Progressive Familial Intrahepatic Cholestasis (PFIC)Phase 3MARCH-PFICNCT03905330
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
A Phase 3 study testing Maralixibat in people with Progressive Familial Intrahepatic Cholestasis (PFIC), run by Mirum Pharmaceuticals, Inc. at 31 sites in 17 countries.
Trial summary
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC). Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Progressive Familial Intrahepatic Cholestasis (PFIC)
- What is being tested?
- Maralixibat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 93 participants
- Where
- 31 sitesin 17 countries
- Study dates
- 9 Jul 2019to 1 Sep 2022
- Phase
- Phase 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 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
- Male or female subjects with a body weight ≥5 kg, who are ≥12 months and <18 years of age at time of baseline
- Cholestasis as manifested by total sBA ≥3× ULN (applies to primary cohort only)
- An average AM ItchRO(Obs) score ≥1.5 during 4 consecutive weeks of the screening period, leading to the baseline visit (Visit 1)
- Completion of at least 21 valid* morning ItchRO(Obs) entries during 4 consecutive weeks of the screening period, leading to the baseline visit (Visit 1) (*valid = completed and not answered as "I don't know"; maximum allowed invalid reports = 7, no more than 2 invalid reports during the last 7 days before randomization)
- Diagnosis of PFIC based on the following:
Show all 14Show fewer
- Chronic cholestasis as manifested by persistent (>6 months) pruritus in addition to biochemical abnormalities and/or pathological evidence of progressive liver disease and
- Primary Cohort: Subjects with genetic testing results consistent with biallelic disease-causing variation in ABCB11 (PFIC2), based on standard of care genotyping
- Supplemental Cohort: i. Subjects with genetic testing results consistent with biallelic disease-causing variation in ATP8B1 (PFIC1), ABCB4 (PFIC3), or TJP2 (PFIC4), based on standard of care genotyping. ii. Subjects with PFIC phenotype without a known mutation or with another known mutation not described above or with intermittent cholestasis as manifested by fluctuating sBA levels. iii. Subjects with PFIC after internal or external biliary diversion surgery or for whom internal or external biliary diversion surgery was reversed.
- Male and females of non-childbearing potential. Males and non-pregnant, non-lactating females of childbearing potential who are sexually active must agree to use acceptable methods of contraception during the study and 30 days following the last dose of the study medication. Females of childbearing potential must have a negative pregnancy test
- Access to email or phone for scheduled remote visits
- Ability to read and understand the questionnaires (both caregivers and subjects above the age of assent)
- Access to consistent caregiver(s) during the study
- Subject and caregiver willingness to comply with all study visits and requirements.
Who cannot join
- Predicted complete absence of bile salt excretion pump (BSEP) function based on the type of ABCB11 mutation (PFIC2), as determined by a standard of care genotyping (applies to primary cohort only). Subjects can enter the study in the Supplemental Cohort (under inclusion criteria 6.ii or 6.iii).
- Recurrent intrahepatic cholestasis, indicated by a history of sBA levels <3x ULN or intermittent pruritus (applies to primary cohort only)
- Current or recent history (<1 year) of atopic dermatitis or other non-cholestatic diseases associated with pruritus.
- History of surgical disruption of the enterohepatic circulation (applies to primary cohort only)
- Chronic diarrhea requiring intravenous fluid or nutritional intervention for the diarrhea and/or its sequelae at screening or during the 6 months prior to screening
- Previous or need for imminent liver transplant
Show all 20Show fewer
- Decompensated cirrhosis (international normalized ratio [INR] >1.5, albumin <30 g/L, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy)
- ALT or total serum bilirubin (TSB) >15× ULN at screening
- Presence of other liver disease
- Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per Investigator discretion
- Possibly malignant liver mass on imaging, including screening ultrasound
- Known diagnosis of human immunodeficiency virus (HIV) infection
- Any prior cancer diagnosis (except for in situ carcinoma) within 5 years of the screening visit (Visit 0)
- Any known history of alcohol or substance abuse
- Administration of bile acids or lipid binding resins, or phenylbutyrates during the screening period
- Criterion has been deleted as of Amendment 3
- Administration of any investigational drug, biologic, or medical device during the screening period
- Previous use of an ileal bile acid transporter inhibitor (IBATi)
- History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator or Sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the subject, or lead to nonadherence with the study protocol or inability to conduct the study procedures
- Known hypersensitivity to maralixibat or any of its excipients.
Trial locations and contacts
31 sites in 17 countries.
