Cavernous Malformations,Cerebral and/or SpinalPhase 1NCT03523650
Oral Propanolol for Surgically Inaccessible Cavernous Malformations
A Phase 1 study testing Propranolol Oral Tablet in people with Cavernous Malformations,Cerebral and/or Spinal, run by University of Virginia at 1 site in United States.
Trial summary
The purpose of this study is to evaluate the role of propranolol as an alternative treatment for cavernous malformation in patients that may not be ideal candidates for surgery. Read more about this study
- Who is it for?
- Ages 1 year to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Cavernous Malformations,Cerebral and/or Spinal
- What is being tested?
- Propranolol Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 346 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Feb 2018to Jan 2019
- Phase
- Phase 1
- 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 1 year to 80 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of isolated or familial cavernous malformation syndrome:
- symptomatic cavernous malformation not amenable to surgical resection.
- familial cavernous malformation with seizure, other neurological symptom or surgically inaccessible lesion.
- Written and informed consent obtained prior to study enrollment.
- Subject is able and willing to return for outpatient visits.
- Negative pregnancy test at time of enrollment for women and child-bearing potential.
Who cannot join
- Age less than 1 year-old.
- Propranolol allergy or allergy to other b-blockers.
- Estimated life expectancy of less than 1 year.
- History of severe anemia, cardiac dysfunction, or diabetes.
- A psychiatric or substance abuse problem that may interfere with study compliance.
- Pregnant and lactating women.
Trial locations and contacts
1 site in 1 country.
United States1 site
Univeristy of Virginia
Charlottesville, Virginia, 22903
About this study
Official title: Oral Propanolol for Surgically Inaccessible Cerebral and Spinal Cavernous Malformations
The purpose of this study is to evaluate the role of propranolol as an alternative treatment for cavernous malformation in patients that may not be ideal candidates for surgery.
Read the detailed description
Patients between the ages of 1 and 80 years of age, that have been diagnosed with a spinal or cerebral cavernous malformation(s) that can not be surgically treated, will be enrolled in this randomized study. All eligible patients will be enrolled in one of two groups. Group 1 will receive oral propranolol tablets 3 times a day for 36 months. Group 2 will receive oral placebo tablets 3 times per day for 36 months. Prior to beginning treatment, all imaging, including MRIs, will be evaluated. During the first visit a blood sample will be collected for genomic analysis. Patients will return to clinic every 6 months. Each time the patient returns to clinic monitoring of how the patient is feeling and evaluation of how the patient is tolerating the propranolol will take place. At the end of the three years, evaluators will determine if the propranolol was able to stop the growth of the cavernous malformation and change the imaging features of the lesion on MRI. Once the study is completed patients will no longer receive the study drug.
- Being tested
- Propranolol Oral Tablet, Placebo Oral Tablet
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Propranolol Oral TabletDrug
A randomized group of patients with cerebral or spinal cavernous malformations, will receive a Propranolol Oral tablets, tid, for 36 months.
Placebo Oral TabletDrug
A randomized group of patients with cerebral or spinal cavernous malformations, will receive a Placebo Oral Tablet, tid, for 36 months.
| Study group | Role | What they receive |
|---|---|---|
| Group 1: Propranolol Group | Experimental | Group 1: Propranolol - group of randomized patients will receive one propranolol pill tid for 36 months. Receives: Drug: Propranolol Oral Tablet |
| Group 2: Placebo Group | Placebo comparator | Group 2: Placebo - group of randomized patients will receive one placebo pill tid for 36 months. Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of symptomatic and silent hemorrhages on MRI
Using MRI imaging the size of the cavernous malformation will be measured. The primary outcome is the number of symptomatic and silent hemorrhages as assessed by review of a fine-cut, axial T2-weighted MRI obtained at each visit.
Measured: 36 months
Secondary outcomes (1)
Rate of de novo lesion formation; changes in rate of breakthrough seizures or other neurological deficits
The secondary outcome is the rate of de novo lesion formation; changes in rate of breakthrough seizures or other neurological deficits; quality of life measured using the SF-36 at each visit, patient satisfaction with treatment; and the incidence of treatment failure.
