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Severe Aplastic AnemiaPhase 4NCT02745717

The Efficacy of Immunosuppressive Therapy Combined With Cord Blood Transfusion in Treatment of Severe Aplastic Anemia

A Phase 4 study testing Thymoglobulin, Cord blood and Cyclosporine Oral Product in people with Severe Aplastic Anemia, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Aim: To evaluate if additional cord blood transfusion could accelerate the hematopoietic reconstitution in severe aplastic anemia(SAA) patients receiving immunosuppressive therapy (IST). Read more about this study

Who is it for?
Ages Up to 60 years
Conditions the study is for
Severe Aplastic Anemia
What is being tested?
Thymoglobulin, Cord blood and Cyclosporine Oral Product
Could I receive a placebo?
No, everyone receives an active treatment
How many people
60 participants
Where
1 siteChina
Study dates
Jan 2016to Dec 2021
Phase
Phase 4
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Up to 60 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 1．Diagnosis of AA confirmed by bone marrow aspirate and biopsy, myelodysplastic syndrome and paroxysmal nocturnal hemoglobinuria were excluded. To confirm severe AA, the patient must fulfill at least two of the criteria: i) ANC<0.5×109/L,ii)PLT<20×109/L and iii) Ret<20×109/L ,in addition, ANC<0.5×109/L must be included.
  • 2. Under 60 years old, male or female.
  • 3. No HLA matched siblings.
  • 4. No previous ATG treatment history.
  • 5. Performance status score no more than 2 (ECOG criteria).
  • 6．Adequate organ function as defined by the following criteria:ALT, AST and total serum bilirubin <2×ULN (upper limit of normal) Serum creatinine and BUN <1.25×ULN.
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  • 7. Adequate cardiac function without acute myocardial infarction, arrhythmia or atrioventricular block, heart failure, active rheumatic heart disease and cardiac dilatation.
  • 8．Signed and dated informed consent document indicating that the patient (or legally acceptable representative) has been informed of all pertinent aspects of the trial prior to enrollment.
  • 9. Willingness and ability to comly with scheduled visits, treatment plans, laboratory tests, and other study procedures.

Who cannot join

  • Presence of any condition inappropriate for HSCT.
  • Presence of any fatal disease, including respiratory failure, heart failure, liver or kidney function failure et al.
  • 3．Severely allergic to biologic products.
  • 4．Pregnancy or breastfeeding.
  • 5．Current treatment on another clinical trail.
  • 6．Any other condition the investigator judged the patient inappropriate for entry into this study.

Trial locations and contacts

1 site in 1 country.

China1 site

  • Shanghai general hospital, Shanghai Jiaotong university school of medicine

    Shanghai, Shanghai Municipality, 200080

Other trials in China

About this study

Aim: To evaluate if additional cord blood transfusion could accelerate the hematopoietic reconstitution in severe aplastic anemia(SAA) patients receiving immunosuppressive therapy (IST).

Study design: open-labed, prospective, multicenter, randomized control study Number of subjects: 60 each group

Treatment:

IST group: ATG (Thymoglobuline®, Genzyme) 3.5mg/kg/d×5d plus oral cyclosporine A (CSA) Cord blood transfusion group: In addition to the same dose and course of ATG and CSA , one unit of cord blood having no more than 2 HLA-A, B or DRB1 mismatches is transfused 24h after last dose of ATG administration.

Read the detailed description

Eligible patients should be under 60 years old with confirmed SAA, without HLA matched siblings and previous ATG treatment history. Patients will be excluded if they present any fatal disease, including respiratory failure, heart failure, liver or kidney function failure et al or severely allergic to biologic products.

To evaluate if additional cord blood transfusion could accelerate the hematopoietic reconstitution in severe aplastic anemia(SAA) patients receiving IST therapy, 120 eligible patients will be randomized to two groups, the IST group and the cord blood transfusion group. Patients in the IST group receive standard IST which including ATG (Thymoglobuline®, Genzyme) 3.5mg/kg/d×5d plus oral cyclosporine A（CSA ) started from 5mg/kg/d and adjusted to maintain trough serum concentration of 200-300ng/ml. While patients in the cord blood transfusion group receive the same dose and course of ATG and CSA as the control group and one unit of cord blood having no more than 2 HLA-A, B and DRB1 mismatches is transfused 24h after last dose of ATG administration.

