Safety of and Immune Response to Dolutegravir in HIV-1 Infected Infants, Children, and Adolescents

NCT ID: NCT01302847

Last Updated: 2024-12-12

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

181 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-04-20

Study Completion Date

2023-10-18

Brief Summary

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Dolutegravir (DTG) is an HIV drug in the integrase inhibitor drug class. This study evaluated the pharmacokinetics (PK), safety, tolerability of and immune response to DTG when used concurrently with optimized background therapy (OBT) in HIV-1 infected infants, children, and adolescents.

Detailed Description

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DTG is an HIV medicine in the integrase inhibitor drug class. The purpose of this study was to evaluate the pharmacokinetics, safety, tolerability, and antiviral activity of DTG when used concurrently with OBT in HIV-1 infected infants, children, and adolescents.

Participants in this study were evaluated for PK, safety and tolerability through 48 weeks, followed by additional long-term study follow-up that lasted for approximately 144 weeks (3 years), for a total of 192 weeks on study. This study had two stages. Stage I provided pharmacokinetics, short-term tolerability and safety data on DTG on a limited number of participants to permit dose selection for further study in Stage II. Once a Stage I dose was accepted, enrollment to Stage II began to complete enrollment to the cohort. Stage II provided longer-term safety and antiviral activity data among a larger number of participants.

Infants, children and adolescents with HIV-1, aged ≥ 4 weeks to \< 18 years enrolled in the age and formulation cohorts specified below:

* Cohort I: Adolescents ≥ 12 to \<18 years of age (film-coated tablets)
* Cohort IIA: Children ≥ 6 to \<12 years of age (film-coated tablets)
* Cohort IIB: Children ≥ 6 to \<12 years of age (granules for suspension)
* Cohort III: Children ≥ 2 to \< 6 years of age (granules for suspension)
* Cohort IV: Children ≥ 6 months to \< 2 years of age (granules for suspension)
* Cohort III-DT: Children ≥ 2 to \< 6 years of age (dispersible tablets)
* Cohort IV-DT: Children ≥ 6 months to \< 2 years of age (dispersible tablets)
* Cohort V-DT: Infants ≥ 4 weeks to \< 6 months (dispersible tablets)

Cohorts were opened sequentially according by age group (starting with the older age group), DTG formulation, and study stage, i.e. Initial study enrollment was for Cohort I and progressed to Cohort IIA once Cohort I Stage I met the PK and safety criteria, followed by opening of Cohort IIB. Each cohort enrolled in two sequential stages: Stage I and II (the only exception is Cohort IIB, which only enrolled through Stage I). Sequential enrollment for Cohort III and IV proceeded in the same manner. Cohort V never enrolled because of the recommended changes in dosing and inclusion of enrollment weight band in the criteria for dose finding.

Stage I participants had physical examinations and had blood draws for safety assessments at study visits: Day 0; Day 5 (+5 days); and Weeks 4, 8, 12, 16, 24, 32, 40, and 48. Stage I participants also had intensive PK sampling with blood samples collected at time 0 (pre-dose), and at 1, 2, 3, 4, 6, 8 and 24 hours post dosing. Once a Stage I treatment dose was accepted, enrollment to Stage II began to complete enrollment to the cohort. Stage II participants had physical examinations and blood draws for safety assessment at study visits: Day 0; Day 10; and Weeks 4, 8, 12, 16, 24, 32, 40, and 48. Blood, plasma, and urine were collected and tested to measure immune response. Females of childbearing potential underwent pregnancy testing at screening and at every study visit.

After 48 weeks, all Stage I and Stage II participants entered the long-term study follow-up and continued to receive DTG. During this time, participants had safety and/or antiviral activity assessments every 12 weeks for up to 3 years.

The study was able to determine a proposed dose (i.e. optimal dose) for Cohorts I, IIA, III-DT, IV-DT, and V-DT but not for Cohorts IIB, III, and IV. Participants on the proposed dose had intensive PK sampling between days 5 and10 of DTG initiation with blood samples collected at time 0 (pre-dose), and at 1, 2, 3, 4, 6, 8 and 24 hours post dosing.

