SOURCES SOUGHT
A -- Lab. to Conduct Quantitative Survival Motor Neuron (SMN) Protein Assays for a Phase I/IIa Dose Escalation Study of Phenylbutyrate in the Treatment of Pediatric Subjects with Spinal Muscular Atrophy
- Notice Date
- 12/12/2005
- Notice Type
- Sources Sought
- NAICS
- 541710
— Research and Development in the Physical, Engineering, and Life Sciences
- Contracting Office
- Department of Health and Human Services, National Institutes of Health, Westat (NINDS PRIME CONTRACTOR), 1650 Research Blvd., Rockville, MD, 20850
- ZIP Code
- 20850
- Solicitation Number
- WESTAT-8079-05-03
- Response Due
- 1/25/2006
- Archive Date
- 2/9/2006
- Description
- SECTION A ? SOLICITATION AND OFFER 1. Type of Solicitation: Negotiated 2. Type of Business: Competition limited to North American organizations only 3. Date of Issue: December 12, 2005, 2005 4. Notification of Intent Due Date: 5pm EST on 12/23/05 (see Attachment 1) 5. Proposal Due Date: 5 pm EST on January 25, 2006. 6. Proposal Submission: Proposals must be submitted as separate technical and business volumes. Each volume shall be submitted as an unbound original and five bound copies. 7. Address Offer to: Westat, Inc., RFP 8079-05-03, 1650 Research Blvd., RE/152, Rockville, MD 20850, Attn: Russell Walker, Sr. Contract Administrator 8. Contract Type: Fixed Unit Price SECTION B ? SUPPLIES OR SERVICES AND PRICES/COSTS This solicitation is for the selection of a central laboratory to perform quantitative SMN protein assays for a Phase I/IIa dose escalation trial of phenylbutyrate in the treatment of pediatric subjects with spinal muscular atrophy (SMA). This study will be performed under a prime contract, HHSN265200423611C, NO: N01-NS-4-2361, NINDS Pilot Therapeutic Trials Network (NPTUNE), which Westat holds with the National Institute of Neurological Disorders and Stroke (NINDS). The selected laboratory will provide services as a subcontractor to Westat. The subcontract that is established will incorporate the required clauses from Westat?s prime contract and the Federal Acquisition Regulations (FAR) as delineated in Attachment 5. A full text of some of these FAR clauses is also provided. A full text of any clause that will be incorporated by reference may be accessed electronically at this address: http://www.arnet.gov/far/ A maximum of 21 subjects will be enrolled and followed in the study. Specimens will be shipped at intervals throughout the study to the selected laboratory for quantitative SMN protein testing. The exact number of specimens to be analyzed will depend on the final protocol sampling requirements and on the number of subjects required to determine the Maximum Tolerated Dose (MTD) in the dose-escalation stage of the protocols. We estimate a maximum of 100 samples will be collected for SMN mRNA analysis by the selected laboratory. Pricing for the subcontract will be a Fixed Unit Price per assay performed. The computation and documentation of all proposed pricing must be contained in the Offeror?s business proposal (see Section D). SECTION C ? STATEMENT OF WORK BRIEF BACKGROUND AND OBJECTIVES FOR NPTUNE The NINDS is establishing NPTUNE to facilitate the rapid conduct of high quality clinical trials and research through the provision of infrastructure for the design and implementation of pilot studies, primarily Phase I and Phase II clinical trials, in a timely and efficient manner. Westat, a research organization located in Rockville, Maryland, is under contract with the NINDS to serve as the Clinical Operations Center (COC) for NPTUNE. Westat will assist the NINDS with investigative site selection, assemble protocol development teams, provide training and site monitoring, provide data management expertise, facilitate investigator meetings, perform data analysis, and provide other clinical operations support services as may be required. STUDY SPECIFIC INFORMATION Brief Study Background The first clinical trial to be conducted under NPTUNE is a Phase I/IIa dose escalation study of phenylbutyrate in the treatment of pediatric subjects with SMA. SMA is an autosomal recessive motor neuron disease characterized by degeneration of the anterior horn cells of the spinal cord resulting in diffuse weakness and muscular atrophy. SMA is one of the most common pediatric neuromuscular conditions, with an incidence of 1 in 6,000 ? 10,000 live births, and is a leading cause of hereditary infant mortality. Symptoms most frequently present in infancy and early childhood, but the clinical spectrum of onset and severity is broad. Classification of SMA type is based on age at onset and severity of disease course. Patients with Type I SMA (Werdnig-Hoffman disease), usually show clinical signs of disease by three months and cannot sit unaided. Type II typically presents before 18 months of age and patients cannot stand or walk unaided. Onset of SMA of Type III (Kugelberg-Welander disease) is after 18 months of age and patients typically reach all motor milestones. At this time, there is no registered pharmacological treatment for SMA. However, several compounds have been identified that appear to up-regulate SMN gene expression or alter gene splicing to produce an increased ratio of full-length SMN to exon-7-deleted (SMN7) mRNA. Some of these compounds are readily available for use in subjects, and have a proven safety record in the treatment of other disorders. The purpose of this study is to determine the maximum tolerated dose (MTD) and safety of phenylbutyrate as well as to evaluate the effects of this drug on SMN protein levels in SMA subjects. The protocol for this study is not yet finalized, and the selected laboratory may be asked to review and provide input as appropriate. The current protocol design is briefly described below but the final approved protocol may vary from that presented here. Design The SMA Type II/III protocol will be conducted in children ages 2 to 12 years. The key objective for the study is to select the ?best dose? based on safety endpoints. This study will assess safety, MTD, and the pharmacokinetics of phenylbutyrate in this population, as well as cellular response by measure of specific biomarkers. The protocol will have two stages. Stage 1 is a dose-escalating stage where subjects will be enrolled sequentially into escalating dose cohorts of three subjects. They will remain on drug for 30 days and will then be followed for a two week period once off study drug. Stage 2 will enroll six subjects after the MTD is identified in Stage 1. These subjects will receive study drug for three months and will be followed for a two week period once off study medication. It is expected that the