Steroid Refractory Acute Graft Versus Host DiseasePhase 3NCT02524847
Methoxsalen and Extracorporeal Photopheresis (ECP) for the Treatment of Pediatric Participants With Steroid Refractory Acute Graft Versus Host Disease
A Phase 3 study testing Methoxsalen and Extracorporeal Photopheresis (ECP) in people with Steroid Refractory Acute Graft Versus Host Disease, run by Therakos, Inc., a Mallinckrodt Company at 32 sites in 8 countries.
Trial summary
This is a single-arm, open-label, multicenter study of the efficacy of UVADEX® (methoxsalen) Sterile Solution in conjunction with THERAKOS® CELLEX® Photopheresis Systems (ECP) in pediatric participants with steroid-refractory aGvHD. The study is composed of Screening, Treatment, and Follow-up Periods. Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Steroid Refractory Acute Graft Versus Host Disease
- What is being tested?
- Methoxsalen and Extracorporeal Photopheresis (ECP)
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 32 sitesin 8 countries
- Study dates
- 20 Jan 2016to 16 Jul 2019
- 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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female 1 to 21 years of age at the time of consent
- Steroid-refractory grade B-D aGvHD.
- Steroid-refractory is defined as a failure to respond to steroid treatment, with failure to respond defined as any grade B-D (IBMTR grading) aGvHD that shows progression ≥ 3 days, or no improvement by 5 days of treatment with 2 mg/kg/day methylprednisolone or equivalent in participants with lower gastrointestinal (GI) or liver disease, or skin disease associated with bullae. Grade D organ involvement will be limited to skin and liver.
- Steroid refractory may also be defined as a failure to respond to 1 mg/kg/day of methylprednisolone or equivalent in participants with disease confined to upper GI disease or lesser degrees of skin GvHD
- Participants with lack of complete response after 2 weeks of steroid treatment
- A Lansky scale Performance Status score ≥ 30
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- Laboratory values are within the following limits, assessed within 3 days of the first study treatment:
- Absolute neutrophil count > 0.5 × 10^9/liter (L)
- Creatinine level < 2 times the upper limit of normal
- For participants with isolated upper GI symptoms, pre-Screening biopsy results to confirm diagnosis of aGvHD
- Female participants of childbearing potential and nonsterilized males who are sexually active with a female partner must be practicing highly effective, reliable, and medically approved contraceptive regimen throughout their participation in the study and for 3 months following the last ECP treatment. Or, for the US only, abstinence may be used in place of an approved contraceptive regimen. Females of childbearing potential are those who have reached the onset of menarche, or 8 years of age, whichever comes first. Approved contraceptive methods for female participants of childbearing potential or nonsterilized males who are sexually active with a female partner are as follows:
- Barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/suppository
- Established use of oral, injectable, or implanted hormonal methods of contraception.
- Placement of an intrauterine device or intrauterine system
- Signed informed consent/assent is obtained before conducting any study procedures; the parent, legal guardian, or legally authorized representative of a minor must also provide written informed consent
Who cannot join
- Currently enrolled in another clinical trial for the treatment of aGvHD
- Use of any experimental regimens or medication(s) for aGvHD treatment
- Treatment with > 2.0 mg/kg/day of methylprednisolone equivalents for aGvHD within 30 days prior to the first study treatment
- Overt signs of relapse of the underlying condition
- Uncontrolled viral, fungal, or bacterial infection
- Platelet count < 20.0 × 10^9/L, despite platelet transfusion
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- Inability to tolerate the extracorporeal volume shifts associated with ECP treatment
- Uncontrolled GI bleeding
- Veno-occlusive liver disease
- Life expectancy < 4 weeks
- Participant requires invasive ventilation or vasopressor support
- Known human immunodeficiency virus (HIV) or hepatitis B or C virus infection (proof of seronegativity within 6 months of screening is required)
- Known allergy or hypersensitivity to methoxsalen, Uvadex, or its excipients
- Known hypersensitivity and allergy to heparin and Anticoagulant Citrate Dextrose Formula-A (ACD-A)
- Co-existing photosensitive disease (e.g., porphyria, systemic lupus erythematosus, albinism) or aphakia
- Coagulation disorders that cannot be corrected with simple transfusion
- Co-existing melanoma, basal cell, or squamous cell skin carcinoma
- Previous splenectomy
- White blood cell count greater than 25,000 cubic millimeter (mm^3)
- Currently being treated with any systemic immunosuppressive or biologic therapy for the treatment of a medical condition other than aGvHD
- Female participant is breastfeeding or pregnant
- Any medical concerns that may pose risk to the participant
- Any psychological, familial, sociological, and/or geographical condition that may potentially hamper compliance with the study protocol and follow-up schedule
Trial locations and contacts
32 sites in 8 countries.
32 sites in 8 countries
United States17 sites
Children's Healthcare of Atlanta, Emory - Children's Center
Atlanta, Georgia, 30322
Boston Children's Hospital
Boston, Massachusetts, 02115
Lurie Children's Hospital
Chicago, Illinois, 60611
University Hospitals Rainbow Babies & Children's
Cleveland, Ohio, 44106
Cleveland Clinic Children's
Cleveland, Ohio, 44195
Hackensack University Medical Center
Hackensack, New Jersey, 07601
MD Anderson Cancer Care Center
Houston, Texas, 77030
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Vanderbilt University Medical Center - Ingram Cancer Institute
Nashville, Tennessee, 37232
Yale University
New Haven, Connecticut, 06520
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Oregon Health and Science University
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84112
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
St. Louis Children's Hospital
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Germany4 sites
Universitätsklinikum Leipzig, Klinik und Poliklinik für Kinder- und Jugendmedizin, Abteilung für Hämatologie und Internistische Onkologie
Leipzig, 04103
Klinikum rechts der Isar, TU München, Klinik- und Poliklinik für Kinder- und Jugendmedizin, Kinderklinik München Schwabing
München, 80804
University Hospital Tuebingen
Tübingen, 72076
Universitaetsklinikum Ulm, Kinder- und Jugendmedizin
Ulm, 89075
Spain4 sites
Vall d'Hebron University Hospital
Barcelona, 8035
Hospital Infantil Universitario "Nino Jesus"
Madrid, 28009
University Hospital Salamanca
Salamanca, 37007
Hospital LA FE
Valencia, 46026
France2 sites
Hopital Necker Enfants Malades
Paris, 75015
Hôpital universitaire Robert Debré
Paris, 75019
Italy2 sites
U.O.C. Clinica di Oncoematologia Pediatrica Azienda Ospedaliera di Padova
Padova, 35128
Pediatric Hospital Bambinu Gesu Rome
Rome, 00165
United Kingdom1 site
Great North Children's Hospital (RVI)
Newcastle, NE1 4LP
About this study
Official title: Single-Arm Study to Assess the Efficacy of UVADEX® (Methoxsalen) Sterile Solution in Conjunction With the THERAKOS® CELLEX® Photopheresis System in Pediatric Patients With Steroid-Refractory Acute Graft-vs-Host Disease (aGvHD)
This is a single-arm, open-label, multicenter study of the efficacy of UVADEX® (methoxsalen) Sterile Solution in conjunction with THERAKOS® CELLEX® Photopheresis Systems (ECP) in pediatric participants with steroid-refractory aGvHD. The study is composed of Screening, Treatment, and Follow-up Periods.
Read the detailed description
Screening:
After the informed consent/assent form (ICF) is signed, the screening assessments will be performed in a single visit to establish the eligibility of the participant, based on inclusion and exclusion criteria, as well as aGvHD grading. Scheduling of the first week of ECP treatments and the arrangements for availability of typed and cross-matched donor packed red blood cells (PRBCs) for transfusion, if required, will be made in advance of participants entering the Treatment Period.
