1.25 times the upper limit of normal value. ECG and echocardiography showed no cardiac insufficiency.
  • The legal guardian of the patient has the desire and requirement for the treatment of allogeneic umbilical cord blood stem cell transplantation, and signs the informed consent before treatment. The informed consent should inform all relevant contents of clinical research, patients are willing and abide by the treatment plan, follow-up plan, laboratory examination, etc.

You may not qualify if:

  • There are any contraindications of hematopoietic stem cell transplantation.
  • There are other serious diseases, such as severe damage to vital organ functions: respiratory failure, cardiac insufficiency, decompensated liver insufficiency, renal insufficiency, uncontrollable infection and so on.
  • Other drug clinical researchers are under way.
  • Simultaneously suffering from other serious acute or chronic physical or mental diseases, or abnormal laboratory examinations, may affect patient's life safety and compliance, and affect informed consent, research participation, follow-up or result interpretation.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, Minhang, 201102, China

Location

Related Publications (11)

  • Bianco AM, Girardelli M, Tommasini A. Genetics of inflammatory bowel disease from multifactorial to monogenic forms. World J Gastroenterol. 2015 Nov 21;21(43):12296-310. doi: 10.3748/wjg.v21.i43.12296.

    PMID: 26604638RESULT

  • Kotlarz D, Beier R, Murugan D, Diestelhorst J, Jensen O, Boztug K, Pfeifer D, Kreipe H, Pfister ED, Baumann U, Puchalka J, Bohne J, Egritas O, Dalgic B, Kolho KL, Sauerbrey A, Buderus S, Gungor T, Enninger A, Koda YK, Guariso G, Weiss B, Corbacioglu S, Socha P, Uslu N, Metin A, Wahbeh GT, Husain K, Ramadan D, Al-Herz W, Grimbacher B, Sauer M, Sykora KW, Koletzko S, Klein C. Loss of interleukin-10 signaling and infantile inflammatory bowel disease: implications for diagnosis and therapy. Gastroenterology. 2012 Aug;143(2):347-55. doi: 10.1053/j.gastro.2012.04.045. Epub 2012 Apr 28.

    PMID: 22549091RESULT

  • Engelhardt KR, Shah N, Faizura-Yeop I, Kocacik Uygun DF, Frede N, Muise AM, Shteyer E, Filiz S, Chee R, Elawad M, Hartmann B, Arkwright PD, Dvorak C, Klein C, Puck JM, Grimbacher B, Glocker EO. Clinical outcome in IL-10- and IL-10 receptor-deficient patients with or without hematopoietic stem cell transplantation. J Allergy Clin Immunol. 2013 Mar;131(3):825-30. doi: 10.1016/j.jaci.2012.09.025. Epub 2012 Nov 14.

    PMID: 23158016RESULT

  • Pigneur B, Escher J, Elawad M, Lima R, Buderus S, Kierkus J, Guariso G, Canioni D, Lambot K, Talbotec C, Shah N, Begue B, Rieux-Laucat F, Goulet O, Cerf-Bensussan N, Neven B, Ruemmele FM. Phenotypic characterization of very early-onset IBD due to mutations in the IL10, IL10 receptor alpha or beta gene: a survey of the Genius Working Group. Inflamm Bowel Dis. 2013 Dec;19(13):2820-8. doi: 10.1097/01.MIB.0000435439.22484.d3.

    PMID: 24216686RESULT

  • Huang Z, Peng K, Li X, Zhao R, You J, Cheng X, Wang Z, Wang Y, Wu B, Wang H, Zeng H, Yu Z, Zheng C, Wang Y, Huang Y. Mutations in Interleukin-10 Receptor and Clinical Phenotypes in Patients with Very Early Onset Inflammatory Bowel Disease: A Chinese VEO-IBD Collaboration Group Survey. Inflamm Bowel Dis. 2017 Apr;23(4):578-590. doi: 10.1097/MIB.0000000000001058.

    PMID: 28267044RESULT

  • Silverberg MS, Satsangi J, Ahmad T, Arnott ID, Bernstein CN, Brant SR, Caprilli R, Colombel JF, Gasche C, Geboes K, Jewell DP, Karban A, Loftus EV Jr, Pena AS, Riddell RH, Sachar DB, Schreiber S, Steinhart AH, Targan SR, Vermeire S, Warren BF. Toward an integrated clinical, molecular and serological classification of inflammatory bowel disease: report of a Working Party of the 2005 Montreal World Congress of Gastroenterology. Can J Gastroenterol. 2005 Sep;19 Suppl A:5A-36A. doi: 10.1155/2005/269076.

