MedPath

Immunotherapy Study for Patients With Stage IV Melanoma

Phase 2
Terminated
Conditions
Stage IV Melanoma
Metastatic Melanoma
Interventions
Drug: HyperAcute®-Melanoma (HAM) Immunotherapy
Registration Number
NCT02054520
Lead Sponsor
NewLink Genetics Corporation
Brief Summary

The purpose of this study is to examine the effectiveness of immune checkpoint inhibitors (drugs called ipilimumab, nivolumab, or pembrolizumab), either given alone, or in combination with the experimental immunotherapy drug, dorgenmeltucel-L, for melanoma. We hypothesize that this form of combinatorial immunotherapy will result in tumor stabilization or shrinkage, significant prolongation of progression-free, disease-free or overall survival compared to the use of immune checkpoint inhibitors alone.

Detailed Description

According to statistics of the American Cancer Society, an estimated 73,800 individuals will be diagnosed with melanoma and 9,900 will die of the disease in 2015 in the Unites States despite current therapy. This protocol attempts to exploit an approach to melanoma immunotherapy using a naturally occurring barrier to xenotransplantation in humans to increase the effectiveness of immunizing patients against their melanoma. The expression of the murine (1,3)galactosyltransferase \[alpha(1,3)GT\] gene results in the cell surface expression of (1,3)galactosyl-epitopes (alpha-gal) on membrane glycoproteins and glycolipids. These epitopes are the major target of the hyperacute rejection response that occurs when organs are transplanted from non-primate donor species into man. Human hosts often have pre-existing anti-alpha-gal antibodies that bind alpha-gal epitopes and lead to rapid activation of complement and cell lysis. The pre-existing anti-alpha-gal antibodies found in most individuals are thought to be due to exposure to alpha-gal epitopes that are naturally expressed on normal gut flora leading to chronic immunological stimulation.

These antibodies may comprise up to 1% of serum IgG. In this phase 2b study, patients with advanced stage melanoma will receive immune checkpoint inhibition consisting of ipilimumab, nivolumab, or pembrolizumab per the treating physician's standard of care. In addition to the immune checkpoint therapy, half of the patients will also receive dorgenmeltucel-L. Dorgenmeltucel-L is composed of irradiated allogeneic melanoma cell lines (HAM-1, HAM-2 and HAM-3). These cell lines have been transduced with a recombinant Moloney murine leukemia virus (MoMLV)-based retroviral vector expressing the murine (1,3)GT gene. Endpoints of the study include safety assessments, efficacy, and immunological responses.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
47
Inclusion Criteria
  • Histological/cytological diagnosis of melanoma. AJCC stage IV (any T, any N, M1), metastatic, progressive, refractory, melanoma.

  • Patients may have advanced unresectable stage IV disease, resectable stave IV disease or recently resected stage IV disease (<10 weeks prior) with no apparent disease.

  • Eastern Cooperative Oncology Group (ECOG) Performance Status ≤1.

  • Serum albumin ≥3.0 gm/dL.

  • Adequate organ function including:

    • A. Marrow: Hemoglobin ≥10.0 gm/dL, absolute granulocyte count (AGC) ≥1,000/mm3, platelets ≥75,000/mm3, absolute lymphocyte count ≥475/mm3.
    • B. Hepatic: Serum total bilirubin ≤2.5 x upper limit of normal (ULN), ALT (SGPT) and AST (SGOT) ≤2.5 x ULN.
    • C. Renal: Serum creatinine (sCr) ≤ 1.5 x upper limit of normal.
  • Prior therapy for melanoma that may include surgery, radiation therapy, immunotherapy including interleukins and interferon, and/or ≤2 different regiments of systemic chemotherapy, targeted therapy, or other experimental systemic therapies. Prior treatment with immune checkpoint inhibitors is not allowed.

  • Patients must be ≥4 weeks since major surgery, radiotherapy, chemotherapy (6 weeks if they were treated with nitrosureas) or biotherapy/targeted therapies.

  • Patients must have the ability to understand the study, its risks, side effects, potential benefits and be able to give written informed consent to participate. Patients may not be consented by a durable power of attorney (DPA).

  • Male and female subjects of child producing potential must agree to use contraception or avoidance of pregnancy measures while enrolled on study and receiving the experimental drug, and for one month after the last immunization.

Exclusion Criteria
  • Age <18-years-old.

  • Active CNS metastases or carcinomatous meningitis. Patients with CNS lesions that have been treated and who have no evidence of progression in the brain on CT/MRI for

    ≥1 month are eligible. Pregnant or nursing women due to the unknown effects of immunization on the developing fetus or newborn infant.

  • Other malignancy within five years, except the following may be eligible:

    • patients curatively treated for localized squamous or basal cell carcinoma of the skin or for carcinoma in situ of the uterine cervix (CIN) or breast,
    • patients with a history of malignant tumor who have been disease free for at least five years and are not currently being treated.
  • History of an allogeneic solid organ transplant or bone marrow transplant, or current active immunosuppressive therapy such as cyclosporine, tacrolimus, etc.

