Long-term Outcomes of Conservative Management in Patients With Moyamoya Disease and Their First-degree Relatives (LAMORA)

Moyamoya Disease Clinical Trial

Official title:

Long-term Outcomes of Conservative Management in Patients With Moyamoya Disease and Their First-degree Relatives

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05332756
• Other study ID #: KY2021-186-02
• Status: Recruiting
• Start date: May 1, 2022
• Est. completion date: December 31, 2026

Study information

• Verified date: June 2022
• Source: Beijing Tiantan Hospital
• Contact: Qian Zhang, MD
• Phone: 8613120012579
• Email: zhangqianchina[@]yahoo.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The purpose of this study is to investigate the long-term outcomes of conservative management in patients with moyamoya disease and their first-degree relatives, and provide potential pathogenesis of moyamoya disease.

Description:

Moyamoya disease (MMD) is a chronic occlusive-stenosis cerebrovascular disease that characterized by the stenosis of internal carotid artery termination and the formation of net-like vessel. It is a multifactorial disease caused by genetic, inflammatory, immunological and other environmental factors. The specific pathogenesis of MMD is still unclear. The treatment modalities of revascularization and conservative management have been used in patients with MMD. However, the long-term outcomes of MMD with conservative management remain unknown. Also, some first-degree relatives who are carriers of genetic variants occasionally manifest with intracranial arterial stenosis. Therefore, it is significant to detect the long-term outcomes of conservative treatment in MMD patients and their first-degree relatives, and thus provide potential pathogenesis of MMD.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: December 31, 2026
• Est. primary completion date: November 30, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 60 Years

Eligibility

Inclusion Criteria:

1. Patients with age between 2-60 years;

2. A clinical diagnosis of moyamoya disease, including unilateral and bilateral disease.

3. Patients are medically treated with antiplatelets, antiepileptics, antihypertensives and vasodilators depending on the presentation.

4. Capable of understanding the purpose and risk of the study and has signed the informed consent. If the participant is not capable of this at the time of enrollment, a legally authorized representative will provide written informed consent in accordance with all regulations.

5. Ability to comply with study follow-up.

Exclusion Criteria:

1. Concomitant other diseases, including systemic vasculitis, neurofibroma, meningitis, sickle cell disease, down's syndrome, and previous basilar radiotherapy.

2. Patients with cardiogenic embolism, including a history of atrial fibrillation, valvular disease or cardiac valve replacement.

3. Patients are allergic to the contrast agents.

4. Patients are treated with direct, indirect, or combined revascularization depending on the presentation.

5. Physical or subjective failure to cooperate with the examination or serious comorbid diseases.

6. Patients are unable or unlikely to return for follow-up visits.

7. Any other reasons that, in the opinion of the investigators, make the participant unsuitable for enrollment.

Related Conditions & MeSH terms

• Moyamoya Disease

Intervention

Other:
• Conservative management
Patients and their first-degree relatives will be medically treated with antiplatelets, antiepileptics, antihypertensives and vasodilators depending on the presentation.

Locations

Country	Name	City	State
China	Beijing Tiantan Hospital Capital Medical University	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Beijing Tiantan Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cerebrovascular events		6 months
Primary	Cerebrovascular events		1 year
Primary	Cerebrovascular events		2 years
Primary	Cerebrovascular events		5 years
Secondary	Identification of RNF213 variants		Baseline
Secondary	Unfavorable neurological outcome (mRS>2)		6 months, 1 year, 2 years, and 5 years during follow-up
Secondary	Change in cerebral perfusion status as assessed by CTP		Baseline, 6 months, 1 year, 2 years, and 5 years during follow-up
Secondary	Change in immunological, inflammatory, angiogenesis biomarkers of peripheral blood	Serum, plasma, RNA, immune cells and cytokines	Baseline, 1 year, 2 years, and 5 years during follow-up
Secondary	Change in angiographic features as assessed by CTA		Baseline, 6 months, 1 year, 2 years, and 5 years during follow-up
Secondary	Change in angiographic characteristics as assessed by MRA		Baseline, 1 year, 2 years, and 5 years during follow-up
Secondary	Change in radiological characteristics as assessed by HR-MRI		Baseline, 1 year, 2 years, and 5 years during follow-up