    PMID: 16151544RESULT

  • Levine A, Griffiths A, Markowitz J, Wilson DC, Turner D, Russell RK, Fell J, Ruemmele FM, Walters T, Sherlock M, Dubinsky M, Hyams JS. Pediatric modification of the Montreal classification for inflammatory bowel disease: the Paris classification. Inflamm Bowel Dis. 2011 Jun;17(6):1314-21. doi: 10.1002/ibd.21493. Epub 2010 Nov 8.

    PMID: 21560194RESULT

  • Uhlig HH, Schwerd T, Koletzko S, Shah N, Kammermeier J, Elkadri A, Ouahed J, Wilson DC, Travis SP, Turner D, Klein C, Snapper SB, Muise AM; COLORS in IBD Study Group and NEOPICS. The diagnostic approach to monogenic very early onset inflammatory bowel disease. Gastroenterology. 2014 Nov;147(5):990-1007.e3. doi: 10.1053/j.gastro.2014.07.023. Epub 2014 Jul 21.

    PMID: 25058236RESULT

  • Kelsen JR, Dawany N, Moran CJ, Petersen BS, Sarmady M, Sasson A, Pauly-Hubbard H, Martinez A, Maurer K, Soong J, Rappaport E, Franke A, Keller A, Winter HS, Mamula P, Piccoli D, Artis D, Sonnenberg GF, Daly M, Sullivan KE, Baldassano RN, Devoto M. Exome sequencing analysis reveals variants in primary immunodeficiency genes in patients with very early onset inflammatory bowel disease. Gastroenterology. 2015 Nov;149(6):1415-24. doi: 10.1053/j.gastro.2015.07.006. Epub 2015 Jul 17.

    PMID: 26193622RESULT

  • Glocker EO, Kotlarz D, Boztug K, Gertz EM, Schaffer AA, Noyan F, Perro M, Diestelhorst J, Allroth A, Murugan D, Hatscher N, Pfeifer D, Sykora KW, Sauer M, Kreipe H, Lacher M, Nustede R, Woellner C, Baumann U, Salzer U, Koletzko S, Shah N, Segal AW, Sauerbrey A, Buderus S, Snapper SB, Grimbacher B, Klein C. Inflammatory bowel disease and mutations affecting the interleukin-10 receptor. N Engl J Med. 2009 Nov 19;361(21):2033-45. doi: 10.1056/NEJMoa0907206. Epub 2009 Nov 4.

    PMID: 19890111RESULT

  • Peng K, Qian X, Huang Z, Lu J, Wang Y, Zhou Y, Wang H, Wu B, Wang Y, Chen L, Zhai X, Huang Y. Umbilical Cord Blood Transplantation Corrects Very Early-Onset Inflammatory Bowel Disease in Chinese Patients With IL10RA-Associated Immune Deficiency. Inflamm Bowel Dis. 2018 Jun 8;24(7):1416-1427. doi: 10.1093/ibd/izy028.

    PMID: 29788474RESULT

Biospecimen

Retention: SAMPLES WITH DNA

blood sample of every patient will be collected pre and post-transplant

MeSH Terms

Conditions

Inflammatory Bowel DiseasesInflammatory Bowel Disease 28, Autosomal Recessive

Interventions

Cord Blood Stem Cell Transplantation

Condition Hierarchy (Ancestors)

GastroenteritisGastrointestinal DiseasesDigestive System DiseasesIntestinal Diseases

Intervention Hierarchy (Ancestors)

Stem Cell TransplantationCell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Study Officials

  • Jiang Hui, Master

    Shanghai Children's Hospital

    STUDY DIRECTOR

  • Hu Shaoyan, PHD

    Children's Hospital of Soochow University

    STUDY DIRECTOR

  • Qin Maoquan, PHD

    Beijing Children's Hospital

    STUDY DIRECTOR

  • Jiang Hua, PHD

    Guangzhou Women and Children's Medical Center

    STUDY DIRECTOR

  • Liu Sixi, Master

    Shenzhen Children's Hospital

    STUDY DIRECTOR

  • Xiong Hao, PHD

    Wuhan Women and Children's Medical Center

    STUDY DIRECTOR

  • Fang Yongjun, PHD

    Children's Hospital of Nanjing Medical University

    STUDY DIRECTOR

  • Wang Dao, PHD

    The First Affiliated Hospital of Zhengzhou University

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
vice president

Study Record Dates

First Submitted

November 12, 2019

First Posted

November 20, 2019

Study Start

December 1, 2019

Primary Completion

October 31, 2022

Study Completion

October 31, 2023

Last Updated

February 17, 2020

Record last verified: 2020-02

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)