Warm Antibody Autoimmune Hemolytic AnemiaPhase 3NCT03764618
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of wAIHA
A Phase 3 study testing Fostamatinib disodium in people with Warm Antibody Autoimmune Hemolytic Anemia, run by Rigel Pharmaceuticals at 102 sites in 22 countries.
Trial summary
The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA). Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Warm Antibody Autoimmune Hemolytic Anemia
- What is being tested?
- Fostamatinib disodium
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 102 sitesin 22 countries
- Study dates
- 24 Apr 2019to 11 Apr 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 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject must have a diagnosis of primary or secondary warm Antibody Autoimmune Hemolytic Anemia (wAIHA) as documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA.
- Have failed or not tolerated at least one prior wAIHA treatment regimen, including steroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, MMF, danazol, vincristine, ESA or splenectomy (folate, iron or other supplements do not fulfill this criterion).
- Have haptoglobin <LLN or total bilirubin >ULN or lactate dehydrogenase (LDH) >ULN.
- At screening, subject's hemoglobin level must be ≤9 g/dL OR if hemoglobin value >9 g/dL and <10 g/dL, subject must be on an allowed wAIHA treatment AND the subject must have documented symptoms related to anemia (e.g., weakness, dizziness, fatigue, shortness of breath, chest pain).
- Karnofsky performance status (KPS) ≥70.
- Subject's concurrent treatment for wAIHA may consist of no more than two of any of the following agents: azathioprine, steroids, ESAs, mycophenolate mofetil, dapsone or danazol at a stable dose
Who cannot join
- Subject with other types of Antibody Autoimmune Hemolytic Anemia (AIHA) (e.g., cold antibody AIHA, cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold hemoglobinuria).
- Subject has AIHA secondary to autoimmune disease, including systemic lupus erythematosus (SLE), or lymphoid malignancy if the underlying disease is not stable or is not well-controlled on current therapy, per investigator medical judgement.
- Subject has uncontrolled or poorly controlled hypertension, defined as systolic blood pressure ≥135 mmHg or diastolic blood pressure ≥85 mmHg, whether or not the subject is receiving anti-hypertensive treatment.
- Subject has one or more of the following laboratory abnormalities at screening: neutrophil count of <1,000/μL or platelet count of <30,000/μL, unless due to Evans syndrome; transaminase levels (i.e., alanine aminotransferase [ALT] or aspartate aminotransferase [AST]) >1.5 x ULN.
- Has documented active hepatitis B or hepatitis C infection or HIV infection.
Trial locations and contacts
102 sites in 22 countries.
102 sites in 22 countries
United States26 sites
Piedmont Cancer Institute
Atlanta, Georgia, 30318
John Hopkins Bayview Medical Center
Baltimore, Maryland, 21205
Massachusetts General Hospital
Boston, Massachusetts, 02114
Medical University of South Carolina
Charleston, South Carolina, 29425
Affiliated Oncologists
Chicago Ridge, Illinois, 60415
Duke Cancer Network
Clayton, North Carolina, 27520
Texas Oncology - Baylor Research Institute
Dallas, Texas, 75246
Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234
Banner MD Anderson Cancer Center at North Colorado Medical Center
Greeley, Colorado, 80538
Cancer Specialists of North Florida
Jacksonville, Florida, 32256
Moores UC San Diego Cancer Center
La Jolla, California, 92037
Comprehensive Cancer Centers of Nevada
Las Vegas, Nevada, 89169
University of Southern California
Los Angeles, California, 90033
Versiti Wisconsin, Inc.
Milwaukee, Wisconsin, 53226
Rutgers - Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901
American Oncology Network Vista Oncology Division
Olympia, Washington, 98506
Mayo Clinic Hospital
Phoenix, Arizona, 85054
Swedish Cancer Institute
Seattle, Washington, 98104
University of Washington
Seattle, Washington, 98109
Marshfield Clinic Cancer Center - Stevens Point
Stevens Point, Wisconsin, 54482
Montefiore Medical Center
The Bronx, New York, 10461
Harbor UCLA - Lundquist Institute
Torrance, California, 90502
Arizona Oncology Associates, PC--HOPE Division
Tucson, Arizona, 85711
Georgetown University - Lombardi Comprehensive Cancer Center
Washington D.C., District of Columbia, 20007
Clear Lake Specialties, Research Dept.