Treatment Period:
Once eligibility is established, participants will enter the 12-week ECP Treatment Period. The availability of typed and cross-matched donor PRBCs for transfusion during treatment, if needed, should be established prior to the scheduling of ECP treatments.
Participants will be allowed to continue standard aGvHD prophylaxis regimens (e.g., cyclosporine, tacrolimus, methotrexate, mycophenolate mofetil) without the addition of new therapies. Participants will be allowed to discontinue prophylaxis regimens for reasons of toxicity, and will also be allowed to switch to another prophylaxis medication within the same class (e.g., the calcineurin inhibitors cyclosporine and tacrolimus) for reasons of toxicity.
All participants enrolled in this trial will have received corticosteroids for the treatment of aGvHD. After entering the treatment period on study, tapering of steroids by total weekly decrements of 12.5% to 25% of the steroid dose at initiation of ECP therapy is permitted after a sustained response of aGvHD has been observed for at least 3 consecutive days, with the suggested goal to decrease the starting steroid dose by at least 50% 4 weeks after initiation of ECP.
Follow-Up Period:
After completion of the 12-week Treatment Period, participants may continue ECP treatment on commercial product at the Principal Investigator's discretion. Acute GvHD status will be assessed 4 weeks after completion of the Treatment Period. Participant survival will be assessed by passive follow-up (chart review) 26 weeks after initiation of ECP treatment.
- Being tested
- Methoxsalen, Extracorporeal Photopheresis (ECP)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MethoxsalenDrug
Also called UVADEX®
Sterile solution used in conjunction with photopheresis procedure.
Extracorporeal Photopheresis (ECP)Procedure or surgery
Also called THERAKOS® CELLEX® Photopheresis System
| Study group | Role | What they receive |
|---|---|---|
| Methoxsalen with ECP | Experimental | Participants receive methoxsalen 20 µg/ml in conjunction with ECP procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12. Receives: Drug: Methoxsalen; Procedure: Extracorporeal Photopheresis (ECP) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4
OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows:
- CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment
- PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
Measured: 4 weeks
Secondary outcomes (8)
Number of Participants With Adverse Events
Clinically significant changes in vital signs, laboratory values and investigations are reported as adverse events. Summary data are provided below, with details listed in the adverse events module.
Measured: 16 weeks
Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8
OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows:
- CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment
- PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
Measured: 8 weeks
Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12
OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows:
- CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment
- PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
Measured: 12 weeks
Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index
Duration of first response is presented for patients whose disease progressed.
Duration of response is defined in the following way:
Patients whose response failed: Date at which 1st disease progression occurs - date of 1st response +1.
Patients whose response did not relapse: Date of 16 week follow-up or final assessment prior to week 16 (if patient withdrew early) - date of 1st response.
Measured: 16 weeks
Overall Response Rate (ORR) According to the Modified Glucksberg Criteria
ORR is defined as the percentage of patients who achieve an overall response after 4 weeks, 8 weeks, and 12 weeks of ECP treatment according to a scoring algorithm applied to calculate the grade of aGvHD using the modified Glucksberg Criteria.
