Safety, Tolerability and Immune Response to LC002, an Experimental Therapeutic Vaccine, in Adults Receiving HAART

NCT ID: NCT00270205

Last Updated: 2021-11-04

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

28 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-01-31

Study Completion Date

2010-09-30

Brief Summary

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LC002 is an experimental therapeutic vaccine designed to boost the immune response of people infected with HIV. The purpose of this study was to determine the safety and tolerability of and immune response to LC002 in HIV-1-infected adults who are currently receiving anti-HIV treatment.

Detailed Description

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The use of highly active antiretroviral therapy (HAART) has dramatically improved the rates of survival, morbidity, and mortality among HIV-infected people throughout the world. However, the costs, long-term toxicity, and problems with adherence associated with HAART regimens make such treatment plans less than optimal for individuals seeking treatment for HIV infection. Also, because viral reservoirs cannot be eradicated, HIV-infected people must usually be on HAART indefinitely in order to keep their infection under control. While the mechanism is still unclear, the immune system weakens as HIV disease progresses. A therapeutic HIV vaccine given to HIV infected people may help to promote better immune responses. LC002 is a novel HIV therapeutic vaccine containing a DNA plasmid that codes for most of HIV-1's proteins. LC002 is a unique vaccine in that it is given through topical administration; this allows for Langerhans cells (immune cells located under the surface of the skin) to pick up the vaccine and deliver it to the lymph nodes, causing an immune reaction. This study evaluated the safety, tolerability, and immunogenicity of LC002 in HIV-infected adults currently receiving HAART.

There were three cohorts in this study which were enrolled sequentially. Participants in a given cohort were randomly assigned to receive either LC002 (6 participants) or placebo (2 participants).

* In Cohort 1, participants received three separate low-dose vaccinations of LC002 (Arm A: 0.1 mg DNA/participant, 0.8 ml total, administered over two skin sites of 80 cm\^2 each, 0.4 ml/site) or 3 separate vaccinations of placebo (Arm B: 0.8 ml total, administered over two skin sites of 80 cm\^2 each, 0.4 ml/site). Vaccinations were given over two skin sites on the left and right upper back. Participants received vaccinations at weeks 1, 7, and 13.
* In Cohort 2, participants received three separate high-dose vaccinations of LC002 (Arm C: 0.4 mg DNA/participant, 3.2 ml total, administered over four skin sites of 80 cm\^2 each, 0.8 ml/site) or three separate vaccinations of placebo (Arm D: 3.2 ml total, administered over four skin sites of 80 cm\^2 each, 0.8 ml/site). Vaccinations were given over four skin sites on the left and right upper back and left and right upper ventral thigh. Participants received vaccinations at weeks 1, 7, and 13.
* In Cohort 3, participants received six separate high-dose vaccinations of LC002 (Arm E: 0.4 mg DNA/participant, 3.2 ml total, administered over four skin sites of 80 cm\^2 each, 0.8 ml/site) or six vaccinations of placebo (Arm F: 3.2 ml total, administered over four skin sites of 80 cm\^2 each, 0.8 ml/site). Vaccinations were given over four skin sites on the left and right upper back and left and right upper ventral thigh. Participants received vaccinations at study entry and weeks 1, 6, 7, 12, and 13.

The decision to open the next cohort was made when all participants in the current cohort have remained on study for \>=14 days after the second vaccination or prematurely discontinued from study or had a primary safety endpoint (see primary outcome measure definition). Dose escalation required no primary safety endpoint and on-study follow-up for \>=6 participants in the previous cohort(s).

Prior to receiving the vaccine, the chosen vaccine administration site on the back or thigh was disinfected and exfoliated. A skin patch was applied to the site, and the vaccine solution was placed on the skin underneath the patch with a needleless syringe. Participants were allowed to remove the skin patch 3 hours post vaccination. For the first and second vaccinations, participants were required to remain at the clinic for 3 hours post-vaccination so study staff can assess for side effects. If no side effects occurred after the first two vaccinations, participants were required to stay at the clinic for only 30 minutes after receiving later vaccinations.

