Biliary AtresiaPROBENCT00061828
A Prospective Database of Infants With Cholestasis
An observational study of Biliary Atresia, run by Arbor Research Collaborative for Health at 16 sites in 2 countries.
Trial summary
Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period. Read more about this study
- Who is it for?
- Ages Up to 6 months
- Conditions the study is for
- Biliary Atresia
- How many people
- 1,000 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 21 Apr 2004to 31 May 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Up to 6 months
- All sexes
- No healthy volunteers
- Child
Study population: This study population will be selected from the patient base of participating specialty care clinics.
Who may be able to join
- Infant's age less than or equal to 180 days at initial presentation at the ChiLDReN clinical site.
- Diagnosis of cholestasis defined by serum direct or conjugated bilirubin greater than or equal to 2 mg/dl and suspected biliary atresia.
- The subject's parent(s)/guardian(s) willing to provide informed written consent.
Who cannot join
- Acute liver failure.
- Previous hepatobiliary surgery with dissection or excision of biliary tissue.
- Diagnoses of bacterial or fungal sepsis (except where associated with metabolic liver disease)
- Diagnoses of hypoxia, shock or ischemic hepatopathy within the past two weeks (If the cholestasis persists beyond two weeks of the initiating event, the infant can be enrolled).
- Diagnosis of any malignancy.
- Presence of any primary hemolytic disease (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).
Show all 9Show fewer
- Diagnosis of any drug or Total parenteral nutrition (TPN)-associated cholestasis (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).
- Diagnosis with Extracorporeal membrane oxygenation (ECMO)-associated cholestasis.
- Birth weight less than 1500g (except when diagnosed with biliary atresia).
Trial locations and contacts
16 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
16 sites in 2 countries
United States15 sites, 9 recruiting
- Recruiting
Children's Healthcare of Atlanta - Emory University
Atlanta, Georgia, 30322
Katelynn Harris, 404-785-0421, katelynn.harris@choa.org
Jordyn Turner, 404-785-3690, jordyn.turner@choa.org
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Cynthia Castanon, 720-777-0587, Cynthia.castanon@childrenscolorado.org
Michelle Landis, 720-777-8884, michelle.landis@childrenscolorado.org
- Completed
Johns Hopkins School of Medicine
Baltimore, Maryland, 21287
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60614
Jacqueline Alexander, 312-227-3523, jaalexander@luriechildrens.org
Angela Anthony, 312-227-4559, aanthony@luriechildrens.org
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Jennifer Hawkins, MS, 513-636-7818, jennifer.hawkins@cchmc.org
Erin Chapman, BAS, 513-803-7482, erin.chapman@cchmc.org
- Recruiting
Baylor College of Medicine
Houston, Texas, 77030
Laurel Cavallo, 832-822-1053, children.network@bcm.edu
Cynthia Tsai, MPH, 832-822-3634, children.network@bcm.edu
- Completed
Riley Hospital for Children
Indianapolis, Indiana, 46202
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Sirikorn Boonsawat, 323-361-2181, sboonsawat@chla.usc.edu
Rohit Kohli, MD
- Completed
Mount Sinai Medical Center
New York, New York, 10029
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Caitlin Griffiths, 267-577-9888, griffithc2@chop.edu
Jordan Moreno, 267-425-1614, morenoj@chop.edu
- Completed
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
- Recruiting
University of Utah
Salt Lake City, Utah, 84113
Ann Rutherford, 801-585-9495, ann.rutherford@hsc.utah.edu
Natalie Fillerup, 801-587-5670, natalie.fillerup@hsc.utah.edu
- Completed
University of California
San Francisco, California, 94143
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Melissa Young, 206-987-1037, melissa.young@seattlechildrens.org
Teresa Chen, 206-987-6649, teresa.chen@seattlechildrens.org
- Completed
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: Childhood Liver Disease Research Network (ChiLDReN): A Prospective Database of Infants With Cholestasis
Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period. The long term goal of the Childhood Liver Disease Research Network (ChiLDReN) is to establish a database of clinical information and plasma, serum, and tissue samples from cholestatic children to facilitate research and to perform clinical, epidemiological and therapeutic trials in these important pediatric liver diseases.
Read the detailed description
This is a multi-center project to establish a prospective database of clinical information and a repository of blood, from children with diagnosis of neonatal liver disease, such as biliary atresia (BA), in order to perform research in this important liver problem. Children (diagnosed with BA or suspicious for BA) will be screened and enrolled at presentation at the participating pediatric liver sites. Participants diagnosed with BA will be followed intensively for the first year, at 18 months of age, and then annually up to 10 years of age, and then biannually, or liver transplantation. Other participants (Non-BA) diagnosed with cholestasis will be exited from the study at the time of diagnosis determination.
Detailed clinical data, laboratory investigations, liver and biliary specimens, and long-term follow-up of outcomes are part of the normal standard of care with respect to the diagnosis and treatment of the subjects with liver problems. This research involves the collection of diagnostic, clinical and outcome data concerning the subject, which is kept without identification (coded) in a national research database of infants with liver disease. Samples of blood will be obtained for later research analysis, whenever possible, at the time of clinically indicated blood draws or when there is IV access for a clinical procedure. All data from this study will be kept in a secure research database at the Scientific Data Coordinating Center (SDCC) and transferred to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) data repository after the study ends.
- Conditions
- Biliary Atresia
- Medical subject headings
- Biliary Atresia
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 |
|---|---|---|
| Biliary Atresia | Infants presenting with cholestasis who are diagnosed with biliary atresia. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in disease severity over time (disease progression)
disease progression defined by transplant date, date of death, worsening liver function, and complications related to worsening liver function
Measured: Measured at baseline, 1 month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, annually through year 10 and then biannually through year 20.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2003 (estimated)
- Study start21 Apr 2004
- Primary completion31 May 2029 (estimated)
- Study completion31 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Biliary AtresiaStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arbor Research Collaborative for Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Saul Karpen, MD, PhD, Study chair, VCU School of MedicineEd Doo, MD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)John Magee, MD, Principal investigator, University of Michigan Medical Center, Ann ArborLisa Henn, PhD, Principal investigator, Arbor Research Collaborative for HealthKatrina Loh, MD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00061828
- Sponsor's protocol ID
- PROBE Study - ChiLDReN Network
- Other IDs
- U01DK103149, U01DK103140, U01DK103135, U01DK084575, U01DK084538, U01DK084536, U01DK062503, U01DK062500
- Acronym
- PROBE
- Status last verified
- Jun 2026
- First submitted
- 5 Jun 2003
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The data will be transferred to NIDDK at the end of the study.
Publications
Tessier MEM, Schraw JM, Beer S, Harpavat S, Kyle Jensen M, Magee JC, Ng V, Scheurer ME, Taylor SA, Shneider BL. The association of human milk intake and outcomes in biliary atresia. J Pediatr Gastroenterol Nutr. 2025 Jan;80(1):163-173. doi: 10.1002/jpn3.12403. Epub 2024 Nov 11.
PubMed 39526563Teckman J, Rosenthal P, Ignacio RV, Spino C, Bass LM, Horslen S, Wang K, Magee JC, Karpen S, Asai A, Molleston JP, Squires RH, Kamath BM, Guthery SL, Loomes KM, Shneider BL, Sokol RJ; ChiLDReN (Childhood Liver Disease Research Network). Neonatal cholestasis in children with Alpha-1-AT deficiency is a risk for earlier severe liver disease with male predominance. Hepatol Commun. 2023 Dec 7;7(12):e0345. doi: 10.1097/HC9.0000000000000345. eCollection 2023 Dec 1.
PubMed 38055647Kemme S, Canniff JD, Feldman AG, Garth KM, Li S, Pan Z, Sokol RJ, Weinberg A, Mack CL. Cytomegalovirus in biliary atresia is associated with increased pretransplant death, but not decreased native liver survival. Hepatol Commun. 2023 Jul 17;7(8):e0175. doi: 10.1097/HC9.0000000000000175. eCollection 2023 Aug 1.
PubMed 37471052Hertel PM, Hawthorne K, Kim S, Finegold MJ, Shneider BL, Squires JE, Gupta NA, Bull LN, Murray KF, Kerkar N, Ng VL, Molleston JP, Bezerra JA, Loomes KM, Taylor SA, Schwarz KB, Turmelle YP, Rosenthal P, Magee JC, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Presentation and Outcomes of Infants With Idiopathic Cholestasis: A Multicenter Prospective Study. J Pediatr Gastroenterol Nutr. 2021 Oct 1;73(4):478-484. doi: 10.1097/MPG.0000000000003248.
PubMed 34310436Ng VL, Sorensen LG, Alonso EM, Fredericks EM, Ye W, Moore J, Karpen SJ, Shneider BL, Molleston JP, Bezerra JA, Murray KF, Loomes KM, Rosenthal P, Squires RH, Wang K, Arnon R, Schwarz KB, Turmelle YP, Haber BH, Sherker AH, Magee JC, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Neurodevelopmental Outcome of Young Children with Biliary Atresia and Native Liver: Results from the ChiLDReN Study. J Pediatr. 2018 May;196:139-147.e3. doi: 10.1016/j.jpeds.2017.12.048. Epub 2018 Mar 5.
PubMed 29519540Shneider BL, Magee JC, Karpen SJ, Rand EB, Narkewicz MR, Bass LM, Schwarz K, Whitington PF, Bezerra JA, Kerkar N, Haber B, Rosenthal P, Turmelle YP, Molleston JP, Murray KF, Ng VL, Wang KS, Romero R, Squires RH, Arnon R, Sherker AH, Moore J, Ye W, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Total Serum Bilirubin within 3 Months of Hepatoportoenterostomy Predicts Short-Term Outcomes in Biliary Atresia. J Pediatr. 2016 Mar;170:211-7.e1-2. doi: 10.1016/j.jpeds.2015.11.058. Epub 2015 Dec 24.
PubMed 26725209Ye W, Rosenthal P, Magee JC, Whitington PF; Childhood Liver Disease Research and Education Network. Factors Determining delta-Bilirubin Levels in Infants With Biliary Atresia. J Pediatr Gastroenterol Nutr. 2015 May;60(5):659-63. doi: 10.1097/MPG.0000000000000690.
PubMed 25564820Bessho K, Mourya R, Shivakumar P, Walters S, Magee JC, Rao M, Jegga AG, Bezerra JA. Gene expression signature for biliary atresia and a role for interleukin-8 in pathogenesis of experimental disease. Hepatology. 2014 Jul;60(1):211-23. doi: 10.1002/hep.27045. Epub 2014 May 27.
PubMed 24493287Shneider BL, Abel B, Haber B, Karpen SJ, Magee JC, Romero R, Schwarz K, Bass LM, Kerkar N, Miethke AG, Rosenthal P, Turmelle Y, Robuck PR, Sokol RJ; Childhood Liver Disease Research and Education Network. Portal hypertension in children and young adults with biliary atresia. J Pediatr Gastroenterol Nutr. 2012 Nov;55(5):567-73. doi: 10.1097/MPG.0b013e31826eb0cf.
PubMed 22903006Zahm AM, Hand NJ, Boateng LA, Friedman JR. Circulating microRNA is a biomarker of biliary atresia. J Pediatr Gastroenterol Nutr. 2012 Oct;55(4):366-9. doi: 10.1097/MPG.0b013e318264e648.
PubMed 22732895
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Childhood Liver Disease Research Network (ChiLDReN): A Prospective Database of Infants With Cholestasis
- ClinicalTrials.gov ID
- NCT00061828
- Acronym / sponsor ID
- PROBE / PROBE Study - ChiLDReN Network
- Conditions
- Biliary Atresia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,000 participants (estimated)
- Lead sponsor
- Arbor Research Collaborative for HealthUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- 21 Apr 2004 (actual)
- Primary completion
- 31 May 2029 (estimated)
- Study completion
- 31 May 2029 (estimated)
- First posted
- 6 Jun 2003 (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.
Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period. The long term goal of the Childhood Liver Disease Research Network (ChiLDReN) is to establish a database of clinical information and plasma, serum, and tissue samples from cholestatic children to facilitate research and to perform clinical, epidemiological and therapeutic trials in these important pediatric liver diseases.
