Safety Study of CALAA-01 to Treat Solid Tumor Cancers

NCT ID: NCT00689065

Last Updated: 2013-11-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-05-31

Study Completion Date

2012-09-30

Brief Summary

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Rationale: CALAA-01 is a targeted therapeutic designed to inhibit tumor growth and/or reduce tumor size. The active ingredient in CALAA-01 is a small interfering RNA (siRNA). This siRNA inhibits tumor growth via RNA interference to reduce expression of the M2 subunit of ribonucleotide reductase (R2). The CALAA-01 siRNA is protected from nuclease degradation within a stabilized nanoparticle targeted to tumor cells.

PURPOSE: This phase I trial will:

* Determine the safety, toxicity, and the maximum tolerated dose (MTD) of CALAA-01 when administered intravenously to patients with relapsed or refractory cancer.
* Characterize the pharmacokinetics (PK) of CALAA-01 after intravenous administration.
* Provide preliminary evidence of efficacy of intravenous CALAA-01 by evaluating tumor response.
* Recommend a dose of intravenous CALAA-01 for future clinical studies.
* Evaluate immune response, by measuring antibody and cytokine levels, and the effect of intravenous CALAA-01 on complement.

Detailed Description

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CALAA-01 is a targeted nanocomplex that contains anti-R2 siRNA. The complete nanocomplex formulation consists of four components:

1. a duplex of synthetic, non-chemically-modified siRNA (C05C)
2. a cyclodextrin-containing polymer (CAL101),
3. a stabilizing agent (AD-PEG), and
4. a targeting agent (AD-PEG-Tf) that contains the human transferrin protein (Tf). The cationic polymer interacts electrostatically with anionic siRNA to assemble into nanocomplexes below approximately 100 nm in diameter that protect the siRNA from nuclease degradation in serum. The siRNA-containing nanocomplexes are targeted to cells that over express the transferrin receptor (TfR). Upon reaching a target cell, transferrin binds to TfRs on the cell surface and the siRNA-containing nanocomplex enters the cell by endocytosis. Inside the cell, chemistry built into the polymer achieves unpackaging of the siRNA from the nanocomplex, permitting it to function via RNA interference.

Conditions

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Cancer Solid Tumor

Keywords

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siRNA RNA interference (RNAi) Cyclodextrin Cancer Neoplasms Solid Tumor Ovarian Cancer Lung Cancer Non Small Cell Lung Cancer Pancreatic Cancer Breast Cancer Colon Cancer Endometrial Cancer Kidney (Renal Cell) Cancer Melanoma Prostate Cancer Skin Cancer Thyroid Cancer Solid Malignancies

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CALAA-01

Group Type EXPERIMENTAL

CALAA-01

Intervention Type DRUG

Subjects with solid tumors who satisfy the eligibility criteria will receive two, 21-day cycles of CALAA-01. A cycle will consist of four (4) 30-minute intravenous infusions administered on days 1, 3, 8, and 10 followed by 11 days of rest. If safe, a second 21-day cycle will be administered consisting of infusions on days 22, 24, 29 and 31 followed by 11 days of rest.

Interventions

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CALAA-01

Subjects with solid tumors who satisfy the eligibility criteria will receive two, 21-day cycles of CALAA-01. A cycle will consist of four (4) 30-minute intravenous infusions administered on days 1, 3, 8, and 10 followed by 11 days of rest. If safe, a second 21-day cycle will be administered consisting of infusions on days 22, 24, 29 and 31 followed by 11 days of rest.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Subjects must be at least eighteen (18) years of age.
* Subjects must have the following:

* Histologically- or cytologically-confirmed solid malignancy that is measurable or non-measurable recurrent or metastatic disease (i.e., evaluable; e.g., cytologically or radiologically-detectable disease, markers, etc.)
* Measurable disease is metastatic or unresectable
* Standard curative or palliative measures do not exist, are no longer effective, or are unlikely to be effective.
* Subjects must have tumors that have recurred after previous surgery and/or radiation.
* Subjects must have received prior adjuvant, neoadjuvant, or any other therapy for metastatic disease. No restriction is placed on the number of cycles or regimens of prior therapy.
* Subjects must have fully recovered from diagnostic or therapeutic surgery (i.e., complete wound healing).
* Subjects must have fully recovered from prior radiotherapy for local symptom palliation.
* Subjects must have recovered from the toxic effects of prior therapy.
* Women and men of child-bearing/conceiving potential must be willing to use highly effective contraceptive methods during the course of the study. Any female who is not sexually active must agree to begin using highly effective contraceptive methods if she becomes sexually active during the study. Females who are post-menopausal (i.e., no longer menstruating) must have been so for two (2) years.
* Females of child-bearing potential (e.g., not surgically sterilized or two (2) years post-menopausal) must have a negative urine pregnancy test at screening. Positive tests will be confirmed serologically.
* Subjects must have adequate marrow, hepatic, and renal function at the time of screening,.
* Subjects must be willing and able, in the opinion of the Investigator, to comply with the protocol tests and procedures.
* Subjects must be willing and able to give written informed consent.

