Duchenne Muscular DystrophyPhase 3TAMDMDNCT03354039
Tamoxifen in Duchenne Muscular Dystrophy
A Phase 3 study testing Tamoxifen in people with Duchenne Muscular Dystrophy, run by University Hospital, Basel, Switzerland at 11 sites in 6 countries.
Trial summary
A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne's muscular dystrophy (DMD) patients that are under stable standard treatment of care with glucocorticoids. Read more about this study
- Who is it for?
- Ages 78 months to 16 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Tamoxifen
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 93 participants
- Where
- 11 sitesin 6 countries
- Study dates
- 12 Jun 2018to 18 Oct 2022
- Phase
- Phase 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 78 months to 16 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Group A (ambulant patients)
- Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
- Stable treatment with glucocorticoids >6 months (no significant change in dosage (>0.2mg/kg)) at screening; dosing adaptations according to weight change are allowed
- Male gender
- 6.5 to 12 years of age at time of screening
- weight >20kg
Show all 20Show fewer
- ambulant patients
- able to walk at least 350 meters in 6 minute walking distance test without assistance at screening
- MFM D1 subdomain of the MFM scale >40% at screening
- Ability to provide informed consent and to comply with study requirements
- Patients harbouring a nonsense mutation treatable with the approved drug ataluren should be under stable ataluren treatment for at least 3 months or in case of nontolerance being off ataluren treatment for at least 3 months before screening
- Group B (non-ambulant patients)
- Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
- Not using glucocorticoids for >6 months
- Male gender
- Non-ambulant patients (walking distance less than 10 meters)
- 10 to 16 years of age at time of screening
- Ability to provide informed consent and to comply with study requirements
- Open label extension
- Recent participation and completion of TAMDMD study
Who cannot join
- Known individual hypersensitivity or allergy to tamoxifen or other ingredients/excipients of IMP
- Female gender
- Use of tamoxifen or testosterone within the last 3 months
- Known or suspected malignancy
- Other chronic disease or clinically relevant limitation of renal, liver or heart function
- Known or suspected non-compliance
Show all 21Show fewer
- Any injury which may impact functional testing, e.g. upper or lower limb fracture
- Planned or expected spinal fusion surgery during the study period (as judged by the Investigator; i.e. due to rapid progressing scoliosis), previous spinal fusion surgery is allowed if it took place more than 6 months prior to screening.
- Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders of the participant/parents (as judged by the investigator)
- Concomitant participation in any other interventional trial (and up to 3 months prior to screening)
- Use of CYP2D6 inhibitors or of CYP3A4 inducers (apart from glucocorticoids), platelet aggregation inhibitors and coumarin-type anti-coagulants
- Use of drugs metabolized by CYP2C9, such as phenprocoumon, phenytoin, warfarin, celecoxib, fluvastatin, ginko biloba, St. John's wort and sulfamethoxazol
- Galactosemia (lack of galactose-1-phosphat-uridylyltransferase or UDP-galactose-4-epimerase or galactokinase; Fanconi-Bickel-syndrome); congenital lack of lactase; glucose-galactose malabsorption
- Presence of one or more of the following eye disorders: cataract, retinopathia, optic neuropathy, alteration of the cornea
- Presence of one or more of the following laboratory abnormalities: anaemia, thrombocytopenia, leukopenia, neutropenia or agranulocytosis
- Group A:
- Glucocorticoid naïve patients
- Start of glucocorticoid treatment or change in dosage <6 month prior to screening (dosing adaptations according to weight change are allowed)
- Group B:
- Glucocorticoid treated patients or patients that stopped glucocorticoid treatment <6 month prior to screening
- Assisted ventilation of any kind necessary
Trial locations and contacts
11 sites in 6 countries.
11 sites in 6 countries
United Kingdom3 sites
Royal Hospital for Children
Glasgow, G51 4TF
The Leeds Teaching Hospitals NHS Trust
Leeds, LS9 7TF
Alder Hey Children's Hospital
Liverpool, L12 2AP
France2 sites
Hôpitaux Raymond Poincaré
Garches, 92380
Hôpital de Hautepierre
Strasbourg, 67098
Germany2 sites
DRK Klinik Berlin Westend
Berlin, 14050
Universitätsklinikum Essen
Essen, 45147
Spain2 sites
Hospital Sant Joan de Déu. UB
Esplugues de Llobregat, Barcelona, 08950
Hospital Universitario Virgen del Rocío
Seville, 41013
About this study
Official title: Tamoxifen in Duchenne Muscular Dystrophy: A Multicenter, Randomised, Double-blind, Placebo-controlled, Phase 3 Safety and Efficacy 48-week Trial
A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne's muscular dystrophy (DMD) patients that are under stable standard treatment of care with glucocorticoids. Furthermore, the investigators plan to include 6-20 non-ambulant patients who do not receive glucocorticoids (as parallel group B), 10 to 16 years old, to obtain efficacy and safety data in a broader DMD population. All patients will receive 20 mg of tamoxifen (TAM) or placebo once daily during 48 weeks.