At the start of the study, participants were asked to keep a diary and record daily any side effects or skin irritation they may have experienced following vaccination. Participants were required to bring their diaries with them to their next clinic visit. Two days after vaccination, participants were followed-up by phone and were asked about any side effects they may have experienced. Participants who experienced side effects were asked to return to the clinic for examination. There were 13 study visits; they occurred at study entry and Weeks 1, 3, 6, 7, 9, 12, 13, 15, 17, 24, 37, and 61. Study visits included medication history, a physical exam, and collection of diaries. Blood and urine collection occurred at selected visits. HAART was not be provided by the study.

Conditions

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HIV Infections

Keywords

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Treatment Experienced HIV Therapeutic Vaccine

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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A: 0.1 mg DNA/participant vaccination at weeks 1,7,13

Participants receiving three separate low-dose vaccinations of LC002 (0.1 mg DNA/participant, 0.8 ml total, administered over two skin sites \[on the left and right upper back\] of 80 cm\^2 each, 0.4 ml/site) at weeks 1, 7, and 13.

Group Type EXPERIMENTAL

LC002 standard vaccination

Intervention Type BIOLOGICAL

0.1 mg DNA/participant, 0.8 ml total administered subcutaneously

B

Participants receiving three separate vaccinations of LC002 placebo (0.8 ml total, administered over two skin sites \[on the left and right upper back\] of 80 cm\^2 each, 0.4 ml/site) at weeks 1, 7, and 13.

Group Type EXPERIMENTAL

LC002 placebo vaccination

Intervention Type BIOLOGICAL

Placebo vaccination administered subcutaneously

C: 0.4 mg DNA/participant vaccination at weeks 1, 7, 13

Participants receiving three separate high-dose vaccinations of LC002 (0.4 mg DNA/participant, 3.2 ml total, administered over four skin sites \[on the left and right upper back and left and right upper ventral thigh\] of 80 cm\^2 each, 0.8 ml/site) at weeks 1, 7, and 13.

Group Type EXPERIMENTAL

LC002 high-dose vaccination

Intervention Type BIOLOGICAL

0.4 mg DNA/participant, 3.2 ml total administered subcutaneously

D

Participants receiving three separate vaccinations of LC002 placebo (3.2 ml total, administered over four skin sites \[on the left and right upper back and left and right upper ventral thigh\] of 80 cm\^2 each, 0.8 ml/site) at weeks 1, 7, and 13.

Group Type EXPERIMENTAL

LC002 placebo vaccination

Intervention Type BIOLOGICAL

Placebo vaccination administered subcutaneously

E: 0.4 mg DNA/participant vaccination at weeks 0,1,6,7,12,13

Participants receiving six separate high-dose vaccinations of LC002 (0.4 mg DNA/participant, 3.2 ml total, administered over four skin sites \[on the left and right upper back and left and right upper ventral thigh\] of 80 cm\^2 each, 0.8 ml/site) at study entry and weeks 1, 6, 7, 12, and 13.

Group Type EXPERIMENTAL

LC002 high-dose vaccination

Intervention Type BIOLOGICAL

0.4 mg DNA/participant, 3.2 ml total administered subcutaneously

F

Participants receiving six separate vaccinations of LC002 placebo (3.2 ml total, administered over four skin sites \[on the left and right upper back and left and right upper ventral thigh\] of 80 cm\^2 each, 0.8 ml/site) at study entry and weeks 1, 6, 7, 12, and 13.

Group Type EXPERIMENTAL

LC002 placebo vaccination

Intervention Type BIOLOGICAL

Placebo vaccination administered subcutaneously

Interventions

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LC002 standard vaccination

0.1 mg DNA/participant, 0.8 ml total administered subcutaneously

Intervention Type BIOLOGICAL

LC002 high-dose vaccination

0.4 mg DNA/participant, 3.2 ml total administered subcutaneously

Intervention Type BIOLOGICAL

LC002 placebo vaccination

Placebo vaccination administered subcutaneously

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* HIV-1-infected
* On a stable HAART regimen without changes or interruptions for more than 4 consecutive days for at least 12 weeks prior to study entry. Patients must be currently taking regimens containing drugs of at least two different classes.
* Two readings of plasma HIV-1 viral load of less than 50 copies/ml within 30 days prior to study entry. More information on this criterion can be found in the protocol.
* CD4 count greater than 350 cells/mm\^3 within 12 weeks prior to study entry
* Lowest CD4 count greater than 250 cells/mm\^3 at any time prior to study entry
* Willing to use acceptable forms of contraception
* Karnofsky performance score 90 or higher obtained within 30 days prior to study entry

Exclusion Criteria

* HIV-1 viral load greater than 500 copies/ml within the 24 weeks prior to study entry
* History of or current active skin disease (e.g., atopic dermatitis, psoriasis) or any chronic autoimmune disease (e.g., Graves' disease). Participants with minor, localized skin conditions that, in the opinion of the investigator, do not represent a safety concern, are not excluded.
* Treatment with topical corticosteroids at the proposed vaccination sites (Cohort 1: left and right upper back; Cohorts 2 and 3: left and right upper back and left and right upper ventral thigh) within 2 weeks of study entry
* Excessive exposure to the sun (e.g., sunbathing, tanning bed) within 2 weeks prior to study entry
* Laser hair removal within 2 weeks prior to study entry
* Use of any local skin treatments (e.g., topical/chemical hair removal, ointments, possible irritants) to the targeted vaccination sites within 7 days prior to study entry
* History of diabetes or bleeding disorders
* Previous CDC Category C event. More information on this criterion can be found in the protocol.
* Use of immunomodulating therapy, including cyclosporine, IgG-containing products, interleukins, interferons, or systemic glucocorticosteroids (including those inhaled) within 6 months prior to study entry
* Exposure to an experimental HIV vaccine within 6 months prior to study entry
* Any vaccine within 30 days prior to study entry
* Investigational products within 12 weeks prior to study entry
* Allergy or sensitivity to study vaccine products, adhesives, or polyester
* Current drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with the study
* Serious illness requiring systemic treatment and/or hospitalization. Participants who complete therapy or are clinically stable on therapy for at least 14 days prior to study entry are not excluded.
* Positive hepatitis B surface antigen or positive anti-hepatitis C antibody at screening
* History of treatment with HAART during primary infection
* History of lymph node irradiation
* Pregnant or breastfeeding
* Certain abnormal laboratory results. More information on this criterion can be found in the protocol
Minimum Eligible Age

18 Years

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Advancing Clinical Therapeutics Globally for HIV/AIDS and Other Infections

NETWORK

Sponsor Role collaborator

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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Benigno Rodriguez, MD

Role: STUDY_CHAIR

Division of Infectious Diseases ACTU, University Hospital of Cleveland, Cleveland, OH, USA

Locations

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Univ. of California Davis Med. Ctr., ACTU

Sacramento, California, United States

Site Status

Chicago Children's CRS

Chicago, Illinois, United States

Site Status

Case CRS

Cleveland, Ohio, United States

Site Status

MetroHealth CRS

Cleveland, Ohio, United States

Site Status

University of Pittsburgh CRS

Pittsburgh, Pennsylvania, United States

Site Status

Countries

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United States

References

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Lisziewicz J, Trocio J, Xu J, Whitman L, Ryder A, Bakare N, Lewis MG, Wagner W, Pistorio A, Arya S, Lori F. Control of viral rebound through therapeutic immunization with DermaVir. AIDS. 2005 Jan 3;19(1):35-43. doi: 10.1097/00002030-200501030-00004.

Reference Type BACKGROUND
PMID: 15627031 (View on PubMed)

Lori F, Trocio J, Bakare N, Kelly LM, Lisziewicz J. DermaVir, a novel HIV immunisation technology. Vaccine. 2005 Mar 18;23(17-18):2030-4. doi: 10.1016/j.vaccine.2005.01.004.

Reference Type BACKGROUND
PMID: 15755566 (View on PubMed)

Lori F, Weiner DB, Calarota SA, Kelly LM, Lisziewicz J. Cytokine-adjuvanted HIV-DNA vaccination strategies. Springer Semin Immunopathol. 2006 Nov;28(3):231-8. doi: 10.1007/s00281-006-0047-y. Epub 2006 Oct 20.

Reference Type BACKGROUND
PMID: 17053912 (View on PubMed)

Rajcani J, Mosko T, Rezuchova I. Current developments in viral DNA vaccines: shall they solve the unsolved? Rev Med Virol. 2005 Sep-Oct;15(5):303-25. doi: 10.1002/rmv.467.

