Cavernous Angioma, FamilialNCT01764529
Modifiers of Disease Severity in Cerebral Cavernous Malformations
An observational study of Cavernous Angioma, Familial and Cerebral Cavernous Malformations, run by University of California, San Francisco at 7 sites in United States.
Trial summary
Cerebral cavernous malformations (CCMs) are clusters of abnormal blood vessels in the brain and spine. CCMs can bleed and cause strokes, seizures, and headaches. CCMs are often caused by an inherited gene mutation (alteration) in one of three CCM genes (CCM1, CCM2, or CCM3). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Cavernous Angioma, Familial, Cerebral Cavernous Malformations, Cerebral Cavernous Hemangioma
- How many people
- 789 participants
- Where
- 7 sitesUnited States
- Study dates
- 27 Apr 2010to 30 Jun 2025
- How long will I take part?
- 15 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study population includes individuals who carry the diagnosis of familial cerebral cavernous malformation (CCM), both symptomatic and asymptomatic.
Who may be able to join
- Individual has a CCM mutation confirmed through DNA testing, or
- Individual meets 2 or more of the following clinical criteria:
- Clinical diagnosis of CCM
- Multi-focal CCMs on MRI
- Family history of CCM
Who cannot join
- Individuals who are incarcerated
- Individuals who are homeless
- Unable or unwilling to sign the informed consent
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
University of New Mexico Health Sciences Center
Albuquerque, New Mexico, 87131
Boston Children's Hospital
Boston, Massachusetts, 02115
Alliance to Cure Cavernous Malformation
Charlottesville, Virginia, 22901
University of Chicago, Medicine and Biological Sciences
Chicago, Illinois, 60637
Cincinnati Children's Hospital, Division of Pediatric Neurosurgery, Cerebrovascular Program
Cincinnati, Ohio, 45229
Barrow Neurological Institute
Phoenix, Arizona, 85013
University of California, San Francisco
San Francisco, California, 94143
About this study
Official title: Modifiers of Disease Severity and Progression in Cerebral Cavernous Malformations
Cerebral cavernous malformations (CCMs) are clusters of abnormal blood vessels in the brain and spine. CCMs can bleed and cause strokes, seizures, and headaches. CCMs are often caused by an inherited gene mutation (alteration) in one of three CCM genes (CCM1, CCM2, or CCM3). There is a wide range of disease severity even among family members with this disease, though the natural history has not been clearly described for this particular population.
This study will continue to enroll and follow participants with familial CCM to identify factors that influence CCM disease severity and progression, focusing on barriers to clinical trial preparedness. Our long-term goal is to identify measurable outcomes and robust biomarkers that will help select high-risk patients and help monitor drug response in future clinical trials.
Read more
The specific goals of this study are to:
- Identify factors that influence lesion progression to symptomatic hemorrhage and other outcomes, including quality of life;
- Investigate the role of the gut microbiome and lesion burden in CCM disease, and
- Identify blood biomarkers predictive of CCM disease severity and progression for clinical trials.
Read the detailed description
This study is one of three projects participating in the Brain Vascular Malformation Consortium (BVMC) funded by the Office of Rare Diseases Research, which is part of the National Center for Advancing Translational Sciences (NCATS), and the National Institute of Neurological Disorders and Stroke (NINDS).
The CCM project is a cross-sectional and longitudinal study of familial CCM patients. The study is currently in the third 5-year cycle. During the first 5 year cycle (BVMC1), the CCM project was focused on recruiting CCM1 cases with the common Hispanic mutation (CHM). In the second 5-year cycle (BVMC2), we expanded recruitment to include not only CCM1-CHM cases, but also other CCM familial patients and mutation carriers. In the third 5-year cycle (BVMC3), we will continue to recruit familial CCM cases and expand to additional recruitment sites.
We collect clinical, genetic, imaging, treatment, and outcome data in participants, and follow enrolled participants over time to understand the natural history of this disease.