study will be completed in approximately one year. Multiple clinical sites will be selected to complete the required enrollment. STATEMENT OF WORK The selected central SMN protein laboratory will be required to perform the appropriate assays to quantitatively measure SMN protein in blood specimens. The laboratory will be required to perform the assays and provide the results in an Excel file for electronic export to the NPTUNE Data Management Center at Westat within two weeks of receiving specimens for analysis. Westat staff will work with the laboratory staff to ensure the Excel reporting format is acceptable for import into the Westat database. Test data transfers will be required and will be coordinated by appropriate Westat staff prior to initial data transfer. Specimens will be obtained from study subjects, processed, stored at and shipped from the clinical sites in accordance with the procedures required by the central laboratory. The central laboratory will be required to coordinate specimen shipping with the clinical sites and will be responsible for appropriately receiving, accessioning, processing, storing and tracking use, integrity and security of specimens received. The selected laboratory will be responsible for providing sufficient staff with appropriate training and experience to ensure proper execution of the assays according to written laboratory SOPs, applicable federal, state and local regulatory requirements and industry standards for good laboratory practices. Laboratory facilities and equipment must be sufficient and appropriate to successfully support the assays being performed. The laboratory must record, collect and maintain all source documentation necessary to allow verification of assay performance, quality control, intermediate and final data. The laboratory will be responsible for ensuring the integrity and security of the study specimens while at the laboratory, as well as tracking of specimen use. At the conclusion of the study, the laboratory will be responsible for providing Westat a copy of the tracking record for all study specimens received at the laboratory, including date of use, reason for use (assay type) and volume used. The laboratory will be required to secure and retain all unused study specimens until instructions for final specimen disposition are provided by Westat. Study specimens are not to be used for any purpose other than that specified herein unless instructed in writing by Westat. All data generated from the testing and analysis of these study specimens is the property of NINDS and may not be used or reported, except to Westat, without prior written approval from Westat. It is anticipated the primary research investigator(s) will be appropriately recognized, e.g., authorship. Satisfactory performance of the work under this contract shall be considered complete upon delivery and acceptance by the Westat Contracting Representative, or duly authorized representative, of the following items in accordance with the delivery schedule. Description Quantity Due Date 1. Final quantitative SMN protein data for all specimens provided A maximum of 100 samples will be provided for testing Data from assays will be reported to the NPTUNE Data Management Center at Westat within two weeks of receiving specimens for analysis. 2. Final disposition of all unused study specimens Variable Instructions to be provided by Westat Expected Award Date and Period of Performance Westat expects to award subcontracts on or about February 15, 2006. The period of performance of this subcontract shall be twenty-four (24) months from award date. SECTION D ? PROPOSAL INSTRUCTIONS, CONDITIONS AND NOTICES Offerors are requested to complete and return to Westat the Notification of Intent (see Attachment 1) by 5:00 pm on December 23, 2005. Proposals should contain sufficient detail in order to demonstrate both a clear understanding of the nature of the work requirements and the experience, staff, facilities and capability to fulfill the work requirements. Attachment 2 contains the instructions for completing the technical and business portions of the proposal, as well as specific items to which the Offeror should respond. SECTION E ? EVALUATION CRITERIA Westat anticipates making a single award for the performance of the quantitative SMN protein assays. Proposals will be evaluated based upon complete documentation provided in response to this RFP. Technical proposals will receive paramount consideration in the selection of subcontractors, but cost may be a factor in the final award decision. Westat reserves the right to make awards to its best advantage, cost and other factors considered. Westat reserves the right to make no awards to any Offerors of this RFP if it deems no respondents meet the requirements. Laboratories under consideration for participation in the SMA study as the central laboratory for quantitative SMN protein analysis will be evaluated based on the following scoring system. The Offeror?s total score will be derived by adding the total scores for the Assay Specific Experience, the Laboratory Personnel Experience and the Laboratory Facilities, with a maximum possible score of 100. A. Assay Specific Experience ( 30 points total) Evidence of an appropriate assay for quantitative measurement of SMN protein in peripheral human blood specimens. (30 points) B. Laboratory Experience ( 45 points total) Availability of qualified staff, as evidenced by training and experience, to perform the volume of assays required for these protocols. (25 points) Indication of sufficient and appropriate experience in, and procedures for, receiving, logging in, processing, storing and tracking specimens and working with other facilities to coordinate specimen shipments. (10 points) Indication of experience in reporting data in Excel format and in working with other organizations to prepare for, test and execute electronic data transfers. (5 points) Evidence (e.g., SOPs) that procedures are in place to ensure good laboratory practices are followed for data quality assurance and documentation of all procedures supporting assay results. (5 points) C. Laboratory Facilities ( 25 points total) Availability of appropriate laboratory facilities and equipment to conduct the required assays. (15 points) Evidence of adequate and appropriate procedures for ensuring specimen integrity and security. (10 points) NOTE: INTERESTED ORGANIZATIONS SHOULD EMAIL THE POC AND REQUEST A PRINTABLE VERSION OF THIS SOLICITATION WHICH WILL INCLUDE THE ATTACHMENTS.
- Record
- SN00950159-W 20051214/051212211821 (fbodaily.com)
- Source
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