AMX0035
Brief Summary:
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
Condition or disease Intervention/treatment Phase
Wolfram SyndromeDrug: AMX0035Phase 2
Detailed Description:
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and to evaluate the effect of AMX0035 on residual beta cell functions by monitoring c-peptide levels during a 0-240 minute mixed-meal tolerance test. The trial will also assess the effects of AMX0035 on changes to diabetic measurements including daily insulin dose, time in good glucose range, and HbA1c levels. Effect on best-corrected visual acuity in both eyes will also be evaluated.
Study Design
Study Type :Interventional (Clinical Trial)
Estimated Enrollment :12 participants
Allocation:N/A
Intervention Model:Single Group Assignment
Intervention Model Description:A 24 week open-label treatment period of AMX0035 administered twice daily
Masking:None (Open Label)
Primary Purpose:Treatment
Official Title:A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Estimated Study Start Date :February 2023
Estimated Primary Completion Date :February 2024
Estimated Study Completion Date :February 2024
Resource links provided by the National Library of MedicineMedlinePlus Genetics related topics: Wolfram syndrome
Genetic and Rare Diseases Information Center resources: Wolfram Syndrome
U.S. FDA Resources
Arm Intervention/treatment Experimental: AMX0035
AMX0035 administered by mouth for 24 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participantDrug: AMX0035
AMX0035
Other Name: Proprietary formulation of taurursodiol and sodium phenylbutyrate
Outcome Measures
Primary Outcome Measures :
Secondary Outcome Measures :
Eligibility Criteria
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: 17 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
CriteriaKey Inclusion Criteria:
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05676034
Contacts
Contact: Amy Gauger857-320-6222clinicaltrials@amylyx.com
Sponsors and Collaborators
Amylyx Pharmaceuticals Inc.
Investigators
Study Director:Mathias Leinders, Dr.Amylyx Pharmaceuticals
Brief Summary:
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
Condition or disease Intervention/treatment Phase
Wolfram SyndromeDrug: AMX0035Phase 2
Detailed Description:
AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and to evaluate the effect of AMX0035 on residual beta cell functions by monitoring c-peptide levels during a 0-240 minute mixed-meal tolerance test. The trial will also assess the effects of AMX0035 on changes to diabetic measurements including daily insulin dose, time in good glucose range, and HbA1c levels. Effect on best-corrected visual acuity in both eyes will also be evaluated.
Study Design
Study Type :Interventional (Clinical Trial)
Estimated Enrollment :12 participants
Allocation:N/A
Intervention Model:Single Group Assignment
Intervention Model Description:A 24 week open-label treatment period of AMX0035 administered twice daily
Masking:None (Open Label)
Primary Purpose:Treatment
Official Title:A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Estimated Study Start Date :February 2023
Estimated Primary Completion Date :February 2024
Estimated Study Completion Date :February 2024
Resource links provided by the National Library of MedicineMedlinePlus Genetics related topics: Wolfram syndrome
Genetic and Rare Diseases Information Center resources: Wolfram Syndrome
U.S. FDA Resources
Arm Intervention/treatment Experimental: AMX0035
AMX0035 administered by mouth for 24 weeks: Once daily for first 3 weeks and then twice daily for the remainder of the study if tolerated by participantDrug: AMX0035
AMX0035
Other Name: Proprietary formulation of taurursodiol and sodium phenylbutyrate
Outcome Measures
Primary Outcome Measures :
- To evaluate the effect of AMX0035 during a 0-240 minutes mixed-meal tolerance test (MMTT) [ Time Frame: 28 weeks ]
- C-peptide AUC response at Week 24
- Change from Baseline in C-peptide at Week 24
- To assess the safety and tolerability of AMX0035in adult participants with Wolfram syndrome [ Time Frame: 28 weeks ]
- Incidence and severity of Adverse Events and Serious Adverse Events
- Incidence of abnormalities in clinical laboratory assessments
Secondary Outcome Measures :
- To assess the effect size of AMX0035 on visual acuity [ Time Frame: 24 weeks ]
Change from Baseline on best-corrected visual acuity (Logmar) at Week 24 - To evaluate the effect of AMX0035 on total daily insulin dose [ Time Frame: 24 weeks ]
Change from Baseline of exogenous insulin dose to Week 24 - To evaluate the effect of AMX0035 on glucose range [ Time Frame: 24 weeks ]Change from Baseline to Week 24
- Time in good range
- Time below range
- Time above range
- To evaluate the effect of AMX0035 on HbA1c levels [ Time Frame: 24 weeks ]
Change from Baseline to Week 24 in HbA1c level
Eligibility Criteria
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: 17 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
CriteriaKey Inclusion Criteria:
- Definitive diagnosis of Wolfram syndrome
- Insulin dependent diabetes mellitus due to Wolfram syndrome
- At least 17 years of age
- Participant must be willing to wear a CGM device for the duration of the study
- Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis)
- Any history of heart failure per New York Heart Association (NYHA)
- History of or family history of breast and/or ovarian cancer
- Participant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgment
- Received treatment with any investigational drug or device within the 30 days (or 5 half-lives, whichever is longer) prior to first dose at Day 1
- Previous treatment with gene or cellular therapy
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05676034
Contacts
Contact: Amy Gauger857-320-6222clinicaltrials@amylyx.com
Sponsors and Collaborators
Amylyx Pharmaceuticals Inc.
