Hunter SyndromePhase 2/3NCT00630747
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
A Phase 2/3 study testing Idursulfase in people with Hunter Syndrome and Mucopolysaccharidosis II (MPS II), run by Shire at 52 sites in 10 countries.
Trial summary
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study. The primary objective of this extension study was to collect long-term safety and clinical outcome data in Mucopolysaccharidosis II (MPS II), also known as Hunter Syndrome, from the Phase… Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
- What is being tested?
- Idursulfase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 94 participants
- Where
- 52 sitesin 10 countries
- Study dates
- 13 Sep 2004to 31 Jan 2008
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 5 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient must have completed the double-blind phase of Study TKT024, defined as completing the Week 53 final evaluations.
- Patient, patient's parent(s), or legally authorized representative must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.
Who cannot join
- Patient has received treatment with an investigational therapy other than iduronate-2-sulfatase in Study TKT024 within the past 60 days.
- Patient is unable to comply with the protocol (e.g., due to a medical condition such as cervical cord compression or uncooperative attitude) or is unlikely to complete the study, as determined by the investigator.
- Patient has experienced an adverse reaction to study drug in Study TKT024, which contraindicates further treatment with idursulfase.
- Patient with known hypersensitivity to any of the components of idursulfase.
Trial locations and contacts
52 sites in 10 countries.
52 sites in 10 countries
United States16 sites
Children's Hospital Boston
Boston, Massachusetts, 02115
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
The Children's Hospital
Denver, Colorado, 80218
Baylor College of Medicine Texas Children's Hospital
Houston, Texas, 77030
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
Franciscan Skemp Healthcare
La Crosse, Wisconsin, 54601
Comprehensive Cancer Centers of Nevada
Las Vegas, Nevada, 89109
Mid-Illinois Hematology and Oncology Associates
Normal, Illinois, 61761
Pediatric Clinical Research Center, Children's Hospital Oakland
Oakland, California, 94609
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
St. Joseph's Hospital
Phoenix, Arizona, 85013
Harbin Clinic
Rome, Georgia, 30165
University of Utah Hospital
Salt Lake City, Utah, 84113
Saint Louis University Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104
Upstate Medical University, State University of New York (SUNY)
Syracuse, New York, 13210
Brazil8 sites
Clinica Casa de Saude Sao Joao
Barreiras, Estado de Bahia, 47800-000
Hospital Universitario da Faculdade de Medicina da Universidade Federal de Mato Grosso do Sul
Campo Grande, Mato Grosso do Sul, 79008-900
Fundacao Universidade de Ciencias da Saude de Alagoas Governador Lamenha Filho / UNCISAL
Maceió, Alagoas, 57010-382
Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica
Porto Alegre, Rio Grande do Sul, 90035-003
Instituto de Puericultura e Pediatria Martagao Gesteira / Hospital Pediatrico
Rio de Janeiro, Rio de Janeiro, 21941-590
c-HUPES/UFBA
Salvador, Estado de Bahia, 40110-060
UNIFESP Instituto de Oncologia Pediatrica
São Paulo, São Paulo, 04023-062
Instituto da Crianca / Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, São Paulo, 05403-000
United Kingdom8 sites
Bath and NE Somerset Primary Care Trust
Bath, BA1 3QE
Addenbrooke's Hospital
Cambridge, CB2 2QQ
Derbyshire Children's Hospital
Derby, DE22 3NE
Royal Hospital for Sick Children
Glasgow, G3 8SJ
Royal Surrey County Hospital
Guildford, Surrey, GU2 5XX
Great Ormond Street Hospital for Sick Children
London, WC1N3JH
Royal Manchester Children's Hospital
Manchester, M27 4HA
Royal Victoria Infirmary
Newcastle, NE1 4LP
Germany6 sites
Universitatsklinikum Aachen Kinderklinik
Aachen, D-52074
Universitatsklinik Dusseldorf Kinderklinik
Düsseldorf, 40225
Justus-Liebig Universitat
Giessen, 35385
Universitatsklinikum Gottingen
Göttingen, D-37075
Universitatsklinikum Hamburg Eppendorf
Hamburg, 20246
Children's University Hospital Mainz AG
Mainz, 55101
Italy4 sites
Universita Milano Bicocca / Ospedale S. Gerardo
Milan, 20052
Universita degli Studi di Napoli Federico II
Naples, 80131
Universita di Padova
Padova, 35121
Ospedale S. S. Annunziata
Savigliano, 12038
France3 sites
Hopital Edouard Herriot
Lyon, 69003
Hopital de Hautepierre
Strasbourg, 67098
Hospital Ducuing
Toulouse, 31076
Canada2 sites
University of Montreal / Hopital Ste-Justine
Montreal, Quebec, H3T 1C5
The Hospital for Sick Children Research Institute
Toronto, Ontario, M5G 1XB
Spain2 sites
University Hospital Germans Trias i Pujol
Badalona, 08916
Servicio de Pediatria
Linares, Jaen, 23700
Sweden2 sites
Drottning Silvias Barnsjukhus
Gothenberg, 41685
Karolinska University Hospital
Stockholm, 14186
About this study
Official title: An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study. The primary objective of this extension study was to collect long-term safety and clinical outcome data in Mucopolysaccharidosis II (MPS II), also known as Hunter Syndrome, from the Phase 2/Phase 3 Study TKT024. All patients enrolling into this study received weekly active treatment with idursulfase, the primary dosing regimen investigated in Study TKT024.