Webster, Texas, 77598
The Oncology Institute of Hope and Innovation
Whittier, California, 90603
France7 sites
CHU Angers
Angers, 49100
Institut d'hématologie de Basse Normandie, CHU Caen
Caen, 14033
CHU Estaing - Chirurgie digestive et Médecine interne
Clermont-Ferrand, 63003
CHU Henri Mondor
Créteil, 94000
Hôpital Saint Antoine
Paris, 75012
CHU de Bordeaux - GH Sud- Hôpital Haut Lévêque Service Médecine Interne et Maladies Infectieuses
Pessac, 33603
CHU Toulouse, IUCT Oncopôle
Toulouse, 31100
Russia6 sites
National Research Center for Hematology
Moscow, 125167
Botkin Moscow City Clinical Hospital
Moscow, 125284
State Budgetary Healthcare Institution of Moscow city "City Clinical Hospital No 40 Department of Healthcare of Moscow city"
Moscow, 129301
State Budgetary Healthcare Institution of Novosibirsk Region "City Clinical Hospital №2"
Novosibirsk, Russian Federation, 630051
State Budgetary Healthcare Institution Oncology Dispensary No. 2 of the Ministry of Health of the Krasnodar Territory
Sochi, 354057
Regional State Autonomous Healthcare Institution "Tomsk Regional Clinical Hospital"
Tomsk, 634063
Spain6 sites
Hospital Vall d'Hebron
Barcelona, 08035
Hospital Clinic de Barcelona
Barcelona, 08036
Hospital Universitario Gregorio Marañón
Madrid, 28009
Hospital Universitario Puerta de Hierro de Majadahonda
Madrid, 28222
Hospital Universitario Virgen del Rocio
Seville, 41013
Hospital Universitario y Politecnico La Fe - Servicio de Hematología
Valencia, 46026
Bulgaria5 sites
University Multiprofile Hospital for Active Treatment - Dr Georgi Stranski EAD, Pleven, Clinic of Clinical Haematology
Pleven, 5800
University Multiprofile Hospital for Active Treatment 'Alexandrovska' EAD, Clinic of Clinical Haematology
Sofia, 1431
University Multiprofile Hospital for Active Treatment 'Sv. Ivan Rilski' EAD, Clinic of Clinical Hematology, Department of Clinical Hematology
Sofia, 1431
Specialized Hospital for Active Treatment of Hematological Diseases EAD, Sofia, Clinic of Clinical Haematology
Sofia, 1756
University Multiprofile Hospital for Active Treatment 'Sveta Marina' EAD, Varna, Clinic of Clinical Haematology
Varna, 9010
Italy5 sites
ASST degli Spedali Civili di Brescia, Ematologia UOC
Brescia, 25123
Fondazione IRCCS Ca'Granda - Ospedale Maggiore Policlinico di Milano - UO Ematologia
Milan, 20122
SCDU Ematologia AOU "Maggiore della Carità"
Novara, 28100
Ospedale Maggiore, SC Ematologia - Azienda Sanitaria Universitaria Giuliano Isontina
Trieste, 34125
ULSS8 Berica Ospedale San Bortolo - Unità Operativa Complessa di Ematologia
Vicenza, 36100
United Kingdom5 sites
East Kent Haemophilia Centre, Kent and Canterbury Hospital
Canterbury, Kent, CT1 3NG
Roald Dahl Haemostasis and Thrombosis Centre, Royal Liverpool University Hospital, Liverpool University Hospitals NHS Foundation Trust
Liverpool, L7 8XP
Hammersmith Hospital
London
The Royal London Hospital, Bart's Health NHS Trust
London
Sandwell and West Birmingham NHS Trust
West Bromwich, B71 4HJ
Australia4 sites
Princess Alexandra Hospital - Cancer Trials Unit
Brisbane, Queensland, 4201
The Alfred Hospital
Melbourne, Victoria, 3004
Royal Perth Hospital
Perth, Western Australia, 6000
Concord Repatriation General Hospital
Sydney, New South Wales, 2139
Austria4 sites
Universitätsklinik Innsbruck - Innere Medizin V
Innsbruck, 6020
Universitätsklinikum Salzburg, 3.Medizin/Onkologie
Salzburg, 5020
Medizinsche Universität Wien
Vienna, 1090
Hanusch-Krankenhaus
Vienna, 1140
Belgium3 sites
Ziekenhuis Network Antwerp, Stuivenberg
Antwerp, 2060
Universitair Ziekenhuis Antwerpen - Hematologie
Edegem, 2650
AZ Nikolaas
Sint-Niklaas, 9100
Canada3 sites
Hamilton Health Sciences- McMaster University Medical Centre
Hamilton, Ontario, L8S 4K1
The Ottawa Hospital
Ottawa, Ontario, K1H8L6
St. Michael's Hospital
Toronto, Ontario, M5B 1W8
Czechia3 sites
Fakultni nemocnice Brno Interni hematologicka a onkologicka klinika
Brno, 625 00
Fakultni nemocnice Ostrava Klinika hematoonkologie
Ostrava, 70852
Ustav Hematologie a Krevni Transfuze
Prague, 128 00
Denmark3 sites
Aalborg University Hopital
