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Langerhans Cell HistiocytosisPhase 2NCT01395004

A Study to Test the Ability of and Safety of GSK2110183 in Treating Langerhans Cell Histiocytosis

A Phase 2 study testing GSK2110183 in people with Langerhans Cell Histiocytosis, run by GlaxoSmithKline at 5 sites 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

The purpose of this study was to assess safety and efficacy at months 3 and 6 in patients with Langerhans Cell Histiocytosis given daily oral doses of GSK2110183. Read more about this study

Who is it for?
Ages 12 years and older
Conditions the study is for
Langerhans Cell Histiocytosis
What is being tested?
GSK2110183
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
17 participants
Where
5 sitesUnited States
Study dates
Nov 2011to Nov 2013
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 12 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Written informed consent is provided. Adolescents will provide assent, with consent provided by parent or legal guardian.
  • Prior therapy restrictions:
  • Refractory/reactivation stratum: Age >= 12 years of age at the time of study enrollment and Tanner Stage >2. Subjects less than 18 years old must weigh at least 40 kg.
  • Treatment-naïve stratum (defined as either no prior treatment or intolerant of first-line treatment with cessation prior to first response evaluation): Age >= 18 years old
  • Histologically- or cytologically-confirmed diagnosis of Langerhans Cell Histiocytosis requiring systemic treatment:
  • SS-LCH with 'CNS-risk' or 'special site' lesions or 'risk organs' (Includes LCH brain lesions or pituitary infiltration provided that disease is evaluable for treatment response. Patients with isolated pulmonary disease should have recent onset (within 3 years) or demonstrate evidence of active disease on PET or other functional imaging. If isolated pulmonary disease without radiographic lesion, then pulmonary function testing must demonstrate vital capacity <80% predicted, FEV1 <80% predicted or DLCO, <70% predicted, and condition is evaluable for treatment response and could not be explained through a diagnosis of asthma.)
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  • SS-LCH with multifocal bone or skin disease
  • MS-LCH with or without involvement of 'risk organs'
  • Archival tumor available for central confirmation of LCH and biomarker analysis or willingness to undergo biopsy.
  • If 18 years or older, performance status score of 0, 1, and 2 according to the Eastern Cooperative Oncology Group (ECOG) scale. If age >=12 and <18, performance status score of >50 according to the Karnofsky performance status (KPS) scale.
  • Able to swallow and retain oral medication.
  • Male subjects with a female partner of childbearing potential must have had a prior vasectomy or agree to use adequate contraception from the time of the first dose of study drug until three months after the last dose of study drug.
  • A female subject is eligible to participate if she is of:
  • Non-childbearing potential (i.e., physiologically incapable of becoming pregnant) defined as pre-menopausal females with a documented tubal ligation or hysterectomy; or postmenopausal defined as 12 months of spontaneous amenorrhea [in questionable cases a blood sample with simultaneous follicle stimulating hormone (FSH) > 40 MlU/mL and estradiol < 40 pg/mL (<140 pmol/L) is confirmatory]. Females on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use one of the contraception methods defined in the protocol if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of post-menopausal status prior to study enrolment. For most forms of HRT, at least two to four weeks will elapse between the cessation of therapy and the blood draw; this interval depends on the type and dosage of HRT. Following confirmation of their post-menopausal status, they can resume use of HRT during the study without use of a contraceptive method.
  • Child-bearing potential, has a negative serum pregnancy test during the screening period, and agrees to use adequate contraception from screening until four weeks after the last dose of study drug.
  • Note: Oral contraceptives are not reliable due to potential drug-drug interaction.
  • Adequate organ-systems function.

