Autologous Mitochondrial Transplant for Cerebral Ischemia

NCT ID: NCT04998357

Last Updated: 2025-05-21

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

RECRUITING

Clinical Phase

NA

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-04-29

Study Completion Date

2026-10-29

Brief Summary

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The investigators propose to infuse healthy autologous mitochondria into cerebral vessels supplying brain tissue experiencing ischemia in patients who undergo standard-of- care endovascular reperfusion therapy.

Detailed Description

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Stroke is one of the leading causes of morbidity and mortality worldwide. More than 80% of strokes are the result of ischemia caused by blockage of one or more cerebral arteries. Lack of blood supply starves brain cells of necessary glucose and oxygen, and disturbs cellular homeostasis, eventually resulting in neuronal death.

Mitochondria are tiny organelles present in nearly all types of human cells and are vital to our survival. Much like a battery, they generate most of our adenosine triphosphate (ATP), the energy currency of the cell. Mitochondria are also involved in other tasks, such as signaling between cells and cell death. All these functions can be impaired during ischemia because of the damage caused to mitochondria.

Based on many preclinical studies in animals, damage caused by ischemia can be reversed after infusing healthy mitochondria into injured tissues. An ongoing clinical trial in human hearts at Boston Children's Hospital has also demonstrated that transplanting autologous mitochondria via infusion or direct injection is well-tolerated and safe.

The investigators at the University of Washington are recruiting subjects for a first-in-human-brain trial to confirm the safety of autologous mitochondrial transplant during brain ischemia. The transplantation will be performed within the same procedure as the clinically indicated reperfusion treatment.

During standard-of-care endovascular treatment for cerebral ischemia, the investigators will obtain a very small biopsy from the muscle tissue adjacent to our surgical access site. Muscle tissue will be processed at bedside to extract the autologous mitochondria as performed in prior human cardiac trial and validated in animal studies. The endovascular treatment proceeds without disruption as clinically indicated, and the mitochondria are infused into the brain artery via micro-catheter during reperfusion.

Conditions

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Cerebral Ischemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Transplantation

Endovascular infusion

Group Type EXPERIMENTAL

Endovascular autologous mitochondrial transplantation

Intervention Type OTHER

During standard-of-care endovascular reperfusion procedure, subjects will have autologous mitochondria infused via microcatheter into ischemic brain.

Interventions

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Endovascular autologous mitochondrial transplantation

During standard-of-care endovascular reperfusion procedure, subjects will have autologous mitochondria infused via microcatheter into ischemic brain.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Eligible for endovascular thrombectomy to treat acute large vessel occlusion
* Eligible for angioplasty (microcatheter-based balloon/mechanical and chemical angioplasty) to treat acute cerebral vasospasm after aneurysmal subarachnoid hemorrhage
* Subjects for whom there is likely to be enough time to obtain meaningful consent from patient or legally-authorized representative

Exclusion Criteria

* Unable to receive a brain MRI scan
* Known mitochondrial disease
* Hemodynamically unstable patients in whom standard of care endovascular reperfusion treatment cannot safely be performed or completed
Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Washington

OTHER

Sponsor Role lead

Responsible Party

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Melanie Walker

Professor, School of Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Melanie S Walker, MD

Role: PRINCIPAL_INVESTIGATOR

University of Washington

Locations

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Harborview Medical Center

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Study Coordinator

Role: CONTACT

206-897-5802

Facility Contacts

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Melanie Walker, MD

Role: primary

206-744-3000

References

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Miralles FJ, Prijoles KL, Winter A, Levitt MR, Sancak Y, Walker M. Periprocedural therapeutics do not impair extracellular mitochondrial viability in transplantation. J Cereb Blood Flow Metab. 2025 May 14:271678X251340232. doi: 10.1177/0271678X251340232. Online ahead of print.

Reference Type BACKGROUND
PMID: 40367391 (View on PubMed)

Walker, M., Federico, E., Sancak, Y. et al. Mitochondrial Transplantation in Ischemic Stroke: Insights from a First-in-Human Brain Trial. Curr Transpl Rep 11, 53-62 (2024). https://doi.org/10.1007/s40472-024-00428-6

Reference Type BACKGROUND

Walker M, Levitt MR, Federico EM, Miralles FJ, Levy SH, Lynne Prijoles K, Winter A, Swicord JK, Sancak Y. Autologous mitochondrial transplant for acute cerebral ischemia: Phase 1 trial results and review. J Cereb Blood Flow Metab. 2024 Dec 4:271678X241305230. doi: 10.1177/0271678X241305230. Online ahead of print.

Reference Type BACKGROUND
PMID: 39628322 (View on PubMed)

Preble JM, Pacak CA, Kondo H, MacKay AA, Cowan DB, McCully JD. Rapid isolation and purification of mitochondria for transplantation by tissue dissociation and differential filtration. J Vis Exp. 2014 Sep 6;(91):e51682. doi: 10.3791/51682.

Reference Type BACKGROUND
PMID: 25225817 (View on PubMed)

Guariento A, Piekarski BL, Doulamis IP, Blitzer D, Ferraro AM, Harrild DM, Zurakowski D, Del Nido PJ, McCully JD, Emani SM. Autologous mitochondrial transplantation for cardiogenic shock in pediatric patients following ischemia-reperfusion injury. J Thorac Cardiovasc Surg. 2021 Sep;162(3):992-1001. doi: 10.1016/j.jtcvs.2020.10.151. Epub 2020 Dec 1.