The study is closed to accrual and study follow-up was completed on Oct 18, 2023.

Conditions

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HIV Infections

Keywords

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Integrase Inhibitors Infant Child Adolescent

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort I

Adolescents 12 to younger than 18 years of age who received DTG film-coated tablets

Group Type EXPERIMENTAL

DTG film-coated tablets

Intervention Type DRUG

DTG film-coated tablets initial starting dose at \~1 mg/kg with a maximum dose of 50 mg; orally once daily. Participants weighing ≥ 35kg received the proposed dose of 50 mg of DTG film-coated tablets.

Cohort IIA

Children 6 to younger than 12 years of age who received DTG film-coated tablets

Group Type EXPERIMENTAL

DTG film-coated tablets

Intervention Type DRUG

DTG film-coated tablets initial starting dose at \~1 mg/kg with a maximum dose of 50 mg; orally once daily. Participants weighing ≥ 35kg received the proposed dose of 50 mg of DTG film-coated tablets.

Cohort IIB

Children 6 to younger than 12 years of age who received DTG granules for suspension

Group Type EXPERIMENTAL

DTG granules for suspension

Intervention Type DRUG

DTG granules for suspension initial starting dose at \~0.64 mg/kg with a maximum dose of 32 mg; orally once daily.

Cohort III

Children 2 to younger than 6 years of age who received DTG granules for suspension

Group Type EXPERIMENTAL

DTG granules for suspension

Intervention Type DRUG

DTG granules for suspension initial starting dose at \~0.64 mg/kg with a maximum dose of 32 mg; orally once daily.

Cohort IV

Children 6 months to younger than 2 years of age who received DTG granules for suspension

Group Type EXPERIMENTAL

DTG granules for suspension

Intervention Type DRUG

DTG granules for suspension initial starting dose at \~0.64 mg/kg with a maximum dose of 32 mg; orally once daily.

Cohort III-DT

Children 2 to younger than 6 years of age who received DTG dispersible tablets

Group Type EXPERIMENTAL

DTG dispersible tablets

Intervention Type DRUG

DTG dispersible tablets initial starting dose of \~0.8 mg/kg with a maximum dose of 30 mg; orally once daily. The proposed weight-band dosing was:

Weight band 3 to \<6 kg: 5 mg DTG dispersible tablets; Weight band 6 to \<10 kg: 15 mg DTG dispersible tablets; Weight band 10 to \<14 kg: 20 mg DTG dispersible tablets; Weight band 14 to \<20 kg: 25 mg DTG dispersible tablets; Weight band ≥20 kg: 30 mg DTG dispersible tablets.

Cohort IV-DT

Children 6 months to younger than 2 years of age who received DTG dispersible tablets

Group Type EXPERIMENTAL

DTG dispersible tablets

Intervention Type DRUG

DTG dispersible tablets initial starting dose of \~1.25 mg/kg with a maximum dose of 30 mg; orally once daily. The proposed weight-band dosing was:

Weight band 3 to \<6 kg: 5 mg DTG dispersible tablets; Weight band 6 to \<10 kg: 15 mg DTG dispersible tablets; Weight band 10 to \<14 kg: 20 mg DTG dispersible tablets; Weight band 14 to \<20 kg: 25 mg DTG dispersible tablets; Weight band ≥20 kg: 30 mg DTG dispersible tablets.

Cohort V-DT

Infants 4 weeks to younger than 6 months of age who received DTG dispersible tablets

Group Type EXPERIMENTAL

DTG dispersible tablets

Intervention Type DRUG

DTG dispersible tablets initial starting dose of \~1.25 mg/kg with a maximum dose of 30 mg; orally once daily. The proposed weight-band dosing was:

Weight band 3 to \<6 kg: 5 mg DTG dispersible tablets; Weight band 6 to \<10 kg: 15 mg DTG dispersible tablets.