Investigators
Study Director:Mathias Leinders, Dr.Amylyx Pharmaceuticals
Valproic Acid
A Clinical Trial of Valproate Treatment to Slow Disease Progression in Children and Adults with Wolfram.
The two possible treatments are:
A- Sodium Valproate treatment
This treatment is for 3 years, until final assessments to see if it works. These are tablets.
B- Placebo treatment
This is also for 3 years. The dummy tablets are identical to the real medicine. The patient and doctor will not know which treatment is which.
This study is in Birmingham, UK; Spain; France; and Poland. 70 people will take part. 2 people will have the medicine to every 1 the placebo, decided by chance.
Stage 1
First visit, up to 28 days before starting
• Ask consent to take part
• Baseline tests and brain scan.
• Check person is eligible to take part
Stage 2
Second visit, randomise to valproate or placebo
• Start treatment
Stage 3
Follow-up visit at 6 weeks
• Visit for safety checks
Stage 4
Visits at 6, 12, 18, 24, 36 months.
• Safety checks, blood tests, eye tests
• MRI brain scans at 12, 24, 36 months
Stage 5
Final visit after treatment finished
• Final safety checks
This trial will tell us if Sodium Valproate is SAFE and EFFECTIVE in Wolfram. If these are shown, then doctors may prescribe this medicine for almost everyone with Wolfram in any country.
A Clinical Trial of Valproate Treatment to Slow Disease Progression in Children and Adults with Wolfram.
The two possible treatments are:
A- Sodium Valproate treatment
This treatment is for 3 years, until final assessments to see if it works. These are tablets.
B- Placebo treatment
This is also for 3 years. The dummy tablets are identical to the real medicine. The patient and doctor will not know which treatment is which.
This study is in Birmingham, UK; Spain; France; and Poland. 70 people will take part. 2 people will have the medicine to every 1 the placebo, decided by chance.
Stage 1
First visit, up to 28 days before starting
• Ask consent to take part
• Baseline tests and brain scan.
• Check person is eligible to take part
Stage 2
Second visit, randomise to valproate or placebo
• Start treatment
Stage 3
Follow-up visit at 6 weeks
• Visit for safety checks
Stage 4
Visits at 6, 12, 18, 24, 36 months.
• Safety checks, blood tests, eye tests
• MRI brain scans at 12, 24, 36 months
Stage 5
Final visit after treatment finished
• Final safety checks
This trial will tell us if Sodium Valproate is SAFE and EFFECTIVE in Wolfram. If these are shown, then doctors may prescribe this medicine for almost everyone with Wolfram in any country.
Liraglutide in WS, USA
This study is currently recruiting participants. (see Contacts and Locations)
Sponsor Washington University School of Medicine
Led by Drs. White and Marshall, this study will focus on the tolerability and safety of liraglutide and possible beneficial effects. We are inviting all participants over 10 years old in the Wolfram Research Clinic to participate in this study.
