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Thrombotic DisorderPhase 3NCT05907486

The Use of N-acetylcysteine for Thrombotic Events After Allogenic Hematopoietic Stem Cell Transplantation

A Phase 3 study testing N-acetyl-cysteine, Busulfan and Cytarabine in people with Thrombotic Disorder, run by The First Affiliated Hospital of Soochow University.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial summary

We aim to assess the the efficiency and safety of N-acetylcysteine for prevention of thrombotic events after allogenic hematopoietic stem cell transplantation. Read more about this study

Who is it for?
Ages 16 to 70 years
Conditions the study is for
Thrombotic Disorder
What is being tested?
N-acetyl-cysteine, Busulfan and Cytarabine
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
260 participants
Where
No sites listed yet
Study dates
1 Aug 2023to 1 Dec 2025
Phase
Phase 3
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 16 to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Aged 16-70 years old
  • Diagnosed as myeloid malignancies, and about to undergo allo-HSCT;
  • ECOG: 0-2;
  • Expected survival longer than 1 month

Who cannot join

  • Allergic to any components of NAC;
  • Severe dysfunction of heart, liver, lung and kidney;
  • Relapse before HSCT;
  • A history of bronchial asthma, bronchospasm or moderate / severe gastrohelcosis.

Trial locations and contacts

No sites are listed yet.

Yaqiong Tang, Doctor contact

18896588075

tangyaqiong@suda.edu.cn

About this study

Official title: The Efficiency and Safety of N-acetylcysteine for Prevention of Thrombotic Events After Allogenic Hematopoietic Stem Cell Transplantation

We aim to assess the the efficiency and safety of N-acetylcysteine for prevention of thrombotic events after allogenic hematopoietic stem cell transplantation.

Read the detailed description

The thrombotic events are increasingly recognized complications of hematopoietic cell transplantation (HCT) associated with significant morbidity and mortality, which include transplantation-associated thrombotic microangiopathy (TA-TMA), sinusoidal obstructive syndrome (SOS), deep vein thrombosis (DVT), pulmonary thromboembolism (PTE), catheter-related thrombosis (CRT), superficial vein thrombosis (SVT), etc. There is a complex interplay on balancing the risk for thrombosis and bleeding in these patients, making treatment decisions particularly challenging. Emerging studies revealed that endothelial injury is the common underlying mechanism among different thrombotic disorders. There is increasing data that N-acetyl-cysteine (NAC) may prevent or improve endothelial dysfunction by inhibiting ROS production and preventing endothelial apoptosis. Our previous study showed low dose NAC could decrease the incidence of TA-TMA. In this study, we aim to assess the the efficiency and safety of N-acetylcysteine for prevention of thrombotic events after allogenic hematopoietic stem cell transplantation.

Being tested
N-acetyl-cysteine, Busulfan, Cytarabine, Cyclophosphamide
Keywords
Thrombotic disorder, Hematopoietic stem cell transplantation
Medical subject headings
Thrombosis

What participants receive

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

  • N-acetyl-cysteineDrug

    Also called NAC

    8g/d (>=45kg); 200mg/kg.d (<45kg), intravenously for at least 4 hours, day -9 to day +45

  • BusulfanDrug

    Also called BU

    3.2mg/kg, day-7 to day -5, intravenously

  • CytarabineDrug

    Also called Ara-C

    2g/m2, day -8, intravenously

  • CyclophosphamideDrug

    Also called CTX

    1.8g/m2, day -4 to day -3, intravenously

Study groups in NCT05907486
Study groupRoleWhat they receive
Arm AExperimentalModified BUCY conditioning regimen: busulfan (3.2mg/kg, day-7 to day -5), cytarabine (2g/m2, day -8), cyclophosphamide (1.8g/m2, day -4 to day -3), followed by stem cells infusion; N-acetyl-cysteine (Zambon Pharma, Hainan, China) : 8g/d (>=45kg); 200mg/kg.d (<45kg), intravenously for at least 4 hours, day -9 to day +45.
Receives: Drug: N-acetyl-cysteine; Drug: Busulfan; Drug: Cytarabine; Drug: Cyclophosphamide
Arm BActive comparatorModified BUCY conditioning regimen: busulfan (3.2mg/kg, day-7 to day -5), cytarabine (2g/m2, day -8), cyclophosphamide (1.8g/m2, day -4 to day -3), followed by stem cells infusion.
Receives: Drug: Busulfan; Drug: Cytarabine; Drug: Cyclophosphamide

What is being measured

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

  1. The incidence of thrombotic disorders

    The incidence of thrombotic disorders (TA-TMA, SOS, DVT, PTE, CRT, SVT) after allogenic hematopoietic stem cell transplantation

    Measured: 1 year

  2. Overall survival

    The rate of overall survival after allogenic hematopoietic stem cell transplantation

    Measured: 1 year

Secondary outcomes (3)

  1. The incidence of relapse

    The incidence of relapse after allogenic hematopoietic stem cell transplantation

    Measured: 1 year

  2. The incidence of GVHD

    The incidence of GVHD after allogenic hematopoietic stem cell transplantation

    Measured: 1 year

  3. The incidence of hematopoietic reconstitution

    The incidence of hematopoietic reconstitution after allogenic hematopoietic stem cell transplantation

