Sickle Cell DiseasePhase 2HIBISCUS KIDSNCT06198712
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
A Phase 2 study testing Etavopivat in people with Sickle Cell Disease, run by Forma Therapeutics, Inc. at 18 sites in 7 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Etavopivat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 95 participants
- Where
- 18 sitesin 7 countries
- Study dates
- 12 Jan 2023to 8 Aug 2029
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Type of Participant and Disease Characteristics
- Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent
- Age greater than or equal to (≥) 6 months and lesser than (<) 18 years of age at time of enrollment, according to the enrolling cohort:
- Cohort 1: age 12 to < 18 years (adolescents)
- Cohort 2: age 6 to < 12 years
- Cohort 3: age 2 to < 6 years
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- Cohort 4: age 6 months to < 2 years
- Patient has confirmed diagnosis of SCD
- Documentation of SCD genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing. Molecular genotyping is not required. SCD genotype may be determined from the results of Hb electrophoresis, high-performance liquid chromatography (HPLC), or similar testing. Note that Hb electrophoresis is performed by the local laboratory at Screening.
- Hemoglobin ≥ 5.5 and lesser than or equal to (≤) 10.5 grams per deciliter (g/dL)
- Pediatric patients with severe SCD, as defined by at least 1 of the following:
- 2-15 episodes of documented VOC within the 12 months prior to screening. Documentation must exist in the patient's medical record prior to screening. Events based solely on patient recall without supporting documentation should not be counted towards eligibility.
- Hospitalization for any SCD-related complication in the last 12 months prior to starting study treatment
- Proteinuria, defined as an albumin:creatinine ratio (ACR) > 100 mg/g on 2 measures (separated by ≥ 1 month) as an indicator of early renal disease
- History of a conditional TCD in the last 12 months prior to starting study treatment, but not currently being treated with chronic transfusion therapy (applicable to participants > 2 years of age). Conditional TCD is defined as a TAMMV of 170-199 cm/s by TCD or 155-184 cm/s by imaging TCD (TCDi).
- For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator
- Patients on crizanlizumab or L-glutamine treatment at the time of consent may be eligible if they:
- Have been on a stable dose for ≥ 12 months at the time of consent (ie, no changes to the dose except for changes to weight or for safety reasons)
- For patients on crizanlizumab, have been ≥ 80% compliant with the planned regimen during the 12 months prior to the time of consent
- Female patients of childbearing potential who are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and male patients who are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug.
Who cannot join
- Medical Conditions
- Female who is breastfeeding or pregnant
- More than 15 VOCs within the 12 months prior to starting study treatment that required a hospital, emergency room (ER), or clinic visit
- Hospitalized for sickle cell crisis or other vaso-occlusive event occurring in the 14 days prior to starting study treatment
- Abnormal TCD in the 12 months prior to starting study treatment
- Prior/Concomitant Therapy
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- Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
- Received any blood products within 30 days of starting study treatment
- Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4/5 within 2 weeks of starting study treatment
- Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study
- Receipt of erythropoietin or other hematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study
- Receipt of prior cellular based therapy (eg, hematopoietic cell transplant, gene modification therapy)
Trial locations and contacts
18 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
18 sites in 7 countries
Kenya4 sites, 4 recruiting
- Recruiting
KEMRI-Walter-Reed Kericho
Kericho, 20200
- Recruiting
Kombewa Clinical Research Centre
Kisumu, 40100
- Recruiting
Ahero Clinical Trials Unit
Kisumu, 40101
- Recruiting
KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital
Siaya, Siaya County, 40600
France3 sites, 3 recruiting
- Not yet recruiting
HCL-HOPITAL LYON SUD_Institut d'Hématologie et d'Oncologie Pédiatrique (IHOPe)
Lyon, 69008
- Not yet recruiting
APHP - Centre de Référence des Syndromes
Paris, 75019
- Not yet recruiting
Centre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle
Roeun, 76031
Nigeria3 sites, 3 recruiting
- Recruiting
University of Nigeria Teaching Hospital (UNTH)
Ituku-Ozalla, Enugu State, 400001
- Recruiting
Lagos University Teaching Hospital, Lagos
Lagos, 102215
- Recruiting
Aminu Kano Teaching Hospital (AKTH)
Tarauni, 700101
United Kingdom3 sites, 3 recruiting
- Recruiting
Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital
London, SE1 7EH
- Recruiting
King's College Hospital - Alex Mowat Research Hub
London, SE5 9RS
- Recruiting
Manchester Royal Infirmary_1
Manchester, M13 9WL
Lebanon2 sites, 2 recruiting
- Recruiting
American University of Beirut Medical center
Beirut, 1107 2020
- Recruiting
Hospital Nini
Tripoli, 113-6044
Turkey (Türkiye)2 sites
- Completed
Hacettepe University pediatric hematology
Ankara, 06100
- Completed
Acıbadem Adana Hastanesi
Seyhan, 1130
About this study
Official title: A Single Arm, Open Label, Phase 1/2 Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
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.
