Health Trials

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

ConditionsSickle Cell DiseaseThalassemia
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2+
SponsorNovo Nordisk A/S

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study.

Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator.

Any participant with dose reduction or temporary discontinuation will need to be successfully rechallenged to the full dose of etavopivat before transferring.

Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they have been on a stable dose in the parent study as defined at the investigator's discretion. Necessary adjustments related to weight or age are accepted. Participants with temporary dose reductions or pauses due to medical reasons may still be considered to have a stable dose, as determined by the investigator, who will assess the impact of these adjustments based on clinical context and the participant's overall health status.

Disqualifiers

Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.

Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study.

Participants on permanent dose reduction (greater than [>] 28 days or more) or ongoing temporary treatment discontinuation.

Use of haemoglobin S (HbS) polymerisation inhibitors within participation of the parent study or anticipated need for this agent during this study.

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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.

Treatment groups

480 Participants
are divided into 5 treatment groups
Group A: Participants greater than or equal to (≥) 12 years old with sickle cell diseaseExperimental treatment 2 interventions
Group B: Participants ≥ 12 years old with sickle cell disease transfusion dependentExperimental treatment 2 interventions
Group C: Participants ≥ 12 years old with transfusion-dependent thalassaemiaExperimental treatment 2 interventions
Group D: Participants ≥ 12 years old with non-transfusion dependent thalassaemiaExperimental treatment 2 interventions
Group E: Participants ≥ 2 years to less than (<) 12 years old with sickle cell diseaseExperimental treatment 3 interventions

Trial outcomes

Primary outcomes

1

Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately

Measured as number of events.

Time frame
Baseline (week 0 of FLORAL) up to end of study (up to week 316)
2

Number of adverse reactions, reported for each indication and age group separately

Measured as number of adverse reactions.

Time frame
Baseline (week 0 of FLORAL) up to end of study (up to week 316)

Secondary outcomes

1

Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately

Measured as count.

Time frame
Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)
2

Change in VOCs, reported for each age group separately

Measured as count.

Time frame
Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
3

Change in hemoglobin (Hb) concentration, reported for each age group separately

Measured as grams per deciliter (g/dL).

Time frame
Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
4

Annualised number of hospitalisations, reported for each age group separately

Measured as count.

Time frame
Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Novo Nordisk A/S

Lead sponsor

Source ClinicalTrials.gov
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