Multi-center Clinical Study of Cord Blood Stem Cell Transplantation for SCID

NCT04172181 | Unknown DMC

• 1 other identifier: CPBMT-SCID-2019
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

Severe combined immunodeficiency (SCID) is a rare disease caused by a group of genetic disorders that leads to early death from recurrent infections in affected children.The only curative therapy for SCID is allogeneic hematopoietic stem cell transplantation.Unrelated umbilical cord blood(UCB) is increasingly used as an alternative to bone marrow.

Conditions

Severe Combined Immunodeficiency Disease

Keywords

cord blood stem cell transplantation; severe combined immunodeficiency disease; primary immunodeficiency

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 (3)

• Disease free survival rate

  3 years after transplantation

• Successful engraftment

  3 years after transplantation

• Immune reconstitution

  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

Study Arms (1)

UCBT-SCID-Case

SCID patients who underwent cord blood stem cell transplantation.The only curative therapy for SCID is allogeneic hematopoietic stem cell transplantation.

Procedure: cord blood stem cell transplantation

Interventions

cord blood stem cell transplantation PROCEDURE

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

UCBT-SCID-Case

Eligibility Criteria

• Age: 1 Month - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Patients were diagnosed as severe combined immunodeficiency disease by immunological function and genetic diagnosis center.

You may qualify if:

• All patients were diagnosed as severe combined immunodeficiency disease by immunological function and genetic diagnosis center.
• Patients have no HLA-matched related donor.
• Each organ functions normally and conforms the following inspection criteria:
• Liver function ALT, AST ≤ 10 times the upper limit of normal value, TBIL ≤ 5 times the upper limit of normal value.
• Renal function BUN, Cr ≤ 1.25 times the upper limit of normal value. ECG, cardiac examination normal

You may not qualify if:

• Patients have any contraindications to hematopoietic stem cell transplantation.
• Patients have other serious diseases, such as serious damage to vital organ function: respiratory failure, cardiac insufficiency, decompensated liver dysfunction, renal insufficiency, uncontrollable infection, etc.
• The patient is undergoing other drug clinical research.
• At the same time suffering from other serious acute or chronic physical or mental illness, or laboratory abnormalities, may affect patient safety and compliance, affecting informed consent, research participation, follow-up or interpretation of results.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead
• Shanghai Children's Hospital: collaborator
• Children's Hospital of Soochow University: collaborator
• Beijing Children's Hospital: collaborator
• Guangzhou Women and Children's Medical Center: collaborator
• Shenzhen Children's Hospital: collaborator
• Wuhan Women and Children's Medical Center: collaborator
• Children's Hospital of Nanjing Medical University: collaborator
• The First Affiliated Hospital of Zhengzhou University: collaborator

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, Minhang, 201102, China

Location

Related Publications (22)

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  PMID: 21479529 RESULT

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• Routes JM, Grossman WJ, Verbsky J, Laessig RH, Hoffman GL, Brokopp CD, Baker MW. Statewide newborn screening for severe T-cell lymphopenia. JAMA. 2009 Dec 9;302(22):2465-70. doi: 10.1001/jama.2009.1806.

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• Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet. 1968 Dec 28;2(7583):1366-9. doi: 10.1016/s0140-6736(68)92673-1. No abstract available.

  PMID: 4177932 RESULT

• Antoine C, Muller S, Cant A, Cavazzana-Calvo M, Veys P, Vossen J, Fasth A, Heilmann C, Wulffraat N, Seger R, Blanche S, Friedrich W, Abinun M, Davies G, Bredius R, Schulz A, Landais P, Fischer A; European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet. 2003 Feb 15;361(9357):553-60. doi: 10.1016/s0140-6736(03)12513-5.

