AzTrials
AzTrials

Duchenne Muscular DystrophyPhase 2NCT03439670

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

A Phase 2 study testing Vamorolone and Prednisone in people with Duchenne Muscular Dystrophy, run by ReveraGen BioPharma, Inc. at 33 sites in 11 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period… Read more about this study

Who is it for?
Ages 4 to 7 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
Vamorolone and Prednisone
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
121 participants
Where
33 sitesin 11 countries
Study dates
29 Jun 2018to 19 Aug 2021
Phase
Phase 2
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 4 to 7 years
  • Men only
  • No healthy volunteers
  • Child

Who may be able to join

  • Subject's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act (HIPAA) authorization, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to local requirements
  • Subject has a centrally confirmed (by TRiNDS central genetic counselor[s]) diagnosis of DMD as defined as:
  • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, OR
  • Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'out-of-frame,' and clinical picture consistent with typical DMD, OR
  • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e., nonsense mutation, deletion/duplication leading to a downstream stop codon), with a clinical picture consistent with typical DMD;
  • Subject is ≥ 4 years and <7 years of age at time of enrollment in the study;
Show all 15Show fewer
  • Subject weighs >13.0 kg and ≤ 39.9 kg at the Screening Visit;
  • Subject is able to walk independently without assistive devices;
  • Subject is able to complete the Time to Stand Test (TTSTAND) without assistance in <10 seconds, as assessed at the Screening Visit;
  • Clinical laboratory test results are within the normal range at the Screening Visit, or if abnormal, are not clinically significant, in the opinion of the Investigator. [Notes: Serum gamma glutamyl transferase (GGT), creatinine, and total bilirubin all must be ≤ upper limit of the normal range at the Screening Visit. An abnormal vitamin D level that is considered clinically significant will not exclude a subject from randomization];
  • Subject has evidence of chicken pox immunity as determined by:
  • Presence of IgG antibodies to varicella, as documented by a positive test result from the local laboratory from blood collected during the Screening Period, OR
  • Documentation, provided at the Screening Visit, that the subject has had 2 doses of varicella vaccine, with or without serologic evidence of immunity; the second of the 2 immunizations must have been given at least 14 days prior to randomization.
  • Subject is able to swallow tablets, as confirmed by successful test swallowing of placebo tablets during the Screening Period; and
  • Subject and parent(s)/guardian(s) are willing and able to comply with scheduled visits, study drug administration plan, and study procedures.

Who cannot join

  • Subject has current or history of major renal or hepatic impairment, diabetes mellitus or immunosuppression;
  • Subject has current or history of chronic systemic fungal or viral infections;
  • Subject has had an acute illness within 4 weeks prior to the first dose of study medication;
  • Subject has used mineralocorticoid receptor agents, such as spironolactone, eplerenone, canrenone (canrenoate potassium), prorenone (prorenoate potassium), mexrenone (mexrenoate potassium) within 4 weeks prior to the first dose of study medication;
  • Subject has a history of primary hyperaldosteronism;
  • Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
Show all 17Show fewer
  • Subject is currently being treated or has received previous treatment with oral glucocorticoids or other immunosuppressive agents [Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 1 month cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis, unless discontinued for intolerance. Inhaled and/or topical glucocorticoids are permitted if last use is at least 4 weeks prior to first dose of study medication or if administered at stable dose beginning at least 4 weeks prior to first dose of study medication and anticipated to be used at the stable dose regimen for the duration of the study];
  • Subject has an allergy or hypersensitivity to the study medication or to any of its constituents;
  • Subject has used idebenone within 4 weeks prior to the first dose of study medication;
  • Subject has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
  • Subject has previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
  • Subject is taking (or has taken within 4 weeks prior to the first dose of study medication) herbal remedies and supplements which can impact muscle strength and function (e.g., Co-enzyme Q10, creatine, etc);
  • Subject is taking (or has taken within 3 months prior to the first dose of study medication) any medication indicated for DMD, including Exondys51 and Translarna;
  • Subject has been administered a live attenuated vaccine within 14 days prior to the first dose of study medication;
  • Subject is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of study medication;
  • Subject has a sibling who is currently enrolled in any vamorolone study or Expanded Access Program, or who intends to enroll in any vamorolone study or Expanded Access Program during the subject's participation in the VBP15-004 study; or
  • Subject has previously been enrolled in the study. Note: Any parameter/test may be repeated at the Investigator's discretion during Screening to determine reproducibility. In addition, subjects may be rescreened if ineligible due to a transient condition which would prevent the subject from participating, such as an upper respiratory tract infection or injury, or if ineligible due to negative anti-varicella IgG antibody test result.

Trial locations and contacts

33 sites in 11 countries.