Interventions

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DTG film-coated tablets

DTG film-coated tablets initial starting dose at \~1 mg/kg with a maximum dose of 50 mg; orally once daily. Participants weighing ≥ 35kg received the proposed dose of 50 mg of DTG film-coated tablets.

Intervention Type DRUG

DTG granules for suspension

DTG granules for suspension initial starting dose at \~0.64 mg/kg with a maximum dose of 32 mg; orally once daily.

Intervention Type DRUG

DTG dispersible tablets

DTG dispersible tablets initial starting dose of \~0.8 mg/kg with a maximum dose of 30 mg; orally once daily. The proposed weight-band dosing was:

Weight band 3 to \<6 kg: 5 mg DTG dispersible tablets; Weight band 6 to \<10 kg: 15 mg DTG dispersible tablets; Weight band 10 to \<14 kg: 20 mg DTG dispersible tablets; Weight band 14 to \<20 kg: 25 mg DTG dispersible tablets; Weight band ≥20 kg: 30 mg DTG dispersible tablets.

Intervention Type DRUG

DTG dispersible tablets

DTG dispersible tablets initial starting dose of \~1.25 mg/kg with a maximum dose of 30 mg; orally once daily. The proposed weight-band dosing was:

Weight band 3 to \<6 kg: 5 mg DTG dispersible tablets; Weight band 6 to \<10 kg: 15 mg DTG dispersible tablets; Weight band 10 to \<14 kg: 20 mg DTG dispersible tablets; Weight band 14 to \<20 kg: 25 mg DTG dispersible tablets; Weight band ≥20 kg: 30 mg DTG dispersible tablets.

Intervention Type DRUG

DTG dispersible tablets

DTG dispersible tablets initial starting dose of \~1.25 mg/kg with a maximum dose of 30 mg; orally once daily. The proposed weight-band dosing was:

Weight band 3 to \<6 kg: 5 mg DTG dispersible tablets; Weight band 6 to \<10 kg: 15 mg DTG dispersible tablets.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Confirmed HIV-1 infection, defined as positive results from two samples collected at different time points (see protocol for more information)
* Participant belonged to one of the ARV exposure groups below:

1. ARV-treatment experienced (not including receipt of ARVs as prophylaxis or for prevention of perinatal transmission)

* Previously took ARVs for treatment, but not taking ARVs at study screening.
* Had been off treatment for greater than or equal to 4 weeks prior to screening, OR
* At screening, taking ARVs for treatment but failing.
* Was on an unchanged, failing therapeutic regimen within the 4 to 12 weeks prior to screening (less than or equal to 1 log drop in HIV-1 RNA within the 4 to 12 weeks prior to screening). OR
* For participants less than 2 years of age, initiated ARVs for treatment less than 4 weeks prior to screening.
2. ARV treatment naive (no exposure to ARVs for treatment; could have received ARVs for prophylaxis or prevention of perinatal transmission)
* If an infant had received nevirapine (NVP) as prophylaxis to prevent perinatal transmission, he or she did not receive NVP for at least 14 days prior to enrollment into Stage I or II.
* HIV-1 RNA viral load greater than 1,000 copies/mL of plasma at screening.
* Demonstrated ability or willingness to swallow assigned study medications.
* Parent or legal guardian were able and willing to provide signed informed consent.
* Female participants of reproductive potential, defined as having reached menarche, and who were engaging in sexual activity that could lead to pregnancy, agreed to use two contraceptive methods while on study and for two weeks after stopping study drug.
* Males engaging in sexual activity that could lead to HIV-1 transmission agreed to use a condom.
* Agreed to stay on optimized background therapy (OBT) while on study:

* Participants who at screening were greater than or equal to 2 years of age and ARV-treatment experienced, had at screening at least one fully active drug for the OBT.
* Participants who were greater than or equal to 2 years of age and ARV-treatment naïve, had genotype testing at screening/entry even with results pending.
* Participants less than 2 years of age (either ARV-treatment experienced or ARV treatment naïve), had genotype testing at screening/entry even with results pending.

