Intravenous N-Acetylcysteine for the Treatment of Acute Ischemic Stroke

NCT ID: NCT04918719

Last Updated: 2021-06-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE2

Total Enrollment

118 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-06-01

Study Completion Date

2023-03-25

Brief Summary

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Stroke is a major cause of death and long-term disability in the developed world. While t-PA and mechanical thrombectomy have been shown to decrease disability in properly selected patients, many patients are left with lifelong symptoms. There are currently limited options available for patients who are not candidates for treatment with t-PA and/or mechanical thrombectomy.

N-Acetylcysteine (NAC) is an FDA approved antioxidant and anti-inflammatory agent that has been used safely for many years in the treatment of acetaminophen overdose. In studies, the oral form has been shown to improve outcomes in acute ischemic stroke and has been shown to decrease the effects of ischemic brain injury in animal models. In a small human trial, it improved outcomes in patients suffering from mild traumatic brain injury (TBI). The intravenous formulation has a long safety record. It is not FDA approved for treating stroke but was reviewed by the FDA and was given an FDA IND for this study.

The investigators propose a prospective randomized, double-blind, placebo-controlled study to evaluate the efficacy of administering intravenous N-acetylcysteine to patients with acute ischemic stroke. Eligible subjects will receive a commercially available form of intravenous NAC (Acetadote®) through for the first 21 hours following their enrollment. Patients enrolled who receive t-PA for thrombolysis will have their intravenous NAC infusion delayed for 24 hours after the completion of their t-PA infusion. Patients undergoing thrombectomy will be excluded from enrollment. Subjects will be evaluated by emergency department and/or division of neurology physicians at the time of enrollment, during their hospitalization, as well as 30 days and 90 days after enrollment. At each visit, subjects will be assessed for functional status and quality of life.

This study is designed to compare the efficacy of intravenous N-acetylcysteine compared to normal treatment in patients with acute ischemic stroke.

Detailed Description

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Stroke is a leading cause of death and long-term disability in the United States, affecting more than 795,000 individuals annually. Of these, about 87% are ischemic strokes. In properly selected patients, intravenous recombinant tissue plasminogen activator (t-PA) has been shown to improve functional outcomes if given within 4.5 hours of the onset of stroke symptoms. Endovascular treatment both via clot retrieval devices and catheter directed t-PA has demonstrated benefit in well-selected patients with a large vessel occlusion and significant salvable tissue, also known as the penumbra. For patients who are not candidates for treatment with t-PA or mechanical thrombectomy, current treatment is centered on supportive care.

N-Acetylcysteine (NAC) is an FDA approved medication that has been used successfully for many years in the treatment of acute acetaminophen overdose. It is generally well tolerated with the exception of rare anaphylactoid type reactions to the intravenous formulation. In mouse studies, N-acetylcysteine (NAC) has led to an increase in glutathione levels in the neurons along with a reduced number of microbleeds in ischemic models. NAC treated rats have been demonstrated to have increased level of glutathione in the astrocytes which provides a neuroprotective effect. This reduced the number of microbleeds and prevented further thrombosis or infarct . The efficacy of NAC by scavenging of ROS and increasing mitochondrial activities has been shown to reduce noise-induced hearing loss in chinchilla models. Another study on transient cerebral ischemia rat models defined a new pharmacokinetic in which the neuroprotective effects of NAC are mediated by the increased protein levels of hypoxia-induced factor 1 (HIF-1), its target erythropoietin (EPO) and glucose transporter (GLUT-3).

Studies have shown consistent, similar results for ischemic brain injury in rat. Consistent human studies showing neuroprotective effects which lead to reduced symptoms after traumatic brain injury, reduced noise-induced hearing loss and as a treatment for Parkinson's disease. NAC has been shown to stimulate mitochondrial Complex 1 and 4 activities in vivo and in vitro in pre-synaptic terminals. The antioxidant effect of NAC by scavenging of ROS may prevent intracellular damage by inhibiting NF-kappaB, TNFalpha and Na+, K+ -ATPase inhibition of the neurons. In a human study of NAC for Parkinson's disease, NAC was found to mitigate the effects of ischemic brain injury in animal models. The antioxidant effect from repletion of intracellular glutathione is thought to preserve mitochondrial function and decrease cellular apoptosis. A placebo-controlled human trial demonstrated that NAC is able to decrease the effects of mild traumatic brain injury 7 days after blast injury. A recent study has shown that sufficient plasma and CSF levels can be achieved at well tolerated doses: 7 mg/kg, 35 mg/kg and 50 mg/kg.

A recent randomized placebo-controlled trial demonstrated improvement in patients given oral N-acetylcysteine every four hours for a total of 72 hours after enrollment in their NIH stroke scale and modified Rankin Score at 90 days. Intravenous N-Acetylcysteine given over 21 hours has proven to be equally efficacious in the treatment of acetaminophen toxicity and is currently the standard of care for that indication. The investigators aim to determine if intravenous NAC completed over a 21-hour course will improve NIH Stroke Scale and modified Rankin scores at 90 days. of NAC following an acute ischemic stroke.

