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AIDS-related Kaposi SarcomaPhase 2NCT00064142

Halofuginone Hydrobromide in Treating Patients With HIV-Related Kaposi's Sarcoma

A Phase 2 study testing halofuginone hydrobromide, laboratory biomarker analysis and pharmacological study in people with AIDS-related Kaposi Sarcoma and Recurrent Kaposi Sarcoma, run by National Cancer Institute (NCI) at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This phase II trial studies how well halofuginone hydrobromide works in treating patients with human immunodeficiency virus (HIV)-related Kaposi's sarcoma. Halofuginone hydrobromide ointment may stop the growth of Kaposi's sarcoma by stopping blood flow to the tumor. Read more about this study

Who is it for?
Ages 16 years and older
What is being tested?
halofuginone hydrobromide, laboratory biomarker analysis and pharmacological study
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
30 participants
Where
1 siteUnited States
Study dates
May 2003to Dec 2006
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 16 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Biopsy-proven Kaposi's sarcoma with at least 14 cutaneous lesions, 12 of which are measurable in two dimensions and can serve as marker lesions; each of the 14 lesions must measure a minimum of 0.5 cm in diameter, so that a 4 mm punch biopsy will be entirely composed of Kaposi's sarcoma
  • Serologic documentation of HIV infection by any of the Food and Drug Administration (FDA) approved tests
  • Karnofsky performance status >= 60%
  • Hemoglobin >= 8 g/dl
  • Absolute neutrophil count >= 750 cells/mm^3
  • Platelet count >= 75,000/mm^3
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  • Creatinine < 1.5 times the upper limit of normal or creatinine clearance >= 60 mL/min
  • Total bilirubin should be =< 1.5 x upper limit of normal (ULN); if, however, the elevated bilirubin is felt to be secondary to indinavir therapy, patients will be allowed to enroll on protocol if the total bilirubin is =< 3.5 mg/dl provided that the direct bilirubin is normal
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) and alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3 x the upper limit of normal
  • Life expectancy >= 3 months
  • Ability and willingness to give informed consent; patients who are younger than 18 years of age will require the consent of a parent or guardian.
  • All women of childbearing potential must have a negative serum b human chorionic gonadotropin (HCG) within 72 hours prior to study entry and must practice adequate birth control to prevent pregnancy while receiving treatment and for three months after treatment is discontinued
  • Patients must, in the opinion of the investigator, be capable of complying with the protocol
  • Patients receiving antiretroviral therapy must be on a stable regimen for at least 12 weeks prior to study entry without showing evidence of ongoing Kaposi's sarcoma (KS) regression (ie, less than 25% decrease in the size, number or nodularity of KS lesions in the opinion of the investigator); patients may receive any FDA approved antiretroviral therapy or agents available through a treatment IND; concurrent treatment with highly active antiretroviral therapy should be strongly encouraged, in accordance with DHHS guidelines (http://www.aids-ed.org/pdfs/adult_2-4-02.pdf) but will not be required for participation

Who cannot join

  • Concurrent, acute, active, untreated opportunistic infection other than oral thrush or genital herpes within 14 days of enrollment
  • Known active visceral Kaposi's sarcoma or symptomatic Kaposi's sarcoma-related edema that interferes with function or requires cytotoxic therapy
  • Concurrent neoplasia requiring cytotoxic therapy
  • Acute treatment for an infection (other than oral thrush or genital herpes) or other serious medical illness within 14 days of study entry
  • Anti-neoplastic treatment for Kaposi's sarcoma (including chemotherapy, radiation therapy, local therapy, biological therapy, or investigational therapy) within four weeks of study entry
  • Previous local therapy of any KS-indicator lesion within 60 days unless the lesion has clearly progressed since treatment
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  • Corticosteroid treatment, other than replacement doses
  • Use of investigational agents other than antiretroviral drugs available under expanded access or compassionate use protocols
  • Pregnant or breast feeding females are excluded from participation in this study since the effects of halofuginone on an unborn or young child are unknown and may potentially be toxic

Trial locations and contacts

1 site in 1 country.

United States1 site

  • AIDS - Associated Malignancies Clinical Trials Consortium

    Rockville, Maryland, 20850

Other trials in United States

About this study

Official title: A Phase II Trial of Topical Halofuginone in Patients With HIV Related Kaposi's Sarcoma

This phase II trial studies how well halofuginone hydrobromide works in treating patients with human immunodeficiency virus (HIV)-related Kaposi's sarcoma. Halofuginone hydrobromide ointment may stop the growth of Kaposi's sarcoma by stopping blood flow to the tumor.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To determine the tumor response rate of acquired immune deficiency syndrome (AIDS)-related Kaposi's sarcoma to topical halofuginone (halofuginone hydrobromide) versus vehicle control.