Exclusion Criteria

* Presence of any active AIDS-defining opportunistic infection
* At enrollment, participant less than 3.0 kg
* Known Grade 3 or greater of any of the following laboratory toxicities within 30 days prior to study entry: neutrophil count, hemoglobin, platelets, aspartate aminotransferase (AST), alanine transaminase (ALT), lipase, serum creatinine and total bilirubin. A single repeat within the 30 days is allowed for eligibility determination. NOTE: Grade 3 total bilirubin was allowable, if the participant was on atazanavir (ATV).
* ANY known Grade 4 laboratory toxicities within 30 days prior to study entry. NOTE: Grade 4 total bilirubin was allowable, if the participant was on ATV.
* The following liver toxicities within 30 days prior to study entry: ALT greater than 3x the upper limit of normal (ULN) AND direct bilirubin was greater than 2x ULN
* Any prior history of malignancy, with the exception of localized malignancies such as squamous cell or basal cell carcinoma of the skin
* Clinical or symptomatic evidence of pancreatitis, as determined by the clinician
* Use of any disallowed medications at time of screening (see the protocol for a complete list of disallowed medications)
* Known history of exposure to integrase inhibitor treatment by the participant or participant's mother prior to delivery/cessation of breastfeeding
* Known resistance to an integrase inhibitor
* Women who were pregnant or breastfeeding
* At screening/entry, participating in or had participated in a study with a compound or device that was not commercially available within 30 days of signing informed consent, unless permission from both Protocol Teams was granted
* Participant was unlikely to adhere to the study procedures, keep appointments, or was planning to relocate during the study to a non-IMPAACT study site
* Any clinically significant diseases (other than HIV infection) or clinically significant findings during the screening medical history or physical examination that, in the investigator's opinion, would compromise the outcome of this study
* At screening/entry had used, or anticipated using, chronic systemic immunosuppressive agents or systemic interferon (e.g., for treatment of hepatitis C virus \[HCV\] infection) within 30 days prior to beginning DTG study treatment. Systemic corticosteroids (e.g., prednisone or equivalent up to 2 mg/kg/day) for replacement therapy or short courses (less than or equal to 30 days) were permitted. (See protocol for more information on disallowed medications.)
* Any condition that in the opinion of the site investigator, placed the participant at an unacceptable risk of injury or render the participant unable to meet the requirements of the protocol.
* Active tuberculosis (TB) disease and/or requirement for treatment that included rifampin at the time of the screening visit. However, participants who needed rifampin treatment while on DTG were allowed to continue in P1093 provided the DTG dose was adjusted according to the protocol.
Minimum Eligible Age

4 Weeks

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Allergy and Infectious Diseases (NIAID)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Andrew Wiznia, M.D.

Role: STUDY_CHAIR

Jacobi Medical Center

Theodore Ruel, M.D.

Role: STUDY_CHAIR

University of California, San Francisco

Locations

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University of California, UC San Diego CRS- Mother-Child-Adolescent HIV Program (Site ID: 4601)

La Jolla, California, United States

Site Status

Miller Children's Hosp. Long Beach CA NICHD CRS (Site ID: 5093)

Long Beach, California, United States

Site Status

David Geffen School of Medicine at UCLA NICHD CRS (Site ID: 5112)

Los Angeles, California, United States

Site Status

Univ. of California San Francisco NICHD CRS (Site ID: 5091)

San Francisco, California, United States

Site Status

Univ. of Colorado Denver NICHD CRS (Site ID: 5052)

Aurora, Colorado, United States

Site Status

Howard Univ. Washington DC NICHD CRS (Site ID: 5044)

Washington D.C., District of Columbia, United States

Site Status

South Florida CDTC Ft Lauderdale NICHD CRS (Site ID: 5055)

Fort Lauderdale, Florida, United States

Site Status

USF - Tampa NICHD CRS (Site ID: 5018)