31 sites in 17 countries
United States11 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Cleveland Clinic - Pediatric Institute
Cleveland, Ohio, 44195
University of Texas - UT Southwestern Medical Center
Dallas, Texas, 75390
Children's Hospital Los Angeles CHLA
Los Angeles, California, 90027
The Children's Hospital at Montefiore Yeshiva University - Montefiore Medical Center
New York, New York, 10461
Advent Health
Orlando, Florida, 32803
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
University of Texas, Health Science Center San Antonio
San Antonio, Texas, 78229
Seattle Children's Hospital
Seattle, Washington, 98105
Medstar Georgetown University Hospital
Washington D.C., District of Columbia, 20007-2113
France3 sites
Hospices Civils de Lyon - Hopital Femme Mere Enfant Service de Gastroenterologie, Hepatologie et Nutrition
Lyon
CHU de Marseille, Hôpital de la Timone
Marseille
CHU de Toulouse - Hôpital des Enfants
Toulouse, 31059
Italy2 sites
Hospital Papa Giovanni XXIII / Unità di Pediatria
Bergamo
Ospedale Pediatrico Bambino Gesu
Roma
United Kingdom2 sites
Birmingham Children's Hospital
Birmingham
King's College Hospital NHS
London
Austria1 site
Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde
Vienna
Belgium1 site
Universite Catholique de Louvain (UCLouvain) - Cliniques Universitaires Saint-Luc
Brussels
Brazil1 site
Sociedade Beneficente de Senhoras Hospital Sírio-Libanês
São Paulo
Canada1 site
University of Alberta - Women and Children's Health Research Institute
Edmonton
Colombia1 site
Fundacion Cardioinfantil - Departamento de Investigaciones
Bogotá
Hungary1 site
Semmelweis Egyetem - Altalanos Orvostudomanyi Kar (SE AOK)
Budapest, 1083
About this study
Official title: MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFIC
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
Read the detailed description
This study was conducted at multiple sites in North America, Europe, Asia and South America.
- Being tested
- Maralixibat, Placebo
- Keywords
- Cholestasis, Maralixibat, Mutation, PFIC, PFIC2, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases, Pediatric
- Medical subject headings
- Cholestasis, progressive familial intrahepatic 1, Cholestasis, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MaralixibatDrug
Also called Formerly LUM001 and SHP625
Maralixibat oral solution (up to 600 mcg/kg) orally twice daily for 26 weeks.
PlaceboOther
Placebo matching to maralixibat orally twice daily for 26 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Maralixibat | Experimental | Participants will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) orally twice daily for 26 weeks. Receives: Drug: Maralixibat |
| Placebo | Placebo comparator | Participants will receive placebo matched to maralixibat oral solution twice daily for 26 weeks. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort
The primary efficacy endpoint is the mean change in the average morning ItchRO(Obs) severity score between baseline and Weeks 15-26, using 4-week average morning ItchRO(Obs) severity scores (Mixed Model Repeated Measures). The baseline average morning ItchRO(Obs) severity score is defined as the 4-week average morning ItchRO(Obs) severity score prior to the first dose of the study medication.
ItchRo(Obs) Severity Score = Itch Reported Outcome Observer assessment severity score; scale between 0 (not itchy at all) and 4 (extremely itchy); the lower the score the better.
Measured: MMRM model was used with inputs of the average changes from baseline (BL) to Weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. The average change from BL over Weeks 15-26 is calculated in the model and presented as a single number.
Secondary outcomes (7)
Mean Change in Total sBA Level in the Primary Cohort.
Mean change in total sBA level between baseline and average of Weeks 18, 22 and 26.
Measured: Baseline and average of Weeks 18, 22 and 26
Mean Change in the Average Morning ItchRO(Obs) Severity Score in Participants With PFIC (PFIC1, Nt-PFIC2, PFIC3, PFIC4 and PFIC6)
Mean change in the average morning ItchRO(Obs) severity score between baseline and Week 15 through Week 26 in participants with PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6).
ItchRo(Obs) Severity Score = Itch Reported Outcome Observer assessment severity score; scale between 0 (not itchy at all) and 4 (extremely itchy); the lower the score the better.
Measured: Between Baseline and Week 15 through Week 26
Mean Change in Total sBA Level in Participants With PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)
Mean change in total sBA level between baseline and average of Weeks 18, 22, and 26 in participants with PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)
Measured: Between Baseline and average of Weeks 18, 22 and 26
Proportion of ItchRO(Obs) Responders in the Primary Cohort
Proportion of ItchRO(Obs) responders from Week 15 to Week 26 in participants with PFIC2 using the average value from the three 4-week periods (Weeks 15-18, 19-22 and 23-26). The number in the Subject Analysis Set refers to the number of responders.