Hunter Syndrome is an X-linked recessive lysosomal storage disease caused by a deficiency of iduronate-2-sulfatase, an enzyme required to catabolize glycosaminoglycans (GAGS) in cells. As a result, GAGs accumulate in the lysosomes leading to cellular engorgement, organomegaly, tissue destruction, and organ system dysfunction. Hunter Syndrome is a rare disease with an estimated incidence of 1 in 162,000 live births.
Read the detailed description
Study TKT024EXT was conducted in 2 phases. The first phase ("Phase I") was 2 years (104 weeks) in duration and consisted of weekly infusions of IV idursulfase (0.5 mg/kg), and the collection of patients' safety and clinical outcomes. Week 105 defined the beginning of the second phase of the study. The second phase ("Phase II") consisted of weekly infusions of IV idursulfase (0.5 mg/kg) and the monitoring of patients for safety (via collection of adverse events, concomitant medications, and vital signs). Study completion was defined as the time a patient either transitioned to commercially available idursulfase or discontinued this study.
Idursulfase was administered to patients as a continuous IV infusion at a dose of 0.5 mg of protein per kg of body weight (0.5 mg/kg). Final evaluations from Study TKT024, the one-year predecessor Phase 2/Phase 3 registration study, served as the baseline assessments for the TKT024EXT study. Forced vital capacity (FVC) and the 6-minute walk test (6MWT) continued to be the primary clinical outcomes of TKT024EXT study. Efficacy outcomes were evaluated over the course of 2 years and were determined at 4-month intervals during the first year (ie, Weeks 18, 36, and 53) and at 6-month intervals in the second year (ie, Weeks 79 and 105). Safety outcomes were assessed throughout the duration of the study. The safety and clinical testing performed in the TKT024EXT study were identical to those performed in the double-blind phase of Study TKT024.
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
- Being tested
- Idursulfase
- Keywords
- Hunter syndrome, hunters syndrome, hunter's syndrome, hunter disease, hunters disease, hunter's disease, MPS II, MPSII, MPS2, MPS 2, mucopolysaccharides, lysosomal storage disease, lysosomal storage disorder, chronic ear infection, enlarged adenoids, mps symptoms, mps diagnosis, mps ii therapy, MPS II treatment, ert treatment
- Medical subject headings
- Mucopolysaccharidosis II, Sudden Infant Death, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IdursulfaseBiological
Also called Elaprase®, iduronate-2-sulfatase
Solution for intravenous infusion, 0.5 mg/kg once-weekly
| Study group | Role | What they receive |
|---|---|---|
| Idursulfase | Experimental | Not described Receives: Biological: Idursulfase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105
Determined by spirometry. The change is calculated as Week 105 minus baseline.
Measured: Baseline and at Week 105
Change From Baseline in Mean Distance Walked in the 6-minute Walk Test (6MWT) at Week 105
Determined on a walking course. The change was calculated as Week 105 minus baseline.
Measured: Baseline and at Week 105
Secondary outcomes (4)
Change From Baseline in Mean Passive Joint Range of Motion (JROM) at Week 105
Change was calculated as Week 105 minus baseline. Global JROM (% normal range of motion) is the average of 11 ratios multiplied by 100. Ratios are Left/Right means of passive range of motion in Shoulder (Flexion/Extension, Abduction, Internal/External Rotation), Elbow (Flexion/Extension), Wrist (Flexion/Extension), Index Finger (Flexion/Extension [Combined Metacarpophalangeal joint (MCP), Proximal interphalangeal joint (PIP), Distal interphalangeal joint (DIP) motion]), Hip (Flexion/Extension, Abduction, Internal/External Rotation), Knee (Flexion/Extension), and Ankle (Dorsiflexion) divided by the normal range (American Academy of Orthopedic Surgeons and American Medical Association).