Tampa, Florida, United States

Site Status

Rush Univ. Cook County Hosp. Chicago NICHD CRS (Site ID: 5083)

Chicago, Illinois, United States

Site Status

Lurie Children's Hospital of Chicago (LCH) CRS (Site ID: 4001)

Chicago, Illinois, United States

Site Status

Children's Hosp. of Boston NICHD CRS (Site ID: 5009)

Boston, Massachusetts, United States

Site Status

Boston Medical Center Ped. HIV Program NICHD CRS (Site ID: 5011)

Boston, Massachusetts, United States

Site Status

Metropolitan Hosp. NICHD CRS (Site ID: 5003)

New York, New York, United States

Site Status

Bronx-Lebanon Hospital Center NICHD CRS (Site ID: 5114)

The Bronx, New York, United States

Site Status

Jacobi Med. Ctr. Bronx NICHD CRS (Site ID: 5013)

The Bronx, New York, United States

Site Status

DUMC Ped. CRS (Site ID: 4701)

Durham, North Carolina, United States

Site Status

Seattle Children's Research Institute CRS (Site ID: 5017)

Seattle, Washington, United States

Site Status

Gaborone CRS (Site ID: 12701)

Gaborone, South-East District, Botswana

Site Status

Molepolole CRS (Site ID: 12702)

Gaborone, , Botswana

Site Status

SOM Federal University Minas Gerais Brazil NICHD CRS (Site ID: 5073)

Belo Horizonte, Minas Gerais, Brazil

Site Status

Hospital Federal dos Servidores do Estado NICHD CRS (Site ID: 5072)

Rio de Janeiro, , Brazil

Site Status

Instituto de Puericultura e Pediatria Martagao Gesteira - UFRJ NICHD CRS (Site ID: 5071)

Rio de Janeiro, , Brazil

Site Status

Hosp. Geral De Nova Igaucu Brazil NICHD CRS (Site ID: 5097)

Rio de Janeiro, , Brazil

Site Status

Univ. of Sao Paulo Brazil NICHD CRS (Site ID: 5074)

São Paulo, , Brazil

Site Status

Kenya Medical Research Institute / Walter Reed Project Clinical Research Center, Kericho CRS (Site ID: 5121)

Kericho, , Kenya

Site Status

Wits RHI Shandukani Research Centre CRS (Site ID: 8051)

Johannesburg, Gauteng, South Africa

Site Status

Umlazi CRS (Site ID: 30300)

Durban, KwaZulu-Natal, South Africa

Site Status

FAMCRU CRS (Site ID: 8950)

Tygerberg, Western Cape, South Africa

Site Status

Kilimanjaro Christian Medical Centre (KCMC) (Site ID: 5118)

Moshi, , Tanzania

Site Status

Siriraj Hospital, Mahidol University NICHD CRS (Site ID: 5115)

Bangkok, Bangkoknoi, Thailand

Site Status

Chiangrai Prachanukroh Hospital NICHD CRS (Site ID: 5116)

Chiang Mai, , Thailand

Site Status

Chiang Mai University HIV Treatment (CMU HIV Treatment) CRS (Site ID: 31784)

Chiang Mai, , Thailand

Site Status

Harare Family Care CRS (Site ID: 31890)

Harare, , Zimbabwe

Site Status

Countries

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Argentina Puerto Rico Uganda United States Botswana Brazil Kenya South Africa Tanzania Thailand Zimbabwe

References

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Bennetto-Hood C, Tabolt G, Savina P, Acosta EP. A sensitive HPLC-MS/MS method for the determination of dolutegravir in human plasma. J Chromatogr B Analyt Technol Biomed Life Sci. 2014 Jan 15;945-946:225-32. doi: 10.1016/j.jchromb.2013.11.054. Epub 2013 Dec 3.