For new study participants, you will be asked to:
- Give permission for study staff to access your medical records to collect clinical information and to obtain copies of MRI scans and reports.
- Fill out a questionnaire about your quality of life, family history, and medical/surgical history.
- Give a blood and/or saliva sample, and stool sample.
- Give permission to store and use your CCM resected tissue for research (if undergoing surgery).
- Participate in annual follow-ups to update medical, surgical, and neurological information.
Eligible cases include those with a known genetic mutation in one of the three CCM genes or those that meet 2 of 3 following clinical criteria:
- Clinical diagnosis of CCM,
- Multi-focal lesions on MRI, and/or
- Family history of CCMs.
Exclusion Criteria:
- Patients who cannot or are unwilling to sign informed consent and for whom no appropriate surrogate is available.
- Prisoners and homeless individuals because of the inability to contact the subject and collect follow-up data using standard procedures.
- Conditions
- Cavernous Angioma, Familial, Cerebral Cavernous Malformations, Cerebral Cavernous Hemangioma
- Medical subject headings
- Hemangioma, Cavernous, Central Nervous System
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| The BVMC FCCM cohort | Aim 1: To investigate the relationship between lesion burden and outcomes in familial CCM. Aim 2: To investigate the role of the gut microbiome in familial CCM disease severity. Aim 3: To establish blood markers predictive of disease severity and progression for medical treatment of CCM. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total CCM lesion number per patient
The number of lesions (or cavernous angiomas) located in the brain will be counted by a neuroradiologist and by an automated algorithm developed as part of this project.
Measured: Baseline
Rate of symptomatic hemorrhage
Symptomatic hemorrhage is defined as diagnostic evidence of new lesional bleeding or hemorrhagic growth, in association with directly attributable symptoms. Rate of symptomatic hemorrhage and the factors that influence hemorrhage rates will be assessed.
Measured: Baseline and annual assessment
Secondary outcomes (2)
Change in lesion number
The number of lesions (or cavernous angiomas) counted on the baseline MRI will be compared to the number of lesions observed in follow up MRIs.
Measured: Baseline, Follow up MRI
Modified Rankin score
The modified Rankin score will be assessed at baseline and at approximately one year intervals while remaining in study
Measured: Baseline and annual assessment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2013 (estimated)
- Study start27 Apr 2010
- Primary completion31 Mar 2025
- Study completion30 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (28)Papers the sponsor has linked to this study.
- Other trials for Cavernous Angioma, FamilialStudies of the same condition, open and under way.
- More trials run by University of California, San FranciscoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 789 participants (actual)
- Target duration
- 15 Years
Sponsor and investigators
- Lead sponsor
- University of California, San Francisco
- Sponsor type
- University, hospital or other
- Collaborators
- University of New Mexico, University of Chicago, National Institute of Neurological Disorders and Stroke (NINDS), Boston Children's Hospital, Children's Hospital Medical Center, Cincinnati, Barrow Neurological Institute, Angioma Alliance
- Responsible party
- Sponsor
- Investigators
- Helen Kim, PhD, Principal investigator, University of California, San FranciscoIssam Awad, MD, Principal investigator, University of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01764529
- Sponsor's protocol ID
- BVMC 6201
- Other IDs
- U54NS065705-15
- Status last verified
- Jul 2026
- First submitted
- 7 Jan 2013
- Last updated
- 27 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Available data will be released to the Rare Diseases Clinical Research Network repository and will become available to the scientific community one year after publication of planned analyses, or after a period of 5 years from the date when the data were collected, whichever comes first
Publications
Akers AL, Ball KL, Clancy M, Comi AM, Faughnan ME, Gopal-Srivastava R, Jacobs TP, Kim H, Krischer J, Marchuk DA, McCulloch CE, Morrison L, Moses M, Moy CS, Pawlikowska L, Young WL. Brain Vascular Malformation Consortium: Overview, Progress and Future Directions. J Rare Disord. 2013 Apr 1;1(1):5.