Measured: Baseline and at Week 105
Change From Baseline in Mean Combined Liver and Spleen Volume at Week 105
Determined by Magnetic Resonance Imaging (MRI). The change was calculated as Week 105 minus baseline.
Measured: Baseline and at Week 105
Change From Baseline in Mean Normalized Urine Glycosaminoglycans (GAG) Levels at Week 105
Determined by urine testing. The change was calculated as Week 105 minus baseline.
Measured: Baseline and at Week 105
Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 105
Determined by echocardiogram. LVMI indexed to body surface area (g/m^2). The change was calculated as Week 105 minus baseline.
Measured: Baseline and at Week 105
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2008 (estimated)
- Study start13 Sep 2004
- Primary completion31 Jan 2008
- Study completion31 Jan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Hunter SyndromeStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00630747
- Sponsor's protocol ID
- TKT024EXT
- Other IDs
- 2004-002743-27, 2004
- Status last verified
- May 2021
- First submitted
- 28 Feb 2008
- Last updated
- 10 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab. 2007 Mar;90(3):329-37. doi: 10.1016/j.ymgme.2006.09.001. Epub 2006 Dec 20.
PubMed 17185020Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Puga AC, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Kimura A. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med. 2006 Aug;8(8):465-73. doi: 10.1097/01.gim.0000232477.37660.fb.
PubMed 16912578Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, Ramaswami U, Vellodi A, Wraith JE, Cleary M, Gucsavas-Calikoglu M, Puga AC, Shinawi M, Ulbrich B, Vijayaraghavan S, Wendt S, Conway AM, Rossi A, Whiteman DA, Kimura A. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genet Med. 2011 Feb;13(2):95-101. doi: 10.1097/GIM.0b013e3181fea459.
PubMed 21150784Beusterien KM, Yeung JE, Pang F, Brazier J. Development of the multi-attribute Adolescent Health Utility Measure (AHUM). Health Qual Life Outcomes. 2012 Aug 28;10:102. doi: 10.1186/1477-7525-10-102.
PubMed 22929184
Study at a glance
From the registry record, last updated by the sponsor on 10 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT00630747
- Acronym / sponsor ID
- TKT024EXT
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
- Keywords
- Hunter syndrome, hunters syndrome, hunter's syndrome, hunter disease, hunters disease, hunter's disease, MPS II, MPSII
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 13 Sep 2004 (actual)
- Primary completion
- 31 Jan 2008 (actual)
- Study completion
- 31 Jan 2008 (actual)
- First posted
- 7 Mar 2008 (estimated)
- Results
- Posted 17 Mar 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study. The primary objective of this extension study was to collect long-term safety and clinical outcome data in Mucopolysaccharidosis II (MPS II), also known as Hunter Syndrome, from the Phase 2/Phase 3 Study TKT024. All patients enrolling into this study received weekly active treatment with idursulfase, the primary dosing regimen investigated in Study TKT024.
Hunter Syndrome is an X-linked recessive lysosomal storage disease caused by a deficiency of iduronate-2-sulfatase, an enzyme required to catabolize glycosaminoglycans (GAGS) in cells. As a result, GAGs accumulate in the lysosomes leading to cellular engorgement, organomegaly, tissue destruction, and organ system dysfunction. Hunter Syndrome is a rare disease with an estimated incidence of 1 in 162,000 live births.
Detailed description
Study TKT024EXT was conducted in 2 phases. The first phase ("Phase I") was 2 years (104 weeks) in duration and consisted of weekly infusions of IV idursulfase (0.5 mg/kg), and the collection of patients' safety and clinical outcomes. Week 105 defined the beginning of the second phase of the study. The second phase ("Phase II") consisted of weekly infusions of IV idursulfase (0.5 mg/kg) and the monitoring of patients for safety (via collection of adverse events, concomitant medications, and vital signs). Study completion was defined as the time a patient either transitioned to commercially available idursulfase or discontinued this study.