An open label extension (OLE) trial for participants of the TAMDMD main study will be performed. All TAMDMD patients on TAM or placebo are offered to enter this OLE.
Read the detailed description
This is a 48-week multicentre, parallel, randomised, double-blind, placebo controlled phase 3 safety and efficacy trial. There are two treatment arms: Tamoxifen (verum) and placebo (control), with treatment allocation of 1:1.
The investigators plan to screen at least 79 and to enroll at least 71 ambulant DMD patients aged between 6.5 and 12 years (group A) and 6 - 20 non-ambulant DMD patients aged between 10 and 16 years (group B). In order to reach statistical power, 60 ambulant patients (group A) need to complete the trial. Treatment with 20 mg Tamoxifen once daily will be given for the total trial duration of 48 weeks.
Only patients with glucocorticoids (standard treatment of care) will be included in group A (ambulant patients) and only non-glucocorticoid users in group B. At baseline as well as at the end of the study clinical, laboratory, and MRI measurements will be performed. These include the Motor Function Measure (MFM) scale, timed function tests, the 6 minute walking distance, quantitative muscle testing (QMT) and quantitative thigh muscle MRI, questionnaires. A physical examination, an ECG, vital signs as well as safety laboratory blood analyses will be performed at every visit. Furthermore, an x-ray of the hand and a dual energy x-ray absorptiometry (DEXA)-scan will be performed at baseline and at the end of the study.
An open label extension (OLE) trial for participants of the TAMDMD main study will be performed. All TAMDMD patients on TAM or placebo are offered to enter this OLE. All OLE patients will receive 20 mg of TAM daily during 48 weeks. The same study specific assessments as in the double-blind randomized phase will be performed during the OLE phase
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Tamoxifen, Matching placebo
- Keywords
- DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TamoxifenDrug
DMD patients randomised to verum will receive 20 mg (0.6mg/kg) of Tamoxifen daily. Treatment will be given for the total period of 48 weeks.
Matching placeboDrug
Patients randomised to placebo will be administered matching placebo. Treatment will be given for the total period of 48 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Tamoxifen 20 mg once daily | Experimental | DMD patients randomised to verum will receive 20 mg (0.6mg/kg) of TAM daily. Receives: Drug: Tamoxifen |
| Matching placebo once daily | Placebo comparator | Patients randomised to placebo will be administered matching placebo. Receives: Drug: Matching placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction of disease progression
To test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 6.5-12 years old ambulant DMD patients (Group A) by at least 50% (using the MFM D1 subscore as primary clinical endpoint).
Measured: Baseline to week 48
Secondary outcomes (9)
Muscle function measured by D2 MFM subscore
D2 MFM subscore from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Muscle function measured by D3 MFM subscore
D3 MFM subscore from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Muscle function measured by North Star Ambulatory Assessment
North Star Ambulatory Assessment from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Muscle function measured by proximal upper limb function
Proximal upper limb function from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Muscle function measured by 6 minute walking distance in meter
6 minute walking distance in meter from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Muscle function measured by 10 meter walking time in seconds
10 meter walking time in seconds from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Muscle function measured by time to rise from lying on the floor / supine up in seconds
time to rise from lying on the floor / supine up in seconds from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Muscle force measured by quantitative muscle testing (using Myogrip)
Quantitative muscle testing (using Myogrip) from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Muscle Degeneration measured by MRI
Quantitative muscle MRI including muscle fat fraction (MFF) and T2 times of thigh muscles visualised by MRI from baseline to week 48 under TAM treatment compared to placebo.
Measured: Baseline to week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2017
- Study start12 Jun 2018
- Primary completion25 Jul 2022
- Study completion18 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University Hospital, Basel, SwitzerlandThe sponsor's other registered studies.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 93 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Basel, Switzerland
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dirk Fischer, MD, Principal investigator, University Children's Hospital Basel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03354039
- Sponsor's protocol ID
- TAMDMD
- Acronym
- TAMDMD
- Status last verified
- Dec 2022
- First submitted
- 10 Oct 2017
- Last updated
- 20 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Zwingli G, Putananickal N, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Amthor H, Childs AM, Deconinck N, Horrocks I, Houwen-van Opstal S, Laugel V, Lopez Lobato M, Nascimento Osorio A, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D, Henzi BC. Safety and Efficacy of Tamoxifen in Patients with Duchenne muscular dystrophy: open Label Extension of TAMDMD Trial. Neuromuscul Disord. 2026 Apr;61:106366. doi: 10.1016/j.nmd.2026.106366. Epub 2026 Feb 1.