Liraglutide is a compound that is a glucagon-like peptide-1 (GLP-1) receptor agonist; that is, it acts like the natural hormone GLP-1. GLP-1 increases the release of insulin from the pancreas after a meal and slows down the digestion of the meal; this lowers blood sugars in people with type 2 diabetes. Liraglutide is approved by the Food and Drug Administration (FDA) for use in adults and children older than 10 years old with type 2 diabetes. In animal models of Wolfram syndrome, liraglutide has also been shown to slow the progression of diabetes, and possibly neurodegeneration. It is not known if this is also true in people with Wolfram Syndrome.
Participation in this study would require injecting liraglutide once a day for 12 months. Being on liraglutide will likely lower blood sugars and insulin doses, but it is unlikely to eliminate the need for insulin completely. In addition, evaluations of insulin secretion, vision, and an MRI would be conducted at the annual research clinic, and data would be shared between those two studies. Participation in this study is voluntary, and you will be free not to participate or to stop participating at any time, and you will still be able to keep participating in the research clinic.
Contact:
Neil H. White, MD (314) 286-1157
Bess A. Marshall, MD (314) 454-6051
Sponsor Washington University School of Medicine
Led by Drs. White and Marshall, this study will focus on the tolerability and safety of liraglutide and possible beneficial effects. We are inviting all participants over 10 years old in the Wolfram Research Clinic to participate in this study.
Liraglutide is a compound that is a glucagon-like peptide-1 (GLP-1) receptor agonist; that is, it acts like the natural hormone GLP-1. GLP-1 increases the release of insulin from the pancreas after a meal and slows down the digestion of the meal; this lowers blood sugars in people with type 2 diabetes. Liraglutide is approved by the Food and Drug Administration (FDA) for use in adults and children older than 10 years old with type 2 diabetes. In animal models of Wolfram syndrome, liraglutide has also been shown to slow the progression of diabetes, and possibly neurodegeneration. It is not known if this is also true in people with Wolfram Syndrome.
Participation in this study would require injecting liraglutide once a day for 12 months. Being on liraglutide will likely lower blood sugars and insulin doses, but it is unlikely to eliminate the need for insulin completely. In addition, evaluations of insulin secretion, vision, and an MRI would be conducted at the annual research clinic, and data would be shared between those two studies. Participation in this study is voluntary, and you will be free not to participate or to stop participating at any time, and you will still be able to keep participating in the research clinic.
Contact:
Neil H. White, MD (314) 286-1157
Bess A. Marshall, MD (314) 454-6051
Dantrolene Sodium
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
This study is currently recruiting participants. (see Contacts and Locations)
Sponsor Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT02829268
Purpose
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration. The purpose of this study is to assess the safety and tolerability of dantrolene sodium in patients with Wolfram syndrome. In addition, we will assess the efficacy of dantrolene sodium on the cardinal manifestations of Wolfram syndrome, including visual acuity, remaining beta cell functions, and neurological functions.
There is a screening period up to 56 days, a 24-week treatment period, and a 4-week safety follow-up period. Study assessments include medical & medication history, physical exams, neurological exams, eye exams, endocrine exams, vital signs, height, weight, electrocardiograms, blood and urine tests, pregnancy test if applicable, and questionnaires.
Official Title A Phase 1b Safety Trial of Dantronele Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Primary Outcome Measures
• Number of participants with treatment-related adverse events as assessed by liver function tests [ Time Frame: 6 months ]
The investigators assess the safety and tolerability of dantrolene sodium administered orally at upper end of therapeutic dose range for 6 months in patients with Wolfram syndrome. More specifically, the investigators perform liver function tests to check the levels of certain enzymes and proteins in participants’ blood. Levels that are higher or lower than normal can indicate liver problems. The liver function tests include: Alanine transaminase (ALT), Aspartate transaminase (AST), Alkaline Phosphatase (AP), and bilirubin.
Secondary Outcome Measures
• Changes in C-peptide levels in participants assessed by the ELISA assay [ Time Frame: 6 months ]
The investigators determine the effect of dantrolene sodium on residual beta cell functions. The investigators monitor base-line C-peptide levels in participants’ blood. The investigators also monitor C-peptide levels in participant’s blood during the oral mixed meal tolerance test. The night before the oral mixed meal tolerance test, the participants will turn their insulin pump basal rate to 50% of the normal rate at midnight or take half of their evening dose of Lantus insulin and fasted from midnight until the test at 8 a.m. The mixed meal consists of 6 ml/kg (maximum 360 ml) of Boost Original (Société des Produits Nestlé S.A., Vevey, Switzerland). Blood for glucose and C-peptide measurement will be drawn at time 0 (fasting) and 30 minutes after the Boost. If a subject’s fasting glucose exceeds 11.1 mmol/l, the test will not be performed, but fasting glucose and C-peptide will be obtained.
