An observational study in Alpha-Thalassemia, Beta-Thalassemia and Amyloidosis, sponsored by xCures. Recruiting at 1 site in United States. Per ClinicalTrials.gov, last updated 2024-11-14.
Sponsored by xCures · Observational
Among 91 studies in beta-Thalassemia with a site in United States — 22 open to participants now, 36 under way, 8 of them phase 3. beta-Thalassemia · United States.
Among the 91 studies in beta-Thalassemia with a site in United States, this one is the 5th largest by planned enrollment of the 17 observational ones, is one of 4 that started in 2024, is one of 17 observational ones, reaches primary completion later than 9 of the 35 other live ones, and was the 84th registered.
294 studies on the registry are indexed under beta-Thalassemia; 69 are open to participants now.
This study's planned enrollment of 1,000 is above the median of 100 across 75 observational studies indexed under beta-Thalassemia.
Browse beta-Thalassemia studies →22 of the 91 studies in beta-Thalassemia with a site in United States are open now, in 12 countries. Each count lists the studies.
Of the 43 beta-Thalassemia studies recruiting now, 10 (23%) set no upper age limit and 31 (72%) stop at 75 or younger; 34 take children under 18; 6 take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →91 studies; the count open now beside each. This study's row is marked.
First in 1995; this study’s year is marked. 4 started in 2024.
Median enrollment 25 across the 71 interventional studies, 150 across the 17 observational. Of the 50 that completed or were terminated, 17 (34%) have results posted.
42 of 91 have an industry lead sponsor. The 10 most frequent:
xCures is the lead sponsor of 6 studies on the registry; 3 are open to participants now.
xCures has 1 study filed under beta-Thalassemia — fewer than ten, so there is no comparison to draw.
11 of the studies in beta-Thalassemia with a site in United States that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05477563Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | 2027-06-09 |
| NCT05356195Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) | 2027-11-14 |
| NCT01962415Paul SzabolcsReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | 2026-11 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
| NCT06421636Regeneron PharmaceuticalsA Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body | 2026-12-03 |
Among the 91 studies in beta-Thalassemia with a site in United States, from their registry updates:
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA/RNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic/digitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.
This study does not require data entry by treating site staff or physicians. Centralized data structuring is completed by xCures study staff. Data elements are aggregated, normalized, and coded to OMOP-based ontologies (SNOMED, LOINC, ICD-10, CTCAE, RxNorm, and MedDRA) in one process, permitting standardization of verbatim terms from medical records. The data collection platform supports 21 CFR Part 11-compliant data annotation with formal QC/QA process, medical review, and source data verification.
Beyond EMR data, raw DICOM images (MRI, CT files) can be collected from all sites of care and anonymized for integration with the clinical data. Molecular profiling and somatic or germline genomics results, and biochemical lab data, when available, are collected from commercial and academic sources and centralized. Additionally, patient- and caregiver-reported outcome surveys (PROs) can be collected to supplement information not found in clinical records.
Together, these clinical, imaging, biomarker, and assessment data will provide a comprehensive and longitudinal documentation of rare diseases in near real-time in a single observational basket study.
Traditional rare disease research registries rely on patients reporting many aspects of their condition via surveys or rely on key opinion leaders at specific institutions managing a team to enroll patients and annotate necessary data. These put unnecessary burdens on patients and strain limited research resources at medical centers. Gathering the necessary data and in sufficient quantities is often a limitation to successfully defining the natural history of a rare disease.
This study will include adult and pediatric patients with known or suspected rare disease.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Alpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Overall Survival (OS)
Time frame: 5 Years
Safety/tolerability of medications
Time frame: 5 years
Changes in normal development milestones
Time frame: 5 years
Changes in functional status
Time frame: 5 years
Changes in motor function
Time frame: 5 years
Changes in symptoms or clinical status
Time frame: 5 years
Started Jun 2024 (actual). Primary completion is estimated for Jun 2026, 24 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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