NCT00471848

Brief Summary

To assess the tolerability and effectiveness of rabbit antithymocyte globulin (ATG, Thymoglobuline) with ciclosporin in the first line treatment of patients with acquired severe aplastic anaemia, and patients with non-severe aplastic anaemia and who are transfusion dependent.

Trial Health

93
On Track

Trial Health Score

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

Enrollment
35

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Aug 2008

Typical duration for phase_2

Geographic Reach
6 countries

14 active sites

Status
completed

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

May 9, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

May 10, 2007

Completed
1.2 years until next milestone

Study Start

First participant enrolled

August 1, 2008

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2011

Completed
1.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2013

Completed
8.7 years until next milestone

Results Posted

Study results publicly available

October 18, 2021

Completed
Last Updated

September 26, 2023

Status Verified

September 1, 2023

Enrollment Period

3.2 years

First QC Date

May 9, 2007

Results QC Date

December 21, 2020

Last Update Submit

September 7, 2023

Conditions

Aplastic Anemia

Keywords

ThymoglobulineRabbit ATGCiclosporinAplastic Anemia

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Response to Rabbit Antithymocyte Globulin (Thymoglobuline)

    Complete Response (CR) defined as: Haemoglobin normal for age and gender, neutrophils \> 1.5 x 10E9/l, platelets \> 150 x 10E9/l. Partial Response (PR) defined as: * transfusion independence (if previously dependent) or * doubling or normalisation of at least one cell line or * increase of baseline haemoglobin of \> 3 g/dl (if initially \<6) + neutrophils of \> 0.5 x 10E9/l + platelets of \> 20 x 10E9/l (if initially \< 20) No Response (MR) is defined as: worse or not meeting criteria above

    at 6months

Secondary Outcomes (1)

  • Failure Free and Overall Survival of Participants to Rabbit Antithymocyte Globulin (Thymoglobuline)

    at 2 years

Study Arms (1)

Treatment Arm

EXPERIMENTAL

Antithymocyte globulin with cyclosporin in first line treatment of patients with acquired severe aplastic anaemia and patients with non-severe aplastic anaemia who are transfusion dependent

Drug: rabbit antithymocyte globulin

Interventions

rabbit antithymocyte globulinDRUG

1.5 vials/10kg daily for 5 days

Also known as: Thymoglobuline
Treatment Arm

Eligibility Criteria

Age16 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Must fulfil definition of aplastic anaemia:
  • There must be at least two of the following:
  • haemoglobin \< 10g/dl
  • platelet count \< 50 x 109/l
  • neutrophil count \< 1.5 x 109/l, and a hypocellular bone marrow on bone marrow biopsy
  • SAA as defined by a hypocellular bone marrow of \<25% cellularity and two of the following:
  • neutrophil count \< 0.5 x 109/l
  • platelets \< 20 x 109/l
  • reticulocytes \< 20 x 109/l
  • NSAA as defined by a hypocellular bone marrow and cytopenia in at least two cell lines and neutrophil count \> 0.5 x 109/l, and red cell and/or platelet transfusion dependence
  • Have acquired aplastic anaemia
  • Time from diagnosis to study registration maximum 6 months
  • No prior treatment except for haemopoietic growth factors given for no more than four weeks, and androgens
  • Age minimum 16 years with no upper age limit

You may not qualify if:

  • Eligibility for an human leukocyte antigens (HLA)-matched sibling donor transplant for SAA patients
  • Prior therapy with ATG or CSA
  • Haematopoeitic growth factors more than 4 weeks before study enrolment
  • Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome
  • Evidence of myelodysplastic disease
  • Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history of Paroxysmal Nocturnal Hemoglobinuria (PNH) associated thrombosis or a PNH clone \>50% by flow cytometry
  • Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)
  • Subject is pregnant (e.g. positive Human Chorionic Gonadotropin (HCG) test) or is breast feeding
  • Severe uncontrolled infection or unexplained fever \>38 degrees Celsius
  • Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (14)

Henri Mondor Hospital

Créteil, France

Location

Hopital St. Louis

Paris, 75475, France

Location

University Hospital Essen

Essen, Germany

Location

University Hospital Eppendorf

Hamburg, Germany

Location

Medical University Hannover

Hanover, Germany

Location

Universitätsklinikum - Institut für klinische Transfusionsmedizin

Ulm, 89081, Germany

Location

Ospedale San Martino

Genova, 16132, Italy

Location

King Faisal Specialist Hospital & Research Cnetre

Riyadh, Saudi Arabia

Location

University Hospital

Basel, 4031, Switzerland

Location

Royal Bournemouth

Bournemouth, United Kingdom

Location

Addenbrooke's Hospital

Cambridge, United Kingdom

Location

St George's Hospital/ St George's University of London

London, Sw17 0RE, United Kingdom

Location

King's College Hospital

London, United Kingdom

Location

Nottingham Universitry Hospital Trust

Nottingham, United Kingdom

Location

Related Publications (1)

  • Marsh JC, Bacigalupo A, Schrezenmeier H, Tichelli A, Risitano AM, Passweg JR, Killick SB, Warren AJ, Foukaneli T, Aljurf M, Al-Zahrani HA, Hochsmann B, Schafhausen P, Roth A, Franzke A, Brummendorf TH, Dufour C, Oneto R, Sedgwick P, Barrois A, Kordasti S, Elebute MO, Mufti GJ, Socie G; European Blood and Marrow Transplant Group Severe Aplastic Anaemia Working Party. Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. Blood. 2012 Jun 7;119(23):5391-6. doi: 10.1182/blood-2012-02-407684. Epub 2012 Apr 27.

    PMID: 22544699RESULT

Related Links

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

thymoglobulinAntilymphocyte Serum

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Intervention Hierarchy (Ancestors)

Immune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex Mixtures

Results Point of Contact

Title
Prof. Judith Marsh
Organization
European Group for Blood and Marrow Transplantation
a.j.barrois@lumc.nl
+31 (0)71 5265005

Study Officials

  • Judith Marsh, Prof. MD.

    King's College Hospital NHS Trust

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NETWORK
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 9, 2007

First Posted

May 10, 2007

Study Start

August 1, 2008

Primary Completion

October 1, 2011

Study Completion

February 1, 2013

Last Updated

September 26, 2023

Results First Posted

October 18, 2021

Record last verified: 2023-09

Locations

GB(5)DE(4)IT(1)SA(1)CH(1)FR(2)