Alpha-thalassemia Advocacy Hub | UniteRare

At a Glance

Live from database

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8Recruiting trials

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7Specialists

Regulatory Watchboard

10 events

Aug 2024Genetic Profile of Alpha Thalassemia Children at Sohag University Hospital .

Sohag University

TrialRECRUITING

Jun 2024FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

xCures — NA

TrialRECRUITING

Oct 2023Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army — EARLY_PHASE1

TrialRECRUITING

Dec 2022A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia

Bristol-Myers Squibb — PHASE2

TrialRECRUITING

Dec 2021A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

Agios Pharmaceuticals, Inc. — PHASE3

TrialACTIVE NOT RECRUITING

Nov 2021A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Agios Pharmaceuticals, Inc. — PHASE3

TrialACTIVE NOT RECRUITING

Oct 2021In-utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Bart's Hydrops Fetalis Syndrome

Chinese University of Hong Kong — NA

TrialRECRUITING

Sep 2019Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

National Institute of Allergy and Infectious Diseases (NIAID)

TrialRECRUITING

Jul 2018Fetal Electrophysiologic Abnormalities in High-Risk Pregnancies Associated With Fetal Demise

Medical College of Wisconsin

TrialRECRUITING

Apr 2017Screening for Alpha Thalassemia in Healthy Volunteers

National Institute of Allergy and Infectious Diseases (NIAID) — NA

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Alpha-thalassemia.
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Clinical Trial Landscape

8 active trials

2Phase 3

1Phase 2

2N/A

2Unknown

1EARLY_PHASE1

8Total recruiting

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Recent News & Research

No recent news articles indexed yet for Alpha-thalassemia.
Search PubMed for Alpha-thalassemia →

Browse all Alpha-thalassemia news →

Specialist Network

Top 6 by expertise

Tippi C MacKenzie, MD

University of California, San Francisco

📍 San Francisco, California

Score 292 trials

Paul Szabolcs, MD

UPMC Children's Hospital of Pittsburgh

📍 Pittsburgh, Pennsylvania

Score 297 trials

Margaret MacMillan

University of Minnesota Masonic Cancer Center

📍 Minneapolis, Minnesota

Score 81 trial12 pubs

Mark Shapiro, MS

xCures

📍 Los Altos, California

Score 4

Tippi MacKenzie, MD

University of California, San Francisco

📍 San Francisco, California

Score 42 trials

Amy P Ruhl, M.D.

National Institute of Allergy and Infectious Diseases (NIAID)

📍 Bethesda, Maryland

Score 31 trial

View all Alpha-thalassemia specialists →

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