Alport Syndrome Treatments and Outcomes Registry
| Status: | Recruiting |
|---|---|
| Conditions: | Nephrology |
| Therapuetic Areas: | Nephrology / Urology |
| Healthy: | No |
| Age Range: | Any - 99 |
| Updated: | 5/3/2018 |
| Start Date: | September 2007 |
| End Date: | January 2022 |
| Contact: | Kristi Rosenthal |
| Email: | krosenth@umn.edu |
| Phone: | 612-626-6135 |
Over the past 30 years much has been learned about the molecular genetics and natural history
of familial forms of hematuria. However, the enhanced understanding of these conditions has
yet to generate effective therapies for Alport syndrome, the form of familial hematuria
associated with end-stage renal disease. Males with Alport syndrome inevitably develop
end-stage kidney failure, with a 50% likelihood of dialysis or kidney transplantation by age
25 years. There is no proven treatment for Alport syndrome, although studies in animals have
suggested several promising potential therapies. Potential drug treatments that might delay
or prevent the development of kidney failure exist, but need to be evaluated through clinical
trails. Conducting clinical trails for proposed treatments for Alport syndrome present many
challenges. Because Alport syndrome is not a common disease, informative clinical trials will
require the collaboration of investigators at multiple centers.
The University of Minnesota, Department of Pediatrics, proposes to create the Alport Syndrome
Treatments and Outcomes Registry (ASTOR) in order to facilitate clinical trials for the
treatment of Alport Syndrome. This registry will be the first of its kind in North America.
Because Alport syndrome is a rare disorder, recruitment of sufficient participants for
meaningful therapeutic trials will require a multicenter effort. The primary objective of
establishing and sustaining this registry is to enable clinical natural history studies and
therapeutic trials to be conducted in children and adolescents with Alport syndrome.
of familial forms of hematuria. However, the enhanced understanding of these conditions has
yet to generate effective therapies for Alport syndrome, the form of familial hematuria
associated with end-stage renal disease. Males with Alport syndrome inevitably develop
end-stage kidney failure, with a 50% likelihood of dialysis or kidney transplantation by age
25 years. There is no proven treatment for Alport syndrome, although studies in animals have
suggested several promising potential therapies. Potential drug treatments that might delay
or prevent the development of kidney failure exist, but need to be evaluated through clinical
trails. Conducting clinical trails for proposed treatments for Alport syndrome present many
challenges. Because Alport syndrome is not a common disease, informative clinical trials will
require the collaboration of investigators at multiple centers.
The University of Minnesota, Department of Pediatrics, proposes to create the Alport Syndrome
Treatments and Outcomes Registry (ASTOR) in order to facilitate clinical trials for the
treatment of Alport Syndrome. This registry will be the first of its kind in North America.
Because Alport syndrome is a rare disorder, recruitment of sufficient participants for
meaningful therapeutic trials will require a multicenter effort. The primary objective of
establishing and sustaining this registry is to enable clinical natural history studies and
therapeutic trials to be conducted in children and adolescents with Alport syndrome.
Detailed Description:
ASTOR is envisioned as a permanent organization sustained by private philanthropic and public
funding sources. Recruitment of participants for the registry will consist of three
approaches. First, pediatric nephrologists in the United States and Canada will be invited to
participate in ASTOR. Activities of participating investigators will include identification
and recruitment of potential study participants, collection and transmission of participant
data to the ASTOR central office at the University of Minnesota, and implementation of study
protocols.
Participating physicians will inform their participants and parents about ASTOR and invite
them to participate in writing. The correspondence will include instructions on how to
contact ASTOR personal about formal participation. Participants and their parents will be
asked to permit sharing of their personal health information with the ASTOR central office.
Second, the ASTOR central office will develop a website for participants and families. The
website will provide participants and families with medical and new research information and
instructions on how to contact registry staff about registry participation. Potential
participants who contact the central office will be asked to give consent for ASTOR staff to
contact their health care providers.
Third, the University of Utah School of Medicine's Department of Nephrology, currently holds
data pertaining to approximately 350 families with a reported history of Alport syndrome.
Department investigators have agreed to provide information that will ultimately assist ASTOR
staff in executing recruitment activities by providing historical medical health information
pertaining the natural progression of the disease and current treatment practices and by
contacting patients in the existing database to determine if any individuals or families are
interested in participating in the registry. This information will be used to support
analysis efforts that may come about as a result of new studies that are developed or
implemented under the aegis of the registry.
Selection and Enrollment of Participants
Each consented participant will be assigned a unique identifier and entered into the ASTOR
database, along with demographic data, health information and urine analysis results.
Referring physicians and their home institutions will also be assigned unique identifiers.
ASTOR coordinator will contact each participant's physician and provide instructions on how
to complete a detailed baseline questionnaire designed to capture the following information:
Family history
- X-chromosomal inheritance
- Autosomal inheritance
- Mode of inheritance unknown
- Mode of inheritance known: Which person of your family is/was effected (or indicate in
family tree), which symptoms (hearing defect, ocular abnormalities, renal disease,
leiomyomatosis)?
- At what age was the diagnosis Alport syndrome was made in effected family members?
- If a family member had end stage renal failure, at what age?
Individual data of treated participant
- At what age the diagnosis of Alport syndrome was made?
- Has a skin biopsy been performed? If yes, list results.
- Has a kidney biopsy been performed? If yes, list results.
- Has a molecular-genetic diagnosis/ mutation analysis been performed?
Clinical data and progress of disease
• Medications and the age these meds were started
Recruitment of participants for the registry will consist of three approaches. First,
pediatric nephrologists in the United States and Canada will be invited to participate in the
Alport Syndrome Treatments and Outcomes Registry (ASTOR). Activities of participating
investigators will include identification and recruitment of potential study participants,
collection and transmission of participant data to the ASTOR registry's central office at the
University of Minnesota. Second, the ASTOR central office will develop a website for
participants and families. The website will provide participants and families with medical
and new research information and instructions on how to contact registry staff about registry
participation. All policies and procedures for the information or samples that are shared,
stored or banked by the ASTOR central office will be prepared in accordance with the
University of Minnesota Office of Information Technology standard for securing private data
(see data safety monitoring plan). Urine samples will be collected by the ASTOR central
office. Instructions and materials for collecting and mailing the specimens will be provided
to each participant. Third, the University of Utah will contact their existing patient base
to determine if any individuals or families are interested in participating in the registry
(IRB approval will be sought to contact potential participants). Interested participants will
be referred to the ASTOR central office. Interested individuals and families will be
contacted in writing about formal participation.
ASTOR central office staff will obtain informed consent form all participants (telephone
consent IRB approval pending). Participants will be seen every six months for assessments.
Clinical assessments will include an initial baseline questionnaire and urine sample followed
with bi-annual updates on medical and medication histories and urine analysis.
Medical and medication histories will be completed by the participant's provider and urine
samples will be forwarded by the participant to the University of Minnesota's central office
for testing. Contractual agreements for the processing of all urine samples will be will be
established with an in-state laboratory .
Data Collection and Site Monitoring
Data to be Collected by Study Personnel:
1. Participant consent for the study (includes participant and parental consent,
participant assent when appropriate)
2. Baseline Questionnaire with bi annual medication and medical updates c. Baseline urine
sample repeated bi annually
Participant's medical and clinical data and urine analysis results will be submitted to
the ASTOR central office from multiple sources. Our goal is that all participants by 4
months would have complete study data (acknowledgement of consent, completed baseline
questionnaire and urine sample results) and data entry into an ASTOR central office data
base. Performance and completion of these components do not have participant or
participant safety implications.
