Clinical trial NCT06749054: Evaluation of Long-Acting Lenacapavir for the Treatment of HIV-1 in Treatment-experienced Adolescents and Children
Official title: A Phase 2, Open-label, Single-Arm Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Antiviral Activity of Long-Acting Lenacapavir in Combination With an Optimized Background Regimen in Treatment-experienced Adolescents and Children With HIV-1
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: HIV-1-infection
Lead sponsor: Gilead Sciences
Sponsor class: INDUSTRY
First posted: 2024-12-27
Enrollment: 12
Sex: ALL
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this clinical study is to learn more about the study drug, lenacapavir (LEN). The study will assess the safety, tolerability, and efficacy of long-acting LEN when combined with other medicines in adolescents and children living with HIV-1 who weigh at least 35 kg and have been treated before for HIV-1. The study will also see how easy it is for participants to take LEN as injection or an oral pill.

The primary objectives are to evaluate the pharmacokinetics and safety of LEN in combination with optimized background regimen (OBR) in TE pediatric participants with HIV-1.


Eligibility criteria: Key Inclusion Criteria:

* Body weight at screening ≥ 35 kg.
* On a stable failing antiretroviral (ARV) regimen for \> 8 weeks before screening and willing to continue the regimen until Day 1.
* Plasma HIV-1 RNA ≥ 400 copies/mL on at least 2 consecutive occasions spanning at least 6 months, including at screening.
* Have previously changed their ARV regimen due to treatment failure.
* ARV treatment options limited due to resistance, tolerability, contraindications, safety, drug access.
* Able and willing to commit to taking LEN in combination with their OBR.
* The following laboratory parameters at screening:

  1. Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m\^2 using Bedside Schwartz Formula.
  2. Absolute neutrophil count \> 0.50 GI/L (\> 500 cells/mm\^3).
  3. Hemoglobin ≥ 85 g/L (\> 8.5 g/dL).
  4. Platelets ≥ 50 GI/L (≥ 50,000/mm\^3).
  5. Hepatic transaminases (aspartate aminotransferase and alanine aminotransferase) ≤ 5 × upper limit of normal.
  6. Total bilirubin ≤ 23 μmol/L (≤ 1.5 mg/dL) and direct bilirubin ≤ 7 μmol/L (≤ 0.4 mg/dL).

Key Exclusion Criteria:

* Life expectancy ≤ 1 year.
* An opportunistic illness requiring treatment within the 30 days prior to screening.
* Evidence of active pulmonary or extra-pulmonary tuberculosis within 3 months prior to screening.
* Hepatitis C virus (HCV) antibody positive with detectable HCV RNA at screening.
* Hepatitis B virus (HBV) surface antigen (HBsAg) positive or HBV core antibody (antibody against hepatitis B core antigen (anti-HBc)) positive; if individual is HBsAg negative and anti-HBc positive but HBV DNA undetectable, individual may be enrolled.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Pharmacokinetic (PK) Parameter: Ctrough, W26 of Lenacapavir (LEN)
timeFrame: Week 26
measure: Percentage of Participants Experiencing Treatment-Emergent Adverse Events (AEs) Through Week 26
timeFrame: First dose date up to Week 26
measure: Percentage of Participants Experiencing Treatment-Emergent Laboratory Abnormalities Through Week 26
timeFrame: First dose date up to Week 26
Secondary outcomes:
measure: PK Parameter: Cmax, D1-W26 of LEN
timeFrame: Day 1 up to Week 26
measure: PK Parameter: AUC D1-W26 of LEN
timeFrame: Day 1 up to Week 26
measure: Percentage of Participants Experiencing Treatment-Emergent AEs Through Week 52
timeFrame: First dose date up to Week 52
measure: Percentage of Participants Experiencing Treatment-Emergent Laboratory Abnormalities Through Week 52
timeFrame: First dose date up to Week 52
measure: Percentage of Participants With Plasma HIV-1 RNA < 50 Copies/mL at Week 26 Based on the US Food and Drug Administration (FDA)-Defined Snapshot Algorithm
timeFrame: Week 26
measure: Percentage of Participants with Plasma HIV-1 RNA < 50 Copies/mL at Week 52 Based on the US FDA-Defined Snapshot Algorithm
timeFrame: Week 52
measure: Change From Baseline in Clusters of Differentiation (CD4)+ Cell Counts at Week 26
timeFrame: Baseline, Week 26
measure: Change From Baseline in CD4+ Cell Counts at Week 52
timeFrame: Baseline, Week 52
measure: Percent Change From Baseline in CD4+ at Week 26
timeFrame: Baseline, Week 26
measure: Percent Change From Baseline in CD4+ at Week 52
timeFrame: Baseline, Week 52
measure: General Acceptability of Oral LEN as Assessed by Percentage of Participants With Acceptability Questionnaire Responses on Day 1
timeFrame: Day 1
measure: General Acceptability of Oral LEN as Assessed by Percentage of Participants With Acceptability Questionnaire Responses on Day 2
timeFrame: Day 2
measure: General Palatability of Oral LEN as Assessed by Percentage of Participants With Palatability Questionnaire Responses on Day 1
timeFrame: Day 1
measure: General Palatability of Oral LEN as Assessed by Percentage of Participants With Palatability Questionnaire Responses on Day 2
timeFrame: Day 2
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Grady Health System, Ponce De Leon Center
state: Georgia
city: Cape Town
country: South Africa
facility: FAMCRU
city: Johannesburg
country: South Africa
facility: Wits RHI Shandukani Research Centre CRS
city: Johannesburg
country: South Africa
facility: Rahima Moosa Mother and Child Hospital
city: KwaZulu - Natal
country: South Africa
facility: Victoria Mxenge Hospital
city: Paarl
country: South Africa
facility: Be Part Research Pty (Ltd)
city: Soweto
country: South Africa
facility: Perinatal HIV Research Unit (PHRU)
Study officials:
affiliation: Gilead Sciences
name: Gilead Study Director
role: STUDY_DIRECTOR
References: