Secondary (AA) AmyloidosisPhase 2/3NCT00035334
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
A Phase 2/3 study testing NC-503 (Anti-amyloidotic (AA) Agent) in people with Secondary (AA) Amyloidosis and Rheumatoid Arthritis, run by Bellus Health Inc. - a GSK company at 26 sites in 12 countries.
Trial summary
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and gastrointestinal functions. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome
- What is being tested?
- NC-503 (Anti-amyloidotic (AA) Agent)
- How many people
- 150 participants
- Where
- 26 sitesin 12 countries
- Study dates
- Oct 2001to Dec 2004
- Phase
- Phase 2/3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
PROTOCOL INCLUSION CRITERIA
- Patients must be 18 years of age or older.
- Males and females. If women of childbearing potential (i.e., not surgically sterilized or post-menopausal greater than one year) the patient must be using effective birth control.
- Diagnosis of AA amyloidosis demonstrated by positive biopsy (Congo red staining) and immunohistochemistry or immunoelectron microscopy at screening visit. Tissue from previous biopsy can be used for confirmation of diagnosis, if available.
- Persistent proteinuria defined as urinary protein excretion ? 1g/24h in two distinct 24-h urine collections at least 1 week apart within 3 months prior to study entry (baseline, Month 0 visit) without evidence of urinary tract infection or overt heart failure (NYHA class III or more); OR creatinine clearance ? 60 mL/min in two distinct measures at least 1 week apart within 3 months prior to study entry (baseline, Month 0 visit).
- Creatinine clearance ? 20 mL/min AND serum creatinine ? 3 mg/dl within 3 months prior to study entry (baseline, Month 0 visit).
- Written informed consent.
PROTOCOL EXCLUSION CRITERIA
- Evidence or suspicion of renal or renovascular diseases other than renal AA amyloidosis.
- Presence of diabetes mellitus (Type I and II).
- Evidence of a cause of potentially reversible reduced renal function, such as accelerated hypertension or drug nephrotoxicity.
- AST, ALT, or ALP > 5 times the upper limit of normal, or total bilirubin 50% above upper limits of normal.
- Presence of any other clinically significant diseases that could interfere with the interpretation of study results or compromise patient safety or any conditions that could reduce life expectancy to less than two years.
- Use of an investigational drug within thirty days prior to the screening visit.
- Active alcohol and/or drug abuse.
- Initiation of or any changes in ACE inhibitor therapy within 3 months prior to the screening visit.
- Initiation of or any changes in cytotoxic agents/colchicine therapy within 3 months prior to the screening visit.
- Inability to provide legal consent.
Trial locations and contacts
26 sites in 12 countries.
26 sites in 12 countries
United States4 sites
Boston Medical Center, Renal Division
Boston, Massachusetts, 02118
Indiana University School of Medicine, Department of Pathology and Laboratory Medicine,
Indianapolis, Indiana, 46202
Mount Sinai Medical Center
New York, New York, 10029
Mayo Clinic
Rochester, Minnesota, 55905
Spain4 sites
Hospital Universitario Germans Trias I Pujol, Servicio de Reumatologia
Badalona, 08916
Hospital Clinic I Provincial de Barcelona, Jefe del Departamento de Reumatologia
Barcelona, 08036
Ciutad Sanitària y Universitària de Bellvitge, Servicio de Reumatologia, Hospitalet de Llobregat
Llobregat, 08907
Hospital Clinico San Carlos de Madrid, Servicio de Reumatologia
Madrid, 28040
France3 sites
Centre Hospitalier du Mans, Service de Rhumatologie
Le Mans, CEDEX 1
Hôpital Claude Huriez, Service de médecine Interne, Clinique Médicale A
Lille, CEDEX 59037
Hôpital Cochin, Centre de Recherche et d'Explorations Fonctionnelles
Paris, 75679 CEDEX 14
Turkey (Türkiye)3 sites
Cerrehpasa Tip Fakultesi
Askaray, Istanbul, Turkey
Istanbul Faculty of Medicine, Department of Internal Medicine, Division of Rheumatology
Istanbul, 34390 CAPA
Marmara University Medical School Hospital, Department of Rheumatology
Uskudar, Altunizade, Istanbul, 81190
Israel2 sites
Bnai Zion Medical Center
Haifa, 31048
Heller Institute of Medical Research, Sheba Medical Center
Tel HaShomer, 52621
Poland2 sites
Instytut Reumatologiczny
Warsaw, 02-632
Okregowy Szpital Kolejowy, Zaklad Reumatologii
Wroclaw, 53-137
Russia2 sites
Institute of Rheumatology RAMS
Moscow, 115522
Regional Hospital No. 1
Yekaterinburg, 320102
United Kingdom2 sites
Royal Free and University College Medical School, Department of Medicine, National Amyloidosis Centre
London, NW3 2PF
Gartnavel General Hospital
Scotland, G12 0YN
Italy1 site
Italian Group for Systemic Amyloidosis, Biotechnology Research Laboratories, IRCCS Policlinico San Matteo, Internal Medicine and Medical Oncology
Pavia, 27100
Netherlands1 site
University Hospital Groningen, Department of Medicine, Division of Rheumatology
Groningen, 9700 RB
About this study
Official title: A Phase II/III Study of the Safety and Efficacy of NC-503 in Patients Suffering From Secondary (AA) Amyloidosis
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and gastrointestinal functions.