PubMed 25221778Flemming KD, Smith E, Marchuk D, Derry WB. Familial Cerebral Cavernous Malformations. 2003 Feb 24 [updated 2023 Jul 27]. In: Adam MP, Bick S, Mirzaa GM, Wallace SE, Amemiya A, editors. GeneReviews(R) [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2026. Available from http://www.ncbi.nlm.nih.gov/books/NBK1293/
PubMed 20301470Choquet H, Pawlikowska L, Lawton MT, Kim H. Genetics of cerebral cavernous malformations: current status and future prospects. J Neurosurg Sci. 2015 Sep;59(3):211-20. Epub 2015 Apr 22.
PubMed 25900426Zafar A, Quadri SA, Farooqui M, Ikram A, Robinson M, Hart BL, Mabray MC, Vigil C, Tang AT, Kahn ML, Yonas H, Lawton MT, Kim H, Morrison L. Familial Cerebral Cavernous Malformations. Stroke. 2019 May;50(5):1294-1301. doi: 10.1161/STROKEAHA.118.022314. No abstract available.
PubMed 30909834Hart BL, Mabray MC, Morrison L, Whitehead KJ, Kim H. Systemic and CNS manifestations of inherited cerebrovascular malformations. Clin Imaging. 2021 Jul;75:55-66. doi: 10.1016/j.clinimag.2021.01.020. Epub 2021 Jan 20.
PubMed 33493737Choquet H, Pawlikowska L, Nelson J, McCulloch CE, Akers A, Baca B, Khan Y, Hart B, Morrison L, Kim H; Brain Vascular Malformation Consortium (BVMC) Study. Polymorphisms in inflammatory and immune response genes associated with cerebral cavernous malformation type 1 severity. Cerebrovasc Dis. 2014;38(6):433-40. doi: 10.1159/000369200. Epub 2014 Dec 3.
PubMed 25472749Choquet H, Nelson J, Pawlikowska L, McCulloch CE, Akers A, Baca B, Khan Y, Hart B, Morrison L, Kim H. Association of cardiovascular risk factors with disease severity in cerebral cavernous malformation type 1 subjects with the common Hispanic mutation. Cerebrovasc Dis. 2014;37(1):57-63. doi: 10.1159/000356839. Epub 2013 Dec 21.
PubMed 24401931Golden M, Saeidi S, Liem B, Marchand E, Morrison L, Hart B. Sensitivity of patients with familial cerebral cavernous malformations to therapeutic radiation. J Med Imaging Radiat Oncol. 2015 Feb;59(1):134-6. doi: 10.1111/1754-9485.12269. Epub 2015 Jan 7.
PubMed 25565562Hart BL, Taheri S, Rosenberg GA, Morrison LA. Dynamic contrast-enhanced MRI evaluation of cerebral cavernous malformations. Transl Stroke Res. 2013 Oct;4(5):500-6. doi: 10.1007/s12975-013-0285-y. Epub 2013 Sep 21.
PubMed 24323376Petersen TA, Morrison LA, Schrader RM, Hart BL. Familial versus sporadic cavernous malformations: differences in developmental venous anomaly association and lesion phenotype. AJNR Am J Neuroradiol. 2010 Feb;31(2):377-82. doi: 10.3174/ajnr.A1822. Epub 2009 Oct 15.
PubMed 19833796Choquet H, Trapani E, Goitre L, Trabalzini L, Akers A, Fontanella M, Hart BL, Morrison LA, Pawlikowska L, Kim H, Retta SF. Cytochrome P450 and matrix metalloproteinase genetic modifiers of disease severity in Cerebral Cavernous Malformation type 1. Free Radic Biol Med. 2016 Mar;92:100-109. doi: 10.1016/j.freeradbiomed.2016.01.008. Epub 2016 Jan 19.
PubMed 26795600Golden MJ, Morrison LA, Kim H, Hart BL. Increased number of white matter lesions in patients with familial cerebral cavernous malformations. AJNR Am J Neuroradiol. 2015 May;36(5):899-903. doi: 10.3174/ajnr.A4200. Epub 2015 Jan 2.
