A Phase 3 interventional study of N-Acetylcysteine and Placebo Oral Tablet in Thrombotic Microangiopathies and Hematologic Diseases, sponsored by The First Affiliated Hospital of Soochow University. Completed at 1 site in China. Per ClinicalTrials.gov, last updated 2022-02-14.
Sponsored by The First Affiliated Hospital of Soochow University · Phase 3, Interventional, and Prevention
HSCT associated thrombotic microangiopathy(TA-TMA) is a heterogeneous, fatal disorder seen within 100 days post-transplant and presents with thrombocytopenia, hemolysis, acute renal failure, mental status changes and involvement of other organs. N-Acetylcysteine (NAC) is a small, simple molecule that began as a generic drug almost 40 years ago. It has since been approved by the FDA for many indications. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of NAC in patients with TA-TMA.
Hematopoietic stem cell transplantation (HSCT) has been commonly used as a potentially curative option in the treatment of various hematological malignancies. However, it may end up with serious complications in various systems, including the hemostatic system. HSCT associated thrombotic microangiopathy(TA-TMA) is a heterogeneous, fatal disorder seen within 100 days post-transplant and presents with thrombocytopenia, hemolysis, acute renal failure, mental status changes and involvement of other organs. Since the pathophysiology has not been clarified, there are no established treatments for TA-TMA several agents seem to have successful results. N-Acetylcysteine (NAC) is a small, simple molecule that began as a generic drug almost 40 years ago. It has since been approved by the FDA for many indications. Studies showed NAC reduce plasma VWF multimers and VWF multimeric size without an effect on the bleeding time in vitro and in vivo, thus proposed as a possible supplementary treatment for TTP. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of NAC in patients with TA-TMA.
52 studies on the registry are indexed under Thrombotic Microangiopathies; 21 are open to participants now.
This study's enrollment of 170 is above the median of 40 across 24 interventional studies indexed under Thrombotic Microangiopathies.
Browse Thrombotic Microangiopathies studies →The First Affiliated Hospital of Soochow University is the lead sponsor of 261 studies on the registry; 150 are open to participants now.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
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You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
N-Acetylcysteine
Drug: N-Acetylcysteine
Placebo Oral Tablet
Drug: Placebo Oral Tablet
50mg/Kg.d, oral
Also known as: NAC
50mg/Kg.d, oral
Also known as: Placebo
The Incidence of TA-TMA.
The incidence of TMA after HSCT.
Time frame: 100 days
The Level of VWF Multimers
The level of VWF multimers in patients post HSCT.
Time frame: 100 days
The Level of Endothelial Micro Particle
The level of endothelial micro particle in patients post HSCT.
Time frame: 100 days
The Level of TNF-α
The level of TNF-α in patients post HSCT.
Time frame: 40 days
The Level of ROS
The level of ROS in patients post HSCT.
Time frame: 100 days
| Milestone | Experimental Group | Control Group |
|---|---|---|
| Started | 80 | 80 |
| Completed | 66 | 74 |
| Not completed | 14 | 6 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Withdrew: Protocol violation | 6 | 0 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Lost to follow-up | 6 | 6 |
The incidence of TMA after HSCT.
| Participants | NAC Group | Placebo Group |
|---|---|---|
| The Incidence of TA-TMA. | 5 | 15 |
The level of VWF multimers in patients post HSCT.
Results for this outcome have not been posted.
The level of endothelial micro particle in patients post HSCT.
Results for this outcome have not been posted.
The level of TNF-α in patients post HSCT.
| pg/mL | NAC Group | Placebo Group |
|---|---|---|
| The Level of TNF-α | 0.4 (0 to 1.4) | 0.3 (0 to 2.1) |
The level of ROS in patients post HSCT.
Results for this outcome have not been posted.
Collected over 100 days. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| NAC Group | 16/66 (24.2%) | 0/66 (0%) | 59/66 (89.4%) |
| Placebo | 27/74 (36.5%) | 0/74 (0%) | 72/74 (97.3%) |
| Event | NAC Group | Placebo |
|---|---|---|
| Abdominal pain or diarrheaGastrointestinal disorders | 36/66 | 49/74 |
| Hemorrhagic cystitisRenal and urinary disorders | 27/66 | 43/74 |
| FeverBlood and lymphatic system disorders | 20/66 | 37/74 |
| Upper gastrointestinal complicationsGastrointestinal disorders | 3/66 | 18/74 |
| ErythraBlood and lymphatic system disorders | 11/66 | 15/74 |
| Cough or expectorationRespiratory, thoracic and mediastinal disorders | 9/66 | 15/74 |
| Dizziness or headacheVascular disorders | 5/66 | 12/74 |
| Gastrointestinal bleedingGastrointestinal disorders | 6/66 | 6/74 |
| Chest tightnessRespiratory, thoracic and mediastinal disorders | 2/66 | 5/74 |
| EpilepsyNervous system disorders | 1/66 | 4/74 |
| Age, Categorical(Participants) | NAC Group | Placebo Group | Total |
|---|---|---|---|
| <=18 years | 7 | 11 | 18 |
| Between 18 and 65 years | 59 | 63 | 122 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | NAC Group | Placebo Group | Total |
|---|---|---|---|
| Female | 30 | 38 | 68 |
| Male | 36 | 36 | 72 |
| Race/Ethnicity, Customized(Participants) | NAC Group | Placebo Group | Total |
|---|---|---|---|
| Yellow Race | 66 | 74 | 140 |
| Region of Enrollment(participants) | NAC Group | Placebo Group | Total |
|---|---|---|---|
| China | 66 | 74 | 140 |
| Primary Diseases(Participants) | NAC Group | Placebo Group | Total |
|---|---|---|---|
| Acute myeloid leukemia | 39 | 38 | 77 |
| Acute lymphoblastic leukemia | 15 | 18 | 33 |
| Aplastic anemia | 3 | 5 | 8 |
| Chronic myelogenous leukemia | 1 | 2 | 3 |
| Myelodysplastic syndrome | 7 | 10 | 17 |
| Lymphoma and multiple myeloma | 1 | 1 | 2 |
| Transplant Type(Participants) | NAC Group | Placebo Group | Total |
|---|---|---|---|
| HLA identical sibling | 14 | 16 | 30 |
| Related haplo-identical | 51 | 55 | 106 |
| Matched unrelated | 1 | 3 | 4 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Nov 2017 to primary completion in Oct 2021, 47 months.
Results were first posted in Feb 2022, 4 months after primary completion.
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This study is completed, as verified in Dec 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Acetylcysteine
Thrombotic Microangiopathies →
The First Affiliated Hospital of Soochow University →