Evaluation of Long-Acting Lenacapavir for the Treatment of HIV-1 in Treatment-experienced Adolescents and Children

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

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.

Study Overview

• Status: Recruiting
• Conditions: HIV-1-infection
• Intervention / Treatment: Drug: Oral Lenacapavir; Drug: Optimized Background Regimen (OBR); Drug: Subcutaneous Lenacapavir

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Gilead Clinical Study Information Center; Phone Number: 1-833-445-3230 (GILEAD-0); Email: GileadClinicalTrials@gilead.com

Study Locations

• South Africa
  • Cape Town, South Africa, 7505
    • Recruiting
    • FAMCRU
  • Germiston, South Africa, 1401
    • Recruiting
    • CRISMO Research Centre
  • Johannesburg, South Africa, 2112
    • Recruiting
    • Rahima Moosa Mother and Child Hospital
  • Johannesburg, South Africa, 2038
    • Recruiting
    • Wits RHI Shandukani Research Centre CRS
  • KwaDukuza, South Africa, 4449
    • Recruiting
    • Clinical Research Institute of South Africa (CRISA)
  • KwaZulu - Natal, South Africa, 4093
    • Recruiting
    • Durban International Clinical Research Site, Enhancing Care Foundation
  • Paarl, South Africa, 7626
    • Recruiting
    • Be Part Research Pty (Ltd)
  • Soweto, South Africa, 2013
    • Withdrawn
    • Perinatal HIV Research Unit (PHRU)
• United States
  • Georgia
    • Atlanta, Georgia, United States, 30308
      • Recruiting
      • Grady Health System, Ponce De Leon Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: LEN; Participants will receive oral LEN 600 mg on Days 1 and 2. Participants will also receive 2 doses of LEN 927 mg as subcutaneous (SC) injection on Day 1 and Week 26 along with their OBR per clinical practice. At the Week 52, participants will be given the option to receive SC LEN every 6 months while continuing their OBR for at least another 2 SC LEN doses in the extension phase.

Drug: Oral Lenacapavir; Tablets administered without regard to food; Other Names: GS-6207; Sunlenca®; Drug: Optimized Background Regimen (OBR); Optimized background regimen as prescribed by the Investigator; Drug: Subcutaneous Lenacapavir; Administered via subcutaneous injections; Other Names: GS-6207; Sunlenca®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetic (PK) Parameter: Ctrough, W26 of Lenacapavir (LEN)	Ctrough, W26 is defined as the plasma concentration at the end of the dosing interval at Week 26.	Week 26
Percentage of Participants Experiencing Treatment-Emergent Adverse Events (AEs) Through Week 26		First dose date up to Week 26
Percentage of Participants Experiencing Treatment-Emergent Laboratory Abnormalities Through Week 26		First dose date up to Week 26

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
PK Parameter: Cmax, D1-W26 of LEN	Cmax, D1-W26 is defined as the maximum observed concentration of drug from Day 1 to Week 26.	Day 1 up to Week 26
PK Parameter: AUC D1-W26 of LEN	AUC D1-W26 is defined as the partial area under the concentration versus time curve from Day 1 to Week 26.	Day 1 up to Week 26
Percentage of Participants Experiencing Treatment-Emergent AEs Through Week 52		First dose date up to Week 52
Percentage of Participants Experiencing Treatment-Emergent Laboratory Abnormalities Through Week 52		First dose date up to Week 52
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		Week 26
Percentage of Participants with Plasma HIV-1 RNA < 50 Copies/mL at Week 52 Based on the US FDA-Defined Snapshot Algorithm		Week 52
Change From Baseline in Clusters of Differentiation (CD4)+ Cell Counts at Week 26		Baseline, Week 26
Change From Baseline in CD4+ Cell Counts at Week 52		Baseline, Week 52
Percent Change From Baseline in CD4+ at Week 26		Baseline, Week 26
Percent Change From Baseline in CD4+ at Week 52		Baseline, Week 52
General Acceptability of Oral LEN as Assessed by Percentage of Participants With Acceptability Questionnaire Responses on Day 1	To assess the acceptability of the study drug, the participants will complete questionnaire including a question on general acceptability of the assigned study drug on an ordinal 5-category scale.	Day 1
General Acceptability of Oral LEN as Assessed by Percentage of Participants With Acceptability Questionnaire Responses on Day 2	To assess the acceptability of the study drug, the participants will complete questionnaire including a question on general acceptability of the assigned study drug on an ordinal 5-category scale.	Day 2
General Palatability of Oral LEN as Assessed by Percentage of Participants With Palatability Questionnaire Responses on Day 1	To assess the palatability of the study drug, the participants will complete questionnaire including a question on general palatability of the assigned study drug on an ordinal 5-category scale.	Day 1
General Palatability of Oral LEN as Assessed by Percentage of Participants With Palatability Questionnaire Responses on Day 2	To assess the palatability of the study drug, the participants will complete questionnaire including a question on general palatability of the assigned study drug on an ordinal 5-category scale.	Day 2

Collaborators and Investigators

• Sponsor: Gilead Sciences
• Investigators: Study Director: Gilead Study Director, Gilead Sciences

Publications and helpful links

Helpful Links

• Gilead Clinical Trials Website

Study record dates

Study Major Dates

• Study Start (Actual): March 26, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): September 1, 2027

Study Registration Dates

• First Submitted: December 19, 2024
• First Submitted That Met QC Criteria: December 19, 2024
• First Posted (Actual): December 27, 2024

Study Record Updates

• Last Update Posted (Actual): April 24, 2026
• Last Update Submitted That Met QC Criteria: April 22, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: lenacapavir
• Other Study ID Numbers: GS-US-200-6712

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No