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161 Participants Needed

ITU512 for Sickle Cell Disease

Overseen ByNovartis Pharmaceuticals

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Novartis Pharmaceuticals

Must not be taking: Hydroxyurea

Disqualifiers: Arrhythmias, Significant illness, others

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

Trial Summary

Will I have to stop taking my current medications?

If you are currently using hydroxyurea or hydroxycarbamide, you will need to stop taking it to participate in this trial. The protocol does not specify about other medications.

Is ITU512 safe for humans?

The research on hydroxyurea (HU), which may be related to ITU512, shows it is generally safe for children and adults with sickle cell disease. Studies found that any side effects were mild, temporary, and reversible, with no life-threatening events reported.¹ ² ³ ⁴ ⁵

What is the purpose of this trial?

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

Eligibility Criteria

This trial is for healthy individuals and those with sickle cell disease. Participants must meet specific health criteria to join, but the exact inclusion and exclusion details are not provided here.

Inclusion Criteria

I have been diagnosed with sickle cell disease.

Part 1: Participants must be in good health as determined by the investigator's assessment of medical history, physical examination, vital signs, ECG, and laboratory tests

I weigh at least 50 kg and my BMI is between 18.0-32.0.

See 1 more

Exclusion Criteria

I have a history of irregular heartbeats.

I have been healthy for the last two weeks.

I am a woman who can become pregnant.

See 4 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase I Treatment

First-in-human study to assess safety, tolerability, and pharmacokinetics of ITU512 in healthy participants

Up to 60 days

Multiple visits for dose administration and monitoring

Phase II Treatment

Evaluation of safety, tolerability, pharmacokinetics, and efficacy of ITU512 in patients with sickle cell disease

Up to 5 months

Regular visits for dose administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Extension

Optional extension part for continued evaluation of ITU512 in patients with sickle cell disease

Treatment Details

Interventions

ITU512

Trial Overview The study is testing ITU512's safety, how it's tolerated by the body, its movement through the body (pharmacokinetics), effect on food intake, and ability to increase fetal hemoglobin in both healthy people and those with sickle cell disease.

Participant Groups

4Treatment groups

Experimental Treatment

Group I: Part 2Experimental Treatment2 Interventions

Part 2 in patients with sickle cell disease

Group II: Part 1CExperimental Treatment1 Intervention

Part 1C in healthy participants

Group III: Part 1BExperimental Treatment2 Interventions

Part 1B in healthy participants

Group IV: Part 1AExperimental Treatment2 Interventions

Part 1A in healthy participants

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Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Findings from Research

In a study of 523 pediatric patients with sickle cell disease, those treated with hydroxyurea (HU) were more likely to experience organ-specific complications, such as cardiovascular, hepatic, renal, and pulmonary issues, compared to those not treated with HU.

Despite the increased likelihood of complications in the HU-treated group, the study concluded that HU is not associated with the development of serious adverse events, suggesting it can be safely administered to severely ill children with sickle cell disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical complications in severe pediatric sickle cell disease and the impact of hydroxyurea.Tripathi, A., Jerrell, JM., Stallworth, JR.[2022]

Hydroxyurea (HU) significantly reduced the rate of vaso-occlusive crises, blood transfusion needs, and hospitalizations in patients with HbSβ(+)-thalassemia after 2 years of treatment, demonstrating its efficacy in managing this condition.

The treatment led to notable improvements in hematological parameters, such as increased %HbF and hemoglobin levels, with minimal toxicity observed, making HU a safe and effective option for patients in resource-limited settings.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Low and fixed dose of hydroxyurea is effective and safe in patients with HbSβ(+) thalassemia with IVS1-5(G→C) mutation.Dehury, S., Purohit, P., Patel, S., et al.[2022]

Hydroxyurea (HU) is effective in reducing complications of sickle cell disease (SCD) in children, with a study of 127 patients showing low rates of acute chest syndromes and cerebrovascular events over 426 patient-years of follow-up.

In a subgroup of 32 patients followed for 6 years, HU demonstrated significant benefits, and among 72 patients assessed for stroke risk, only 1 experienced a cerebrovascular event, suggesting HU may help prevent strokes even in very young children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Hydroxyurea for sickle cell disease in children and for prevention of cerebrovascular events: the Belgian experience.Gulbis, B., Haberman, D., Dufour, D., et al.[2022]