Reference Type BACKGROUND
PMID: 15906276 (View on PubMed)

Somogyi E, Xu J, Gudics A, Toth J, Kovacs AL, Lori F, Lisziewicz J. A plasmid DNA immunogen expressing fifteen protein antigens and complex virus-like particles (VLP+) mimicking naturally occurring HIV. Vaccine. 2011 Jan 17;29(4):744-53. doi: 10.1016/j.vaccine.2010.11.019. Epub 2010 Nov 23.

Reference Type BACKGROUND
PMID: 21109034 (View on PubMed)

Lorincz O, Toke ER, Somogyi E, Horkay F, Chandran PL, Douglas JF, Szebeni J, Lisziewicz J. Structure and biological activity of pathogen-like synthetic nanomedicines. Nanomedicine. 2012 May;8(4):497-506. doi: 10.1016/j.nano.2011.07.013. Epub 2011 Aug 10.

Reference Type BACKGROUND
PMID: 21839051 (View on PubMed)

Toke ER, Lorincz O, Somogyi E, Lisziewicz J. Rational development of a stable liquid formulation for nanomedicine products. Int J Pharm. 2010 Jun 15;392(1-2):261-7. doi: 10.1016/j.ijpharm.2010.03.048. Epub 2010 Mar 25.

Reference Type BACKGROUND
PMID: 20347027 (View on PubMed)

Cristillo AD, Lisziewicz J, He L, Lori F, Galmin L, Trocio JN, Unangst T, Whitman L, Hudacik L, Bakare N, Whitney S, Restrepo S, Suschak J, Ferrari MG, Chung HK, Kalyanaraman VS, Markham P, Pal R. HIV-1 prophylactic vaccine comprised of topical DermaVir prime and protein boost elicits cellular immune responses and controls pathogenic R5 SHIV162P3. Virology. 2007 Sep 15;366(1):197-211. doi: 10.1016/j.virol.2007.04.012. Epub 2007 May 11.

Reference Type BACKGROUND
PMID: 17499328 (View on PubMed)

Lisziewicz J, Trocio J, Whitman L, Varga G, Xu J, Bakare N, Erbacher P, Fox C, Woodward R, Markham P, Arya S, Behr JP, Lori F. DermaVir: a novel topical vaccine for HIV/AIDS. J Invest Dermatol. 2005 Jan;124(1):160-9. doi: 10.1111/j.0022-202X.2004.23535.x.

Reference Type BACKGROUND
PMID: 15654970 (View on PubMed)

Lisziewicz J, Rosenberg E, Lieberman J, Jessen H, Lopalco L, Siliciano R, Walker B, Lori F. Control of HIV despite the discontinuation of antiretroviral therapy. N Engl J Med. 1999 May 27;340(21):1683-4. doi: 10.1056/NEJM199905273402114. No abstract available.

Reference Type BACKGROUND
PMID: 10348681 (View on PubMed)

Calarota SA, Foli A, Maserati R, Baldanti F, Paolucci S, Young MA, Tsoukas CM, Lisziewicz J, Lori F. HIV-1-specific T cell precursors with high proliferative capacity correlate with low viremia and high CD4 counts in untreated individuals. J Immunol. 2008 May 1;180(9):5907-15. doi: 10.4049/jimmunol.180.9.5907.

Reference Type BACKGROUND
PMID: 18424710 (View on PubMed)

Gudmundsdotter L, Wahren B, Haller BK, Boberg A, Edback U, Bernasconi D, Butto S, Gaines H, Imami N, Gotch F, Lori F, Lisziewicz J, Sandstrom E, Hejdeman B. Amplified antigen-specific immune responses in HIV-1 infected individuals in a double blind DNA immunization and therapy interruption trial. Vaccine. 2011 Jul 26;29(33):5558-66. doi: 10.1016/j.vaccine.2011.01.064. Epub 2011 Feb 5.

Reference Type BACKGROUND
PMID: 21300092 (View on PubMed)

Natz E, Lisziewicz J. Rational Design of Formulated DNA Vaccines: The DermaVir Approach. In J. Thalhamer, R. Weiss & S. Scheiblhofer (Eds.), Gene Vaccines. In press.

Reference Type RESULT

Other Identifiers

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10126

Identifier Type: REGISTRY

Identifier Source: secondary_id

ACTG A5176

Identifier Type: -

Identifier Source: secondary_id

A5176

Identifier Type: -

Identifier Source: org_study_id