Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors
NCT ID: NCT01962103
Last Updated: 2019-12-27
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
107 participants
INTERVENTIONAL
2013-12-04
2018-11-06
Brief Summary
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Detailed Description
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The Phase 2 is using a Simon 2-stage design to monitor patient enrollment for each group separately. The rhabdomyosarcoma group, neuroblastoma or Ewing's sarcoma groups did not reach the expected number of 2 responders out of 14 efficacy eligible patients. Consequently, the groups were stopped.
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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nab-paclitaxel
nab-paclitaxel 100-240 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle.
nab-paclitaxel
nab-paclitaxel 120-270 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle
Phase 1: Nab-Paclitaxel 120 mg/m^2
nab-paclitaxel 120 mg/m\^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
nab-paclitaxel
IV infusion
Phase 1: Nab-Paclitaxel 150 mg/m^2
nab-paclitaxel 150 mg/m\^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
nab-paclitaxel
IV infusion
Phase 1: Nab-Paclitaxel 180 mg/m^2
nab-paclitaxel 180 mg/m\^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
nab-paclitaxel
IV infusion
Phase 1: Nab-Paclitaxel 210 mg/m^2
nab-paclitaxel 210 mg/m\^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
nab-paclitaxel
IV infusion
Phase 1: Nab-Paclitaxel 240 mg/m^2
nab-paclitaxel 240 mg/m\^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
nab-paclitaxel
IV infusion
Phase 1: Nab-Paclitaxel 270 mg/m^2
nab-paclitaxel 270 mg/m\^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
nab-paclitaxel
IV infusion
Phase 2: Ewing's Sarcoma
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m\^2 in participants weighing \> 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
nab-paclitaxel
IV infusion
Phase 2: Neuroblastoma
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m\^2 in participants weighing \> 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
nab-paclitaxel
IV infusion
Phase 2: Rhabdomyosarcoma
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m\^2 in participants weighing \> 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
nab-paclitaxel
IV infusion
Interventions
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nab-paclitaxel
nab-paclitaxel 120-270 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle
nab-paclitaxel
IV infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
1. Patient has a confirmed solid tumor diagnosis according to the
following:
1. Phase 1: patient has a recurrent or refractory solid tumor that has
progressed or did not respond to standard therapy, or for which no
standard anticancer therapy exists
2. Phase 2: patient has radiologically documented measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 (for neuroblastoma, evaluable disease by 123\^I-metaiodobenzylguanidine \[MIBG\]/Curie score is also acceptable) in 1 of the following tumor types and has failed up to 3 lines of treatment: Group 1: neuroblastoma, Group 2: rhabdomyosarcoma; Group 3: Ewing's sarcoma.
2. The patient has a Lansky/Karnofsky performance status score of ≥ 70%.
3. The patient has adequate serum chemistry levels, evidenced by the
following laboratory values
1. aspartate aminotransferase (AST)/serum glutamic-oxaloacetric
transaminase (SGOT), alanine aminotransferase (ALT)/serum glutamic
pyruvate transaminase (SGPT) ≤ 2.5 × upper limit of normal range (ULN)
2. Total bilirubin ≤ 1.5 × ULN
3. Creatinine ≤ 1.5 × ULN
4. The patient has adequate bone marrow function, evidenced by the
following:
1. Absolute neutrophil count ≥ 1.0 × 10\^9 cells/L
2. Platelets ≥ 80 × 10\^9 cells/L (transfusion independent, defined as not
receiving platelet transfusions within 7 days prior to laboratory sample). In the phase 2 portion, for patients with known bone marrow involvement, platelets ≥ 50 × 10\^9 cells/L
3. Hemoglobin ≥ 8 g/dL (transfusion is permitted to fulfill this criterion).
5. The patient (when applicable) or patient's parent(s) or legal guardian(s)
understand(s) and voluntarily signed an informed consent document prior
to any study-related assessments/procedures being conducted. Where
locally applicable, the patient also understands and voluntarily provides
his/her assent prior to any study-related assessments/procedures being
conducted.
6. Male patients of childbearing potential must use a condom during
sexual intercourse and shall not father a child during the study and for 6 months after the last dose of study medication.
7. Female patients of childbearing potential \[defined as all female
patients ≥ 12 years old or who have reached menarche, whichever occurs
first\] must have both of the following:
a. Agree to the use of two physician-approved contraceptive methods
simultaneously or practice complete abstinence while on study medication or for a longer period if required by regulations.
i. True abstinence: When this is in line with the preferred and usual
lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation,
symptothermal, postovulation methods) and withdrawal are not
acceptable methods of contraception.
ii. Acceptable contraceptive methods include: oral, injectable, or
implantable hormonal contraceptive; tubal ligation; intra-uterine device;
barrier contraceptive with spermicide; or vasectomized partner) including
at least one barrier method.
b. Have negative serum pregnancy test result at screening confirmed by
negative urine pregnancy dipstick within 72 hours prior to first dose of
investigational product (if serum test occurred \> 72 hours from first
dose); pregnancy test with sensitivity of at least 25 mIU/mL.