• Changes in Visual Functioning in participants assessed by Visual Functioning Questionnaire-25. [ Time Frame: 6 months ]
Visual functions will be assessed by Visual Functioning Questionnaire – 25.
• Changes in best-corrected visual acuity in participants measured by Snellen optotype [ Time Frame: 6 months ]
Best-corrected visual acuity will be measured by Snellen optotype. Higher logMar scores indicate worse vision.
• Changes in Neurological Functions in participants assessed by the Wolfram Unified Rating Scale (WURS) [ Time Frame: 6 months ]
Neurological functions will be assessed by the Wolfram Unified Rating Scale (WURS) and standard neurological assessments.
Eligibility
Ages Eligible for Study: 5 Years to 60 Years (Child, Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria
Patients must meet all of the following criteria to be eligible for enrolment:
1.The patient has a definitive diagnosis of Wolfram syndrome, as determined by the following:
a. Documented functionally relevant recessive mutations on both alleles of the WFS1 gene or dominant mutation on one allele of the WFS1 gene based on historical test results (if available) or from a qualified laboratory at screening.
2.The patient is at least 5 years of age (biological age) at the time of written informed consent.
3.The patient, patient’s parent(s), or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. The guardians’ consent and patient’s assent, as relevant, must be obtained.
Exclusion Criteria
Patients who meet any of the following criteria are not eligible for this study:
1.The patient has clinically significant non-Wolfram related CNS involvement which is judged by the investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
2.The patient has a known defect in oxidative phosphorylation (such as a confirmed mitochondrial myopathy)
3.The patient has abnormal liver function (defined as serum transaminases more than twice the upper limit of normal for the reference laboratory)
4.The patient has a significant medical or psychiatric co-morbidity that might affect study data or confound the integrity of study results.
5.The patient has received treatment with any investigational drug within the 30 days prior to study entry.
6.The patient has received blood product transfusions within 90 days prior to screening.
7.The patient is unable to comply with the protocol, (e.g. has a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, known clinically significant psychiatric/behavioural instability, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator.
8.The patient has a known history of central apnea and/or ventilation requirements.
9.The patient has a known history of chronic obstructive pulmonary disease, pleural effusion, and/or myocardial disease.
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02829268
Contacts
Contact Ashley N Simpson, RN, BSN, CPN +1-314-286-1550 ashley.simpson@wustl.edu
Contact Teresa Arb, RN, BSN, CCRC +1-314-747-1217 arbt@wustl.edu
Locations
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Ashley Simpson, RN, BSN, CPN 314-286-1550 ashley.simpson@wustl.edu
Contact: Fumihiko Urano, MD 314-362-8683 urano@wustl.edu
Principal Investigator: Fumihiko Urano, MD
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
This study is currently recruiting participants. (see Contacts and Locations)
Sponsor Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT02829268
Purpose
Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration. The purpose of this study is to assess the safety and tolerability of dantrolene sodium in patients with Wolfram syndrome. In addition, we will assess the efficacy of dantrolene sodium on the cardinal manifestations of Wolfram syndrome, including visual acuity, remaining beta cell functions, and neurological functions.
There is a screening period up to 56 days, a 24-week treatment period, and a 4-week safety follow-up period. Study assessments include medical & medication history, physical exams, neurological exams, eye exams, endocrine exams, vital signs, height, weight, electrocardiograms, blood and urine tests, pregnancy test if applicable, and questionnaires.
Official Title A Phase 1b Safety Trial of Dantronele Sodium in Pediatric and Adult Patients With Wolfram Syndrome
Primary Outcome Measures
• Number of participants with treatment-related adverse events as assessed by liver function tests [ Time Frame: 6 months ]
The investigators assess the safety and tolerability of dantrolene sodium administered orally at upper end of therapeutic dose range for 6 months in patients with Wolfram syndrome. More specifically, the investigators perform liver function tests to check the levels of certain enzymes and proteins in participants’ blood. Levels that are higher or lower than normal can indicate liver problems. The liver function tests include: Alanine transaminase (ALT), Aspartate transaminase (AST), Alkaline Phosphatase (AP), and bilirubin.
