A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Conditions

• Sickle Cell Disease
• Thalassemia

Eligibility Criteria

Sex

ALL

Age

2 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Etavopivat A — DRUG
  Participants will receive an oral dose of Etavopivat A.
• Etavopivat B — DRUG
  Participants will receive an oral dose of Etavopivat B.
• Etavopivat C — DRUG
  Participants will receive an oral dose of Etavopivat C.

Study Details

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

Key Dates

Start date

Jan 10, 2025

Status verified

Jun 2026

Primary completion

Dec 30, 2030

Completion

Dec 30, 2030

Study Design

Enrollment

480 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Participants greater than or equal to (≥) 12 years old with sickle cell disease
  Participants will receive an oral dose of Etavopivat A or C.
• Experimental: Participants ≥ 12 years old with sickle cell disease transfusion dependent
  Participants will receive an oral dose of Etavopivat A or C.
• Experimental: Participants ≥ 12 years old with transfusion-dependent thalassaemia
  Participants will receive an oral dose of Etavopivat A or C.
• Experimental: Participants ≥ 12 years old with non-transfusion dependent thalassaemia
  Participants will receive an oral dose of Etavopivat A or C.
• Experimental: Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease
  Participants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants \< 12 years of age will receive an oral dose of Etavopivat B.

Primary Outcome Measure

Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately [ Time Frame: Baseline (week 0 of FLORAL) up to end of study (up to week 316) ]

Central Contacts

• Novo Nordisk
  (+1) 866-867-7178
  [email protected]

Locations (39)

Find similar trials in Birmingham, AL

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