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Evaluating the Efficacy and Safety of Dolutegravir-Containing Versus Efavirenz-Containing Antiretroviral Therapy Regimens in HIV-1-Infected Pregnant Women and Their Infants

Registration Number
NCT03048422
Lead Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Brief Summary

The purpose of this study was to compare the virologic efficacy and safety of three antiretroviral (ARV) regimens, dolutegravir plus emtricitabine/tenofovir alafenamide, dolutegravir plus emtricitabine/tenofovir disoproxil fumarate, and efavirenz/emtricitabine/tenofovir disoproxil fumarate in pregnant women living with HIV-1 and to compare the safety of these regimens for their infants.

Detailed Description

This study compared the virologic efficacy and safety of three ARV regimens in pregnant women living with HIV: dolutegravir (DTG) plus emtricitabine/tenofovir alafenamide (FTC/TAF), DTG plus emtricitabine/tenofovir disoproxil fumarate (FTC/TDF), and efavirenz/emtricitabine/tenofovir disoproxil fumarate (EFV/FTC/TDF). The study also compared the safety of these regimens for their infants.

At study entry, mothers were randomly assigned to either receive DTG plus FTC/TAF (Arm 1), DTG plus FTC/TDF (Arm 2), or EFV/FTC/TDF (Arm 3) during pregnancy, through delivery, and for 50 weeks postpartum.

Mothers completed study visits at study entry and every four weeks during pregnancy. Study visits for mothers and their infants occurred at delivery and at 6, 14, 26, 38, and 50 weeks postpartum. Visits for mothers and infants included physical examinations and blood collection. Select study visits also included breast milk collection from mothers who breastfed, hair and urine collection, ultrasound scans, pregnancy testing, contraception counseling, and, for a subset of participants, dual energy x-ray absorptiometry (DXA) scans for mothers and their infants.

For pregnancy outcome measures, mothers and infants were evaluated together as mother-infant pairs, with any outcome between the two counting as an event (for example, if an infant was born small for gestational age, this would be a pregnancy outcome event for the mother-infant pair). For all other outcome measures, women and infants were evaluated separately.

Recruitment & Eligibility

Status
COMPLETED
Sex
Female
Target Recruitment
643
Inclusion Criteria
  • Mother is able to provide written informed consent for her and her infant's participation in this study

  • Mother has confirmed HIV-1 infection based on documented testing of two samples collected at different time points:

    • Sample #1 may be tested using any of the following:
    • Two rapid antibody tests from different manufacturers or based on different principles and epitopes
    • One enzyme immunoassay (EIA) OR Western blot OR immunofluorescence assay OR chemiluminescence assay
    • One HIV DNA polymerase chain reaction (PCR)
    • One quantitative HIV RNA PCR (above the limit of detection of the assay)
    • One qualitative HIV RNA PCR
    • One total HIV nucleic acid test
    • Sample #2 may be tested using any of the following:
    • One rapid antibody test. If this option is used in combination with two rapid tests for Sample #1, at least one of the three rapid tests must be FDA-approved and the third rapid test must be from a third manufacturer or based on a third principle or epitope.
    • One EIA OR Western blot OR immunofluorescence assay OR chemiluminescence assay
    • One HIV DNA PCR
    • One quantitative HIV RNA PCR (above the limit of detection of the assay)
    • One qualitative HIV RNA PCR
    • One total HIV nucleic acid test.
    • See the protocol for more information on this inclusion criterion.
  • At screening, mother is ART-naive, defined as having not received prior antiretroviral therapy other than ARVs received during prior pregnancies or prior periods of breastfeeding (i.e., receipt of any single, dual, or triple ARV regimen during prior time-limited periods of pregnancy and breastfeeding is permitted). Receipt of up to 14 days of ARVs during the current pregnancy is permitted prior to study entry so that initiation of ARVs during the current pregnancy is not delayed during the study screening period. Note: Non-study ART may be initiated in the current pregnancy prior to initiation of the study screening process. For eligible participants, enrollment must occur within 14 days of non-study ART initiation. Note: Receipt of ARVs during a prior pregnancy or prior period of breastfeeding must have concluded at least six months prior to study entry. Receipt of TDF or FTC/TDF for pre-exposure prophylaxis at any time in the past is not exclusionary (even if received within six months prior to study entry).