Reference Type BACKGROUND
PMID: 24361860 (View on PubMed)

Viani RM, Alvero C, Fenton T, Acosta EP, Hazra R, Townley E, Steimers D, Min S, Wiznia A; P1093 Study Team. Safety, Pharmacokinetics and Efficacy of Dolutegravir in Treatment-experienced HIV-1 Infected Adolescents: Forty-eight-week Results from IMPAACT P1093. Pediatr Infect Dis J. 2015 Nov;34(11):1207-13. doi: 10.1097/INF.0000000000000848.

Reference Type BACKGROUND
PMID: 26244832 (View on PubMed)

Viani RM, Ruel T, Alvero C, Fenton T, Acosta EP, Hazra R, Townley E, Palumbo P, Buchanan AM, Vavro C, Singh R, Graham B, Anthony P, George K, Wiznia A; P1093 Study Team. Long-Term Safety and Efficacy of Dolutegravir in Treatment-Experienced Adolescents With Human Immunodeficiency Virus Infection: Results of the IMPAACT P1093 Study. J Pediatric Infect Dis Soc. 2020 Apr 30;9(2):159-165. doi: 10.1093/jpids/piy139.

Reference Type BACKGROUND
PMID: 30951600 (View on PubMed)

Ruel TD, Acosta EP, Liu JP, Gray KP, George K, Montanez N, Popson S, Buchanan AM, Bartlett M, Dayton D, Anthony P, Brothers C, Vavro C, Singh R, Koech L, Vhembo T, Mmbaga BT, Pinto JA, Dobbels EFM, Archary M, Chokephaibulkit K, Ounchanum P, Deville JG, Hazra R, Townley E, Wiznia A; IMPAACT P1093 team. Pharmacokinetics, safety, tolerability, and antiviral activity of dolutegravir dispersible tablets in infants and children with HIV-1 (IMPAACT P1093): results of an open-label, phase 1-2 trial. Lancet HIV. 2022 May;9(5):e332-e340. doi: 10.1016/S2352-3018(22)00044-3.

Reference Type DERIVED
PMID: 35489377 (View on PubMed)

Singh RP, Adkison KK, Baker M, Parasrampuria R, Wolstenholme A, Davies M, Sewell N, Brothers C, Buchanan AM. Development of Dolutegravir Single-entity and Fixed-dose Combination Formulations for Children. Pediatr Infect Dis J. 2022 Mar 1;41(3):230-237. doi: 10.1097/INF.0000000000003366.

Reference Type DERIVED
PMID: 34817414 (View on PubMed)

Vavro C, Ruel T, Wiznia A, Montanez N, Nangle K, Horton J, Buchanan AM, Stewart EL, Palumbo P. Emergence of Resistance in HIV-1 Integrase with Dolutegravir Treatment in a Pediatric Population from the IMPAACT P1093 Study. Antimicrob Agents Chemother. 2022 Jan 18;66(1):e0164521. doi: 10.1128/AAC.01645-21. Epub 2021 Oct 25.

Reference Type DERIVED
PMID: 34694878 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol: Protocol V5.0

View Document

Document Type: Study Protocol: Protocol V5.0_Letter of Amendment 1

View Document

Document Type: Statistical Analysis Plan: Primary Statistical Analysis Plan

View Document

Document Type: Statistical Analysis Plan: Pharmacokinetics (PK) Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Related Links

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https://rsc.niaid.nih.gov/clinical-research-sites/daids-adverse-event-grading-tables

The Division of AIDS Table for Grading the Severity of Adult and Pediatric Adverse Events (DAIDS AE Grading Table), Version 1.0, December 2004 (Clarification, August 2009).

https://rsc.niaid.nih.gov/clinical-research-sites/manual-expedited-reporting-adverse-events-daids

Manual for Expedited Reporting of Adverse Events to DAIDS (DAIDS EAE Manual), Version 2.0, January 2010

Other Identifiers

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11773

Identifier Type: REGISTRY

Identifier Source: secondary_id

2010-020988-20

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

IMPAACT P1093

Identifier Type: -

Identifier Source: secondary_id

P1093

Identifier Type: -

Identifier Source: org_study_id