    Measured: 1 year

Timeline

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

  1. First posted18 Jun 2023
  2. Study start1 Aug 2023 (estimated)
  3. Primary completion1 Aug 2025 (estimated)
  4. Study completion1 Dec 2025 (estimated)

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 3
Primary purpose
Prevention
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
260 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05907486
Sponsor's protocol ID
SOOCHOW-HY-2023-05-30
Status last verified
May 2023
First submitted
8 Jun 2023
Last updated
18 Jun 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Labrador J, Lopez-Anglada L, Perez-Lopez E, Lozano FS, Lopez-Corral L, Sanchez-Guijo FM, Vazquez L, Perez Rivera JA, Martin-Herrero F, Sanchez-Barba M, Guerrero C, del Canizo MC, Caballero MD, San Miguel JF, Alberca I, Gonzalez-Porras JR. Analysis of incidence, risk factors and clinical outcome of thromboembolic and bleeding events in 431 allogeneic hematopoietic stem cell transplantation recipients. Haematologica. 2013 Mar;98(3):437-43. doi: 10.3324/haematol.2012.069559. Epub 2012 Aug 16.

    PubMed 22899581

  • Laskin BL, Goebel J, Davies SM, Jodele S. Small vessels, big trouble in the kidneys and beyond: hematopoietic stem cell transplantation-associated thrombotic microangiopathy. Blood. 2011 Aug 11;118(6):1452-62. doi: 10.1182/blood-2011-02-321315. Epub 2011 May 19.

    PubMed 21596850

  • George JN, Li X, McMinn JR, Terrell DR, Vesely SK, Selby GB. Thrombotic thrombocytopenic purpura-hemolytic uremic syndrome following allogeneic HPC transplantation: a diagnostic dilemma. Transfusion. 2004 Feb;44(2):294-304. doi: 10.1111/j.1537-2995.2004.00700.x.

    PubMed 14962323

  • Coppell JA, Richardson PG, Soiffer R, Martin PL, Kernan NA, Chen A, Guinan E, Vogelsang G, Krishnan A, Giralt S, Revta C, Carreau NA, Iacobelli M, Carreras E, Ruutu T, Barbui T, Antin JH, Niederwieser D. Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome. Biol Blood Marrow Transplant. 2010 Feb;16(2):157-68. doi: 10.1016/j.bbmt.2009.08.024. Epub 2009 Sep 18.

    PubMed 19766729

  • Tsirigotis PD, Resnick IB, Avni B, Grisariu S, Stepensky P, Or R, Shapira MY. Incidence and risk factors for moderate-to-severe veno-occlusive disease of the liver after allogeneic stem cell transplantation using a reduced intensity conditioning regimen. Bone Marrow Transplant. 2014 Nov;49(11):1389-92. doi: 10.1038/bmt.2014.168. Epub 2014 Jul 28.

    PubMed 25068424

  • Carreras E, Diaz-Beya M, Rosinol L, Martinez C, Fernandez-Aviles F, Rovira M. The incidence of veno-occlusive disease following allogeneic hematopoietic stem cell transplantation has diminished and the outcome improved over the last decade. Biol Blood Marrow Transplant. 2011 Nov;17(11):1713-20. doi: 10.1016/j.bbmt.2011.06.006. Epub 2011 Jun 25.

    PubMed 21708110

  • Jodele S, Laskin BL, Dandoy CE, Myers KC, El-Bietar J, Davies SM, Goebel J, Dixon BP. A new paradigm: Diagnosis and management of HSCT-associated thrombotic microangiopathy as multi-system endothelial injury. Blood Rev. 2015 May;29(3):191-204. doi: 10.1016/j.blre.2014.11.001. Epub 2014 Nov 28.

    PubMed 25483393

  • Ho VT, Cutler C, Carter S, Martin P, Adams R, Horowitz M, Ferrara J, Soiffer R, Giralt S. Blood and marrow transplant clinical trials network toxicity committee consensus summary: thrombotic microangiopathy after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2005 Aug;11(8):571-5. doi: 10.1016/j.bbmt.2005.06.001.

    PubMed 16041306

  • Sartain S, Shubert S, Wu MF, Srivaths P, Teruya J, Krance R, Martinez C. Therapeutic Plasma Exchange does not Improve Renal Function in Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy: An Institutional Experience. Biol Blood Marrow Transplant. 2019 Jan;25(1):157-162. doi: 10.1016/j.bbmt.2018.08.016. Epub 2018 Aug 23.

    PubMed 30144562

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing N-acetyl-cysteine, Busulfan and Cytarabine in people with Thrombotic Disorder, run by The First Affiliated Hospital of Soochow University.

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 4 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 18 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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

Are results available for this study?

Results are not available in the source record.

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

Contact Yaqiong Tang, Doctor (18896588075, tangyaqiong@suda.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The incidence of thrombotic disorders (time frame: 1 year). The study lists 2 primary outcomes in all. 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 NCT05907486, which also lists 9 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 18 Jun 2023.

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