  • At screening, mother has the following laboratory test results (based on testing of samples collected within 14 days prior to study entry):

    • Grade 1 or lower (less than 2.5 times upper limit of normal [ULN]) alanine aminotransferase (ALT) and aspartate aminotransferase (AST)
    • Grade 2 or lower (less than or equal to 1.8 times ULN) creatinine
    • Grade 2 or lower (greater than or equal to 60 mL/min) estimated creatinine clearance (CrCl; Cockcroft-Gault formula). See the protocol for guidance on severity grading. Laboratory tests may be repeated during the study screening period, with the latest result used for eligibility determination.
  • At screening and at study entry, no evidence of multiple gestation or fetal anomalies, as assessed by best available method

  • At study entry, gestational age of 14-28 weeks, defined as greater than 13 weeks plus six days and less than 28 completed weeks gestation, estimated by best available method. Note: For this inclusion criterion and the previous inclusion criterion, fetal ultrasound is preferred but not required for purposes of eligibility determination. If ultrasound cannot be performed during the study screening period prior to study entry, it must be performed within 14 days after study entry. As further explained in the protocol, enrolled participants will not be withdrawn from the study based on ultrasound findings obtained after study entry.

  • At study entry, mother expects to remain in the geographic area of the study site during pregnancy and for 50 weeks postpartum [Eligibility criteria added per Letter of Amendment 1 to V2; July 2018]:

  • At study entry, mother reports that she does not wish to become pregnant again for at least 50 weeks after her current pregnancy and that she is willing to use effective contraception during this period. Effective contraception may include surgical sterilization (i.e., hysterectomy, bilateral oophorectomy, tubal ligation, or salpingectomy) or any of the following methods:

    • Contraceptive intrauterine device (IUD) or intrauterine system (IUS)
    • Subdermal contraceptive implant
    • Progestogen injections
    • Progestogen only oral contraceptive pills
    • Combined estrogen and progestogen oral contraceptive pills
    • Percutaneous contraceptive patches
    • Contraceptive vaginal rings
    • Note: IUDs, IUSs, implants, and injections are strongly recommended due to their lower failure rates with typical use. Male or female condom use is recommended with all contraceptive methods for dual protection against pregnancy and to avoid transmission of HIV and other sexually transmitted infections.
Exclusion Criteria
  • Mother is currently incarcerated or involuntarily confined in a medical facility

  • Mother is currently receiving:

    • A psychoactive medication for treatment of a psychiatric illness
    • Treatment for active tuberculosis
    • Treatment for active hepatitis C infection
  • Mother is expected to require treatment with interferon and/or ribavirin for hepatitis C infection during the study follow-up period

  • Mother has a history of any of the following, as determined by the site investigator or designee based on maternal report and available medical records:

    • Hypersensitivity or clinically significant adverse reaction to any of the ARVs included in the three study drug regimens (ever)
    • Antiretroviral drug resistance mutations that would impact selection of ART regimen (ever)
    • Clinically significant heart disease and/or known prolonged corrected QT (QTc) interval (ever)
    • Suicidal ideation or attempt (ever)
    • HIV-2 infection (ever)
    • Zika virus infection, diagnosed or suspected, during the current pregnancy
    • Receipt of any antiretroviral medication within six months prior to study entry, with two exceptions: receipt of any duration of TDF or FTC/TDF for pre-exposure prophylaxis or receipt of up to 14 days of ARVs during the current pregnancy
    • Receipt of any prohibited medication within 14 days prior to study entry (see the protocol for more information)
    • Clinically significant acute illness requiring systemic treatment and/or hospitalization (i.e., major medical condition that is likely to lead to hospitalization and/or to an adverse pregnancy outcome) within 14 days prior to study entry
    • Unstable liver disease (defined by the presence of ascites, encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, or persistent jaundice) or known biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones) within 14 days prior to study entry
    • Note: Testing to rule out HIV-2 infection is not required.
  • Mother or fetus has any other condition that, in the opinion of the site investigator or designee, would make participation in the study unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm 1: Maternal DTG+FTC/TAFEmtricitabine/tenofovir alafenamideMothers randomized to receive dolutegravir (DTG) plus emtricitabine/tenofovir alafenamide (FTC/TAF) during pregnancy, through delivery, and for 50 weeks postpartum.
Arm 2: Maternal DTG+FTC/TDFDolutegravirMothers randomized to receive DTG plus emtricitabine/tenofovir disoproxil fumarate (FTC/TDF) during pregnancy, through delivery, and for 50 weeks postpartum.
Arm 2: Maternal DTG+FTC/TDFEmtricitabine/tenofovir disoproxil fumarateMothers randomized to receive DTG plus emtricitabine/tenofovir disoproxil fumarate (FTC/TDF) during pregnancy, through delivery, and for 50 weeks postpartum.
Arm 3: Maternal EFV/FTC/TDFEfavirenz/emtricitabine/tenofovir disoproxil fumarateMothers randomized to receive efavirenz/emtricitabine/tenofovir disoproxil fumarate (EFV/FTC/TDF) during pregnancy, through delivery, and for 50 weeks postpartum.
Arm 1: Maternal DTG+FTC/TAFDolutegravirMothers randomized to receive dolutegravir (DTG) plus emtricitabine/tenofovir alafenamide (FTC/TAF) during pregnancy, through delivery, and for 50 weeks postpartum.
Primary Outcome Measures
NameTimeMethod
Percentage of Mothers With HIV-1 RNA Viral Load Less Than 200 Copies/mL at DeliveryDelivery

Percentage of mothers with plasma HIV-1 RNA viral load less than 200 copies/mL at delivery determined using real-time test results obtained at site laboratories. This outcome was evaluated in the non-inferiority (primary outcome) and superiority (secondary outcome) analyses. The intention-to-treat analysis included all randomized women who had viral load data available. The per-protocol analysis excluded women who modified randomized treatment (stopped, paused, switched, added any treatment) before viral load evaluation at delivery, with the exception of women who modified randomized treatment for use of a concomitant medication.

Percentage of Mother-infant Pairs With an Adverse Pregnancy OutcomeDelivery

Percentage of mother-infant pairs with an adverse pregnancy outcome. Adverse pregnancy outcome includes spontaneous abortion (\<20 weeks gestation), stillbirth (≥20 weeks gestation), preterm delivery (\<37 completed weeks), or small for gestational age (\<10th percentile by INTERGROWTH 21st Standards)

Cumulative Probability of Women Experiencing Grade 3 or Higher Adverse EventFrom randomization up to 74 weeks

The Kaplan-Meier estimate of the cumulative probability of women experiencing grade 3 or higher adverse events, including events resulting in death due to any cause.

Time to first maternal grade 3 or higher adverse event was defined as the first grade 3 or higher adverse event that occurred after randomization and before 74 weeks of follow-up. The timeframe of 74 weeks was determined by adding up 56 weeks of postpartum follow-up to the mean duration of antepartum follow-up, which was 18 weeks.

Cumulative Probability of Infants Experiencing Grade 3 or Higher Adverse EventFrom birth through Week 50 postpartum

The Kaplan-Meier estimate of the cumulative probability of infants experiencing grade 3 or higher adverse events, including events resulting in death due to any cause.

Secondary Outcome Measures
NameTimeMethod
Count of Mother-infant Pairs in the Classified Ranked Composite Safety OutcomeBirth through 50 weeks postpartum

Infant and pregnancy outcomes were classified on a scale of 1 to 10, with mother-infant pairs categorized by the worst outcome they experienced (worst category being 1 and best being 10): 1) Infant death; 2) Spontaneous abortion (\<20 weeks gestation) or stillbirth (≥20 weeks gestation); 3) Infant HIV infection; 4) Extremely and very early preterm (\<32 completed weeks); 5) Major congenital anomaly; 6) Preterm delivery (\<37 completed weeks); 7) Small for gestational age (\<10th percentile); 8) Infant hospitalization; 9) Infant grade 3 or 4 adverse event; 10) None of the above. If a mother-infant pair experienced more than one safety outcome, only the worst was reported.