Secondary Outcome Measures
• Changes in C-peptide levels in participants assessed by the ELISA assay [ Time Frame: 6 months ]
The investigators determine the effect of dantrolene sodium on residual beta cell functions. The investigators monitor base-line C-peptide levels in participants’ blood. The investigators also monitor C-peptide levels in participant’s blood during the oral mixed meal tolerance test. The night before the oral mixed meal tolerance test, the participants will turn their insulin pump basal rate to 50% of the normal rate at midnight or take half of their evening dose of Lantus insulin and fasted from midnight until the test at 8 a.m. The mixed meal consists of 6 ml/kg (maximum 360 ml) of Boost Original (Société des Produits Nestlé S.A., Vevey, Switzerland). Blood for glucose and C-peptide measurement will be drawn at time 0 (fasting) and 30 minutes after the Boost. If a subject’s fasting glucose exceeds 11.1 mmol/l, the test will not be performed, but fasting glucose and C-peptide will be obtained.
• Changes in Visual Functioning in participants assessed by Visual Functioning Questionnaire-25. [ Time Frame: 6 months ]
Visual functions will be assessed by Visual Functioning Questionnaire – 25.
• Changes in best-corrected visual acuity in participants measured by Snellen optotype [ Time Frame: 6 months ]
Best-corrected visual acuity will be measured by Snellen optotype. Higher logMar scores indicate worse vision.
• Changes in Neurological Functions in participants assessed by the Wolfram Unified Rating Scale (WURS) [ Time Frame: 6 months ]
Neurological functions will be assessed by the Wolfram Unified Rating Scale (WURS) and standard neurological assessments.
Eligibility
Ages Eligible for Study: 5 Years to 60 Years (Child, Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Criteria
Inclusion Criteria
Patients must meet all of the following criteria to be eligible for enrolment:
1.The patient has a definitive diagnosis of Wolfram syndrome, as determined by the following:
a. Documented functionally relevant recessive mutations on both alleles of the WFS1 gene or dominant mutation on one allele of the WFS1 gene based on historical test results (if available) or from a qualified laboratory at screening.
2.The patient is at least 5 years of age (biological age) at the time of written informed consent.
3.The patient, patient’s parent(s), or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board/Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient. The guardians’ consent and patient’s assent, as relevant, must be obtained.
Exclusion Criteria
Patients who meet any of the following criteria are not eligible for this study:
1.The patient has clinically significant non-Wolfram related CNS involvement which is judged by the investigator to be likely to interfere with the accurate administration and interpretation of protocol assessments.
2.The patient has a known defect in oxidative phosphorylation (such as a confirmed mitochondrial myopathy)
3.The patient has abnormal liver function (defined as serum transaminases more than twice the upper limit of normal for the reference laboratory)
4.The patient has a significant medical or psychiatric co-morbidity that might affect study data or confound the integrity of study results.
5.The patient has received treatment with any investigational drug within the 30 days prior to study entry.
6.The patient has received blood product transfusions within 90 days prior to screening.
7.The patient is unable to comply with the protocol, (e.g. has a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, known clinically significant psychiatric/behavioural instability, is unable to return for safety evaluations, or is otherwise unlikely to complete the study), as determined by the Investigator.
8.The patient has a known history of central apnea and/or ventilation requirements.
9.The patient has a known history of chronic obstructive pulmonary disease, pleural effusion, and/or myocardial disease.
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02829268
Contacts
Contact Ashley N Simpson, RN, BSN, CPN +1-314-286-1550 ashley.simpson@wustl.edu
Contact Teresa Arb, RN, BSN, CCRC +1-314-747-1217 arbt@wustl.edu
Locations
United States, Missouri
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Ashley Simpson, RN, BSN, CPN 314-286-1550 ashley.simpson@wustl.edu
Contact: Fumihiko Urano, MD 314-362-8683 urano@wustl.edu
Principal Investigator: Fumihiko Urano, MD
For More information