Exclusion Criteria
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1. The patient has a primary brain tumor(s) or brain metastasis (unless metastasis is treated and stable for \> 28 days). In patients who are symptomatic, a brain scan is required to exclude metastasis.
2. The patient has received therapeutic dose chemotherapy or radiotherapy ≤ 21 days prior to start of investigational product.
3. The patient has received maintenance dose chemotherapy (e.g., low dose cyclophosphamide) ≤ 7 days from the first dose of investigational product.
4. The patient has received any investigational therapy ≤ 28 days prior to start of investigational product. Investigational therapy is defined as any medicinal product that is not approved in the country of treatment for any indication, adult or pediatric.
5. The patient has received any biological therapy ≤ 7 days prior to the start of investigational product, or monoclonal antibody ≤ 3 half-lives or 28 days, whichever is shorter, prior to the first dose of investigational product.
6. The patient has received any hematopoietic stem cell transplantation (HSCT) ≤ 3 months prior to start of investigational product.
7. The patient has received allogeneic hematopoietic stem cell transplantation (HSCT) ≤ 3 months or autologous HSCT ≤ 21 days prior to start of investigational product.
8. The patient has not recovered from the acute toxic effects of prior chemotherapy, radiation, or major surgery/significant trauma.
9. The patient has had minor surgery ≤ 7 days from the start of study treatment (excluding the placement of central/peripheral lines, skin biopsy).
10. The patient has a known history of stroke, myocardial infarction, peripheral vascular disease, or recent (within 3 months) uncontrolled deep venous thrombosis.
11. The patient has a known history or current diagnosis of human immunodeficiency virus (HIV) infection, regardless of treatment status.
12. The patient has an uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
13. The patient has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study.
14. The patient has any condition, including the presence of laboratory abnormalities, that places the patient at unacceptable risk if he/she were to participate in the study.
15. The patient has any condition that confounds the ability to interpret data from the study.
16. The patient or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator.
17. The patient has ≥ Grade 2 peripheral neuropathy by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) at screening.
6 Months
24 Years
ALL
No
Sponsors
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Celgene
INDUSTRY
Responsible Party
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Principal Investigators
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Ileana Elias, M.D.
Role: STUDY_DIRECTOR
Celgene Corporation
Locations
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Phoenix Childrens Hospital
Phoenix, Arizona, United States
Columbia University Medical Center
New York, New York, United States
The Hospital for Sick Children
Toronto, Ontario, Canada
Institute for Pediatric Hematology - Oncology, Leon Berard Cancer Center
Lyon, , France
Hopital d'Enfants, CHU Nancy
Nancy, , France
Institut Curie
Paris, , France
Institut Gustave Roussy
Villejuif, , France
Azienda Ospedaliera Universitaria Meyer
Florence, , Italy
Children's Hospital Largo
Genova, , Italy
Istituto Nazionale Tumori
Milan, , Italy
Clinica di Oncoematologia
Padua, , Italy
Policlinico Agostino Gemelli
Rome, , Italy
l'Azienda Ospedaliera Regina Margherita - Sant Anna
Torino, , Italy
Hospital Universitario Vall D Hebron
Barcelona, , Spain
Hospital Sant Joan de Deu
Barcelona, , Spain
Spanish National Cancer Research Centre
Madrid, , Spain
Hospital Universitario Virgen Del Rocio
Seville, , Spain
Unidad de Oncologia Pediatrica, Hospital Universitario la Fe
Valencia, , Spain
Universitäts-Kinderklinik
Zurich, , Switzerland
Royal Marsden Hospital
Sutton, , United Kingdom
Countries
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References
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Moreno L, Casanova M, Chisholm JC, Berlanga P, Chastagner PB, Baruchel S, Amoroso L, Gallego Melcon S, Gerber NU, Bisogno G, Fagioli F, Geoerger B, Glade Bender JL, Aerts I, Bergeron C, Hingorani P, Elias I, Simcock M, Ferrara S, Le Bruchec Y, Slepetis R, Chen N, Vassal G. Phase I results of a phase I/II study of weekly nab-paclitaxel in paediatric patients with recurrent/refractory solid tumours: A collaboration with innovative therapies for children with cancer. Eur J Cancer. 2018 Sep;100:27-34. doi: 10.1016/j.ejca.2018.05.002. Epub 2018 Jun 21.
Amoroso L, Castel V, Bisogno G, Casanova M, Marquez-Vega C, Chisholm JC, Doz F, Moreno L, Ruggiero A, Gerber NU, Fagioli F, Hingorani P, Melcon SG, Slepetis R, Chen N, le Bruchec Y, Simcock M, Vassal G. Phase II results from a phase I/II study to assess the safety and efficacy of weekly nab-paclitaxel in paediatric patients with recurrent or refractory solid tumours: A collaboration with the European Innovative Therapies for Children with Cancer Network. Eur J Cancer. 2020 Aug;135:89-97. doi: 10.1016/j.ejca.2020.04.031. Epub 2020 Jun 15.
Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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ABI-007-PST-001
Identifier Type: -
Identifier Source: org_study_id