What is the mechanism of action of this treatment?

The most common treatments for Acute Promyelocytic Leukemia (APL) are all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). ATRA promotes the differentiation of immature promyelocytes into mature blood cells, while ATO induces apoptosis of the leukemic cells. Both treatments target the PML-RARA fusion protein, a genetic abnormality specific to APL, leading to high cure rates. Understanding these mechanisms is crucial for APL patients as they directly address the disease's root cause, offering effective and targeted therapy.

What side effects are associated with this type of intervention?

Common treatments for Acute Promyelocytic Leukemia (APL) include all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). Side effects of ATRA can include headache, fever, dry skin, and retinoic acid syndrome, which is characterized by fever, respiratory distress, and fluid buildup. Arsenic trioxide may cause fatigue, nausea, QT prolongation, and differentiation syndrome, which presents with fever, weight gain, and respiratory issues. There is no direct mention of PI3Kδ inhibitors like Leniolisib being used for APL in the provided context.

What prior FDA approvals exist?

The FDA has approved treatments for Acute Promyelocytic Leukemia (APL) that primarily include all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). These treatments target the differentiation of promyelocytes and the degradation of the PML-RARα fusion protein, respectively. There are no FDA-approved treatments for APL that are similar to Leniolisib, a PI3Kδ inhibitor.

What other types of research exist?

For Acute Promyelocytic Leukemia (APL) patients, promising novel alternatives to Leniolisib (PI3Kδ Inhibitor) include investigational agents such as regorafenib, cabozantinib, sorafenib, and lenvatinib, which have shown some activity in related hematologic malignancies. Additionally, combination treatments involving these agents with traditional chemotherapeutics like etoposide and ifosfamide may offer new therapeutic options.

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PI3K Inhibitor

Leniolisib for Activated PI3K Delta Syndrome

Phase 3

Waitlist Available

Research Sponsored by Pharming Technologies B.V.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patient weighs ≥13 kg and <45 kg at baseline.

Patient has at least 1 measurable nodal lesion on magnetic resonance imaging/low-dose computed tomography within 6 months of screening.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to end of 12 weeks, & from baseline to through study completion, an average of 1 year

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing leniolisib, an oral medicine, in children aged 4 to 11 with a genetic condition called APDS. The medicine aims to calm an overactive part of their immune system to improve their health. Leniolisib has shown benefits in patients with APDS in previous clinical trials.

Who is the study for?

This trial is for pediatric patients aged 4 to 11 with Activated PI3K Delta Syndrome (APDS). They must weigh between 13 kg and <45 kg, have a measurable nodal lesion, and a confirmed genetic mutation in the PIK3CD or PIK3R1 gene. Participants should not be on other trials or certain immunosuppressants, and girls who reach menarche must use contraception.

What is being tested?

The study tests Leniolisib's safety, tolerability, how it's processed by the body (pharmacokinetics), its effects on the disease (pharmacodynamics), and effectiveness in at least 15 children with APDS. It's an open-label trial where everyone gets Leniolisib; there are no comparison groups.

What are the potential side effects?

Potential side effects of Leniolisib aren't specified here but may include reactions related to immune system suppression given its pharmacological target. Side effects could also relate to drug components like lactose monohydrate or hypromellose.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I weigh between 13 kg and 45 kg.

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I have a measurable cancerous node identified by MRI or CT scan in the last 6 months.

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I can take pills without any trouble.

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Your heart rate is within a normal range.

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My heart rate is between 60 and 140 beats per minute.

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I am 10 years or older with a heart rate between 50 to 100 bpm.

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I have given my written consent for the study, as approved by an ethics committee.

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I am a woman not able to have children or a man willing to use contraception.

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I, or my child, will follow study rules about menstruation if it starts after screening.

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I will not engage in heterosexual activity for the study duration and 6 months after.

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I will use effective birth control for 30 days after my last study procedure.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to end of 12 weeks, & from baseline to through study completion, an average of 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to end of 12 weeks, & from baseline to through study completion, an average of 1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to end of 12 weeks, & from baseline to through study completion, an average of 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Part I & II: Change from baseline in clinical laboratory test results

Part I & II: Change from baseline in electrocardiograms (ECGs)

Part I & II: Change from baseline in growth and physical development

+5 more

Secondary study objectives

Part I and II: Key secondary efficacy outcomes for Part I include incidence of infections and use of antibiotics.

Part I and II: Pediatric Quality of Life Inventory (PedsQLTM) Parent Report for Children Questionnaire 4.0 Generic Core Scales

Part I: To assess the maximum concentration (Cmax) of leniolisib in pediatric patients (aged 4 to 11 years) with APDS

+6 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: LeniolisibExperimental Treatment1 Intervention

Leniolisib - Film Coated Tablets Leniolisib tablets in 10 and 30 mg strengths administered orally BID by body weight for 12 weeks for Part I and for 1 year for Part II.

0 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Acute Promyelocytic Leukemia (APL) are all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). ATRA promotes the differentiation of immature promyelocytes into mature blood cells, while ATO induces apoptosis of the leukemic cells. Both treatments target the PML-RARA fusion protein, a genetic abnormality specific to APL, leading to high cure rates. Understanding these mechanisms is crucial for APL patients as they directly address the disease's root cause, offering effective and targeted therapy.

Emerging Epigenetic Therapeutic Targets in Acute Myeloid Leukemia.Molecular targeting in acute myeloid leukemia.