  PMID: 12598139 RESULT

• Fernandes JF, Rocha V, Labopin M, Neven B, Moshous D, Gennery AR, Friedrich W, Porta F, Diaz de Heredia C, Wall D, Bertrand Y, Veys P, Slatter M, Schulz A, Chan KW, Grimley M, Ayas M, Gungor T, Ebell W, Bonfim C, Kalwak K, Taupin P, Blanche S, Gaspar HB, Landais P, Fischer A, Gluckman E, Cavazzana-Calvo M; Eurocord and Inborn Errors Working Party of European Group for Blood and Marrow Transplantation. Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? Blood. 2012 Mar 22;119(12):2949-55. doi: 10.1182/blood-2011-06-363572. Epub 2012 Feb 3.

  PMID: 22308292 RESULT

• Chiesa R, Gilmour K, Qasim W, Adams S, Worth AJ, Zhan H, Montiel-Equihua CA, Derniame S, Cale C, Rao K, Hiwarkar P, Hough R, Saudemont A, Fahrenkrog CS, Goulden N, Amrolia PJ, Veys P. Omission of in vivo T-cell depletion promotes rapid expansion of naive CD4+ cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant. Br J Haematol. 2012 Mar;156(5):656-66. doi: 10.1111/j.1365-2141.2011.08994.x. Epub 2012 Jan 9.

  PMID: 22224700 RESULT

• Gennery AR, Slatter MA, Grandin L, Taupin P, Cant AJ, Veys P, Amrolia PJ, Gaspar HB, Davies EG, Friedrich W, Hoenig M, Notarangelo LD, Mazzolari E, Porta F, Bredius RG, Lankester AC, Wulffraat NM, Seger R, Gungor T, Fasth A, Sedlacek P, Neven B, Blanche S, Fischer A, Cavazzana-Calvo M, Landais P; Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation; European Society for Immunodeficiency. Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol. 2010 Sep;126(3):602-10.e1-11. doi: 10.1016/j.jaci.2010.06.015. Epub 2010 Jul 31.

  PMID: 20673987 RESULT

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  PMID: 17697973 RESULT

• Beck JC, Wagner JE, DeFor TE, Brunstein CG, Schleiss MR, Young JA, Weisdorf DH, Cooley S, Miller JS, Verneris MR. Impact of cytomegalovirus (CMV) reactivation after umbilical cord blood transplantation. Biol Blood Marrow Transplant. 2010 Feb;16(2):215-22. doi: 10.1016/j.bbmt.2009.09.019. Epub 2009 Sep 26.

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  PMID: 18162223 RESULT

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• Chung B, Barbara-Burnham L, Barsky L, Weinberg K. Radiosensitivity of thymic interleukin-7 production and thymopoiesis after bone marrow transplantation. Blood. 2001 Sep 1;98(5):1601-6. doi: 10.1182/blood.v98.5.1601.

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• Jimenez M, Martinez C, Ercilla G, Carreras E, Urbano-Ispizua A, Aymerich M, Villamor N, Amezaga N, Rovira M, Fernandez-Aviles F, Gaya A, Martino R, Sierra J, Montserrat E. Reduced-intensity conditioning regimen preserves thymic function in the early period after hematopoietic stem cell transplantation. Exp Hematol. 2005 Oct;33(10):1240-8. doi: 10.1016/j.exphem.2005.06.016.

  PMID: 16219547 RESULT

Biospecimen

Retention: SAMPLES WITH DNA

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

MeSH Terms

Conditions

X-Linked Combined Immunodeficiency Diseases; Primary Immunodeficiency Diseases

Interventions

Cord Blood Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Severe Combined Immunodeficiency; Infant, Newborn, Diseases; Immunologic Deficiency Syndromes; Immune System Diseases

Intervention Hierarchy (Ancestors)

Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical 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, master

  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: clinical professor

Study Record Dates

• First Submitted: November 12, 2019
• First Posted: November 21, 2019
• Study Start: December 1, 2019
• Primary Completion: October 31, 2022
• Study Completion: October 31, 2023
• Last Updated: January 28, 2020

Record last verified: 2020-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)