Conditions

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Acute Ischemic Stroke

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Randomized double blinded clinical trial
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Sylvia Youn, Pharm.D., the ED Pharmacist will maintain drug accountability logs in compliance with FDA regulations. She will instruct other ED pharmacists about the study and the randomization log which will be kept in the pharmacy room in the ED. She will also create study drug labels to keep the nurses, physicians and patients blinded to the study medication.

Study Groups

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NAC arm

Both arms are subjects who present with neurological deficits consistent with stroke without hemorrhage less than 24 hours since symptom onset. Both arms will receive standard of care with the study intervention being considered an "add-on" therapy. Thirty minutes after enrollment, eligible subjects will be randomized into one of two arms: placebo or N-Acetylcysteine (NAC). Any patient receiving t-PA who enrolls in the study will have their study drug infusion delayed by 24 hours after the completion of the t-PA infusion.

The dosing of NAC will be similar to the standard intravenous acetaminophen toxicity dosing: 150mg/kg in 200 milliliters of 5% Dextrose (D5W) infused over 1 hour, immediately followed by 50mg/kg in 500mL D5W infused over 4 hours, then 100mg/kg in 1000 milliliters D5W infused over 16 hours.

Group Type EXPERIMENTAL

Acetylcysteine

Intervention Type DRUG

The dosing of Acetylcysteine will be similar to the standard intravenous acetaminophen toxicity dosing: 150mg/kg in 200 milliliters of 5% Dextrose (D5W) infused over 1 hour, immediately followed by 50mg/kg in 500mL D5W infused over 4 hours, then 100mg/kg in 1000 milliliters D5W infused over 16 hours. (Any patient receiving t-PA who enrolls in the study will have their study drug infusion delayed by 24 hours after the completion of the t-PA infusion. )

Placebo arm

Both arms are subjects who present with neurological deficits consistent with stroke without hemorrhage less than 24 hours since symptom onset. Both arms will receive standard of care with the study intervention being considered an "add-on" therapy. Thirty minutes after enrollment, eligible subjects will be randomized into one of two arms: placebo or N-Acetylcysteine(NAC). Any patient receiving t-PA who enrolls in the study will have their study drug infusion delayed by 24 hours after the completion of the t-PA infusion.

The placebo will consist of a 5% Dextrose in Water (D5W) instead of NAC (dosage and timings are the same as the NAC arm).

Group Type PLACEBO_COMPARATOR

Dextrose in Water

Intervention Type DRUG

The placebo will consist of a 5% Dextrose in Water instead of NAC (dosage and timigs are the same as the NAC arm). (Any patient receiving t-PA who enrolls in the study will have their study drug infusion delayed by 24 hours after the completion of the t-PA infusion. )

Interventions

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Acetylcysteine

The dosing of Acetylcysteine will be similar to the standard intravenous acetaminophen toxicity dosing: 150mg/kg in 200 milliliters of 5% Dextrose (D5W) infused over 1 hour, immediately followed by 50mg/kg in 500mL D5W infused over 4 hours, then 100mg/kg in 1000 milliliters D5W infused over 16 hours. (Any patient receiving t-PA who enrolls in the study will have their study drug infusion delayed by 24 hours after the completion of the t-PA infusion. )

Intervention Type DRUG

Dextrose in Water

The placebo will consist of a 5% Dextrose in Water instead of NAC (dosage and timigs are the same as the NAC arm). (Any patient receiving t-PA who enrolls in the study will have their study drug infusion delayed by 24 hours after the completion of the t-PA infusion. )

Intervention Type DRUG

Other Intervention Names

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N-acetylcysteine D5W 5% Dextrose in Water

Eligibility Criteria

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Inclusion Criteria

* Consecutive subjects between ages 18 years or older evaluated in the emergency department at Harbor-UCLA Medical Center with signs and symptoms of acute ischemic stroke presenting within 24 hours of symptom onset. All individuals regardless of gender or race will be included. For adults who cannot consent for themselves because of the severity of their stroke, one of the investigators will offer participation in the study to the family member who possesses the authority for medical decision making for the patient. There is no financial incentive for either the patient or the investigator. It will also be explained that the potential benefit of giving N-acetylcysteine is unknown and that the patient will receive excellent care (standard for their presentation) whether they participate or not and outside of not receiving the study drug.

Our patients tend to be economically disadvantaged but since there are no financial incentives either for the patient or for the investigators, this bias has been minimized. Patients will receive the same care whether they participate in the study or decline.

Exclusion Criteria

* Evidence of hemorrhagic stroke on initial CT scan
* Need for thrombectomy as determined by the treating neurologist -Known allergy to NAC
* Protected populations, including age \< 18, known pregnancy, and prisoners
* Patients who are DNR/DNI at the time of presentation with a short life expectancy
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

OTHER

Sponsor Role lead

Responsible Party

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David Tanen

Associate Residency Director & Senior Physician, Department of Emergency Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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David Tanen, MD

Role: PRINCIPAL_INVESTIGATOR

Lundquist LABiomed

Central Contacts

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David Tanen, MD

Role: CONTACT

424-306-5412

Elizabeth Burrola

Role: CONTACT

310-222-3624

References

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Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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https://www.pedro.org.au/wp-content/uploads/CPG_stroke.pdf

Foundation NS. Clinical Guidelines For Stroke Management. Melbourne Australia2010.

Other Identifiers

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32358-01

Identifier Type: -

Identifier Source: org_study_id

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