Exclusion Criteria

* Pregnant or nursing females.
* Clinically-evident (e.g., abdominal distention, bulging and/or fluid wave) ascites or Grade 3 peripheral edema.
* Allergy(ies) to contrast media required for protocol testing.
* History of significant weight loss within four (4) weeks prior to baseline.
* Evidence of active, uncontrolled infection or unstable or severe intercurrent medical conditions.
* Peripheral venous access insufficient to permit infusion of intravenous CALAA-01 and acquisition of laboratory specimens.
* Alcoholism (dependency), alcohol or substance abuse within twelve (12) months prior to screening that has caused health consequences.
* Immunocompromised subjects, subjects with known autoimmune conditions, active hepatitis or human immunodeficiency virus (HIV) seropositivity.
* Prior gene transfer therapy or prior therapy with a cytolytic virus of any type.
* Any electrocardiogram (ECG) abnormality at screening documented by the Principal Investigator as clinically significant.
* Vaccinations of any kind within thirty (30) days of baseline.
* Use of any investigational agent or device within thirty (30) days of CALAA-01 administration.
* Any concomitant medical or psychiatric condition or social situation that would make it difficult to comply with protocol requirements.
* Subjects requiring anticonvulsants.
* Radiotherapy, cytotoxic chemotherapy, biologic, hormonal or immunotherapy or bone marrow transplantation within four (4) weeks of baseline; nitroureas within six (6) weeks. Current use of growth factors.
* A myocardial infarction within six (6) months prior to enrollment or having New York Hospital Association (NYHA) Class III or IV heart failure, uncontrolled angina, cardiomyopathy, severe uncontrolled ventricular arrhythmias, left bundle branch block, or electrocardiographic evidence of acute ischemic or active conduction system abnormalities (e.g., Long QT interval, Torsade de Pointes).
* Poorly controlled hypertension
* Prior corticosteroids as anticancer therapy within seven (7) days of baseline.
* Active CNS metastases or currently receiving dexamethasone for CNS disease.
* Major surgery within four (4) weeks of baseline.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Calando Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Antoni Ribas, M.D.

Role: PRINCIPAL_INVESTIGATOR

UCLA Jonsson Comprehensive Cancer Center

Anthony W Tolcher, M.D.

Role: PRINCIPAL_INVESTIGATOR

START (South Texas Accelerated Research Therapeutics)

Yun Yen, M.D., Ph.D.

Role: PRINCIPAL_INVESTIGATOR

City of Hope National Medical Center

Locations

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City of Hope National Medical Center

Duarte, California, United States

Site Status

UCLA Jonsson Comprehensive Cancer Center

Los Angeles, California, United States

Site Status

START (South Texas Accelerated Research Therapeutics)

San Antonio, Texas, United States

Site Status

Countries

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United States

References

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Heidel JD, Yu Z, Liu JY, Rele SM, Liang Y, Zeidan RK, Kornbrust DJ, Davis ME. Administration in non-human primates of escalating intravenous doses of targeted nanoparticles containing ribonucleotide reductase subunit M2 siRNA. Proc Natl Acad Sci U S A. 2007 Apr 3;104(14):5715-21. doi: 10.1073/pnas.0701458104. Epub 2007 Mar 22.

Reference Type BACKGROUND
PMID: 17379663 (View on PubMed)

Heidel JD, Liu JY, Yen Y, Zhou B, Heale BS, Rossi JJ, Bartlett DW, Davis ME. Potent siRNA inhibitors of ribonucleotide reductase subunit RRM2 reduce cell proliferation in vitro and in vivo. Clin Cancer Res. 2007 Apr 1;13(7):2207-15. doi: 10.1158/1078-0432.CCR-06-2218.

Reference Type BACKGROUND
PMID: 17404105 (View on PubMed)

Bartlett DW, Davis ME. Impact of tumor-specific targeting and dosing schedule on tumor growth inhibition after intravenous administration of siRNA-containing nanoparticles. Biotechnol Bioeng. 2008 Mar 1;99(4):975-85. doi: 10.1002/bit.21668.

Reference Type BACKGROUND
PMID: 17929316 (View on PubMed)

Heidel JD. Linear cyclodextrin-containing polymers and their use as delivery agents. Expert Opin Drug Deliv. 2006 Sep;3(5):641-6. doi: 10.1517/17425247.3.5.641.

Reference Type BACKGROUND
PMID: 16948559 (View on PubMed)

Davis ME, Zuckerman JE, Choi CH, Seligson D, Tolcher A, Alabi CA, Yen Y, Heidel JD, Ribas A. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature. 2010 Apr 15;464(7291):1067-70. doi: 10.1038/nature08956. Epub 2010 Mar 21.

Reference Type RESULT
PMID: 20305636 (View on PubMed)

Related Links

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http://www.calandopharma.com/

Calando Pharmaceuticals Website

Other Identifiers

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CALAA-01-ST-001

Identifier Type: -

Identifier Source: org_study_id