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2018
- Study start7 Feb 2018
- Primary completionJan 2019 (estimated)
- Study completionJan 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cavernous Malformations,Cerebral and/or SpinalStudies of the same condition, open and under way.
- 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 346 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Virginia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yashar Kalani, MD, University of Virginia
- Investigators
- Yashar Kalani, MD, PHD, Principal investigator, University of Virginia Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03523650
- Sponsor's protocol ID
- 20195
- Status last verified
- May 2018
- First submitted
- 13 Apr 2018
- Last updated
- 14 May 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Oral Propanolol for Surgically Inaccessible Cerebral and Spinal Cavernous Malformations
- ClinicalTrials.gov ID
- NCT03523650
- Acronym / sponsor ID
- 20195
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 346 participants (estimated)
- Lead sponsor
- University of VirginiaUniversity, hospital or other
- Study start
- 7 Feb 2018 (actual)
- Primary completion
- Jan 2019 (estimated)
- Study completion
- Jan 2019 (estimated)
- First posted
- 14 May 2018 (actual)
- 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.
The purpose of this study is to evaluate the role of propranolol as an alternative treatment for cavernous malformation in patients that may not be ideal candidates for surgery.
Detailed description
Patients between the ages of 1 and 80 years of age, that have been diagnosed with a spinal or cerebral cavernous malformation(s) that can not be surgically treated, will be enrolled in this randomized study. All eligible patients will be enrolled in one of two groups. Group 1 will receive oral propranolol tablets 3 times a day for 36 months. Group 2 will receive oral placebo tablets 3 times per day for 36 months. Prior to beginning treatment, all imaging, including MRIs, will be evaluated. During the first visit a blood sample will be collected for genomic analysis. Patients will return to clinic every 6 months. Each time the patient returns to clinic monitoring of how the patient is feeling and evaluation of how the patient is tolerating the propranolol will take place. At the end of the three years, evaluators will determine if the propranolol was able to stop the growth of the cavernous malformation and change the imaging features of the lesion on MRI. Once the study is completed patients will no longer receive the study drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1: Propranolol Group | Experimental | Drug: Propranolol Oral TabletGroup 1: Propranolol - group of randomized patients will receive one propranolol pill tid for 36 months. |
| Group 2: Placebo Group | Placebo comparator | Drug: Placebo Oral TabletGroup 2: Placebo - group of randomized patients will receive one placebo pill tid for 36 months. |
Who may qualify
- Ages
- 1 year to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of isolated or familial cavernous malformation syndrome:
- Written and informed consent obtained prior to study enrollment.
- Subject is able and willing to return for outpatient visits.
- Negative pregnancy test at time of enrollment for women and child-bearing potential.
Key exclusion criteria (6 listed)
- Age less than 1 year-old.
- Propranolol allergy or allergy to other b-blockers.
- Estimated life expectancy of less than 1 year.
- History of severe anemia, cardiac dysfunction, or diabetes.
- A psychiatric or substance abuse problem that may interfere with study compliance.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Propranolol Oral TabletA randomized group of patients with cerebral or spinal cavernous malformations, will receive a Propranolol Oral tablets, tid, for 36 months. | Drug | Group 1: Propranolol Group |
| Placebo Oral TabletA randomized group of patients with cerebral or spinal cavernous malformations, will receive a Placebo Oral Tablet, tid, for 36 months. | Drug | Group 2: Placebo Group |
Primary outcome
Number of symptomatic and silent hemorrhages on MRI
Time frame: 36 months
Using MRI imaging the size of the cavernous malformation will be measured. The primary outcome is the number of symptomatic and silent hemorrhages as assessed by review of a fine-cut, axial T2-weighted MRI obtained at each visit.
1 secondary outcome
Rate of de novo lesion formation; changes in rate of breakthrough seizures or other neurological deficits
Time frame: 36 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2018.
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.
Yashar Kalani, MD, PHD principal investigator
University of Virginia Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Propranolol Oral Tablet in people with Cavernous Malformations,Cerebral and/or Spinal, run by University of Virginia at 1 site 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 6 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 14 May 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Number of symptomatic and silent hemorrhages on MRI (time frame: 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 14 May 2018.