• Data accuracy: For each participant, the data entered will match with the hard copy of
the questionnaire and lab and results. Opportunities for error will be minimized by the
elimination of the need to re-copy data to multiple locations. Copies of all source
documents will be filed in double locked environments.
• Data Confidentiality: Every participant will be assigned a unique identifying registry
number. These unique participant identifiers will be kept within individual participant
folders. These participant folders are kept in files that are locked, with only study
specific personnel having access to them.
Participant data in computer databases are accessible only by study personnel require
and require a password. Participant specific information will not be included in
publications or in presentations at local or national meetings or conferences.
• Data Use: Registry participant data regarding medical history, urine analysis, and
demographic data will be used to define and categorize participants for future research
studies. Participant will be informed of the intended use and sign a formal consent to
participate in any future research endeavors that may be developed and implemented.
The database will also be useful in providing study personnel with the specific dates
for ensuing visits beginning with baseline and all subsequent bi-annual evaluations. No
one outside of immediate study staff will have access to the participant data, and
password protection is in place on each of the study computers to reduce the chance of
any breach in confidentiality.
There may be occasional requests from investigators outside the immediate study
personnel to share information. Information will be provided to investigators in
accordance with University of Minnesota Data Monitoring and Safety policies.
ASTOR is envisioned as a permanent organization sustained by private philanthropic and public
funding sources. Recruitment of participants for the registry will consist of three
approaches. First, pediatric nephrologists in the United States and Canada will be invited to
participate in ASTOR. Activities of participating investigators will include identification
and recruitment of potential study participants, collection and transmission of participant
data to the ASTOR central office at the University of Minnesota, and implementation of study
protocols.
Participating physicians will inform their participants and parents about ASTOR and invite
them to participate in writing. The correspondence will include instructions on how to
contact ASTOR personal about formal participation. Participants and their parents will be
asked to permit sharing of their personal health information with the ASTOR central office.
Second, the ASTOR central office will develop a website for participants and families. The
website will provide participants and families with medical and new research information and
instructions on how to contact registry staff about registry participation. Potential
participants who contact the central office will be asked to give consent for ASTOR staff to
contact their health care providers.
Third, the University of Utah School of Medicine's Department of Nephrology, currently holds
data pertaining to approximately 350 families with a reported history of Alport syndrome.
Department investigators have agreed to provide information that will ultimately assist ASTOR
staff in executing recruitment activities by providing historical medical health information
pertaining the natural progression of the disease and current treatment practices and by
contacting patients in the existing database to determine if any individuals or families are
interested in participating in the registry. This information will be used to support
analysis efforts that may come about as a result of new studies that are developed or
implemented under the aegis of the registry.
Selection and Enrollment of Participants
Each consented participant will be assigned a unique identifier and entered into the ASTOR
database, along with demographic data, health information and urine analysis results.
Referring physicians and their home institutions will also be assigned unique identifiers.
ASTOR coordinator will contact each participant's physician and provide instructions on how
to complete a detailed baseline questionnaire designed to capture the following information:
Family history
- X-chromosomal inheritance
- Autosomal inheritance
- Mode of inheritance unknown
- Mode of inheritance known: Which person of your family is/was effected (or indicate in
family tree), which symptoms (hearing defect, ocular abnormalities, renal disease,
leiomyomatosis)?
- At what age was the diagnosis Alport syndrome was made in effected family members?
- If a family member had end stage renal failure, at what age?
Individual data of treated participant
- At what age the diagnosis of Alport syndrome was made?
- Has a skin biopsy been performed? If yes, list results.
- Has a kidney biopsy been performed? If yes, list results.
- Has a molecular-genetic diagnosis/ mutation analysis been performed?