33 sites in 11 countries

United States10 sites

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Ann & Robert H. Lurie Children's Hospital

    Chicago, Illinois, 60611

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75207

  • University of California Davis

    Davis, California, 95616

  • Duke Children's Hospital

    Durham, North Carolina, 27710

  • University of California Los Angeles

    Los Angeles, California, 90095

  • Nemours Children's Hospital

    Orlando, Florida, 32827

  • Children's Hospital of Virginia of Virginia Commonwealth University

    Richmond, Virginia, 23219

  • Gillette Children's Speciality Health Care

    Saint Paul, Minnesota, 55101

  • Seattle Children's Hospital

    Seattle, Washington, 98105

Other trials in United States

United Kingdom6 sites

  • Birmingham Heartlands Hospital

    Birmingham

  • Royal Hospital for Children

    Glasgow, G51 4TF

  • Leeds Teaching Hospital Trust

    Leeds

  • Alder Hey Children's NHS Foundation Trust

    Liverpool, L12 2AP

  • Great Ormond Street Hospital

    London

  • Newcastle upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, NE7 7DN

Other trials in United Kingdom

Canada4 sites

  • Alberta's Children Hospital

    Calgary, Alberta, AB T3B 6A8

  • Montreal Children's Hospital

    Montreal, Quebec, H4A 3J1

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1

  • British Columbia Children's Hospital

    Vancouver, British Columbia, V6H 3N1

Other trials in Canada

Australia2 sites

  • Royal Children's Hospital

    Melbourne

  • Sydney Children's Hospital

    Westmead

Other trials in Australia

Belgium2 sites

  • Ghent University Hospital

    Ghent

  • University Hospitals Leuven

    Leuven

Other trials in Belgium

Czechia2 sites

  • University Hospital Brno

    Brno

  • Charles University

    Prague

Other trials in Czechia

Netherlands2 sites

  • Leiden University Medical Center

    Leiden

  • Radboud University

    Nijmegen

Other trials in Netherlands

Spain2 sites

  • Sant Joan de Deu Hospital - Barcelona, Spain

    Barcelona

  • Hospital Universitario y Politécnico La Fe

    Valencia

Other trials in Spain

Greece1 site

  • Agia Sofia Children's Hospital

    Athens

Other trials in Greece

Israel1 site

  • Schneider Children's Medical Center

    Petah Tikvah, 49202

Other trials in Israel

Sweden1 site

  • Queen Silvia Children's Hospital

    Gothenburg, 41685

Other trials in Sweden

About this study

Official title: A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)

Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years with DMD.

Read the detailed description

This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years with DMD.

The study is comprised of a 5-week Pretreatment Screening Period, a 1-day Pretreatment Baseline Period, a 24-week Treatment Period #1 (Weeks 1-24), a 4-week Transition Period (Weeks 25-28), a 20-week Treatment Period #2 (Weeks 28 + 1 day to 48), and a 4-week Dose-tapering Period (Weeks 49-52).

Subjects will be randomized to one of six treatment groups in a 2:2:1:1:1:1 ratio, where the two prednisone groups in Treatment Period #1 (Groups 3 and 4) will be combined and the two placebo groups in Treatment Period #1 (Groups 5 and 6) will be combined, effectively resulting in a 1:1:1:1 randomization (vamorolone 2.0 mg/kg/day : vamorolone 6.0 mg/kg/day : prednisone 0.75 mg/kg/day : placebo) for Treatment Period #1.

Subjects will be stratified based on age at study entry (<6 vs. ≥ 6 years). During the 4-week Transition Period between Treatment Period #1 and Treatment Period #2, all subjects will continue on the same oral suspension (vamorolone 2.0 mg/kg or 6.0 mg/kg, or matching placebo) they received during Treatment Period #1 and all subjects will have their tablet dose tapered to zero. Thus, subjects randomized to receive vamorolone during Treatment Period #1 (Groups 1 and 2) will continue to receive vamorolone at the same dose, while subjects randomized to receive prednisone will have their dose tapered to zero, and subjects randomized to placebo will continue to receive placebo.

The prednisone group will be used as an active control comparison for safety and efficacy endpoints as requested by the European Medicines Agency (EMA). The placebo group will be used as comparator for efficacy endpoints (superiority model) as requested by the EMA and Food and Drug Administration (FDA) protocol advisory board. Although glucocorticoids are part of the care recommendations for DMD, their adverse effect profile has limited their use. The age at which glucocorticoids should be started in DMD boys is uncertain, ranging from 4 to 7 years, based on a balance between benefits and side effects. In view of the age inclusion criteria and duration of the placebo-controlled study period (6 months), the use of a placebo group has been considered acceptable as in clinical practice it will not cause a real delay in prescription of an accepted treatment for this condition. Any exposure of placebo longer than 6 months was considered unethical.

At the end of the Treatment Period #2, subjects may be given access to vamorolone through an additional study or general access program, or given the option to transition to standard of care treatment for DMD (may include glucocorticoids). Subjects completing VBP15-004 and enrolling directly into an additional vamorolone study or general access program to receive vamorolone will not need to taper their vamorolone dose prior to enrollment. All other subjects will begin a 4-week double-blind Dose-tapering Period during which the dose of study medication will be progressively reduced and discontinued.

Being tested
Vamorolone, Prednisone, Placebo
Keywords
Duchenne Muscular Dystrophy, Vamorolone
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.

  • VamoroloneDrug

    Also called VBP15

    Oral administration of 2.0 mg/kg/day for the duration of the study.

  • PrednisoneDrug

    Oral administration of 0.75 mg/kg/day for 24 weeks.

  • PlaceboOther

    Oral administration of placebo daily for 24 weeks.

  • VamoroloneDrug

    Also called VBP15

    Oral administration of 6.0 mg/kg/day for the duration of the study.

  • PrednisoneDrug

    Oral administration of 0.75 mg/kg/day for 24 weeks.

  • PlaceboOther

    Oral administration of placebo daily for 24 weeks.

  • VamoroloneDrug

    Also called VBP15

    Oral administration of 2.0 mg/kg/day for 20 weeks.

  • VamoroloneDrug

    Also called VBP15

    Oral administration of 6.0 mg/kg/day for 20 weeks.

Study groups in NCT03439670
Study groupRoleWhat they receive
Treatment Group 1ExperimentalPatients enrolled in Treatment Group 1 (experimental group) will receive vamorolone 2.0 mg/kg/day for the duration of the study.
Receives: Drug: Vamorolone
Treatment Group 2ExperimentalPatients enrolled in Treatment Group 2 (experimental group) will receive vamorolone at 6.0 mg/kg/day for the duration of the study.
Receives: Drug: Vamorolone
Treatment Group 3Active comparatorPatients enrolled in Treatment Group 3 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by 20 weeks of treatment with 2.0 mg/kg/day vamorolone.
Receives: Drug: Prednisone; Drug: Vamorolone
Treatment Group 4Active comparatorPatients enrolled in Treatment Group 4 (active comparator group) will receive prednisone 0.75 mg/kg/day for 24 weeks followed by 20 weeks treatment with 6.0 mg/kg/day vamorolone.
Receives: Drug: Prednisone; Drug: Vamorolone
Treatment Group 5Placebo comparatorPatients enrolled in Treatment Group 5 (placebo comparator group) will receive placebo daily for 24 weeks followed by 20 weeks treatment with 2.0 mg/kg/day vamorolone.
Receives: Other: Placebo; Drug: Vamorolone
Treatment Group 6Placebo comparatorPatients enrolled in Treatment Group 6 (placebo comparator group) will receive placebo daily for 24 weeks followed by 20 weeks treatment with 6.0 mg/kg/day vamorolone.
Receives: Other: Placebo; Drug: Vamorolone

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline

    Vamorolone at 6.0mg/kg/day vs. placebo group in change from baseline to the Week 24 assessment

    Measured: 24 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Feb 2018
  2. Study start29 Jun 2018
  3. Primary completion23 Feb 2021
  4. Study completion19 Aug 2021

Resources

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
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
121 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03439670
Sponsor's protocol ID
VBP15-004
Status last verified
Mar 2023
First submitted
9 Jan 2018
Last updated
9 Mar 2023
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Mah JK, Korngut L, Dykeman J, Day L, Pringsheim T, Jette N. A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 2014 Jun;24(6):482-91. doi: 10.1016/j.nmd.2014.03.008. Epub 2014 Mar 22.

    PubMed 24780148

  • Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987 Dec 24;51(6):919-28. doi: 10.1016/0092-8674(87)90579-4.

    PubMed 3319190

  • Evans NP, Misyak SA, Robertson JL, Bassaganya-Riera J, Grange RW. Immune-mediated mechanisms potentially regulate the disease time-course of duchenne muscular dystrophy and provide targets for therapeutic intervention. PM R. 2009 Aug;1(8):755-68. doi: 10.1016/j.pmrj.2009.04.010.

    PubMed 19695529

  • Kumar A, Boriek AM. Mechanical stress activates the nuclear factor-kappaB pathway in skeletal muscle fibers: a possible role in Duchenne muscular dystrophy. FASEB J. 2003 Mar;17(3):386-96. doi: 10.1096/fj.02-0542com.