- Conditions
- Sickle Cell Disease
- Being tested
- Etavopivat
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EtavopivatDrug
Participants will receive oral tablets or granules of etavopivat once daily.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Etavopivat (12 to less than [<] 18 years) | Experimental | Participants aged 12 to less than 18 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group. Receives: Drug: Etavopivat |
| Cohort 2: Etavopivat (6 to <12 years) | Experimental | Participants aged 6 to less than 12 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in this age group. Receives: Drug: Etavopivat |
| Cohort 3: Etavopivat (2 to <6 years) | Experimental | Participants aged 2 to less than 6 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in younger children. Receives: Drug: Etavopivat |
| Cohort 4: Etavopivat (6 months to <2 years) | Experimental | Participants aged 6 months to less than 2 years with sickle cell disease will receive etavopivat to evaluate pharmacokinetics and safety in infants. Receives: Drug: Etavopivat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Single-dose: maximum plasma concentration (Cmax)
Measured: 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)
Measured: During the 24-week primary treatment period
Single-dose: area under the plasma concentration time curve from zero to time infinity (AUC0-inf)
Measured: During the 24-week primary treatment period
Steady-state maximum plasma concentration (Cmax,ss)
Measured: During the 24-week primary treatment period
Steady-state area under the concentration time curve over the dosing interval (AUCtau,ss)
Measured: During the 24-week primary treatment period
Steady-state average plasma concentration (Cavg,ss)
Measured: During the 24-week primary treatment period
Steady-state minimum plasma concentration (Cmin,ss)
Measured: During the 24-week primary treatment period
Incidence of adverse events (AEs), serious adverse events (SAEs), and AEs related to etavopivat
Measured: During the 24-week primary treatment period
Number of premature discontinuations
Measured: During the 24-week primary treatment period
Number of dose interruptions
Measured: During the 24-week primary treatment period
Number of dose reductions
Measured: During the 24-week primary treatment period
Secondary outcomes (10)
Incidence of AEs, SAEs, and AEs related to etavopivat
Measured: During the 72-week treatment extension period
Number of premature discontinuations
Measured: During the 72-week treatment extension period
Number of dose interruptions
Measured: During the 72-week treatment extension period
Number of dose reductions
Measured: During the 72-week treatment extension period
Hemoglobin (Hb) response rate
Measured: Baseline, week 12 and 24
Change in Hb from baseline
Measured: Baseline, week 12 and 24
Change from baseline in number of vaso-occlusive crises (VOCs)
Measured: Baseline and week 24
Change from baseline in Annualized Rate of VOC
Measured: 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.
Measured: Baseline, week 12 and 24
Change from baseline in time-averaged mean of the maximum velocity (TAMMV) by transcranial Doppler ultrasonography (TCD)
Measured: Baseline, week 24, 48, and 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2024
- Study start12 Jan 2023
- Primary completion23 Feb 2028 (estimated)
- Study completion8 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 95 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Forma Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Novo Nordisk A/S. This study is currently undergoing a sponsor transition from Forma Therapeutics, Inc. to Novo Nordisk A/S.
- Responsible party
- Sponsor
- Investigators
- Clinical Transparency (dept. 2834), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06198712
- Sponsor's protocol ID
- 4202-HEM-202
- Other IDs
- 2022-001689-36, 2024-519881-32
- Acronym
- HIBISCUS KIDS
- Status last verified
- Sep 2026
- First submitted
- 27 Dec 2023
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes