SOURCES SOUGHT
A -- Laboratory to Conduct Survival Motor Neuron (SMN) mRNA Assays for Two Phase I/IIa Dose Escalation Studies of Phenylbutyrate in the Treatment of Pediatric Subjects with Spinal Muscular Atrophy (SMA)
- 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-8079-05-02
- 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. Notification of Intent Due Date: 5pm EST on 12/23/05 (see Attachment 1) 4. Proposal Due Date: 5 pm EST on January 25, 2006. 5. 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. 6. Address Offer to: Westat, Inc., RFP 8079-05-02, 1650 Research Blvd., RE/152, Rockville, MD 20850, Attn: Russell Walker Sr. Contract Administrator 7. 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 SMN mRNA assays for two Phase I/IIa trials of phenylbutyrate in the treatment of pediatric subjects with spinal muscular atrophy (SMA). These studies will be performed under a prime contract, HHSN265200423611C, NO: NO1-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 42 subjects will be enrolled and followed in the two studies. Specimens will be batch-shipped at intervals throughout the studies to the selected laboratory for SMN mRNA 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 200 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 (Section D). SECTION C ? STATEMENT OF WORK BRIEF BACKGROUND FOR NPTUNE The NINDS has established 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 trials to be conducted under NPTUNE are two Phase I/IIa dose escalation studies 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. The classification of SMA is based on age at onset and severity of disease course. Patients with Type I SMA (Werdnig-Hoffman disease), usually show clinical signs 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 Type III (Kugelberg-Welander disease) is after 18 months of age and patients typically reach all motor milestones. At this time, there is no known 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 the two planned studies is to determine the maximum tolerated dose (MTD) and safety of phenylbutyrate as well as to evaluate the effects of this drug on SMN mRNA and protein levels in subjects with SMA. The protocol for the two studies will be very similar, with the exceptions of eligibility criteria and frequency of specimen collection. The protocols for these studies are 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 protocols may vary from that presented here. Design The SMA Type I protocol will be conducted in subjects under 2 years of age, and the SMA Type II/III protocol will be conducted in children ages 2 to 12 years. The SMA Type II/III study is expected to begin enrollment first in order to establish an effective time course of SMN mRNA blood draws. Data from the SMN mRNA analysis on the first subjects enrolled in the SMA Type II/III protocol will be used to determine the blood draw schedule for the SMA Type I protocol. The primary objective for these studies is to select the ?best dose? based on safety endpoints. These studies will assess safety, MTD, and the pharmacokinetics of phenylbutyrate in these populations, as well as cellular response by measure of specific biomarkers. Each 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 each. 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 at the MTD as identified in Stage 1. These subjects will receive study drug for three months and will then be followed for a two week period once off study medication. It is expected that each study will be completed in approximately one year. Multiple clinical sites will be selected to complete the required enrollment in each protocol. STATEMENT OF WORK The selected central laboratory will be required to perform the appropriate assays to measure the relative units of both full-length SMN mRNA and SMN7 mRNA (exon 7 deleted) transcripts 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. Specimens will be shipped to the central laboratory from each site when all samples for a subject have been collected. 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 SMN mRNA central laboratory will be responsible for providing sufficient staff with appropriate training and experience to ensure proper execution of the assays according to written laboratory Standard Operating Procedures (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 records for all study specimens received at the laboratory, including date of use, reason for use (assay) and volume used. The lab 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 here 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 data on the relative units of full-length SMN and SMN7 mRNA transcripts from specimens provided A maximum of 200 samples are expected to 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 portion 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 mRNA 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 studies as the central laboratory for SMN mRNA 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 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 measurement of relative units of both full-length SMN and SMN7 mRNA transcripts. (30 points) B. Laboratory Personnel and Experience ( 45 points total) Availability of sufficient 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
- SN00950160-W 20051214/051212211823 (fbodaily.com)
- Source
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