Reference Type BACKGROUND
PMID: 33349443 (View on PubMed)

Weixler V, Lapusca R, Grangl G, Guariento A, Saeed MY, Cowan DB, Del Nido PJ, McCully JD, Friehs I. Autogenous mitochondria transplantation for treatment of right heart failure. J Thorac Cardiovasc Surg. 2021 Jul;162(1):e111-e121. doi: 10.1016/j.jtcvs.2020.08.011. Epub 2020 Aug 10.

Reference Type BACKGROUND
PMID: 32919774 (View on PubMed)

Doulamis IP, Guariento A, Duignan T, Kido T, Orfany A, Saeed MY, Weixler VH, Blitzer D, Shin B, Snay ER, Inkster JA, Packard AB, Zurakowski D, Rousselle T, Bajwa A, Parikh SM, Stillman IE, Del Nido PJ, McCully JD. Mitochondrial transplantation by intra-arterial injection for acute kidney injury. Am J Physiol Renal Physiol. 2020 Sep 1;319(3):F403-F413. doi: 10.1152/ajprenal.00255.2020. Epub 2020 Jul 20.

Reference Type BACKGROUND
PMID: 32686525 (View on PubMed)

Orfany A, Arriola CG, Doulamis IP, Guariento A, Ramirez-Barbieri G, Moskowitzova K, Shin B, Blitzer D, Rogers C, Del Nido PJ, McCully JD. Mitochondrial transplantation ameliorates acute limb ischemia. J Vasc Surg. 2020 Mar;71(3):1014-1026. doi: 10.1016/j.jvs.2019.03.079. Epub 2019 Jul 26.

Reference Type BACKGROUND
PMID: 31353269 (View on PubMed)

Ramirez-Barbieri G, Moskowitzova K, Shin B, Blitzer D, Orfany A, Guariento A, Iken K, Friehs I, Zurakowski D, Del Nido PJ, McCully JD. Alloreactivity and allorecognition of syngeneic and allogeneic mitochondria. Mitochondrion. 2019 May;46:103-115. doi: 10.1016/j.mito.2018.03.002. Epub 2018 Mar 26.

Reference Type BACKGROUND
PMID: 29588218 (View on PubMed)

McCully JD, Levitsky S, Del Nido PJ, Cowan DB. Mitochondrial transplantation for therapeutic use. Clin Transl Med. 2016 Mar;5(1):16. doi: 10.1186/s40169-016-0095-4. Epub 2016 Apr 29.

Reference Type BACKGROUND
PMID: 27130633 (View on PubMed)

Hayakawa K, Esposito E, Wang X, Terasaki Y, Liu Y, Xing C, Ji X, Lo EH. Transfer of mitochondria from astrocytes to neurons after stroke. Nature. 2016 Jul 28;535(7613):551-5. doi: 10.1038/nature18928.

Reference Type BACKGROUND
PMID: 27466127 (View on PubMed)

Pluchino S, Peruzzotti-Jametti L, Frezza C. Astrocyte power fuels neurons during stroke. Swiss Med Wkly. 2016 Nov 10;146:w14374. doi: 10.4414/smw.2016.14374. eCollection 2016. No abstract available.

Reference Type BACKGROUND
PMID: 27878792 (View on PubMed)

Bambrick L, Kristian T, Fiskum G. Astrocyte mitochondrial mechanisms of ischemic brain injury and neuroprotection. Neurochem Res. 2004 Mar;29(3):601-8. doi: 10.1023/b:nere.0000014830.06376.e6.

Reference Type BACKGROUND
PMID: 15038607 (View on PubMed)

Wu M, Gu X, Ma Z. Mitochondrial Quality Control in Cerebral Ischemia-Reperfusion Injury. Mol Neurobiol. 2021 Oct;58(10):5253-5271. doi: 10.1007/s12035-021-02494-8. Epub 2021 Jul 18.

Reference Type BACKGROUND
PMID: 34275087 (View on PubMed)

Norat P, Soldozy S, Sokolowski JD, Gorick CM, Kumar JS, Chae Y, Yagmurlu K, Prada F, Walker M, Levitt MR, Price RJ, Tvrdik P, Kalani MYS. Mitochondrial dysfunction in neurological disorders: Exploring mitochondrial transplantation. NPJ Regen Med. 2020 Nov 23;5(1):22. doi: 10.1038/s41536-020-00107-x.

Reference Type BACKGROUND
PMID: 33298971 (View on PubMed)

Norat P, Sokolowski JD, Gorick CM, Soldozy S, Kumar JS, Chae Y, Yagmurlu K, Nilak J, Sharifi KA, Walker M, Levitt MR, Klibanov AL, Yan Z, Price RJ, Tvrdik P, Kalani MYS. Intraarterial Transplantation of Mitochondria After Ischemic Stroke Reduces Cerebral Infarction. Stroke Vasc Interv Neurol. 2023 May;3(3):e000644. doi: 10.1161/svin.122.000644. Epub 2023 Mar 2.

Reference Type BACKGROUND
PMID: 37545759 (View on PubMed)

Related Links

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https://sanscenter.org/

The Stroke \& Applied NeuroScience Center at the University of Washington

Other Identifiers

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STUDY00006638

Identifier Type: -

Identifier Source: org_study_id

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