Who cannot join

  • Chemotherapy, radiotherapy, or immunotherapy within 28 days (or 42 days for prior nitrosoureas or mitomycin C) prior to the first dose of study drug. Corticosteroids may be administered up to seven days prior to the first dose of study drug. Chemotherapy regimens given continuously or on a weekly basis with limited potential for delayed toxicity (in the medical opinion of the investigator) are permitted, if administered at least 14 days prior to the first dose of study drug.
  • Use of an investigational anti-cancer drug within 28 days or five half-lives, whichever is longer, preceding the first dose of GSK2110183.
  • Current use of a prohibited medication or requires any of these medications during treatment with GSK2110183.
  • Presence of active gastrointestinal disease or other condition that could affect gastrointestinal absorption (e.g. malabsorption syndrome) or predispose subject to gastrointestinal ulceration.
  • Any major surgery within the last four weeks (excluding diagnostic biopsy).
  • Unresolved toxicity (except alopecia) >= Grade 2 from previous therapy, except where the Investigator considers that the ongoing toxicity will not introduce additional risk factors and will not interfere with the study procedures.
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  • Fasting serum glucose >126 mg/dL (7 mmol/L), or for known diabetic patients with stable disease, fasting serum glucose >250 mg/dL (14 mmol/L) and Hemoglobin A1c > 9%.
  • Current use of oral corticosteroids, with the exception of stress dose hydrocortisone (20 mg BID). Inhaled steroids permitted if patient is currently on a stable dose/regimen. Topical steroids are permitted; however, they should not be used as concurrent treatment for LCH skin disease.
  • Any serious or unstable pre-existing medical, psychiatric, or other condition (including lab abnormalities) that could interfere with subject safety or with obtaining informed consent.
  • Evidence of LCH-related neurodegenerative disease if patient has CNS SS-LCH, however, subjects with CNS SS-LCH who were previously treated for CNS involvement, and are currently asymptomatic without anti-epileptic medications or steroids for at least two months are eligible.
  • Evidence of severe or uncontrolled systemic diseases (e.g., unstable or uncompensated respiratory, hepatic, renal, or cardiac disease).
  • Known infection with HIV, HBV or HCV.
  • QTc interval >= 470 msecs.
  • History of myocardial infarction, acute coronary syndromes (including unstable angina), coronary angioplasty, or stenting or bypass grafting within the past six months.
  • Class III or IV heart failure as defined by the New York Heart Association (NYHA) functional classification system.
  • Pregnant or lactating female.
  • History of previous treatment with a small molecule AKT inhibitor (this does not include perifosine), PI3K inhibitors, or mTOR inhibitors.
  • Other associated conditions which in the opinion of the investigator could significantly impair the ability to evaluate the impact of treatment on the clinical course of the disease.

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

United States5 sites

  • Johns Hopkins Hospital

    Baltimore, Maryland, 21287

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

  • Baylor College of Medicine

    Houston, Texas, 77030

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Staten Island University Hospital

    Staten Island, New York, 10305

Other trials in United States

About this study

Official title: A Phase 2a, Open Label, Multicenter Study to Assess the Efficacy and Safety of the Oral AKT Inhibitor GSK2110183 in Subjects With Langerhans Cell Histiocytosis

The purpose of this study was to assess safety and efficacy at months 3 and 6 in patients with Langerhans Cell Histiocytosis given daily oral doses of GSK2110183.

Read the detailed description

LCH115397 was a phase 2a, open label, multicenter study testing 125 mg (starting dose) daily oral GSK2110183 in adult and adolescent patients with Langerhans Cell Histiocytosis. The primary objectives of the study were efficacy (at 3 and 6 months) and safety. For the purpose of efficacy analysis, patients were stratified into two groups: treatment-naïve (Stratum 1) and refractory or reactivation disease (Stratum 2). Adolescent participation was limited to patients with refractory or reactivation disease (Stratum 2).

Being tested
GSK2110183
Medical subject headings
Histiocytosis, Langerhans-Cell

What participants receive

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

  • GSK2110183Drug

    GSK2110183 was orally administered at 125 mg once daily.

Study groups in NCT01395004
Study groupRoleWhat they receive
Active Drug - GSK2110183ExperimentalThis was an open-label study of oral GSK211083 administered at the maximum tolerated dose of 125 mg once daily.
Receives: Drug: GSK2110183

What is being measured

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

  1. Objective disease response at 3 and 6 months

    Measured: Up to 6 months

  2. Proportion of subjects experiencing greater than or equal to grade 3 neutropenia

    Measured: Up to week 53

  3. Proportion of patients who experience microbial infections

    Measured: Up to week 53

  4. Proportion of subjects experiencing newly diagnosed or worsening neuropathy

    Measured: Up to week 53

Timeline

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

  1. First posted15 Jul 2011 (estimated)
  2. Study startNov 2011
  3. Primary completionNov 2013
  4. Study completionNov 2013

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
Basic science
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
17 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01395004
Sponsor's protocol ID
115397
Status last verified
Sep 2020
First submitted
12 Jul 2011
Last updated
9 Sep 2020
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing GSK2110183 in people with Langerhans Cell Histiocytosis, run by GlaxoSmithKline at 5 sites 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 17 inclusion and 18 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 9 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites 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. 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?

Objective disease response at 3 and 6 months (time frame: Up to 6 months). The study lists 4 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 NCT01395004.

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 9 Sep 2020.

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