Cumulative Probability of Infant HIV-infectionBirth through 50 weeks after birth

The Kaplan-Meier estimate of the cumulative probability of infants acquiring HIV-1 infection from birth through 50 weeks after birth based on nucleic acid test results.

Cumulative Probability of Infant DeathsBirth through 50 weeks after birth

The Kaplan-Meier estimate of the cumulative probability of infant deaths from birth through 50 weeks after birth.

Maternal Change in Creatinine ClearanceBaseline to 50 weeks postpartum

Maternal change in creatinine clearance per week based on generalized estimating equations

Infant Creatinine ClearanceDelivery and 26 weeks postpartum

Infant creatinine clearance based on Schwartz formula

Percentage of Mothers With HIV-1 ARV Drug Resistance Mutations at the Time of Maternal Virologic FailureFrom 24 weeks after randomization through Week 50 postpartum

Percentage of mothers with HIV-1 antiretroviral (ARV) drug resistance mutations at the time of maternal virologic failure. Virologic failure was defined as two consecutive plasma HIV-1 RNA viral loads \<200 copies/mL on or after 24 weeks on study. Drug resistance mutations were assessed using the Stanford algorithm, and all ARV regimens were assessed for mutations.

Count of Infants With HIV-1 Antiretroviral Drug Resistance Mutations at the Time of Infant HIV DiagnosisFrom birth through 50 weeks postpartum

Count of infants with HIV-1 antiretroviral drug resistance mutations (to any antiretroviral drug) at the time of infant HIV diagnosis, based on laboratory blood test results.

Percentage of Mother-Infant Pairs With Preterm DeliveriesDelivery

Percentage of mother-infant pairs with preterm deliveries (\<37 weeks gestation) resulting in live born infant

Percentage of Infants Born Small for Gestational AgeBirth

Percentage of infants born small for gestational age (\<10th percentile adjusted for sex assigned at birth) based on Intergrowth 21st Standards

Change in Maternal Weight AntepartumBaseline through before delivery (up to one day prior)

Change in maternal antepartum weight per week based on generalized estimating equations

Change in Maternal Weight PostpartumDelivery to 50 weeks postpartum

Change in maternal postpartum weight per week based on generalized estimating equations

Change in Maternal Weight OverallBaseline to 50 weeks postpartum

Change in maternal weight per week based on generalized estimating equations

Percentage of Mothers With HIV-1 RNA Less Than 50 Copies/mL at Delivery Measured at Central LaboratoryDelivery

Percentage of mothers with HIV-1 RNA less than 50 copies/mL at delivery using batched test results obtained from central laboratory

Percentage of Mothers With HIV-1 RNA Less Than 200 Copies/mL at 50 Weeks Postpartum50 weeks postpartum

Percentage of mothers with HIV-1 RNA less than 200 copies/mL at 50 weeks postpartum using real-time test results obtained from site laboratories

Time to First HIV-1 RNA Less Than 200 Copies/mL Through DeliveryRandomization to delivery

Time to first viral HIV-1 RNA less than 200 copies/mL through delivery, determined using real-time results obtained from site laboratories

Percentage of Mothers With Virologic Success of HIV-1 RNA Less Than 200 Copies/mL at Delivery Based on FDA Snapshot AlgorithmDelivery

Percentage of mothers with virologic success of HIV-1 RNA less than 200 copies/mL at delivery based on FDA snapshot algorithm using real-time test results obtained from site laboratories

Percentage of Mothers With Virologic Success of HIV-1 RNA Less Than 200 Copies/mL at 50 Weeks Postpartum Based on FDA Snapshot Algorithm50 weeks postpartum

Percentage of mothers with virologic success of HIV-1 RNA less than 200 copies/mL at 50 weeks postpartum based on FDA snapshot algorithm using real-time test results obtained from site laboratories

Percentage of Mother-Infant Pairs With an Adverse Pregnancy OutcomeDelivery

Percentage of mother-infant pairs with an adverse pregnancy outcome. Adverse pregnancy outcome includes spontaneous abortion (\<20 weeks gestation), stillbirth (≥20 weeks gestation), preterm delivery (\<37 completed weeks), or small for gestational age (\<10th percentile per INTERGROWTH 21st Standards)

Cumulative Probability of Women Experiencing Grade 3 or Higher Adverse EventFrom randomization up to 74 weeks

The Kaplan-Meier estimate of the cumulative probability of women experiencing grade 3 or higher adverse events, including events resulting in death due to any cause.

