Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Aplastic Anemia Patients

NCT03295058 | Unknown Phase 2

• 1 other identifier: OPALLSCT
• Study Type: interventional
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief Summary

Aplastic anemia is a syndrome of bone marrow failure characterized by peripheral pancytopenia and marrow hypoplasia.The theoretical basis for marrow failure includes primary defects in or damage to the stem cell or the marrow microenvironment

Conditions

Severe Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• Overall Survival rate

  two years

Secondary Outcomes (3)

• GVHD occurrence (Acute or Chronic), engraftment and transplant related mortality

  two years

• Relapse rate

  two years

• Progression free survival

  two years

Study Arms (2)

Non ATG regimen

EXPERIMENTAL

the first 50% of patients will receive Fludarabine + cyclophosphamide prior to hematopoietic cell transplantation. GVHD Prophylaxis: Cyclosporine A All the patient will do allogenic stem cell transplantation from peripheral blood

Drug: Non ATG Conditioning regimen; Drug: GVHD Prophylaxis; Procedure: Allogenic Stem Cell Transplantation

ATG regimen

EXPERIMENTAL

the other 50% will receive Cyclophosphamide + ATG prior to hematopoietic cell transplantation. GVHD Prophylaxis: Cyclosporine A All the patient will do allogenic stem cell transplantation from peripheral blood

Drug: GVHD Prophylaxis; Procedure: Allogenic Stem Cell Transplantation; Drug: ATG conditioning regimen

Interventions

Non ATG Conditioning regimen DRUG

50% of the patients will receive Fludarabine + cyclophosphamide (F-araA 120 mg/m2 total dose for 3 days (d-3,-2,-1) over 1 h infusion and cyclophosphamide 25 mg/kg/d (d-5 to d-2) for 4 days over 1-h infusion prior to hematopoietic cell transplantation , then post transplant cyclophosphamide dose 50 mg/kg on days +3 and +4

Non ATG regimen

GVHD Prophylaxis DRUG

Cyclosporine A (CsA 3 mg/kg) start day +5

ATG regimen; Non ATG regimen

Allogenic Stem Cell Transplantation PROCEDURE

using peripheral blood stem cells

ATG regimen; Non ATG regimen

ATG conditioning regimen DRUG

the other 50% of the patients will receive Cyclophosphamide + ATG

ATG regimen

Eligibility Criteria

• Age: 16 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• All patients with severe and very severe aplastic anemia for stem cell therapy.

You may not qualify if:

• Contraindication to stem cell transplantation. 2 - Patients associated co-morbidities.

Sponsors & Collaborators

• Assiut University: lead

Related Publications (7)

• Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, Hillmen P, Ireland R, Kulasekararaj A, Mufti G, Snowden JA, Samarasinghe S, Wood A, Marsh JC; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207. doi: 10.1111/bjh.13853. Epub 2015 Nov 16. No abstract available.

  PMID: 26568159 BACKGROUND

• George B, Mathews V, Viswabandya A, Abraham A, Ganapule A, Fouzia NA, Korula A, Lakshmi KN, Chandy M, Srivastava A. Immunosuppressive Therapy and Bone Marrow Transplantation for Aplastic Anaemia--The CMC Experience. J Assoc Physicians India. 2015 Mar;63(3 Suppl):36-40.

  PMID: 26529866 BACKGROUND

• Barone A, Lucarelli A, Onofrillo D, Verzegnassi F, Bonanomi S, Cesaro S, Fioredda F, Iori AP, Ladogana S, Locasciulli A, Longoni D, Lanciotti M, Macaluso A, Mandaglio R, Marra N, Martire B, Maruzzi M, Menna G, Notarangelo LD, Palazzi G, Pillon M, Ramenghi U, Russo G, Svahn J, Timeus F, Tucci F, Cugno C, Zecca M, Farruggia P, Dufour C, Saracco P; Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association. Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP). Blood Cells Mol Dis. 2015 Jun;55(1):40-7. doi: 10.1016/j.bcmd.2015.03.007. Epub 2015 Mar 31.

  PMID: 25976466 BACKGROUND

• Socie G. Allogeneic BM transplantation for the treatment of aplastic anemia: current results and expanding donor possibilities. Hematology Am Soc Hematol Educ Program. 2013;2013:82-6. doi: 10.1182/asheducation-2013.1.82.

  PMID: 24319167 BACKGROUND

• Wu Y, Yu J, Zhang L, Luo Q, Xiao JW, Liu XM, Xian Y, Dai BT, Xu YH, Su YC. [Hematopoiesis support of mesenchymal stem cells in children with aplastic anemia]. Zhongguo Dang Dai Er Ke Za Zhi. 2008 Aug;10(4):455-9. Chinese.

  PMID: 18706160 BACKGROUND

• Couban S, Simpson DR, Barnett MJ, Bredeson C, Hubesch L, Howson-Jan K, Shore TB, Walker IR, Browett P, Messner HA, Panzarella T, Lipton JH; Canadian Bone Marrow Transplant Group. A randomized multicenter comparison of bone marrow and peripheral blood in recipients of matched sibling allogeneic transplants for myeloid malignancies. Blood. 2002 Sep 1;100(5):1525-31. doi: 10.1182/blood-2002-01-0048.

  PMID: 12176866 BACKGROUND

• Watanabe T, Takaue Y, Kawano Y, Koike K, Kikuta A, Imaizumi M, Watanabe A, Eguchi H, Ohta S, Horikoshi Y, Iwai A, Makimoto A, Kuroda Y; Peripheral Blood Stem Cell Transplantation Study Group of Japan. HLA-identical sibling peripheral blood stem cell transplantation in children and adolescents. Biol Blood Marrow Transplant. 2002;8(1):26-31. doi: 10.1053/bbmt.2002.v8.pm11846353.

  PMID: 11846353 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Central Study Contacts

Osama A Ibraheim, MD

CONTACT

00201006372498; oibrahiem@yahoo.com

Rania M Hafez, MD

CONTACT

00201000019198; raniahafez@ymail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: severe aplastic anemia patients will receive two different conditioning regimens before doing allogenic stem cell transplantation

Study Record Dates

• First Submitted: September 24, 2017
• First Posted: September 27, 2017
• Study Start: January 1, 2019
• Primary Completion: December 1, 2019
• Study Completion: June 1, 2020
• Last Updated: November 27, 2018

Record last verified: 2018-11