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beta-thalassemia intermedia - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

beta-thalassemia intermedia

MONDO:0016487

Find Clinical TrialsTrials

Overview

Beta-thalassemia (BT) intermedia is a form of BT characterized by mild to moderate anemia which does not or only occasionally requires transfusion.

TreatmentsNo approved treatments · 1 in pipeline

Clinical Trials32 registered · 12 recruiting

Diagnosis

Biomarker and diagnostic research for beta-thalassemia intermedia has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for beta-thalassemia intermedia. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for beta-thalassemia intermedia, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for beta-thalassemia intermedia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Estimated prevalence: Unknown (Unknown prevalence).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

32 clinical trials registered, 12 recruiting. Interventions under study include drug therapy, other interventions, biologic therapy, and gene therapy. Pipeline includes 4 PHASE3, 6 PHASE2, 7 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06213402](https://clinicaltrials.gov/study/NCT06213402)

Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 5:31 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:231222

GARD

Genetic and Rare Diseases Info Center

GARD:17163

Questions for Your Care Team

→Are any of the 32 active clinical trials a good fit for my situation?
→What is the current development status of Lentiviral vector encoded with a human beta-globin gene plasmid, and when might it become available?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Research Type	Count	% of Total
Patient case studies	13	37%
Disease patterns and progression	9	26%
Laboratory research	5	14%
Research summaries	3	9%
Testing and diagnosis research	2	6%
Other research	1	3%
Clinical study results	1	3%
New treatment approaches	1	3%

Kisho

beta-thalassemia intermedia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Clinical base editing for β-hemoglobinopathies across different genetic backgrounds.

Gene Therapy Pricing | Rare Disease Access Barriers | Trial Friend

Building a gene editing lexicon: a model for rare and inherited disorders | Gene Therapy

beta-thalassemia intermedia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Clinical base editing for β-hemoglobinopathies across different genetic backgrounds.

Gene Therapy Pricing | Rare Disease Access Barriers | Trial Friend

Building a gene editing lexicon: a model for rare and inherited disorders | Gene Therapy