Clinical data and progress of disease
• Medications and the age these meds were started
Recruitment of participants for the registry will consist of three approaches. First,
pediatric nephrologists in the United States and Canada will be invited to participate in the
Alport Syndrome Treatments and Outcomes Registry (ASTOR). Activities of participating
investigators will include identification and recruitment of potential study participants,
collection and transmission of participant data to the ASTOR registry's central office at the
University of Minnesota. Second, the ASTOR central office will develop a website for
participants and families. The website will provide participants and families with medical
and new research information and instructions on how to contact registry staff about registry
participation. All policies and procedures for the information or samples that are shared,
stored or banked by the ASTOR central office will be prepared in accordance with the
University of Minnesota Office of Information Technology standard for securing private data
(see data safety monitoring plan). Urine samples will be collected by the ASTOR central
office. Instructions and materials for collecting and mailing the specimens will be provided
to each participant. Third, the University of Utah will contact their existing patient base
to determine if any individuals or families are interested in participating in the registry
(IRB approval will be sought to contact potential participants). Interested participants will
be referred to the ASTOR central office. Interested individuals and families will be
contacted in writing about formal participation.
ASTOR central office staff will obtain informed consent form all participants (telephone
consent IRB approval pending). Participants will be seen every six months for assessments.
Clinical assessments will include an initial baseline questionnaire and urine sample followed
with bi-annual updates on medical and medication histories and urine analysis.
Medical and medication histories will be completed by the participant's provider and urine
samples will be forwarded by the participant to the University of Minnesota's central office
for testing. Contractual agreements for the processing of all urine samples will be will be
established with an in-state laboratory .
Data Collection and Site Monitoring
Data to be Collected by Study Personnel:
1. Participant consent for the study (includes participant and parental consent,
participant assent when appropriate)
2. Baseline Questionnaire with bi annual medication and medical updates c. Baseline urine
sample repeated bi annually
Participant's medical and clinical data and urine analysis results will be submitted to
the ASTOR central office from multiple sources. Our goal is that all participants by 4
months would have complete study data (acknowledgement of consent, completed baseline
questionnaire and urine sample results) and data entry into an ASTOR central office data
base. Performance and completion of these components do not have participant or
participant safety implications.
• Data accuracy: For each participant, the data entered will match with the hard copy of
the questionnaire and lab and results. Opportunities for error will be minimized by the
elimination of the need to re-copy data to multiple locations. Copies of all source
documents will be filed in double locked environments.
• Data Confidentiality: Every participant will be assigned a unique identifying registry
number. These unique participant identifiers will be kept within individual participant
folders. These participant folders are kept in files that are locked, with only study
specific personnel having access to them.
Participant data in computer databases are accessible only by study personnel require
and require a password. Participant specific information will not be included in
publications or in presentations at local or national meetings or conferences.
• Data Use: Registry participant data regarding medical history, urine analysis, and
demographic data will be used to define and categorize participants for future research
studies. Participant will be informed of the intended use and sign a formal consent to
participate in any future research endeavors that may be developed and implemented.
The database will also be useful in providing study personnel with the specific dates
for ensuing visits beginning with baseline and all subsequent bi-annual evaluations. No
one outside of immediate study staff will have access to the participant data, and
password protection is in place on each of the study computers to reduce the chance of
any breach in confidentiality.
There may be occasional requests from investigators outside the immediate study
personnel to share information. Information will be provided to investigators in
accordance with University of Minnesota Data Monitoring and Safety policies.
Inclusion criteria:
- Diagnosis of Alport syndrome, confirmed by skin biopsy, kidney biopsy, or molecular
genetic analysis or diagnosis of Alport syndrome, based on presence of hematuria and
confirmed diagnosis of Alport syndrome in a first-degree relative
- Normal renal function, as measured by serum creatinine or estimated or measured
creatinine clearance
Exclusion Criteria:
- Uncertain diagnosis of Alport syndrome
We found this trial at
1
site
Minneapolis, Minnesota 55455
(612) 625-5000
Principal Investigator: Clifford Kashtan, MD
Univ of Minnesota With a flagship campus in the heart of the Twin Cities, and...
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