Read the detailed description
AA amyloidosis is associated with chronic inflammatory conditions (rheumatoid arthritis, ankylosing spondylitis, inflammatory bowel disease), chronic infection (tuberculosis, osteomyelitis), and Familial Mediterranean Fever. Rheumatoid arthritis is the major cause of AA amyloidosis in Western Europe and North America. The most common clinical feature of AA amyloidosis is renal dysfunction manifested as nephrotic-range proteinuria or renal insufficiency at the time of diagnosis. End-stage renal failure is the cause of death in 40-60% of cases. Gastrointestinal involvement is also frequent and is usually manifested as chronic diarrhea, body weight loss and malabsorption. Enlargement of the liver and spleen may also occur in some patients. The median survival time from diagnosis varies from 2 to 8 years depending on the stage of the disease at time of diagnosis. The goal of the current therapy in AA amyloidosis is the control of the associated disease. However, the current approaches for the treatment of AA amyloidosis are unspecific, toxic, invasive, and not sufficiently effective in many cases. NC-503 was specifically designed to compete with the naturally occurring sulfated GAGs for the binding to amyloidogenic precursor proteins, and to inhibit amyloid deposition into tissues. The proposed therapy with NC-503 is based on the prevention of the amyloid fibril formation. The objective of this clinical phase II/III study is to determine the efficacy and safety of NC-503 compared to a placebo in patients suffering from secondary (AA) amyloidosis by the assessment of clinical improvement/ worsening of both renal and gastrointestinal functions.
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome, Kidney Diseases, Gastrointestinal Diseases
- Being tested
- NC-503 (Anti-amyloidotic (AA) Agent)
- Keywords
- Familial Mediterranean Fever, Amyloidosis, Secondary (AA) Amyloidosis, Nephrotic Syndrome
- Medical subject headings
- Neoplasm Metastasis, Amyloidosis, Arthritis, Rheumatoid, Nephrotic Syndrome, Kidney Diseases, Gastrointestinal Diseases, Familial Mediterranean Fever
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NC-503 (Anti-amyloidotic (AA) Agent)Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2002 (estimated)
- Study startOct 2001
- Study completionDec 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Secondary (AA) AmyloidosisStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Enrolment
- 150 participants
Sponsor and investigators
- Lead sponsor
- Bellus Health Inc. - a GSK company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- FDA Office of Orphan Products Development
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00035334
- Sponsor's protocol ID
- CL-503004
- Status last verified
- Feb 2006
- First submitted
- 2 May 2002
- Last updated
- 14 Feb 2006
- Results posted
- No
Publications
Safety, Tolerability and Pharmacokinetic Profile of FibrillexTM (Anti-AA Amyloid Agent) in Healthy and Renal Impaired Subjects. Garceau D., Gurbindo C., Laurin J. Neurochem Inc. Reference: Proceedings from the IXth International Symposium on Amyloidosis , 2001 (Budapest, Hungary)
Dember LM, Hawkins PN, Hazenberg BP, Gorevic PD, Merlini G, Butrimiene I, Livneh A, Lesnyak O, Puechal X, Lachmann HJ, Obici L, Balshaw R, Garceau D, Hauck W, Skinner M; Eprodisate for AA Amyloidosis Trial Group. Eprodisate for the treatment of renal disease in AA amyloidosis. N Engl J Med. 2007 Jun 7;356(23):2349-60. doi: 10.1056/NEJMoa065644.