II. To evaluate the safety and tolerability of topical halofuginone and vehicle in patients with AIDS-related Kaposi's sarcoma.

SECONDARY OBJECTIVES:

  1. To determine the ability of topical halofuginone to inhibit expression of matrix metallopeptidase 2 (MMP-2) and Collagen type I in AIDS-related Kaposi's sarcoma.

II. To explore the relationship between baseline cluster of differentiation (CD) 4 and CD8 counts, HIV viral load and human herpesvirus 8 (HHV-8) viral load and response to halofuginone.

III. To characterize the pharmacokinetics of halofuginone.

OUTLINE: Twelve treatable Kaposi's sarcoma lesions are selected on each patient, and these 12 lesions are randomized equally to 1 of 2 treatment arms (6 lesions receive study treatment and 6 lesions receive placebo); each patient serves as his/her own control.

ARM I: Patients apply topical halofuginone hydrobromide ointment to each of 6 lesions twice a day for 12 weeks.

ARM II: Patients apply topical placebo ointment to each of 6 lesions twice a day for 12 weeks.

Patients with stable or responding disease in either or both groups of treated lesions (halofuginone hydrobromide ointment or placebo ointment) may receive open-label treatment with topical halofuginone hydrobromide ointment to all 12 lesions for an additional 12 weeks as above in the absence of disease progression or unacceptable toxicity.

Patients are followed for at least 1 month.

Being tested
halofuginone hydrobromide, placebo, laboratory biomarker analysis, pharmacological study
Medical subject headings
AIDS-related Kaposi sarcoma, Sarcoma, Kaposi

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • halofuginone hydrobromideDrug

    Also called halofuginone, halofuginone HBr, RU 19110, Tempostatin

    Applied topically

  • placeboOther

    Also called PLCB

    Applied topically

  • laboratory biomarker analysisOther

    Correlative studies

  • pharmacological studyOther

    Also called pharmacological studies

    Correlative studies

Study groups in NCT00064142
Study groupRoleWhat they receive
Arm I (halofuginone hydrobromide)ExperimentalPatients apply topical halofuginone hydrobromide ointment to each of 6 lesions twice a day for 12 weeks.
Receives: Drug: halofuginone hydrobromide; Other: laboratory biomarker analysis; Other: pharmacological study
Arm II (placebo)Placebo comparatorPatients apply topical placebo ointment to each of 6 lesions twice a day for 12 weeks.
Receives: Other: placebo; Other: laboratory biomarker analysis; Other: pharmacological study

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Response rate

    McNemar's chi-square test will be used to compare vehicle control and halofuginone with respect to response rates.

    Measured: Up to 30 days

  2. Safety of topical halofuginone as assessed by Common Terminology Criteria for Adverse Events (CTCAE) version 3.0

    McNemar's chi-square test will be used to compare the two treatments with respect to the incidence of specific adverse events.

    Measured: Up to 30 days after completion of treatment

Secondary outcomes (3)

  1. Change in MMP-2 and collagen type I levels

    Measured: From baseline to 4 weeks

  2. Change in MMP-2 and collagen type I levels

    Changes from baseline in MMP-2 and Collagen type I for halofuginone and vehicle control lesions will be compared using the Wilcoxon rank sum test.

    Measured: From baseline to 12 weeks

  3. Relationship of CD4, CD8, HIV viral load and HHV-8 viral load on response

    Logistic regression analysis will be used.

    Measured: Up to 30 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Jul 2003 (estimated)
  2. Study startMay 2003
  3. Primary completionDec 2006

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Double blind
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00064142
Sponsor's protocol ID
NCI-2012-02925
Other IDs
AMC-036, CDR0000309055, U01CA070019
Status last verified
Jun 2013
First submitted
8 Jul 2003
Last updated
5 Jun 2013
Results posted
No

Publications

  • Young SK, Baird TD, Wek RC. Translation Regulation of the Glutamyl-prolyl-tRNA Synthetase Gene EPRS through Bypass of Upstream Open Reading Frames with Noncanonical Initiation Codons. J Biol Chem. 2016 May 13;291(20):10824-35. doi: 10.1074/jbc.M116.722256. Epub 2016 Mar 21.

    PubMed 27002157

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing halofuginone hydrobromide, laboratory biomarker analysis and pharmacological study in people with AIDS-related Kaposi Sarcoma and Recurrent Kaposi Sarcoma, run by National Cancer Institute (NCI) at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Jun 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Response rate (time frame: Up to 30 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00064142, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jun 2013.

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