Daprodustat for Pediatric Anemia

(ASCEND-P Trial)

No longer recruiting at 67 trial locations

Overseen By EU GSK Clinical Trials Call Center

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: GlaxoSmithKline

Disqualifiers: Kidney transplant, Malignancy, Hypertension, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of daprodustat in treating anemia (a shortage of red blood cells) in children and teens with chronic kidney disease (CKD). Researchers aim to understand how the body processes the drug and assess its safety and effectiveness over a year. The trial consists of two parts: one for participants not on dialysis and another for those who are. Children and teens with CKD-related anemia, whether on dialysis or not, might be suitable candidates. As a Phase 3 trial, this is the final step before FDA approval, offering participants an opportunity to contribute to a potentially groundbreaking treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that daprodustat is likely to be safe for children and adolescents with anemia associated with chronic kidney disease?

Research has shown that daprodustat is generally safe for people with anemia due to chronic kidney disease. One study found that serious heart problems were slightly less common in patients taking daprodustat (25.2%) compared to those who were not (26.7%), indicating that daprodustat does not significantly increase heart risks. Another study found that daprodustat effectively increased hemoglobin levels, which is important for managing anemia, without major safety issues. These results suggest that daprodustat is relatively safe, with manageable side effects. However, this trial focuses on children, and safety for them might differ. Discuss potential risks and benefits with a healthcare provider. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Daprodustat is unique because it targets anemia differently than traditional treatments like erythropoiesis-stimulating agents (ESAs). While ESAs work by directly stimulating red blood cell production, daprodustat operates by stabilizing hypoxia-inducible factors (HIFs), which play a role in the body's response to low oxygen levels, thereby enhancing natural erythropoiesis. This novel mechanism could offer a more balanced approach to treating anemia, potentially reducing the risks associated with the overproduction of red blood cells. Researchers are excited about daprodustat because it may provide a safer and more effective option for managing pediatric anemia.

What evidence suggests that daprodustat might be an effective treatment for pediatric anemia associated with CKD?

Research has shown that daprodustat effectively treats anemia in people with chronic kidney disease (CKD). One study found that it kept hemoglobin levels stable over 24 weeks in patients, whether they were starting treatment or switching from other therapies. Another study showed that hemoglobin levels increased by 0.28 grams per deciliter from the start to weeks 28 through 52. These results suggest that daprodustat can help manage anemia in CKD by improving hemoglobin levels. These findings provide strong evidence of daprodustat's effectiveness for this condition. ^{1 2 3 4 6}

Who Is on the Research Team?

GSK Clinical Trials

Principal Investigator

GlaxoSmithKline

Are You a Good Fit for This Trial?

This trial is for children and adolescents aged 3 months to under 18 years with anemia due to chronic kidney disease (CKD) stages 3-5, not on dialysis, or those on dialysis. Participants must have specific hemoglobin levels depending on their treatment history with erythropoiesis stimulating agents.

Inclusion Criteria

I have anemia due to chronic kidney disease and meet the specified hemoglobin levels.

Written informed consent or assent as appropriate

I am between 3 months and 18 years old.

Exclusion Criteria

Transferrin saturation (TSAT) < 20 percent (%), or Ferritin <25 nanogram (ng)/milliliter (mL)

Active hemolysis

I am currently on medication for an infection.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

Up to 4 weeks

Pharmacokinetics (PK) Evaluation

Evaluation of pharmacokinetics of daprodustat, including plasma concentration measurements

4 weeks

Regular visits for blood sample collection

Treatment

Participants receive daprodustat for safety and hemoglobin response evaluation

52 weeks

Regular visits for safety and efficacy assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Daprodustat

Trial Overview The study tests Daprodustat's effects over a year in managing anemia in young patients with CKD, both undergoing and not undergoing dialysis. It will measure how the body processes the drug, its safety, and its impact on hemoglobin levels across different age groups.

How Is the Trial Designed?

Group I: Daprodustat Experimental Treatment 1 Intervention

All participants will receive daprodustat for up to 52 weeks.

Who Is Running the Clinical Trial?

GlaxoSmithKline

Lead Sponsor

Trials

4,834

Recruited

8,389,000+

Headquarters

London, UK

Known For

Vaccines & Medicines

Top Products

**Advair (salmeterol, fluticasone propionate)**, **Shingrix (shingles vaccine)**, **Augmentin (amoxicillin/clavulanate potassium)**, **Ventolin (salbutamol sulfate)

Dame Emma Walmsley

GlaxoSmithKline

Chief Executive Officer since 2017

MA in Classics and Modern Languages from Oxford University

Dr. Hal Barron

GlaxoSmithKline

Chief Medical Officer since 2018

MD from Harvard Medical School

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S246802492300387X

WCN23-0672 DAPRODUSTAT FOR TREATMENT OF ... The ASCEND-P single arm basket trial of daprodustat for anemia of CKD will evaluate the safety and efficacy of daprodustat for the treatment of renal anemia in ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6366145/

Daprodustat for anemia: a 24-week, open-label ... Daprodustat effectively maintained target hemoglobin over 24 weeks in CKD patients with anemia who were rhEPO naïve or had switched from existing rhEPO therapy.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05682326?term=DAPRODUSTAT&viewType=Table&rank=5

Anemia Studies in CKD: Erythropoiesis Via a Novel PHI ... Members are chosen based on the scientific skills and knowledge needed to monitor the particular trial. Also called a data safety and monitoring board, or DSMB.

4. nejm.org nejm.org/doi/full/10.1056/NEJMoa2113379

Daprodustat for the Treatment of Anemia in Patients ... Primary Efficacy Outcome. The mean (±SE) change in the hemoglobin level from baseline to weeks 28 through 52 was 0.28±0.02 g per deciliter ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11143073/

Efficacy of daprodustat for patients on dialysis with anemia The eligible trials provided data about safety and efficacy outcomes associated with each dose of daprodustat, rhEPO, and placebo. Our ...

6. jamanetwork.com jamanetwork.com/journals/jamainternalmedicine/fullarticle/2790862

Efficacy and Safety of Daprodustat for Treatment of Anemia ... Patients who received daprodustat had an increase in total iron-binding capacity and a reduction in ferritin level, whereas transferrin ...