    PubMed 12631578

  • Acharyya S, Villalta SA, Bakkar N, Bupha-Intr T, Janssen PM, Carathers M, Li ZW, Beg AA, Ghosh S, Sahenk Z, Weinstein M, Gardner KL, Rafael-Fortney JA, Karin M, Tidball JG, Baldwin AS, Guttridge DC. Interplay of IKK/NF-kappaB signaling in macrophages and myofibers promotes muscle degeneration in Duchenne muscular dystrophy. J Clin Invest. 2007 Apr;117(4):889-901. doi: 10.1172/JCI30556. Epub 2007 Mar 22.

    PubMed 17380205

  • Dogra C, Changotra H, Wergedal JE, Kumar A. Regulation of phosphatidylinositol 3-kinase (PI3K)/Akt and nuclear factor-kappa B signaling pathways in dystrophin-deficient skeletal muscle in response to mechanical stretch. J Cell Physiol. 2006 Sep;208(3):575-85. doi: 10.1002/jcp.20696.

    PubMed 16741926

  • Pahl HL. Activators and target genes of Rel/NF-kappaB transcription factors. Oncogene. 1999 Nov 22;18(49):6853-66. doi: 10.1038/sj.onc.1203239.

    PubMed 10602461

  • Spencer MJ, Montecino-Rodriguez E, Dorshkind K, Tidball JG. Helper (CD4(+)) and cytotoxic (CD8(+)) T cells promote the pathology of dystrophin-deficient muscle. Clin Immunol. 2001 Feb;98(2):235-43. doi: 10.1006/clim.2000.4966.

    PubMed 11161980

  • Spencer MJ, Tidball JG. Do immune cells promote the pathology of dystrophin-deficient myopathies? Neuromuscul Disord. 2001 Sep;11(6-7):556-64. doi: 10.1016/s0960-8966(01)00198-5.

    PubMed 11525885

  • Malik V, Rodino-Klapac LR, Mendell JR. Emerging drugs for Duchenne muscular dystrophy. Expert Opin Emerg Drugs. 2012 Jun;17(2):261-77. doi: 10.1517/14728214.2012.691965.

    PubMed 22632414

  • Reeves EKM, Hoffman EP, Nagaraju K, Damsker JM, McCall JM. VBP15: preclinical characterization of a novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem. 2013 Apr 15;21(8):2241-2249. doi: 10.1016/j.bmc.2013.02.009. Epub 2013 Feb 18.

    PubMed 23498916

  • Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, Kaul A, Kinnett K, McDonald C, Pandya S, Poysky J, Shapiro F, Tomezsko J, Constantin C; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010 Jan;9(1):77-93. doi: 10.1016/S1474-4422(09)70271-6. Epub 2009 Nov 27.

    PubMed 19945913

  • Pane M, Mazzone ES, Sivo S, Sormani MP, Messina S, D'Amico A, Carlesi A, Vita G, Fanelli L, Berardinelli A, Torrente Y, Lanzillotta V, Viggiano E, D Ambrosio P, Cavallaro F, Frosini S, Barp A, Bonfiglio S, Scalise R, De Sanctis R, Rolle E, Graziano A, Magri F, Palermo C, Rossi F, Donati MA, Sacchini M, Arnoldi MT, Baranello G, Mongini T, Pini A, Battini R, Pegoraro E, Previtali S, Bruno C, Politano L, Comi GP, Bertini E, Mercuri E. Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes. PLoS One. 2014 Oct 1;9(10):e108205. doi: 10.1371/journal.pone.0108205. eCollection 2014.

    PubMed 25271887

  • Manzur AY, Kuntzer T, Pike M, Swan A. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database Syst Rev. 2008 Jan 23;(1):CD003725. doi: 10.1002/14651858.CD003725.pub3.

    PubMed 18254031

  • Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, Sali A, Miller BK, Phadke A, Scheffer L, Quinn J, Tatem K, Jordan S, Dadgar S, Rodriguez OC, Albanese C, Calhoun M, Gordish-Dressman H, Jaiswal JK, Connor EM, McCall JM, Hoffman EP, Reeves EK, Nagaraju K. VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med. 2013 Oct;5(10):1569-85. doi: 10.1002/emmm.201302621. Epub 2013 Sep 9.

    PubMed 24014378

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Vamorolone and Prednisone in people with Duchenne Muscular Dystrophy, run by ReveraGen BioPharma, Inc. at 33 sites in 11 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 15 inclusion and 17 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 9 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 33 sites in United States, United Kingdom, Canada, Australia, Belgium and 6 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 13 Jul 2022). 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?

Efficacy Measured by Time to Stand Test (TTSTAND) Velocity in Rises/Second Change From Baseline (time frame: 24 weeks). 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 NCT03439670, which also lists 48 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 9 Mar 2023.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in