Aalborg, 9000
Aarhus University Hospital - Dept of Hematology
Aarhus N, 8200
Herlev and Gentofte Hospital
Herlev, DK-2730
Hungary3 sites
Semmelweis Egyetem, Általános Orvostudományi Kar, I.sz. Belgyógyászati Klinika, Haematológiai Osztály,
Budapest, 1083
Szabolcs-Szatmár- Bereg Megyei Kórházak és Egyetemi Oktatókórház, Jósa András Oktatókórház
Nyíregyháza, 4400
Pécsi Tudományegyetem, Klinikai Központ, I.számú Belgyógyászati Klinikai, Hematológiai Tanszék
Pécs, 7624
Norway3 sites
Ålesund Hospital
Ålesund, 6017
Haukeland University Hospital
Bergen, 5021
Østfold Hopital
Grålum, 1714
Romania3 sites
Coltea Clinical Hospital
Bucharest, 030171
Emergency University Hospital Bucharest
Bucharest, 050098
"Prof. Dr. Ion Chiricuta" Institute of Oncology Cluj-Napoca
Cluj-Napoca, 400015
Serbia3 sites
University Hospital Medical Center "Bezanijska Kosa", Department of Hematology
Belgrade, 11080
Clinical Center Nis, Clinic for Hematology
Niš, 18000
Clinical Centre of Vojvodina, Clinic for Hematology
Novi Sad, 21000
Ukraine3 sites
Cherkasy Regional Oncology Dispensary, Hematology Center
Cherkasy, 18009
City Clinical Hospital № 4, Hematology Center
Dnipro, 49102
Kyiv City Clinical Hospital №9, hematology department №1
Kyiv, 04112
Belarus2 sites
Vitebsk Regional Clinical Hospital
Vitebsk, 210037
Vitebsk Regional Clinical Oncology Dispensary
Vitebsk, 210603
Georgia2 sites
M. Zodelava Hematology Centre, Tbilisi
Tbilisi, 0112
LTD Multiprofile Clinic Consilium Medulla
Tbilisi, 0186
Germany2 sites
Universitätsklinikum Essen
Essen, 45147
University of Leipzig Medical Center, Medical Department I - Haematology and Cell Therapy, Medical Oncology, Hemophilia
Leipzig, 04103
About this study
Official title: A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).
- Being tested
- Fostamatinib disodium, Placebo
- Keywords
- wAIHA, Warm Antibody Autoimmune Hemolytic Anemia, Warm Autoimmune Hemolytic Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fostamatinib disodiumDrug
Also called R935788, Fostamatinib
Fostamatinib (100mg PO bid or 150 mg PO bid)
The dose may be reduced at any time to a dose as low as fostamatinib 100 mg PO qd or matching placebo if dose limiting adverse events are observed.
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Fostamatinib | Experimental | Initial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to fostamatinib 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator. Receives: Drug: Fostamatinib disodium |
| Placebo | Placebo comparator | Initial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to placebo 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Durable Hemoglobin Response
Proportion of subjects achieving a hemoglobin level ≥ 10 g/dL with an increase from Baseline in hemoglobin level of ≥ 2 g/dL on 3 consecutive available visits during the 24-week treatment period.
Measured: 24 Weeks
Secondary outcomes (5)
A Hemoglobin Response by Week 24
Proportion of subjects with a hemoglobin response by Week 24.
Measured: 24 weeks
Change From Baseline in Hemoglobin Level of 2 g/dL or Greater
Proportion of subjects with change from baseline in hemoglobin level of 2 g/dL or greater.
Measured: 24 weeks
Change in Hemoglobin From Baseline to End of Treatment
Change in mean hemoglobin from baseline to end of treatment.
Measured: 24 weeks
Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4
Proportion of subjects free of rescue AIHA regimens used after Week 4.
Measured: 24 weeks
Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F)
Change from Baseline to Week 24 in Functional Assessment of Chronic Illness Therapy - Fatigue scale (FACIT-F). The FACIT-F scale is a short, 13-item, easy to administer tool that measures an individual's level of fatigue during their usual daily activities over the past week. Each item is rated using a 5-point Likert-type scale. The scale range is 0 to 52, with 0 being the worst possible score and 52 being the best possible score indicating no fatigue. Total Score = [Sum of item scores] x [N of items in subscale] ÷ [N of items answered].
