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Clinical Trial News

Innovative Strategies for Patient Recruitment in Rare Disease Trials: Lessons from the Alkaptonuria Experience

  • The National Alkaptonuria Centre (NAC) and DevelopAKUre clinical trials face significant recruitment challenges, needing to identify patients from a limited global pool of only 1,000 known cases.
  • Novel recruitment strategies include GP fax campaigns, professional medical network outreach, and dedicated microsites, though initial physician response rates remain low at 6%.
  • Patient retention proves more challenging than recruitment, requiring dedicated support staff and comprehensive benefits communication to maintain participation in long-term trials.

Strategic Clinical Trial Design: Bridging the Gap Between Regulatory Approval and Market Access

• Clinical trial data now serves dual purposes: regulatory approval and reimbursement decisions, requiring pharmaceutical companies to rethink their trial design strategies.
• Analysis of Multiple Sclerosis trials revealed significant misalignment between clinical trial outcomes and Health Technology Assessment (HTA) priorities, highlighting the need for better trial planning.
• Effective trial design must incorporate both clinical efficacy measures and economic modeling parameters to successfully navigate regulatory approval and secure market access.

Oasis Diagnostics Receives Michael J. Fox Foundation Grant for Saliva-Based Parkinson's Test

Neurology
  • Oasis Diagnostics has been awarded a research grant from The Michael J. Fox Foundation to validate their non-invasive, saliva-based rapid test for Parkinson's disease detection and diagnosis.
  • The company will collaborate with Dr. Charles Adler's team at Mayo Clinic in Scottsdale to study proprietary salivary biomarkers, with research suggesting that just three of 50 identified biomarkers can provide accurate diagnosis.
  • The VerOFy® Rapid Test Platform could potentially transform Parkinson's diagnosis, offering a non-invasive alternative to current expensive and invasive diagnostic methods while potentially accelerating clinical trials of disease-modifying treatments.

Niraparib Maintenance Therapy Demonstrates Sustained Benefit in Recurrent Ovarian Cancer

  • Niraparib maintenance therapy significantly improves progression-free survival (PFS) in patients with recurrent, platinum-sensitive ovarian cancer, regardless of BRCA mutation status.
  • The NOVA trial, a phase 3 study, demonstrated a statistically significant and clinically meaningful benefit with niraparib compared to placebo in prolonging time to disease progression.
  • Niraparib showed efficacy in both patients with and without germline BRCA mutations, highlighting its potential as a broad maintenance therapy option.
  • The safety profile of niraparib was manageable, supporting its use as a maintenance strategy to extend remission in recurrent ovarian cancer.

Advancements in Natural Killer Cell Cancer Immunotherapy

Recent studies highlight the potential of Natural Killer (NK) cells in cancer immunotherapy, focusing on enhancing their anti-tumor responses through adoptive transfer, cytokine activation, and blocking inhibitory signals. Clinical trials have shown promising results, especially in treating hematologic malignancies, by improving NK cell targeting, functional competence, and expansion in patients.

QUILT-3.005: A Study of ALT-803 in Patients With Relapsed or Refractory Multiple Myeloma

NCT02099539Unknown StatusPhase 1
Altor BioScience
Posted 10/1/2014

A Phase I Study of NK Cell Infusion Following Allogeneic Peripheral Blood Stem Cell Transplantation From Related or Matched Unrelated Donors in Pediatric Patients With Solid Tumors and Leukemias

NCT01287104CompletedPhase 1
National Cancer Institute (NCI)
Posted 1/29/2011

NK Cell Infusions With Trastuzumab for Patients With HER2+ Breast and Gastric Cancer

NCT02030561Unknown StatusPhase 1
National University Hospital, Singapore
Posted 1/1/2014

Cytokine-induced Memory-like NK Cells in Patients With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

NCT01898793TerminatedPhase 1
Washington University School of Medicine
Posted 8/11/2014

NK Cell Based Non-Myeloablative Transplantation in Acute Myeloid Diseases

NCT01370213CompletedPhase 2
Masonic Cancer Center, University of Minnesota
Posted 9/1/2011

NK Cells in Cord Blood Transplantation

NCT01619761Unknown StatusPhase 1
M.D. Anderson Cancer Center
Posted 5/3/2013

Fludarabine Phosphate, Cyclophosphamide, Total-Body Irradiation, and Donor Bone Marrow Transplant Followed by Donor Natural Killer Cell Therapy, Mycophenolate Mofetil, and Tacrolimus in Treating Patients With Hematologic Cancer

NCT00789776CompletedPhase 1
Fred Hutchinson Cancer Center
Posted 10/13/2008

IL-15 Super Agonist ALT-803 to Treat Relapse Of Hematologic Malignancy After Allogeneic SCT

NCT01885897CompletedPhase 1
Masonic Cancer Center, University of Minnesota
Posted 11/11/2013

Donor Natural Killer Cells and Donor Stem Cell Transplant in Treating Patients With High Risk Myeloid Malignancies

NCT01823198CompletedPhase 1
M.D. Anderson Cancer Center
Posted 6/11/2013

Haploidentical Donor Natural Killer Cell Infusion With IL-15 in Acute Myelogenous Leukemia (AML)