PubMed 25556204Zou X, Hart BL, Mabray M, Bartlett MR, Bian W, Nelson J, Morrison LA, McCulloch CE, Hess CP, Lupo JM, Kim H. Automated algorithm for counting microbleeds in patients with familial cerebral cavernous malformations. Neuroradiology. 2017 Jul;59(7):685-690. doi: 10.1007/s00234-017-1845-8. Epub 2017 May 22.
PubMed 28534135Strickland CD, Eberhardt SC, Bartlett MR, Nelson J, Kim H, Morrison LA, Hart BL. Familial Cerebral Cavernous Malformations Are Associated with Adrenal Calcifications on CT Scans: An Imaging Biomarker for a Hereditary Cerebrovascular Condition. Radiology. 2017 Aug;284(2):443-450. doi: 10.1148/radiol.2017161127. Epub 2017 Mar 20.
PubMed 28318403Akers A, Al-Shahi Salman R, A Awad I, Dahlem K, Flemming K, Hart B, Kim H, Jusue-Torres I, Kondziolka D, Lee C, Morrison L, Rigamonti D, Rebeiz T, Tournier-Lasserve E, Waggoner D, Whitehead K. Synopsis of Guidelines for the Clinical Management of Cerebral Cavernous Malformations: Consensus Recommendations Based on Systematic Literature Review by the Angioma Alliance Scientific Advisory Board Clinical Experts Panel. Neurosurgery. 2017 May 1;80(5):665-680. doi: 10.1093/neuros/nyx091.
PubMed 28387823
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Modifiers of Disease Severity and Progression in Cerebral Cavernous Malformations
- ClinicalTrials.gov ID
- NCT01764529
- Acronym / sponsor ID
- BVMC 6201
- Conditions
- Cavernous Angioma, Familial, Cerebral Cavernous Malformations, Cerebral Cavernous Hemangioma
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 789 participants (actual)
- Lead sponsor
- University of California, San FranciscoUniversity, hospital or other
- Collaborators
- University of New Mexico, University of Chicago, National Institute of Neurological Disorders and Stroke (NINDS), Boston Children's Hospital, Children's Hospital Medical Center, Cincinnati, Barrow Neurological Institute, Angioma Alliance
- Study start
- 27 Apr 2010 (actual)
- Primary completion
- 31 Mar 2025 (actual)
- Study completion
- 30 Jun 2025 (actual)
- First posted
- 9 Jan 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Cerebral cavernous malformations (CCMs) are clusters of abnormal blood vessels in the brain and spine. CCMs can bleed and cause strokes, seizures, and headaches. CCMs are often caused by an inherited gene mutation (alteration) in one of three CCM genes (CCM1, CCM2, or CCM3). There is a wide range of disease severity even among family members with this disease, though the natural history has not been clearly described for this particular population.
This study will continue to enroll and follow participants with familial CCM to identify factors that influence CCM disease severity and progression, focusing on barriers to clinical trial preparedness. Our long-term goal is to identify measurable outcomes and robust biomarkers that will help select high-risk patients and help monitor drug response in future clinical trials.
The specific goals of this study are to:
- Identify factors that influence lesion progression to symptomatic hemorrhage and other outcomes, including quality of life;
- Investigate the role of the gut microbiome and lesion burden in CCM disease, and
- Identify blood biomarkers predictive of CCM disease severity and progression for clinical trials.
Detailed description
This study is one of three projects participating in the Brain Vascular Malformation Consortium (BVMC) funded by the Office of Rare Diseases Research, which is part of the National Center for Advancing Translational Sciences (NCATS), and the National Institute of Neurological Disorders and Stroke (NINDS).
The CCM project is a cross-sectional and longitudinal study of familial CCM patients. The study is currently in the third 5-year cycle. During the first 5 year cycle (BVMC1), the CCM project was focused on recruiting CCM1 cases with the common Hispanic mutation (CHM). In the second 5-year cycle (BVMC2), we expanded recruitment to include not only CCM1-CHM cases, but also other CCM familial patients and mutation carriers. In the third 5-year cycle (BVMC3), we will continue to recruit familial CCM cases and expand to additional recruitment sites.