  • Subjects taking systemic (parentally or orally) corticosteroid therapy for any reason, including replacement therapy for hypoadrenalism, are not eligible. Topical steroids are acceptable as are intranasal steroids.

  • Active infection or antibiotics within 48 hours prior to study enrollment, including unexplained fever (temp > 38.1°C), if deemed clinically significant by the treating physician.

  • Evidence of active autoimmune disease (e.g., systemic lupus erythematosis, rheumatoid arthritis, with the exception of vitiligo. Patients with a remote history of asthma or mild asthma are eligible.

  • Other serious medical conditions that may be expected to limit life expectancy to less than 2 years (e.g., liver cirrhosis).

  • Any condition, psychiatric or otherwise, that would preclude informed consent, consistent follow-up or compliance with any aspect of the study (e.g., untreated schizophrenia or other significant cognitive impairment, etc).

  • Patients having previously undergone splenectomy.

  • Patients with known hepatitis or unstable liver disease, and/or positive serologies for Hepatitis B or C and HIV.

  • Patients with sickle-cell anemia or thalassemia major.

  • Subjects who received prior treatment with immune checkpoint inhibition, consisting of ipilimumab, tremelimumab, nivolumab, pembrolizumab or other antibody to CTLA4 or PD-1.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm 2B Nivolumab aloneNivolumabArm 2B will receive nivolumab alone at 3 mg/kg given every 2 weeks
Arm 1B HyperAcute®-Melanoma (HAM) + nivolumabHyperAcute®-Melanoma (HAM) ImmunotherapyArm 1B will receive nivolumab alone at 3 mg/kg given every 2 weeks and 300 Million HyperAcute®-Melanoma (HAM) Immunotherapy cells per each immunization, given every week for 4 weeks, every 2 weeks for 5 months, every month for 6 months, and every 3 months for one year.
Arm 1B HyperAcute®-Melanoma (HAM) + nivolumabNivolumabArm 1B will receive nivolumab alone at 3 mg/kg given every 2 weeks and 300 Million HyperAcute®-Melanoma (HAM) Immunotherapy cells per each immunization, given every week for 4 weeks, every 2 weeks for 5 months, every month for 6 months, and every 3 months for one year.
Arm 1C HyperAcute®-Melanoma (HAM) + pembrolizumabHyperAcute®-Melanoma (HAM) ImmunotherapyArm 1C will receive pembrolizumab at 2 mg/kg given every 3 weeks and 300 Million HyperAcute®-Melanoma (HAM) Immunotherapy cells per each immunization, given every week for 4 weeks, every 2 weeks for 5 months, every month for 6 months, and every 3 months for one year.
Arm 1A HyperAcute®-Melanoma (HAM) + IpilimumabHyperAcute®-Melanoma (HAM) ImmunotherapyArm 1A will receive ipilimumab at 3 mg/kg given every 3 weeks for 4 weeks and 300 Million HyperAcute®-Melanoma (HAM) Immunotherapy cells per each immunization, given every week for 4 weeks, every 2 weeks for 5 months, every month for 6 months, and every 3 months for one year.
Arm 2A Ipilimumab AloneIpilimumabArm 2A will receive ipilimumab alone at 3 mg/kg every 3 weeks for a total of four doses.
Arm 1A HyperAcute®-Melanoma (HAM) + IpilimumabIpilimumabArm 1A will receive ipilimumab at 3 mg/kg given every 3 weeks for 4 weeks and 300 Million HyperAcute®-Melanoma (HAM) Immunotherapy cells per each immunization, given every week for 4 weeks, every 2 weeks for 5 months, every month for 6 months, and every 3 months for one year.
Arm 1C HyperAcute®-Melanoma (HAM) + pembrolizumabPembrolizumabArm 1C will receive pembrolizumab at 2 mg/kg given every 3 weeks and 300 Million HyperAcute®-Melanoma (HAM) Immunotherapy cells per each immunization, given every week for 4 weeks, every 2 weeks for 5 months, every month for 6 months, and every 3 months for one year.
Arm 2C Pembrolizumab alonePembrolizumabArm 2C will receive pembrolizumab at 2 mg/kg given every 3 weeks
Primary Outcome Measures
NameTimeMethod
Safety and Tolerability Assessed by Development of AEs and Laboratory Parameters2 years

To determine the safety of administration of immune checkpoint inhibition consisting of ipilimumab, nivolumab, or pembrolizumab with or without dorgenmeltucel-L immunotherapy for patients with stage IV melanoma

Clinical Response Rate2 years

To estimate the clinical response rate of metastatic melanoma patients after immunotherapy with dorgenmeltucel-L immunotherapy plus immune checkpoint inhibition

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (5)

University of Tennessee Medical Center

🇺🇸

Knoxville, Tennessee, United States

University of Kansas Cancer Center

🇺🇸

Westwood, Kansas, United States

Oncology Specialists

🇺🇸

Niles, Illinois, United States

University of Iowa Hospitals and Clinics

🇺🇸

Iowa City, Iowa, United States

Wake Forest Baptist Health

🇺🇸

Winston-Salem, North Carolina, United States

© Copyright 2025. All Rights Reserved by MedPath