Trial Outcomes & Findings for A Safety and Efficacy Study of NAC in Patients With TA-TMA (NCT NCT03252925)

NCT ID: NCT03252925

Last Updated: 2022-02-14

Results Overview

The incidence of TMA after HSCT.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 170 participants
• Primary outcome timeframe: 100 days
• Results posted on: 2022-02-14

Participant Flow

Participant milestones

Measure	Experimental Group N-Acetylcysteine N-Acetylcysteine: 50mg/Kg.d, oral	Control Group Placebo Oral Tablet Placebo Oral Tablet: 50mg/Kg.d, oral
Overall Study STARTED	80	80
Overall Study COMPLETED	66	74
Overall Study NOT COMPLETED	14	6

Reasons for withdrawal

Measure	Experimental Group N-Acetylcysteine N-Acetylcysteine: 50mg/Kg.d, oral	Control Group Placebo Oral Tablet Placebo Oral Tablet: 50mg/Kg.d, oral
Overall Study Withdrawal by Subject	1	0
Overall Study Protocol Violation	6	0
Overall Study Adverse Event	1	0
Overall Study Lost to Follow-up	6	6

Baseline Characteristics

A Safety and Efficacy Study of NAC in Patients With TA-TMA

Baseline characteristics by cohort

Measure	NAC Group n=66 Participants N-Acetylcysteine 50mg/Kg.d, oral	Placebo Group n=74 Participants Placebo oral tablet 50mg/Kg.d, oral	Total n=140 Participants Total of all reporting groups
Age, Categorical <=18 years	7 Participants n=5 Participants	11 Participants n=7 Participants	18 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	59 Participants n=5 Participants	63 Participants n=7 Participants	122 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Sex: Female, Male Female	30 Participants n=5 Participants	38 Participants n=7 Participants	68 Participants n=5 Participants
Sex: Female, Male Male	36 Participants n=5 Participants	36 Participants n=7 Participants	72 Participants n=5 Participants
Race/Ethnicity, Customized Yellow Race	66 Participants n=5 Participants	74 Participants n=7 Participants	140 Participants n=5 Participants
Region of Enrollment China	66 participants n=5 Participants	74 participants n=7 Participants	140 participants n=5 Participants
Primary Diseases Acute myeloid leukemia	39 Participants n=5 Participants	38 Participants n=7 Participants	77 Participants n=5 Participants
Primary Diseases Acute lymphoblastic leukemia	15 Participants n=5 Participants	18 Participants n=7 Participants	33 Participants n=5 Participants
Primary Diseases Aplastic anemia	3 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants
Primary Diseases Chronic myelogenous leukemia	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Primary Diseases Myelodysplastic syndrome	7 Participants n=5 Participants	10 Participants n=7 Participants	17 Participants n=5 Participants
Primary Diseases Lymphoma and multiple myeloma	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Transplant Type HLA identical sibling	14 Participants n=5 Participants	16 Participants n=7 Participants	30 Participants n=5 Participants
Transplant Type Related haplo-identical	51 Participants n=5 Participants	55 Participants n=7 Participants	106 Participants n=5 Participants
Transplant Type Matched unrelated	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: 100 days

The incidence of TMA after HSCT.

Outcome measures

Measure	NAC Group n=66 Participants N-Acetylcysteine 50mg/Kg.d, oral	Placebo Group n=77 Participants Placebo Oral Tablet 50mg/Kg.d, oral
The Incidence of TA-TMA.	5 Participants	15 Participants

SECONDARY outcome

Timeframe: 100 days

The level of VWF multimers in patients post HSCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 100 days

The level of endothelial micro particle in patients post HSCT.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 40 days

The level of TNF-α in patients post HSCT.

Outcome measures

Measure	NAC Group n=66 Participants N-Acetylcysteine 50mg/Kg.d, oral	Placebo Group n=74 Participants Placebo Oral Tablet 50mg/Kg.d, oral
The Level of TNF-α	0.4 pg/mL Interval 0.0 to 1.4	0.3 pg/mL Interval 0.0 to 2.1

SECONDARY outcome

Timeframe: 100 days

The level of ROS in patients post HSCT.

Outcome measures

Outcome data not reported

Adverse Events

NAC Group

Serious events: 0 serious events

Other events: 59 other events

Deaths: 16 deaths

Placebo

Serious events: 0 serious events

Other events: 72 other events

Deaths: 27 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	NAC Group n=66 participants at risk N-Acetylcysteine 50mg/Kg.d, oral	Placebo n=74 participants at risk Placebo Oral Tablet 50mg/Kg.d, oral
Gastrointestinal disorders Abdominal pain or diarrhea	54.5% 36/66 • 100 days	66.2% 49/74 • 100 days
Renal and urinary disorders Hemorrhagic cystitis	40.9% 27/66 • 100 days	58.1% 43/74 • 100 days
Blood and lymphatic system disorders Fever	30.3% 20/66 • 100 days	50.0% 37/74 • 100 days
Gastrointestinal disorders Upper gastrointestinal complications	4.5% 3/66 • 100 days	24.3% 18/74 • 100 days
Blood and lymphatic system disorders Erythra	16.7% 11/66 • 100 days	20.3% 15/74 • 100 days
Respiratory, thoracic and mediastinal disorders Cough or expectoration	13.6% 9/66 • 100 days	20.3% 15/74 • 100 days
Vascular disorders Dizziness or headache	7.6% 5/66 • 100 days	16.2% 12/74 • 100 days
Gastrointestinal disorders Gastrointestinal bleeding	9.1% 6/66 • 100 days	8.1% 6/74 • 100 days
Respiratory, thoracic and mediastinal disorders Chest tightness	3.0% 2/66 • 100 days	6.8% 5/74 • 100 days
Nervous system disorders Epilepsy	1.5% 1/66 • 100 days	5.4% 4/74 • 100 days

Additional Information

Yue Han

The First Affiliated Hospital of Soochow University

Phone: 13901551669

Email: hanyue@suda.edu.cn

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place