We collect clinical, genetic, imaging, treatment, and outcome data in participants, and follow enrolled participants over time to understand the natural history of this disease.
For new study participants, you will be asked to:
- Give permission for study staff to access your medical records to collect clinical information and to obtain copies of MRI scans and reports.
- Fill out a questionnaire about your quality of life, family history, and medical/surgical history.
- Give a blood and/or saliva sample, and stool sample.
- Give permission to store and use your CCM resected tissue for research (if undergoing surgery).
- Participate in annual follow-ups to update medical, surgical, and neurological information.
Eligible cases include those with a known genetic mutation in one of the three CCM genes or those that meet 2 of 3 following clinical criteria:
- Clinical diagnosis of CCM,
- Multi-focal lesions on MRI, and/or
- Family history of CCMs.
Exclusion Criteria:
- Patients who cannot or are unwilling to sign informed consent and for whom no appropriate surrogate is available.
- Prisoners and homeless individuals because of the inability to contact the subject and collect follow-up data using standard procedures.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 15 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| The BVMC FCCM cohort | – | –Aim 1: To investigate the relationship between lesion burden and outcomes in familial CCM. Aim 2: To investigate the role of the gut microbiome in familial CCM disease severity. Aim 3: To establish blood markers predictive of disease severity and progression for medical treatment of CCM. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study population includes individuals who carry the diagnosis of familial cerebral cavernous malformation (CCM), both symptomatic and asymptomatic.
Key inclusion criteria (5 listed)
- Individual has a CCM mutation confirmed through DNA testing, or
- Individual meets 2 or more of the following clinical criteria:
Key exclusion criteria (3 listed)
- Individuals who are incarcerated
- Individuals who are homeless
- Unable or unwilling to sign the informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Total CCM lesion number per patient
Time frame: Baseline
The number of lesions (or cavernous angiomas) located in the brain will be counted by a neuroradiologist and by an automated algorithm developed as part of this project.
Rate of symptomatic hemorrhage
Time frame: Baseline and annual assessment
Symptomatic hemorrhage is defined as diagnostic evidence of new lesional bleeding or hemorrhagic growth, in association with directly attributable symptoms. Rate of symptomatic hemorrhage and the factors that influence hemorrhage rates will be assessed.
2 secondary outcomes
Change in lesion number
Time frame: Baseline, Follow up MRI
Modified Rankin score
Time frame: Baseline and annual assessment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2026.
- Background: PubMed 25221778Akers AL, Ball KL, Clancy M, Comi AM, Faughnan ME, Gopal-Srivastava R, Jacobs TP, Kim H, Krischer J, Marchuk DA, McCulloch CE, Morrison L, Moses M, Moy CS, Pawlikowska L, Young WL. Brain Vascular Malformation Consortium: Overview, Progress and Future Directions. J Rare Disord. 2013 Apr 1;1(1):5.
- Background: PubMed 20301470Flemming KD, Smith E, Marchuk D, Derry WB. Familial Cerebral Cavernous Malformations. 2003 Feb 24 [updated 2023 Jul 27]. In: Adam MP, Bick S, Mirzaa GM, Wallace SE, Amemiya A, editors. GeneReviews(R) [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2026. Available from http://www.ncbi.nlm.nih.gov/books/NBK1293/
- Background: PubMed 25900426Choquet H, Pawlikowska L, Lawton MT, Kim H. Genetics of cerebral cavernous malformations: current status and future prospects. J Neurosurg Sci. 2015 Sep;59(3):211-20. Epub 2015 Apr 22.
- Background: PubMed 30909834Zafar A, Quadri SA, Farooqui M, Ikram A, Robinson M, Hart BL, Mabray MC, Vigil C, Tang AT, Kahn ML, Yonas H, Lawton MT, Kim H, Morrison L. Familial Cerebral Cavernous Malformations. Stroke. 2019 May;50(5):1294-1301. doi: 10.1161/STROKEAHA.118.022314. No abstract available.