NCT01385423CompletedPhase 1
Masonic Cancer Center, University of Minnesota
Posted 9/1/2011

Expanded Natural Killer (NK) Cells for Multiple Myeloma Study

NCT01040026CompletedPhase 1
University Hospital, Basel, Switzerland
Posted 10/1/2012

Natural Killer Cells Before and After Donor Stem Cell Transplant in Treating Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Chronic Myelogenous Leukemia

NCT01904136CompletedPhase 1
M.D. Anderson Cancer Center
Posted 4/22/2014

Efficacy of Lenalidomide With Rituximab in Refractory or Relapse of Primary Central Nervous System Lymphoma

NCT01956695CompletedPhase 2
Institut Curie
Posted 9/18/2013

Phase I/II Carfilzomib Plus Lenalidomide and Rituximab in the Treatment of Relapsed/Refractory Mantle Cell Lymphoma

NCT01729104TerminatedPhase 1
M.D. Anderson Cancer Center
Posted 4/25/2013

CNDO-109-AANK for AML in First Complete Remission (CR1)

NCT01520558Unknown StatusPhase 1
Coronado Biosciences, Inc.
Posted 12/1/2012

QUILT-3.002: N-803 in Patients With Relapse/Refractory iNHL in Conjunction With Rituximab

NCT02384954TerminatedPhase 1
Altor BioScience
Posted 4/17/2015

Haploidentical Donor Hematopoietic Progenitor Cell and NK Cell Transplantation for Hematologic Malignancy

NCT01807611CompletedPhase 2
St. Jude Children's Research Hospital
Posted 5/16/2013

QUILT-3.018: Neukoplast™ (NK-92) for the Treatment of Refractory or Relapsed Acute Myeloid Leukemia

NCT00900809CompletedPhase 1
ImmunityBio, Inc.
Posted 5/12/2014

Phase I of Infusion of Selected Donor NK Cells After Allogeneic Stem Cell Transplantation

NCT01853358CompletedPhase 1
Institut Paoli-Calmettes
Posted 4/1/2013

Urelumab (CD137 mAb) With Rituximab for Relapsed, Refractory or High-risk Untreated Chronic Lymphocytic Leukemia (CLL) Patients

NCT02420938WithdrawnPhase 2
M.D. Anderson Cancer Center
Posted 7/1/2015

Cord Blood Natural Killer (NK) Cells in Leukemia/Lymphoma

NCT02280525CompletedPhase 1
M.D. Anderson Cancer Center
Posted 3/5/2015

Donor Natural Killer Cells in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

NCT01787474CompletedPhase 1
M.D. Anderson Cancer Center
Posted 5/19/2014

Phase I Study of Adoptive Immunotherapy With Enriched and Expanded Autologous Natural Killer (NK) Cells for Patients With Ph+ Acute Lymphoblastic Leukemia (ALL)

NCT02185781CompletedPhase 1
Gruppo Italiano Malattie EMatologiche dell'Adulto
Posted 1/28/2015

Contraceptive Implant on Day of Mifepristone Administration Shows High Satisfaction

  • A pilot study evaluated the acceptability of initiating the etonogestrel implant at the time of mifepristone administration for medical abortion.
  • The study found that 87.5% of participants were satisfied with the timing of implant insertion.
  • At one year, 87.5% of participants with available follow-up data continued using the implant.
  • These findings suggest that initiating the contraceptive implant on the first day of medical abortion is a well-received approach.

Rare Disease Research Faces Critical Funding Gap Despite Scientific Value

  • Major UK medical research funders allocate less than 1% of their budgets to rare disease research, despite these conditions consuming 10% of the NHS specialized services budget.
  • Research into rare diseases like alkaptonuria (AKU) has proven valuable for understanding common conditions such as osteoarthritis, demonstrating their broader scientific importance.
  • Public awareness initiatives like the French Telethon, which raised €94 million in 2011, show the potential for increased rare disease funding through effective advocacy campaigns.

Ruxolitinib Demonstrates Sustained Efficacy in Myelofibrosis Treatment

  • Ruxolitinib, a JAK1/JAK2 inhibitor, has shown significant and durable improvements in splenomegaly and disease-related symptoms in patients with myelofibrosis (MF).
  • Long-term follow-up data confirms ruxolitinib's benefits across various patient subgroups, regardless of age, MF type, risk category, or JAK2 V617F mutation status.
  • Clinical trials indicate that ruxolitinib may improve survival in intermediate-2 or high-risk MF patients compared to placebo or best available therapy.
  • While ruxolitinib can cause dose-dependent anemia and thrombocytopenia, these adverse events are generally manageable and rarely lead to treatment discontinuation.

Bristol-Myers Squibb: 150 Years of Pharmaceutical Innovation and Global Healthcare Leadership

  • Bristol-Myers Squibb, founded in 1858, has evolved from a small Brooklyn pharmaceutical company to a global healthcare leader with approximately 28,000 employees and focus on six core therapeutic areas.
  • The company's landmark merger in 1989 created a pharmaceutical powerhouse with combined profits of $1.3 billion, leading to over 60 product lines generating $50+ million in annual sales by 1995.
  • BMS maintains a robust pipeline with 40 compounds in exploratory development and 6 in late-stage development, despite facing patent expiration challenges for key drugs like Plavix and Avapro.

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