Idursulfase was administered to patients as a continuous IV infusion at a dose of 0.5 mg of protein per kg of body weight (0.5 mg/kg). Final evaluations from Study TKT024, the one-year predecessor Phase 2/Phase 3 registration study, served as the baseline assessments for the TKT024EXT study. Forced vital capacity (FVC) and the 6-minute walk test (6MWT) continued to be the primary clinical outcomes of TKT024EXT study. Efficacy outcomes were evaluated over the course of 2 years and were determined at 4-month intervals during the first year (ie, Weeks 18, 36, and 53) and at 6-month intervals in the second year (ie, Weeks 79 and 105). Safety outcomes were assessed throughout the duration of the study. The safety and clinical testing performed in the TKT024EXT study were identical to those performed in the double-blind phase of Study TKT024.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Idursulfase | Experimental | Biological: Idursulfase |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Patient must have completed the double-blind phase of Study TKT024, defined as completing the Week 53 final evaluations.
- Patient, patient's parent(s), or legally authorized representative must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.
Key exclusion criteria (4 listed)
- Patient has received treatment with an investigational therapy other than iduronate-2-sulfatase in Study TKT024 within the past 60 days.
- Patient is unable to comply with the protocol (e.g., due to a medical condition such as cervical cord compression or uncooperative attitude) or is unlikely to complete the study, as determined by the investigator.
- Patient has experienced an adverse reaction to study drug in Study TKT024, which contraindicates further treatment with idursulfase.
- Patient with known hypersensitivity to any of the components of idursulfase.
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 |
|---|---|---|
| IdursulfaseAlso: Elaprase®, iduronate-2-sulfataseSolution for intravenous infusion, 0.5 mg/kg once-weekly | Biological | Idursulfase |
Primary outcome
Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105
Time frame: Baseline and at Week 105
Determined by spirometry. The change is calculated as Week 105 minus baseline.
Change From Baseline in Mean Distance Walked in the 6-minute Walk Test (6MWT) at Week 105
Time frame: Baseline and at Week 105
Determined on a walking course. The change was calculated as Week 105 minus baseline.
4 secondary outcomes
Change From Baseline in Mean Passive Joint Range of Motion (JROM) at Week 105
Time frame: Baseline and at Week 105
Change From Baseline in Mean Combined Liver and Spleen Volume at Week 105
Time frame: Baseline and at Week 105
Change From Baseline in Mean Normalized Urine Glycosaminoglycans (GAG) Levels at Week 105
Time frame: Baseline and at Week 105
Change From Baseline in Mean Cardiac Left Ventricular Mass Index (LVMI) at Week 105
Time frame: Baseline and at Week 105
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jun 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 17185020Muenzer J, Gucsavas-Calikoglu M, McCandless SE, Schuetz TJ, Kimura A. A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab. 2007 Mar;90(3):329-37. doi: 10.1016/j.ymgme.2006.09.001. Epub 2006 Dec 20.
- Background: PubMed 16912578Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Puga AC, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Kimura A. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med. 2006 Aug;8(8):465-73. doi: 10.1097/01.gim.0000232477.37660.fb.
- Results publication: PubMed 21150784Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, Ramaswami U, Vellodi A, Wraith JE, Cleary M, Gucsavas-Calikoglu M, Puga AC, Shinawi M, Ulbrich B, Vijayaraghavan S, Wendt S, Conway AM, Rossi A, Whiteman DA, Kimura A. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genet Med. 2011 Feb;13(2):95-101. doi: 10.1097/GIM.0b013e3181fea459.
- Derived from this study: PubMed 22929184Beusterien KM, Yeung JE, Pang F, Brazier J. Development of the multi-attribute Adolescent Health Utility Measure (AHUM). Health Qual Life Outcomes. 2012 Aug 28;10:102. doi: 10.1186/1477-7525-10-102.
Related trials for Hunter Syndrome
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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
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An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
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A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
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A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
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Active, not recruitingPhase 2/3
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Mucopolysaccharidosis II
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.
Study Director study director
Takeda
52 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Idursulfase in people with Hunter Syndrome and Mucopolysaccharidosis II (MPS II), run by Shire at 52 sites in 10 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 2 inclusion and 4 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: finished, as of the sponsor's last update on 10 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 52 sites in United States, Brazil, United Kingdom, Germany, Italy and 5 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?
Yes. Results are posted in the registry (first posted 17 Mar 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change From Baseline in Mean Percent Predicted Forced Vital Capacity (FVC) at Week 105 (time frame: Baseline and at Week 105). The study lists 2 primary outcomes in all. 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 NCT00630747, which also lists 4 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 10 Jun 2021.