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Dec 2018
- Study start24 Apr 2019
- Primary completion11 Apr 2022
- Study completion11 Apr 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.
- Other trials for Warm Antibody Autoimmune Hemolytic AnemiaStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rigel Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03764618
- Sponsor's protocol ID
- C-935788-057
- Status last verified
- May 2023
- First submitted
- 3 Dec 2018
- Last updated
- 25 May 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
- ClinicalTrials.gov ID
- NCT03764618
- Acronym / sponsor ID
- C-935788-057
- Keywords
- wAIHA, Warm Antibody Autoimmune Hemolytic Anemia, Warm Autoimmune Hemolytic Anemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- Rigel PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 24 Apr 2019 (actual)
- Primary completion
- 11 Apr 2022 (actual)
- Study completion
- 11 Apr 2022 (actual)
- First posted
- 5 Dec 2018 (actual)
- Results
- Posted 25 May 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).
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 |
|---|---|---|
| Fostamatinib | Experimental | Drug: Fostamatinib disodiumInitial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to fostamatinib 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator. |
| Placebo | Placebo comparator | Drug: PlaceboInitial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to placebo 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Subject must have a diagnosis of primary or secondary warm Antibody Autoimmune Hemolytic Anemia (wAIHA) as documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA.
- Have failed or not tolerated at least one prior wAIHA treatment regimen, including steroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, MMF, danazol, vincristine, ESA or splenectomy (folate, iron or other supplements do not fulfill this criterion).
- Have haptoglobin <LLN or total bilirubin >ULN or lactate dehydrogenase (LDH) >ULN.
- At screening, subject's hemoglobin level must be ≤9 g/dL OR if hemoglobin value >9 g/dL and <10 g/dL, subject must be on an allowed wAIHA treatment AND the subject must have documented symptoms related to anemia (e.g., weakness, dizziness, fatigue, shortness of breath, chest pain).
- Karnofsky performance status (KPS) ≥70.
Key exclusion criteria (5 listed)
- Subject with other types of Antibody Autoimmune Hemolytic Anemia (AIHA) (e.g., cold antibody AIHA, cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold hemoglobinuria).
- Subject has AIHA secondary to autoimmune disease, including systemic lupus erythematosus (SLE), or lymphoid malignancy if the underlying disease is not stable or is not well-controlled on current therapy, per investigator medical judgement.
- Subject has uncontrolled or poorly controlled hypertension, defined as systolic blood pressure ≥135 mmHg or diastolic blood pressure ≥85 mmHg, whether or not the subject is receiving anti-hypertensive treatment.
- Subject has one or more of the following laboratory abnormalities at screening: neutrophil count of <1,000/μL or platelet count of <30,000/μL, unless due to Evans syndrome; transaminase levels (i.e., alanine aminotransferase [ALT] or aspartate aminotransferase [AST]) >1.5 x ULN.
- Has documented active hepatitis B or hepatitis C infection or HIV 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 |
|---|---|---|
| Fostamatinib disodiumAlso: R935788, FostamatinibFostamatinib (100mg PO bid or 150 mg PO bid) The dose may be reduced at any time to a dose as low as fostamatinib 100 mg PO qd or matching placebo if dose limiting adverse events are observed. | Drug | Fostamatinib |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
Durable Hemoglobin Response
Time frame: 24 Weeks
Proportion of subjects achieving a hemoglobin level ≥ 10 g/dL with an increase from Baseline in hemoglobin level of ≥ 2 g/dL on 3 consecutive available visits during the 24-week treatment period.
5 secondary outcomes
A Hemoglobin Response by Week 24
Time frame: 24 weeks
Change From Baseline in Hemoglobin Level of 2 g/dL or Greater
Time frame: 24 weeks
Change in Hemoglobin From Baseline to End of Treatment
Time frame: 24 weeks
Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4
Time frame: 24 weeks
Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F)
Time frame: 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 May 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
102 sites in 22 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Fostamatinib disodium in people with Warm Antibody Autoimmune Hemolytic Anemia, run by Rigel Pharmaceuticals at 102 sites in 22 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 6 inclusion and 5 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 25 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 102 sites in United States, France, Russia, Spain, Bulgaria and 17 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 25 May 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?
Durable Hemoglobin Response (time frame: 24 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 25 May 2023.