Measured: 4 weeks, 8 weeks, and 12 weeks
Cumulative Dose of Daily Steroids
Steroids administered from diagnosis of aGvHD to 12 Weeks after initiation of ECP treatment
Measured: From diagnosis of aGvHD to 12 Weeks
Number of Patients With Skin Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria
Number of patients whose skin was rated as Stage 0 - 4 using the modified Glucksberg criteria based on the Graft versus Host Disease (GvHD) rash - Stages are defined as 0=No GvHD rash, 1=Maculopapular rash on <25% body surface area (BSA), 2=Maculopapular rash on 25-50% BSA, 3=Maculopapular rash on >50% BSA, and 4=Generalized erythroderma plus bullous formation, which are blisters bigger than 5 mm across
Measured: at 4, 8 and 12 weeks
Number of Patients With Liver Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria
Number of patients whose liver was rated as Stage 0 - 4 on the modified Glucksberg criteria - Stages are based on level of bilirubin, defined as: Stage 0 = Bilirubin < 2.0 mg/dL, Stage 1 = Bilirubin 2.0-3.0 mg/dL, Stage 2 = Bilirubin 3.1-6.0 mg/dL, Stage 3 = Bilirubin 6.1-15.0 mg/dL, and Stage 4 = Bilirubin > 15.0 mg/dL
Measured: at 4, 8 and 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2015 (estimated)
- Study start20 Jan 2016
- Primary completion16 Jul 2019
- Study completion16 Jul 2019
Why it stopped: Slow enrollment
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.
- Other trials for Steroid Refractory Acute Graft Versus Host DiseaseStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Therakos, Inc., a Mallinckrodt Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Team Leader, Study director, Mallinckrodt
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02524847
- Sponsor's protocol ID
- TKS-2014-001
- Status last verified
- Jul 2020
- First submitted
- 13 Aug 2015
- Last updated
- 11 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Because we work in the rare disease space, to eliminate risk of patient identification we do not share individual patient data. We post summary aggregate results for applicable clinical trials in the registry, and statistical endpoints and discussion in publications; with each referencing the other.
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Single-Arm Study to Assess the Efficacy of UVADEX® (Methoxsalen) Sterile Solution in Conjunction With the THERAKOS® CELLEX® Photopheresis System in Pediatric Patients With Steroid-Refractory Acute Graft-vs-Host Disease (aGvHD)
- ClinicalTrials.gov ID
- NCT02524847
- Acronym / sponsor ID
- TKS-2014-001
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow enrollment
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Therakos, Inc., a Mallinckrodt CompanyIndustry (pharma, biotech, devices)
- Study start
- 20 Jan 2016 (actual)
- Primary completion
- 16 Jul 2019 (actual)
- Study completion
- 16 Jul 2019 (actual)
- First posted
- 17 Aug 2015 (estimated)
- Results
- Posted 11 Sep 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single-arm, open-label, multicenter study of the efficacy of UVADEX® (methoxsalen) Sterile Solution in conjunction with THERAKOS® CELLEX® Photopheresis Systems (ECP) in pediatric participants with steroid-refractory aGvHD. The study is composed of Screening, Treatment, and Follow-up Periods.
Detailed description
Screening:
After the informed consent/assent form (ICF) is signed, the screening assessments will be performed in a single visit to establish the eligibility of the participant, based on inclusion and exclusion criteria, as well as aGvHD grading. Scheduling of the first week of ECP treatments and the arrangements for availability of typed and cross-matched donor packed red blood cells (PRBCs) for transfusion, if required, will be made in advance of participants entering the Treatment Period.
Treatment Period:
Once eligibility is established, participants will enter the 12-week ECP Treatment Period. The availability of typed and cross-matched donor PRBCs for transfusion during treatment, if needed, should be established prior to the scheduling of ECP treatments.
Participants will be allowed to continue standard aGvHD prophylaxis regimens (e.g., cyclosporine, tacrolimus, methotrexate, mycophenolate mofetil) without the addition of new therapies. Participants will be allowed to discontinue prophylaxis regimens for reasons of toxicity, and will also be allowed to switch to another prophylaxis medication within the same class (e.g., the calcineurin inhibitors cyclosporine and tacrolimus) for reasons of toxicity.
All participants enrolled in this trial will have received corticosteroids for the treatment of aGvHD. After entering the treatment period on study, tapering of steroids by total weekly decrements of 12.5% to 25% of the steroid dose at initiation of ECP therapy is permitted after a sustained response of aGvHD has been observed for at least 3 consecutive days, with the suggested goal to decrease the starting steroid dose by at least 50% 4 weeks after initiation of ECP.