PubMed 17554116
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2006. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II/III Study of the Safety and Efficacy of NC-503 in Patients Suffering From Secondary (AA) Amyloidosis
- ClinicalTrials.gov ID
- NCT00035334
- Acronym / sponsor ID
- CL-503004
- Conditions
- Secondary (AA) Amyloidosis, Rheumatoid Arthritis, Nephrotic Syndrome, Familial Mediterranean Syndrome, Kidney Diseases, Gastrointestinal Diseases
- Keywords
- Familial Mediterranean Fever, Amyloidosis, Secondary (AA) Amyloidosis, Nephrotic Syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 150 participants
- Lead sponsor
- Bellus Health Inc. - a GSK companyIndustry (pharma, biotech, devices)
- Collaborators
- FDA Office of Orphan Products Development
- Study start
- Oct 2001
- Study completion
- Dec 2004
- First posted
- 3 May 2002 (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.
The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and gastrointestinal functions.
Detailed description
AA amyloidosis is associated with chronic inflammatory conditions (rheumatoid arthritis, ankylosing spondylitis, inflammatory bowel disease), chronic infection (tuberculosis, osteomyelitis), and Familial Mediterranean Fever. Rheumatoid arthritis is the major cause of AA amyloidosis in Western Europe and North America. The most common clinical feature of AA amyloidosis is renal dysfunction manifested as nephrotic-range proteinuria or renal insufficiency at the time of diagnosis. End-stage renal failure is the cause of death in 40-60% of cases. Gastrointestinal involvement is also frequent and is usually manifested as chronic diarrhea, body weight loss and malabsorption. Enlargement of the liver and spleen may also occur in some patients. The median survival time from diagnosis varies from 2 to 8 years depending on the stage of the disease at time of diagnosis. The goal of the current therapy in AA amyloidosis is the control of the associated disease. However, the current approaches for the treatment of AA amyloidosis are unspecific, toxic, invasive, and not sufficiently effective in many cases. NC-503 was specifically designed to compete with the naturally occurring sulfated GAGs for the binding to amyloidogenic precursor proteins, and to inhibit amyloid deposition into tissues. The proposed therapy with NC-503 is based on the prevention of the amyloid fibril formation. The objective of this clinical phase II/III study is to determine the efficacy and safety of NC-503 compared to a placebo in patients suffering from secondary (AA) amyloidosis by the assessment of clinical improvement/ worsening of both renal and gastrointestinal functions.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| NC-503 (Anti-amyloidotic (AA) Agent) | Drug | – |
Primary outcome
Not reported in the source record.
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 14 Feb 2006.
- BackgroundSafety, Tolerability and Pharmacokinetic Profile of FibrillexTM (Anti-AA Amyloid Agent) in Healthy and Renal Impaired Subjects. Garceau D., Gurbindo C., Laurin J. Neurochem Inc. Reference: Proceedings from the IXth International Symposium on Amyloidosis , 2001 (Budapest, Hungary)
- Derived from this study: PubMed 17554116Dember LM, Hawkins PN, Hazenberg BP, Gorevic PD, Merlini G, Butrimiene I, Livneh A, Lesnyak O, Puechal X, Lachmann HJ, Obici L, Balshaw R, Garceau D, Hauck W, Skinner M; Eprodisate for AA Amyloidosis Trial Group. Eprodisate for the treatment of renal disease in AA amyloidosis. N Engl J Med. 2007 Jun 7;356(23):2349-60. doi: 10.1056/NEJMoa065644.
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.
The record lists no contact. Use the official record to reach the sponsor.
26 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing NC-503 (Anti-amyloidotic (AA) Agent) in people with Secondary (AA) Amyloidosis and Rheumatoid Arthritis, run by Bellus Health Inc. - a GSK company at 26 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 14 Feb 2006. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in United States, Spain, France, Turkey (Türkiye), Israel and 7 more countries. 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.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00035334, which also lists 2 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 14 Feb 2006.