PubMed 41691937Henzi BC, Putananickal N, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Amthor H, Childs AM, Deconinck N, Horrocks I, Houwen-van Opstal S, Laugel V, Lobato ML, Osorio AN, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Safety and efficacy of tamoxifen in non-ambulant patients with Duchenne muscular dystrophy: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial (TAMDMD Group B). Neuromuscul Disord. 2025 Feb;47:105275. doi: 10.1016/j.nmd.2025.105275. Epub 2025 Jan 16.
PubMed 39879732Henzi BC, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Putananickal N, Stimpson G; North Star Consortium; Amthor H, Childs AM, Deconinck N, de Groot I, Horrocks I, Houwen-van Opstal S, Laugel V, Lopez Lobato M, Madruga Garrido M, Nascimento Osorio A, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2023 Oct;22(10):890-899. doi: 10.1016/S1474-4422(23)00285-5.
PubMed 37739572Nagy S, Hafner P, Schmidt S, Rubino-Nacht D, Schadelin S, Bieri O, Fischer D. Tamoxifen in Duchenne muscular dystrophy (TAMDMD): study protocol for a multicenter, randomized, placebo-controlled, double-blind phase 3 trial. Trials. 2019 Nov 21;20(1):637. doi: 10.1186/s13063-019-3740-6.
PubMed 31752977
Study at a glance
From the registry record, last updated by the sponsor on 20 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tamoxifen in Duchenne Muscular Dystrophy: A Multicenter, Randomised, Double-blind, Placebo-controlled, Phase 3 Safety and Efficacy 48-week Trial
- ClinicalTrials.gov ID
- NCT03354039
- Acronym / sponsor ID
- TAMDMD / TAMDMD
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 93 participants (actual)
- Lead sponsor
- University Hospital, Basel, SwitzerlandUniversity, hospital or other
- Study start
- 12 Jun 2018 (actual)
- Primary completion
- 25 Jul 2022 (actual)
- Study completion
- 18 Oct 2022 (actual)
- First posted
- 27 Nov 2017 (actual)
- 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.
A randomised, double blind, placebo controlled, 48-week clinical trial with a core population (group A) of 79 ambulant 6.5 to 12 years old Duchenne's muscular dystrophy (DMD) patients that are under stable standard treatment of care with glucocorticoids. Furthermore, the investigators plan to include 6-20 non-ambulant patients who do not receive glucocorticoids (as parallel group B), 10 to 16 years old, to obtain efficacy and safety data in a broader DMD population. All patients will receive 20 mg of tamoxifen (TAM) or placebo once daily during 48 weeks.
An open label extension (OLE) trial for participants of the TAMDMD main study will be performed. All TAMDMD patients on TAM or placebo are offered to enter this OLE.
Detailed description
This is a 48-week multicentre, parallel, randomised, double-blind, placebo controlled phase 3 safety and efficacy trial. There are two treatment arms: Tamoxifen (verum) and placebo (control), with treatment allocation of 1:1.
The investigators plan to screen at least 79 and to enroll at least 71 ambulant DMD patients aged between 6.5 and 12 years (group A) and 6 - 20 non-ambulant DMD patients aged between 10 and 16 years (group B). In order to reach statistical power, 60 ambulant patients (group A) need to complete the trial. Treatment with 20 mg Tamoxifen once daily will be given for the total trial duration of 48 weeks.
Only patients with glucocorticoids (standard treatment of care) will be included in group A (ambulant patients) and only non-glucocorticoid users in group B. At baseline as well as at the end of the study clinical, laboratory, and MRI measurements will be performed. These include the Motor Function Measure (MFM) scale, timed function tests, the 6 minute walking distance, quantitative muscle testing (QMT) and quantitative thigh muscle MRI, questionnaires. A physical examination, an ECG, vital signs as well as safety laboratory blood analyses will be performed at every visit. Furthermore, an x-ray of the hand and a dual energy x-ray absorptiometry (DEXA)-scan will be performed at baseline and at the end of the study.
