A Phase 2 interventional study of Etavopivat in Sickle Cell Disease, sponsored by Forma Therapeutics, Inc.. Recruiting at 18 sites in 7 countries. Open to participants aged 6 Months to 18 Years. Per ClinicalTrials.gov, last updated 2026-09-18.
Sponsored by Forma Therapeutics, Inc. (part of Novo Nordisk A/S) · Phase 2, Interventional, and Treatment
The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD). Participants will receive etavopivat and will be enrolled in a staggered manner, starting with the oldest age group and followed sequentially by younger cohorts after review of pharmacokinetic and safety data from the preceding cohort. All participants will undergo a 24-week primary treatment period followed by a 72-week extension treatment period to further evaluate long-term safety and pharmacokinetics of etavopivat. The total duration of the study will be approximately 96 weeks.
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's planned enrollment of 95 is above the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Forma Therapeutics, Inc. is the lead sponsor of 10 studies on the registry; 2 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 3 (60%) have results posted.
First submitted after Forma Therapeutics, Inc. became part of Novo Nordisk A/S.
Counted across the registry records on this site, refreshed daily.
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.
Type of Participant and Disease Characteristics
Age greater than or equal to (≥) 6 months and lesser than (\<) 18 years of age at time of enrollment, according to the enrolling cohort:
Patient has confirmed diagnosis of SCD
Pediatric patients with severe SCD, as defined by at least 1 of the following:
Patients on crizanlizumab or L-glutamine treatment at the time of consent may be eligible if they:
Exclusion Criteria:
Medical Conditions
Abnormal TCD in the 12 months prior to starting study treatment
Prior/Concomitant Therapy
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.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
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.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants aged 12 to less than 18 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group.
Drug: Etavopivat
Participants aged 6 to less than 12 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group.
Drug: Etavopivat
Participants aged 2 to less than 6 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in younger children.
Drug: Etavopivat
Participants aged 6 months to less than 2 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in infants.
Drug: Etavopivat
Participants will receive oral tablets or granules of etavopivat once daily.
Single-dose: maximum plasma concentration (Cmax)
Time frame: During the 24-week primary treatment period
Single-dose: area under the plasma concentration time curve from dosing (time 0) to time t ((AUC)0-t)
Time frame: During the 24-week primary treatment period
Single-dose: area under the plasma concentration time curve from zero to time infinity (AUC0-inf)
Time frame: During the 24-week primary treatment period
Steady-state maximum plasma concentration (Cmax,ss)
Time frame: During the 24-week primary treatment period
Steady-state area under the concentration time curve over the dosing interval (AUCtau,ss)
Time frame: During the 24-week primary treatment period
Steady-state average plasma concentration (Cavg,ss)
Time frame: During the 24-week primary treatment period
Steady-state minimum plasma concentration (Cmin,ss)
Time frame: During the 24-week primary treatment period
Incidence of adverse events (AEs), serious adverse events (SAEs), and AEs related to etavopivat
Time frame: During the 24-week primary treatment period
Number of premature discontinuations
Time frame: During the 24-week primary treatment period
Number of dose interruptions
Time frame: During the 24-week primary treatment period
Number of dose reductions
Time frame: During the 24-week primary treatment period
Incidence of AEs, SAEs, and AEs related to etavopivat
Time frame: During the 72-week treatment extension period
Number of premature discontinuations
Time frame: During the 72-week treatment extension period
Number of dose interruptions
Time frame: During the 72-week treatment extension period
Number of dose reductions
Time frame: During the 72-week treatment extension period
Hemoglobin (Hb) response rate
Time frame: Baseline, week 12 and 24
Change in Hb from baseline
Time frame: Baseline, week 12 and 24
Change from baseline in number of vaso-occlusive crises (VOCs)
Time frame: Baseline and week 24
Change from baseline in Annualized Rate of VOC
Time frame: Baseline and week 24
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Scale
PROMIS is a 10-item patient-reported health outcome measurement system used to evaluate quality of life in both children and adults. The assessment is based on the responses - 1. Never, 2. Almost never, 3. Sometimes, 4. Often and 5. Almost always. 'Never' response on the PROMIS fatigue scale indicates better quality of life.
Time frame: Baseline, week 12 and 24
Change from baseline in time-averaged mean of the maximum velocity (TAMMV) by transcranial Doppler ultrasonography (TCD)
Time frame: Baseline, week 24, 48, and 96
Not enrolling at this site.
Not enrolling at this site.
Not enrolling at this site.
Plan to share: Yes
No publications or documents are linked to this record.
Started Jan 2023 (actual). Primary completion is estimated for Feb 2028, 61 months after the start.
Of 34 industry-sponsored phase 2 interventional studies under Anemia, Sickle Cell, half reached primary completion within 28 months of starting (the median), counting those still running; a quarter within 16 and three quarters within 41. This study's planned 61 months is above that.
Counting every sponsor, half of 92 studies reached it within 46 months; a quarter within 24 and three quarters within 73.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Etavopivat