- Background: PubMed 33493737Hart BL, Mabray MC, Morrison L, Whitehead KJ, Kim H. Systemic and CNS manifestations of inherited cerebrovascular malformations. Clin Imaging. 2021 Jul;75:55-66. doi: 10.1016/j.clinimag.2021.01.020. Epub 2021 Jan 20.
- Results publication: PubMed 25472749Choquet H, Pawlikowska L, Nelson J, McCulloch CE, Akers A, Baca B, Khan Y, Hart B, Morrison L, Kim H; Brain Vascular Malformation Consortium (BVMC) Study. Polymorphisms in inflammatory and immune response genes associated with cerebral cavernous malformation type 1 severity. Cerebrovasc Dis. 2014;38(6):433-40. doi: 10.1159/000369200. Epub 2014 Dec 3.
- Results publication: PubMed 24401931Choquet H, Nelson J, Pawlikowska L, McCulloch CE, Akers A, Baca B, Khan Y, Hart B, Morrison L, Kim H. Association of cardiovascular risk factors with disease severity in cerebral cavernous malformation type 1 subjects with the common Hispanic mutation. Cerebrovasc Dis. 2014;37(1):57-63. doi: 10.1159/000356839. Epub 2013 Dec 21.
- Results publication: PubMed 25565562Golden M, Saeidi S, Liem B, Marchand E, Morrison L, Hart B. Sensitivity of patients with familial cerebral cavernous malformations to therapeutic radiation. J Med Imaging Radiat Oncol. 2015 Feb;59(1):134-6. doi: 10.1111/1754-9485.12269. Epub 2015 Jan 7.
- Results publication: PubMed 24323376Hart BL, Taheri S, Rosenberg GA, Morrison LA. Dynamic contrast-enhanced MRI evaluation of cerebral cavernous malformations. Transl Stroke Res. 2013 Oct;4(5):500-6. doi: 10.1007/s12975-013-0285-y. Epub 2013 Sep 21.
- Results publication: PubMed 19833796Petersen TA, Morrison LA, Schrader RM, Hart BL. Familial versus sporadic cavernous malformations: differences in developmental venous anomaly association and lesion phenotype. AJNR Am J Neuroradiol. 2010 Feb;31(2):377-82. doi: 10.3174/ajnr.A1822. Epub 2009 Oct 15.
- Results publication: PubMed 26795600Choquet H, Trapani E, Goitre L, Trabalzini L, Akers A, Fontanella M, Hart BL, Morrison LA, Pawlikowska L, Kim H, Retta SF. Cytochrome P450 and matrix metalloproteinase genetic modifiers of disease severity in Cerebral Cavernous Malformation type 1. Free Radic Biol Med. 2016 Mar;92:100-109. doi: 10.1016/j.freeradbiomed.2016.01.008. Epub 2016 Jan 19.
- Results publication: PubMed 25556204Golden MJ, Morrison LA, Kim H, Hart BL. Increased number of white matter lesions in patients with familial cerebral cavernous malformations. AJNR Am J Neuroradiol. 2015 May;36(5):899-903. doi: 10.3174/ajnr.A4200. Epub 2015 Jan 2.
- Angioma Alliance is an organization by and for those affected by cavernous angiomas and their loved ones, health professionals, and researchers.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. Available data will be released to the Rare Diseases Clinical Research Network repository and will become available to the scientific community one year after publication of planned analyses, or after a period of 5 years from the date when the data were collected, whichever comes first
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Helen Kim, PhD principal investigator
University of California, San Francisco
Issam Awad, MD principal investigator
University of Chicago
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cavernous Angioma, Familial and Cerebral Cavernous Malformations, run by University of California, San Francisco at 7 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total CCM lesion number per patient (time frame: Baseline). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01764529, which also lists 28 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2026.