The neutrophil recovery day is defined as the first day of 3 consecutive days during which the absolute neutrophil count (ANC) is >0.5×109/L, without G-CSF adminstration. Platelet recovery day is defined to have occurred on the first of 7 consecutive days with a blood platelet count (BPC) of >20×109/L, without transfusion support. Response (CR, PR or NR) is evaluated on 3, 4, 6,9, 12, 18 and 24months after treatment.

The primary end point is the neutrophil recovery day and second end points are response rate (CR+PR), treatment related mortality, disease free survival and overall survival.

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Being tested
Thymoglobulin, Cord blood, Cyclosporine Oral Product
Keywords
Severe aplastic anemia, immunosuppressive therapy, cord blood transfusion
Medical subject headings
Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ThymoglobulinDrug

    Also called Thymoglobuline

    administration of ATG 3.5mg/kg/d (Thymoglobuline®, Genzyme) intravenously for 5 days

  • Cord bloodProcedure or surgery

    transfusion of one unit of at least 4/6 HLA loci matched cord blood.

  • Cyclosporine Oral ProductDrug

    Also called cyclosporine A

    administration of cyclosporine A 5mg/kg orally, and maintain the trough serum concentration between 200ng/ml to 300ng/ml.

Study groups in NCT02745717
Study groupRoleWhat they receive
cord blood and IST groupExperimentalAdministration of antithymocyte ( Thymoglobulin ) 3.5mg/kg/d for 5 days, Cyclosporine Oral Product 5mg/kg/d with trough serum concentration of 200-300ng/ml, plus one unit of at least 4/6 HLA loci matched cord blood transfusion 24 hours after last dose of ATG.
Receives: Drug: Thymoglobulin; Procedure: Cord blood; Drug: Cyclosporine Oral Product
IST groupActive comparatorAntithymocyte ( Thymoglobulin ) 3.5mg/kg/d for 5 days , Cyclosporine Oral Product 5mg/kg/d with trough serum concentration of 200-300ng/ml.
Receives: Drug: Thymoglobulin; Drug: Cyclosporine Oral Product

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. neutrophil recovery day

    the first day of 3 consecutive days when the absolute neutrophil count (ANC) reaches 0.5×10^9/L, without G-CSF administration .

    The day of first dose of ATG administration is record as day 0.

    Measured: 5 days to 365 days

Secondary outcomes (2)

  1. overall response rate

    overall response rate is the percentage of patients who acquire complete remission and partial remission according to the criteria of British Committee for Standards in Haematology (BCSH).

    Measured: 3 months to 24 months

  2. overall survival

    The length of time from the start of treatment for patients with SAA are still alive.

    Measured: 24month

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Apr 2016 (estimated)
  2. Study startJan 2016
  3. Primary completionDec 2021
  4. Study completionDec 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
60 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02745717
Sponsor's protocol ID
Shanghai1st-48
Status last verified
Mar 2023
First submitted
7 Apr 2016
Last updated
17 Mar 2023
Results posted
No
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 26568159

  • Liu HL, Sun ZM, Geng LQ, Wang XB, Ding KY, Tang BI, Tong J, Wang ZY. Unrelated cord blood transplantation for newly diagnosed patients with severe acquired aplastic anemia using a reduced-intensity conditioning: high graft rejection, but good survival. Bone Marrow Transplant. 2012 Sep;47(9):1186-90. doi: 10.1038/bmt.2011.251. Epub 2012 Jan 16.

    PubMed 22246086

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Thymoglobulin, Cord blood and Cyclosporine Oral Product in people with Severe Aplastic Anemia, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 17 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

neutrophil recovery day (time frame: 5 days to 365 days). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02745717, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Mar 2023.

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