An open label extension (OLE) trial for participants of the TAMDMD main study will be performed. All TAMDMD patients on TAM or placebo are offered to enter this OLE. All OLE patients will receive 20 mg of TAM daily during 48 weeks. The same study specific assessments as in the double-blind randomized phase will be performed during the OLE phase
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Tamoxifen 20 mg once daily | Experimental | Drug: TamoxifenDMD patients randomised to verum will receive 20 mg (0.6mg/kg) of TAM daily. |
| Matching placebo once daily | Placebo comparator | Drug: Matching placeboPatients randomised to placebo will be administered matching placebo. |
Who may qualify
- Ages
- 78 months to 16 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (20 listed)
- Group A (ambulant patients)
- Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
- Stable treatment with glucocorticoids >6 months (no significant change in dosage (>0.2mg/kg)) at screening; dosing adaptations according to weight change are allowed
- Male gender
- 6.5 to 12 years of age at time of screening
Key exclusion criteria (21 listed)
- Known individual hypersensitivity or allergy to tamoxifen or other ingredients/excipients of IMP
- Female gender
- Use of tamoxifen or testosterone within the last 3 months
- Known or suspected malignancy
- Other chronic disease or clinically relevant limitation of renal, liver or heart function
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 |
|---|---|---|
| TamoxifenDMD patients randomised to verum will receive 20 mg (0.6mg/kg) of Tamoxifen daily. Treatment will be given for the total period of 48 weeks. | Drug | Tamoxifen 20 mg once daily |
| Matching placeboPatients randomised to placebo will be administered matching placebo. Treatment will be given for the total period of 48 weeks. | Drug | Matching placebo once daily |
Primary outcome
Reduction of disease progression
Time frame: Baseline to week 48
To test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 6.5-12 years old ambulant DMD patients (Group A) by at least 50% (using the MFM D1 subscore as primary clinical endpoint).
9 secondary outcomes
Muscle function measured by D2 MFM subscore
Time frame: Baseline to week 48
Muscle function measured by D3 MFM subscore
Time frame: Baseline to week 48
Muscle function measured by North Star Ambulatory Assessment
Time frame: Baseline to week 48
Muscle function measured by proximal upper limb function
Time frame: Baseline to week 48
Muscle function measured by 6 minute walking distance in meter
Time frame: Baseline to week 48
Muscle function measured by 10 meter walking time in seconds
Time frame: Baseline to week 48
Muscle function measured by time to rise from lying on the floor / supine up in seconds
Time frame: Baseline to week 48
Muscle force measured by quantitative muscle testing (using Myogrip)
Time frame: Baseline to week 48
Muscle Degeneration measured by MRI
Time frame: Baseline to week 48
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 20 Dec 2022.
- Derived from this study: PubMed 41691937Zwingli G, Putananickal N, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Amthor H, Childs AM, Deconinck N, Horrocks I, Houwen-van Opstal S, Laugel V, Lopez Lobato M, Nascimento Osorio A, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D, Henzi BC. Safety and Efficacy of Tamoxifen in Patients with Duchenne muscular dystrophy: open Label Extension of TAMDMD Trial. Neuromuscul Disord. 2026 Apr;61:106366. doi: 10.1016/j.nmd.2026.106366. Epub 2026 Feb 1.
- Derived from this study: PubMed 39879732Henzi BC, Putananickal N, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Amthor H, Childs AM, Deconinck N, Horrocks I, Houwen-van Opstal S, Laugel V, Lobato ML, Osorio AN, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Safety and efficacy of tamoxifen in non-ambulant patients with Duchenne muscular dystrophy: a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial (TAMDMD Group B). Neuromuscul Disord. 2025 Feb;47:105275. doi: 10.1016/j.nmd.2025.105275. Epub 2025 Jan 16.
- Derived from this study: PubMed 37739572Henzi BC, Schmidt S, Nagy S, Rubino-Nacht D, Schaedelin S, Putananickal N, Stimpson G; North Star Consortium; Amthor H, Childs AM, Deconinck N, de Groot I, Horrocks I, Houwen-van Opstal S, Laugel V, Lopez Lobato M, Madruga Garrido M, Nascimento Osorio A, Schara-Schmidt U, Spinty S, von Moers A, Lawrence F, Hafner P, Dorchies OM, Fischer D. Safety and efficacy of tamoxifen in boys with Duchenne muscular dystrophy (TAMDMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2023 Oct;22(10):890-899. doi: 10.1016/S1474-4422(23)00285-5.
- Derived from this study: PubMed 31752977Nagy S, Hafner P, Schmidt S, Rubino-Nacht D, Schadelin S, Bieri O, Fischer D. Tamoxifen in Duchenne muscular dystrophy (TAMDMD): study protocol for a multicenter, randomized, placebo-controlled, double-blind phase 3 trial. Trials. 2019 Nov 21;20(1):637. doi: 10.1186/s13063-019-3740-6.
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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. This study is not recruiting at the moment.
Dirk Fischer, MD principal investigator
University Children's Hospital Basel
11 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Tamoxifen in people with Duchenne Muscular Dystrophy, run by University Hospital, Basel, Switzerland at 11 sites in 6 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 20 inclusion and 21 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 20 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United Kingdom, France, Germany, Spain, Netherlands and 1 more country. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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.
What does the primary outcome measure?
Reduction of disease progression (time frame: Baseline to week 48). 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 NCT03354039, 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 20 Dec 2022.