Follow-Up Period:
After completion of the 12-week Treatment Period, participants may continue ECP treatment on commercial product at the Principal Investigator's discretion. Acute GvHD status will be assessed 4 weeks after completion of the Treatment Period. Participant survival will be assessed by passive follow-up (chart review) 26 weeks after initiation of ECP treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Methoxsalen with ECP | Experimental | Drug: Methoxsalen, Procedure: Extracorporeal Photopheresis (ECP)Participants receive methoxsalen 20 µg/ml in conjunction with ECP procedure three times per week for Weeks 1 to 4, and two times per week for Weeks 5 to 12. |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- Male or female 1 to 21 years of age at the time of consent
- Steroid-refractory grade B-D aGvHD.
- A Lansky scale Performance Status score ≥ 30
- Laboratory values are within the following limits, assessed within 3 days of the first study treatment:
- For participants with isolated upper GI symptoms, pre-Screening biopsy results to confirm diagnosis of aGvHD
Key exclusion criteria (23 listed)
- Currently enrolled in another clinical trial for the treatment of aGvHD
- Use of any experimental regimens or medication(s) for aGvHD treatment
- Treatment with > 2.0 mg/kg/day of methylprednisolone equivalents for aGvHD within 30 days prior to the first study treatment
- Overt signs of relapse of the underlying condition
- Uncontrolled viral, fungal, or bacterial infection
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 |
|---|---|---|
| MethoxsalenAlso: UVADEX®Sterile solution used in conjunction with photopheresis procedure. | Drug | Methoxsalen with ECP |
| Extracorporeal Photopheresis (ECP)Also: THERAKOS® CELLEX® Photopheresis System | Procedure or surgery | Methoxsalen with ECP |
Primary outcome
Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4
Time frame: 4 weeks
OR using the modified IBMTR Severity Index is defined as complete response (CR) + partial response (PR) as follows: * CR: complete resolution of all signs and symptoms of aGvHD in all evaluable organs without addition of next-line systemic treatment * PR: improvement of 1 stage in 1 or more aGvHD target organs without progression in others and without addition of next-line systemic treatment
8 secondary outcomes
Number of Participants With Adverse Events
Time frame: 16 weeks
Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 8
Time frame: 8 weeks
Percentage of Participants Achieving Overall Response (OR) Using Modified IBMTR Severity Index at Week 12
Time frame: 12 weeks
Duration of Response (Days) Within 16 Weeks Using Modified IBMTR Severity Index
Time frame: 16 weeks
Overall Response Rate (ORR) According to the Modified Glucksberg Criteria
Time frame: 4 weeks, 8 weeks, and 12 weeks
Cumulative Dose of Daily Steroids
Time frame: From diagnosis of aGvHD to 12 Weeks
Number of Patients With Skin Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria
Time frame: at 4, 8 and 12 weeks
Number of Patients With Liver Rated as Stage 0 - 4 Using the Modified Glucksberg Criteria
Time frame: at 4, 8 and 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No. Because we work in the rare disease space, to eliminate risk of patient identification we do not share individual patient data. We post summary aggregate results for applicable clinical trials in the registry, and statistical endpoints and discussion in publications; with each referencing the other.
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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.
Clinical Team Leader study director
Mallinckrodt
32 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Methoxsalen and Extracorporeal Photopheresis (ECP) in people with Steroid Refractory Acute Graft Versus Host Disease, run by Therakos, Inc., a Mallinckrodt Company at 32 sites in 8 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 23 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: stopped early, as of the sponsor's last update on 11 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, Germany, Spain, France, Italy and 3 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 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 Sep 2020). 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?
Number of Participants Achieving Overall Response (OR) Using the Modified International Bone Marrow Transplant Registry (IBMTR) Severity Index at Week 4 (time frame: 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 Sep 2020.