Time to first maternal grade 3 or higher adverse event was defined as the first grade 3 or higher adverse event that occurred after randomization and before 74 weeks of follow-up. The timeframe of 74 weeks was determined by adding up 56 weeks of postpartum follow-up to the mean duration of antepartum follow-up, which was 18 weeks.

Cumulative Probability of Infants Experiencing Grade 3 or Higher Adverse EventBirth through Week 50 postpartum

The Kaplan-Meier estimate of the cumulative probability of infants experiencing grade 3 or higher adverse events, including events resulting in death due to any cause.

Percentage of Mother-infant Pairs With an Adverse Pregnancy Outcome or Major Congenital AnomalyDelivery through 50 weeks postpartum

Percentage of mother-infant pairs with an adverse pregnancy outcome or major congenital anomaly. Adverse pregnancy outcomes include spontaneous abortions (\<20 weeks gestation), stillbirths (≥20 weeks gestation), preterm deliveries (\<37 weeks gestation), and infants small for gestational age (\<10th percentile per INTERGROWTH 21st Standards). Major congenital anomaly was defined consistent with the definition of malformation provided by Holmes and Westgate (i.e., a structural abnormality with surgical, medical, or cosmetic importance) and evaluated by an internal study team blinded to treatment arm.

Trial Locations

Locations (21)

Molepolole CRS

🇧🇼

Gaborone, Botswana

Chiang Mai University HIV Treatment (CMU HIV Treatment) CRS

🇹🇭

Chiang Mai, Thailand

St Mary's CRS

🇿🇼

Chitungwiza, Zimbabwe

Harare Family Care CRS

🇿🇼

Harare, Zimbabwe

Instituto de Puericultura e Pediatria Martagao Gesteira - UFRJ NICHD CRS

🇧🇷

Rio de Janeiro, Brazil

SOM Federal University Minas Gerais Brazil NICHD CRS

🇧🇷

Belo Horizonte, Minas Gerais, Brazil

Soweto IMPAACT CRS

🇿🇦

Johannesburg, Gauteng, South Africa

Pediatric Perinatal HIV Clinical Trials Unit CRS

🇺🇸

Miami, Florida, United States

Hospital Federal dos Servidores do Estado NICHD CRS

🇧🇷

Rio De Janeiro, Brazil

Hosp. Geral De Nova Igaucu Brazil NICHD CRS

🇧🇷

Rio de Janeiro, Brazil

Baylor-Uganda CRS

🇺🇬

Kampala, Uganda

Wits RHI Shandukani Research Centre CRS

🇿🇦

Johannesburg, Gauteng, South Africa

Umlazi CRS

🇿🇦

Durban, Kwa Zulu Natal, South Africa

Seke North CRS

🇿🇼

Chitungwiza, Zimbabwe

Famcru Crs

🇿🇦

Tygerberg, Western Cape Province, South Africa

Gaborone CRS

🇧🇼

Gaborone, South-East District, Botswana

Univ. of Florida Jacksonville NICHD CRS

🇺🇸

Jacksonville, Florida, United States

Chiangrai Prachanukroh Hospital NICHD CRS

🇹🇭

Chiang Mai, Thailand

Siriraj Hospital ,Mahidol University NICHD CRS

🇹🇭

Bangkok, Bangkoknoi, Thailand

Kilimanjaro Christian Medical Centre (KCMC)

🇹🇿

Moshi, Tanzania

Byramjee Jeejeebhoy Medical College (BJMC) CRS

🇮🇳

Pune, Maharashtra, India

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