Detailed description
This is a multi-center project to establish a prospective database of clinical information and a repository of blood, from children with diagnosis of neonatal liver disease, such as biliary atresia (BA), in order to perform research in this important liver problem. Children (diagnosed with BA or suspicious for BA) will be screened and enrolled at presentation at the participating pediatric liver sites. Participants diagnosed with BA will be followed intensively for the first year, at 18 months of age, and then annually up to 10 years of age, and then biannually, or liver transplantation. Other participants (Non-BA) diagnosed with cholestasis will be exited from the study at the time of diagnosis determination.
Detailed clinical data, laboratory investigations, liver and biliary specimens, and long-term follow-up of outcomes are part of the normal standard of care with respect to the diagnosis and treatment of the subjects with liver problems. This research involves the collection of diagnostic, clinical and outcome data concerning the subject, which is kept without identification (coded) in a national research database of infants with liver disease. Samples of blood will be obtained for later research analysis, whenever possible, at the time of clinically indicated blood draws or when there is IV access for a clinical procedure. All data from this study will be kept in a secure research database at the Scientific Data Coordinating Center (SDCC) and transferred to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) data repository after the study ends.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Biliary Atresia | – | –Infants presenting with cholestasis who are diagnosed with biliary atresia. |
Who may qualify
- Ages
- Up to 6 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- This study population will be selected from the patient base of participating specialty care clinics.
Key inclusion criteria (3 listed)
- Infant's age less than or equal to 180 days at initial presentation at the ChiLDReN clinical site.
- Diagnosis of cholestasis defined by serum direct or conjugated bilirubin greater than or equal to 2 mg/dl and suspected biliary atresia.
- The subject's parent(s)/guardian(s) willing to provide informed written consent.
Key exclusion criteria (9 listed)
- Acute liver failure.
- Previous hepatobiliary surgery with dissection or excision of biliary tissue.
- Diagnoses of bacterial or fungal sepsis (except where associated with metabolic liver disease)
- Diagnoses of hypoxia, shock or ischemic hepatopathy within the past two weeks (If the cholestasis persists beyond two weeks of the initiating event, the infant can be enrolled).
- Diagnosis of any malignancy.
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
Change in disease severity over time (disease progression)
Time frame: Measured at baseline, 1 month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, annually through year 10 and then biannually through year 20.
disease progression defined by transplant date, date of death, worsening liver function, and complications related to worsening liver function
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
- Derived from this study: PubMed 39526563Tessier MEM, Schraw JM, Beer S, Harpavat S, Kyle Jensen M, Magee JC, Ng V, Scheurer ME, Taylor SA, Shneider BL. The association of human milk intake and outcomes in biliary atresia. J Pediatr Gastroenterol Nutr. 2025 Jan;80(1):163-173. doi: 10.1002/jpn3.12403. Epub 2024 Nov 11.
- Derived from this study: PubMed 38055647Teckman J, Rosenthal P, Ignacio RV, Spino C, Bass LM, Horslen S, Wang K, Magee JC, Karpen S, Asai A, Molleston JP, Squires RH, Kamath BM, Guthery SL, Loomes KM, Shneider BL, Sokol RJ; ChiLDReN (Childhood Liver Disease Research Network). Neonatal cholestasis in children with Alpha-1-AT deficiency is a risk for earlier severe liver disease with male predominance. Hepatol Commun. 2023 Dec 7;7(12):e0345. doi: 10.1097/HC9.0000000000000345. eCollection 2023 Dec 1.
- Derived from this study: PubMed 37471052Kemme S, Canniff JD, Feldman AG, Garth KM, Li S, Pan Z, Sokol RJ, Weinberg A, Mack CL. Cytomegalovirus in biliary atresia is associated with increased pretransplant death, but not decreased native liver survival. Hepatol Commun. 2023 Jul 17;7(8):e0175. doi: 10.1097/HC9.0000000000000175. eCollection 2023 Aug 1.
- Derived from this study: PubMed 34310436Hertel PM, Hawthorne K, Kim S, Finegold MJ, Shneider BL, Squires JE, Gupta NA, Bull LN, Murray KF, Kerkar N, Ng VL, Molleston JP, Bezerra JA, Loomes KM, Taylor SA, Schwarz KB, Turmelle YP, Rosenthal P, Magee JC, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Presentation and Outcomes of Infants With Idiopathic Cholestasis: A Multicenter Prospective Study. J Pediatr Gastroenterol Nutr. 2021 Oct 1;73(4):478-484. doi: 10.1097/MPG.0000000000003248.
- Derived from this study: PubMed 29519540Ng VL, Sorensen LG, Alonso EM, Fredericks EM, Ye W, Moore J, Karpen SJ, Shneider BL, Molleston JP, Bezerra JA, Murray KF, Loomes KM, Rosenthal P, Squires RH, Wang K, Arnon R, Schwarz KB, Turmelle YP, Haber BH, Sherker AH, Magee JC, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Neurodevelopmental Outcome of Young Children with Biliary Atresia and Native Liver: Results from the ChiLDReN Study. J Pediatr. 2018 May;196:139-147.e3. doi: 10.1016/j.jpeds.2017.12.048. Epub 2018 Mar 5.
- Derived from this study: PubMed 26725209Shneider BL, Magee JC, Karpen SJ, Rand EB, Narkewicz MR, Bass LM, Schwarz K, Whitington PF, Bezerra JA, Kerkar N, Haber B, Rosenthal P, Turmelle YP, Molleston JP, Murray KF, Ng VL, Wang KS, Romero R, Squires RH, Arnon R, Sherker AH, Moore J, Ye W, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Total Serum Bilirubin within 3 Months of Hepatoportoenterostomy Predicts Short-Term Outcomes in Biliary Atresia. J Pediatr. 2016 Mar;170:211-7.e1-2. doi: 10.1016/j.jpeds.2015.11.058. Epub 2015 Dec 24.
- Derived from this study: PubMed 25564820Ye W, Rosenthal P, Magee JC, Whitington PF; Childhood Liver Disease Research and Education Network. Factors Determining delta-Bilirubin Levels in Infants With Biliary Atresia. J Pediatr Gastroenterol Nutr. 2015 May;60(5):659-63. doi: 10.1097/MPG.0000000000000690.
- Derived from this study: PubMed 24493287Bessho K, Mourya R, Shivakumar P, Walters S, Magee JC, Rao M, Jegga AG, Bezerra JA. Gene expression signature for biliary atresia and a role for interleukin-8 in pathogenesis of experimental disease. Hepatology. 2014 Jul;60(1):211-23. doi: 10.1002/hep.27045. Epub 2014 May 27.
- Derived from this study: PubMed 22903006Shneider BL, Abel B, Haber B, Karpen SJ, Magee JC, Romero R, Schwarz K, Bass LM, Kerkar N, Miethke AG, Rosenthal P, Turmelle Y, Robuck PR, Sokol RJ; Childhood Liver Disease Research and Education Network. Portal hypertension in children and young adults with biliary atresia. J Pediatr Gastroenterol Nutr. 2012 Nov;55(5):567-73. doi: 10.1097/MPG.0b013e31826eb0cf.
- Derived from this study: PubMed 22732895Zahm AM, Hand NJ, Boateng LA, Friedman JR. Circulating microRNA is a biomarker of biliary atresia. J Pediatr Gastroenterol Nutr. 2012 Oct;55(4):366-9. doi: 10.1097/MPG.0b013e318264e648.
- Childhood Liver Disease Research Network (ChiLDReN) websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. The data will be transferred to NIDDK at the end of the study.
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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.
Saul Karpen, MD, PhD study chair
VCU School of Medicine
Ed Doo, MD study director
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
John Magee, MD principal investigator
University of Michigan Medical Center, Ann Arbor
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Biliary Atresia, run by Arbor Research Collaborative for Health at 16 sites in 2 countries.
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 3 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Canada. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Melissa Sexton, BBA, CCRP (734-693-3811, melissa.sexton@arborresearch.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in disease severity over time (disease progression) (time frame: Measured at baseline, 1 month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, annually through year 10 and then biannually through year 20.). 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 NCT00061828, which also lists 10 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 4 Jun 2026.