A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Recruiting
Phase 2Interventional Study
Sickle Cell Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
6 Months – 18
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
95 (estimated)
Sponsor
Forma Therapeutics, Inc. · Industry
Who this trial is looking for
This trial is looking for children and adolescents with sickle cell disease. Participants will take a drug called etavopivat for about 24 weeks, followed by a 72-week extended treatment period to check how safe it is and how the body processes it.
Are You a Good Fit for This Trial?
You may be able to join if
I am between 6 months and 18 years old.
I have been diagnosed with sickle cell disease.
My hemoglobin level is between 5.5 and 10.5 grams per deciliter.
I have had severe complications from sickle cell disease.
I have consent from my parent or guardian.
I can give age-appropriate assent to participate.
I have been stable on hydroxyurea for the past 90 days, if applicable.
I agree to use contraception if I could become pregnant or father a child.
You may not be able to join if
I am pregnant or breastfeeding.
I have had more than 15 vaso-occlusive events in the past 12 months.
I was hospitalized for a sickle cell crisis in the last 14 days.
I have had an abnormal transcranial Doppler test in the past 12 months.
I receive regular blood transfusions.
I received any blood products in the last 30 days.
I am taking medications that speed up certain liver enzymes within the last 2 weeks.
I have had a cell-based therapy in the past.
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
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 w…
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.
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Eligibility Criteria
Inclusion Criteria:
* Type of Participant and Disease Characteristics
1. Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent
2. Age greater than or equal to (≥) 6 months and lesser than (\<) 18 yea…
Inclusion Criteria:
* Type of Participant and Disease Characteristics
1. Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent
2. 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
* Cohort 4: age 6 months to \< 2 years
3. 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.
4. Hemoglobin ≥ 5.5 and lesser than or equal to (≤) 10.5 grams per deciliter (g/dL)
5. 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).
6. 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
7. 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
8. 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.
Exclusion Criteria:
* Medical Conditions
1. Female who is breastfeeding or pregnant
2. More than 15 VOCs within the 12 months prior to starting study treatment that required a hospital, emergency room (ER), or clinic visit
3. Hospitalized for sickle cell crisis or other vaso-occlusive event occurring in the 14 days prior to starting study treatment
4. Abnormal TCD in the 12 months prior to starting study treatment
Prior/Concomitant Therapy
5. Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion)
6. Received any blood products within 30 days of starting study treatment
7. Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4/5 within 2 weeks of starting study treatment
8. Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study
9. 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
10. Receipt of prior cellular based therapy (eg, hematopoietic cell transplant, gene modification therapy)
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