ItchRO responders are defined as a subject having a 4-week average morning ItchRO(Obs) severity change from baseline of ≤-1.0 or an average severity score of ≤1.0.
Measured: Week 15 to Week 26 using the average value from the three 4-week periods (Weeks 15-18, 19-22 and 23-26)
Proportion of sBA Responders in the Primary Cohort
Proportion of sBA responders from Week 18 to Week 26 in participants with PFIC2, using the average value from Weeks 18, 22 and 26 values. The number in the Subject Analysis Set refers to the number of responders.
sBA responders are defined as a subject having an average sBA level of <102 umol/L (applies only if baseline sBA level was ≥102 umol/L), OR a ≤-75% average percent change from baseline.
Measured: Average value from Weeks 18, 22 and 26
Proportion of ItchRO(Obs) Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)
Proportion of ItchRO(Obs) responders from Week 15 to Week 26 in participants with PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6). The number in the Subject Analysis Set refers to the number of responders.
ItchRO responders are defined as a subject having a 4-week average morning ItchRO(Obs) severity change from baseline of ≤-1.0 OR an average severity score of ≤1.0.
Measured: From Week 15 to Week 26
Proportion of sBA Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)
Proportion of sBA responders from Week 18 to Week 26 in participants with PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6). The number in the Subject Analysis Set refers to the number of responders.
sBA responders are defined as a subject having an average sBA level of <102 umol/L (applies only if baseline sBA level was ≥102 umol/L), or a ≤-75% average percent change from baseline.
Measured: Week 18 to Week 26
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2019
- Study start9 Jul 2019
- Primary completion1 Sep 2022
- Study completion1 Sep 2022
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 (1)Papers the sponsor has linked to this study.
- Other trials for Progressive Familial Intrahepatic Cholestasis (PFIC)Studies of the same condition, open and under way.
- More trials run by Mirum Pharmaceuticals, Inc.The 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
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03905330
- Sponsor's protocol ID
- MRX-502
- Other IDs
- 2019-001211-22, 2019
- Acronym
- MARCH-PFIC
- Status last verified
- Dec 2023
- First submitted
- 1 Apr 2019
- Last updated
- 11 Dec 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Miethke AG, Moukarzel A, Porta G, Covarrubias Esquer J, Czubkowski P, Ordonez F, Mosca A, Aqul AA, Squires RH, Sokal E, D'Agostino D, Baumann U, D'Antiga L, Kasi N, Laborde N, Arikan C, Lin CH, Gilmour S, Mittal N, Chiou FK, Horslen SP, Huber WD, Jaecklin T, Nunes T, Lascau A, Longpre L, Mogul DB, Garner W, Vig P, Hupertz VF, Gonzalez-Peralta RP, Ekong U, Hartley J, Laverdure N, Ovchinsky N, Thompson RJ. Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2024 Jul;9(7):620-631. doi: 10.1016/S2468-1253(24)00080-3. Epub 2024 May 6.
PubMed 38723644
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MRX-502: Randomized Double-blind Placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFIC
- ClinicalTrials.gov ID
- NCT03905330
- Acronym / sponsor ID
- MARCH-PFIC / MRX-502
- Keywords
- Cholestasis, Maralixibat, Mutation, PFIC, PFIC2, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 93 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 9 Jul 2019 (actual)
- Primary completion
- 1 Sep 2022 (actual)
- Study completion
- 1 Sep 2022 (actual)
- First posted
- 5 Apr 2019 (actual)
- Results
- Posted 11 Dec 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
Detailed description
This study was conducted at multiple sites in North America, Europe, Asia and South America.
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 |
|---|---|---|
| Maralixibat | Experimental | Drug: MaralixibatParticipants will receive Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) orally twice daily for 26 weeks. |
| Placebo | Placebo comparator | Other: PlaceboParticipants will receive placebo matched to maralixibat oral solution twice daily for 26 weeks. |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- Informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee (IRB/EC)
- Male or female subjects with a body weight ≥5 kg, who are ≥12 months and <18 years of age at time of baseline
- Cholestasis as manifested by total sBA ≥3× ULN (applies to primary cohort only)
- An average AM ItchRO(Obs) score ≥1.5 during 4 consecutive weeks of the screening period, leading to the baseline visit (Visit 1)
- Completion of at least 21 valid* morning ItchRO(Obs) entries during 4 consecutive weeks of the screening period, leading to the baseline visit (Visit 1) (*valid = completed and not answered as "I don't know"; maximum allowed invalid reports = 7, no more than 2 invalid reports during the last 7 days before randomization)
Key exclusion criteria (20 listed)
- Predicted complete absence of bile salt excretion pump (BSEP) function based on the type of ABCB11 mutation (PFIC2), as determined by a standard of care genotyping (applies to primary cohort only). Subjects can enter the study in the Supplemental Cohort (under inclusion criteria 6.ii or 6.iii).
- Recurrent intrahepatic cholestasis, indicated by a history of sBA levels <3x ULN or intermittent pruritus (applies to primary cohort only)
- Current or recent history (<1 year) of atopic dermatitis or other non-cholestatic diseases associated with pruritus.
- History of surgical disruption of the enterohepatic circulation (applies to primary cohort only)
- Chronic diarrhea requiring intravenous fluid or nutritional intervention for the diarrhea and/or its sequelae at screening or during the 6 months prior to screening
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 |
|---|---|---|
| MaralixibatAlso: Formerly LUM001 and SHP625Maralixibat oral solution (up to 600 mcg/kg) orally twice daily for 26 weeks. | Drug | Maralixibat |
| PlaceboPlacebo matching to maralixibat orally twice daily for 26 weeks. | Other | Placebo |
Primary outcome
Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort
Time frame: MMRM model was used with inputs of the average changes from baseline (BL) to Weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. The average change from BL over Weeks 15-26 is calculated in the model and presented as a single number.
The primary efficacy endpoint is the mean change in the average morning ItchRO(Obs) severity score between baseline and Weeks 15-26, using 4-week average morning ItchRO(Obs) severity scores (Mixed Model Repeated Measures). The baseline average morning ItchRO(Obs) severity score is defined as the 4-week average morning ItchRO(Obs) severity score prior to the first dose of the study medication. ItchRo(Obs) Severity Score = Itch Reported Outcome Observer assessment severity score; scale between 0 (not itchy at all) and 4 (extremely itchy); the lower the score the better.
7 secondary outcomes
Mean Change in Total sBA Level in the Primary Cohort.
Time frame: Baseline and average of Weeks 18, 22 and 26
Mean Change in the Average Morning ItchRO(Obs) Severity Score in Participants With PFIC (PFIC1, Nt-PFIC2, PFIC3, PFIC4 and PFIC6)
Time frame: Between Baseline and Week 15 through Week 26
Mean Change in Total sBA Level in Participants With PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)
Time frame: Between Baseline and average of Weeks 18, 22 and 26
Proportion of ItchRO(Obs) Responders in the Primary Cohort
Time frame: Week 15 to Week 26 using the average value from the three 4-week periods (Weeks 15-18, 19-22 and 23-26)
Proportion of sBA Responders in the Primary Cohort
Time frame: Average value from Weeks 18, 22 and 26
Proportion of ItchRO(Obs) Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)
Time frame: From Week 15 to Week 26
Proportion of sBA Responders PFIC (PFIC1, PFIC2, PFIC3, PFIC4 and PFIC6)
Time frame: Week 18 to Week 26
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38723644Miethke AG, Moukarzel A, Porta G, Covarrubias Esquer J, Czubkowski P, Ordonez F, Mosca A, Aqul AA, Squires RH, Sokal E, D'Agostino D, Baumann U, D'Antiga L, Kasi N, Laborde N, Arikan C, Lin CH, Gilmour S, Mittal N, Chiou FK, Horslen SP, Huber WD, Jaecklin T, Nunes T, Lascau A, Longpre L, Mogul DB, Garner W, Vig P, Hupertz VF, Gonzalez-Peralta RP, Ekong U, Hartley J, Laverdure N, Ovchinsky N, Thompson RJ. Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Gastroenterol Hepatol. 2024 Jul;9(7):620-631. doi: 10.1016/S2468-1253(24)00080-3. Epub 2024 May 6.
- Genetics Home Reference - PFICLink provided by the sponsor.
- US FDA ResourcesLink provided by the sponsor.
- Mirum Pharmaceuticals homepageLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
31 sites in 17 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Maralixibat in people with Progressive Familial Intrahepatic Cholestasis (PFIC), run by Mirum Pharmaceuticals, Inc. at 31 sites in 17 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 14 inclusion and 20 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 11 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, France, Italy, United Kingdom, Argentina and 12 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 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 11 Dec 2023). 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?
Mean Change in the Average Morning ItchRO(Obs) Severity Score in the Primary Cohort (time frame: MMRM model was used with inputs of the average changes from baseline (BL) to Weeks 1-6, 7-10, 11-14, 15-18, 19-22, 23-26, and other covariates. The average change from BL over Weeks 15-26 is calculated in the model and presented as a single number.). 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 NCT03905330, which also lists 1 linked publication.
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 11 Dec 2023.