scholarly journals LB773 Treating pemphigus vulgaris (PV) and foliaceus (PF) by inhibiting the neonatal Fc receptor: Phase 2 multicentre open-label trial with efgartigimod

2021 ◽  
Vol 141 (9) ◽  
pp. B16
Author(s):  
M. Goebeler ◽  
Z. Bata-Csörgo ◽  
C. de Simone ◽  
B. Didona ◽  
E. Remenyik ◽  
...  
Keyword(s):  
Pemphigus Vulgaris ◽  
Fc Receptor ◽  
Phase 2 ◽  
Neonatal Fc Receptor ◽  
Open Label ◽  
Open Label Trial ◽  
Receptor Phase

scholarly journals Efficacy and safety of memantine in children with autism spectrum disorder: Results from three phase 2 multicenter studies

Autism ◽  
2019 ◽  
Vol 23 (8) ◽  
pp. 2096-2111 ◽  
Author(s):  
Antonio Y Hardan ◽  
Robert L Hendren ◽  
Michael G Aman ◽  
Adelaide Robb ◽  
Raun D Melmed ◽  
...  
Keyword(s):  
Extended Release ◽  
Children With Autism ◽  
Autism Spectrum ◽  
Social Responsiveness Scale ◽  
Social Responsiveness ◽  
Phase 2 ◽  
Open Label ◽  
Double Blind ◽  
Three Phase ◽  
Open Label Trial

Three phase 2 trials were conducted to assess the efficacy and long-term safety of weight-based memantine extended release (ER) treatment in children with autism spectrum disorder. MEM-MD-91, a 50-week open-label trial, identified memantine extended-release treatment responders for enrollment into MEM-MD-68, a 12-week randomized, double-blind, placebo-controlled withdrawal trial. MEM-MD-69 was an open-label extension trial in which participants from MEM-MD-68, MEM-MD-91, and open-label trial MEM-MD-67 were treated ⩽48 weeks with memantine extended release. In MEM-MD-91, 517 (59.6%) participants were confirmed Social Responsiveness Scale responders at week 12; mean Social Responsiveness Scale total raw scores improved two to three times a minimal clinically important difference of 10 points. In MEM-MD-68, there was no difference between memantine and placebo on the primary efficacy parameter, the proportion of patients with a loss of therapeutic response (defined as ⩾10-point increase from baseline in Social Responsiveness Scale total raw score). MEM-MD-69 exploratory analyses revealed mean standard deviation improvement in Social Responsiveness Scale total raw score of 32.4 (26.4) from baseline of the first lead-in study. No new safety concerns were evident. While the a priori–defined efficacy results of the double-blind trial were not achieved, the considerable improvements in mean Social Responsiveness Scale scores from baseline in the open-label trials were presumed to be clinically important.

Preliminary product parameter and safety results from NeuACT, a phase 2 randomized, open-label trial of DN24-02 in patients with surgically resected HER2+ urothelial cancer at high risk for recurrence.

2014 ◽  
Vol 32 (15_suppl) ◽  
pp. 4541-4541 ◽  
Author(s):  
Dean F. Bajorin ◽  
Leonard G. Gomella ◽  
Padmanee Sharma ◽  
Elizabeth R. Plimack ◽  
Peter H. O'Donnell ◽  
...  
Keyword(s):  
High Risk ◽  
Urothelial Cancer ◽  
Phase 2 ◽  
Open Label ◽  
Product Parameter ◽  
Open Label Trial

scholarly journals 035 Humanized anti-Desmoglein-3 antibodies as tools for research on the role of the neonatal Fc receptor in pemphigus vulgaris

2021 ◽  
Vol 141 (10) ◽  
pp. S154
Author(s):  
A. Zakrzewicz ◽  
C. Würth ◽  
B. Beckert ◽  
S. Feldhoff ◽  
P. Verheesen ◽  
...  
Keyword(s):  
Pemphigus Vulgaris ◽  
Fc Receptor ◽  
Neonatal Fc Receptor ◽  
Desmoglein 3

scholarly journals LB5. A Long-Time Coming: Final 2-year Analysis of Efficacy, Durability, and Microbiome Changes in a Controlled Open-Label Trial of Investigational Microbiota-Based Drug RBX2660 for Recurrent Clostridioides difficile Infections

2019 ◽  
Vol 6 (Supplement_2) ◽  
pp. S994-S995
Author(s):  
Robert Orenstein ◽  
Sarah Mische ◽  
Sarah Mische ◽  
Ken Blount ◽  
Ken Blount ◽  
...  
Keyword(s):  
Historical Control ◽  
Control Group ◽  
Fisher Exact Test ◽  
Phase 2 ◽  
Open Label ◽  
Clostridioides Difficile ◽  
Stool Samples ◽  
Open Label Trial ◽  
Pre Treatment ◽  
Age And Sex

Abstract Background Recurrent Clostridioides difficile infection (rCDI) is an urgent public health threat associated with significant mortality and medical cost. Microbiota therapy is gaining acceptance as a strategy to reduce rCDI recurrence. We present the final 24-month analysis of clinical safety, efficacy, and microbiome restoration from a Phase 2 open-label trial of RBX2660 for prevention of CDI recurrence. Methods Participants with multi-recurrent CDI received <2 doses of RBX2660 delivered via enema 7 days apart in this multicenter, open-label Phase 2 study. Efficacy was defined as the absence of CDI recurrence through 56 days after the last dose and was compared with 8-week recurrence-free rates for a historical control cohort that received standard-of-care antibiotic therapy. Fisher exact test compared the proportion of treatment participants who were CDI-free by age and sex. Durability was defined as continued absence of CDI episodes beyond 8 weeks. Safety and durability assessments occurred at 3, 6, 12, and 24 months. Participant stool samples were collected prior to and for up to 720 days after treatment, and microbiome changes were assessed by shallow shotgun sequencing. Results The efficacy of RBX2660 to prevent rCDI at 8 weeks (78.9%; 112/142) was higher than the CDI-free rate in the historical control group (30.7%, 23/75; P < 0.0001). Age and sex did not impact efficacy. Among participants who achieved treatment success at 8 weeks and were evaluable for long-term durability (n = 95), 8 experienced a new CDI episode by the 24-month follow-up for an overall durability of 91.6%. The safety profile was consistent with previous reports for RBX2660. In total, 503 stool samples from 110 treatment responders were analyzed. Within 7 days of treatment, the relative abundance of Bacteroidia and Clostridia remained shifted higher than pre-treatment levels while Gammaproteobacteria and Bacilli declined sharply after treatment, and these changes persisted to at least 24 months. Conclusion RBX2660, a microbiota-based drug, was safe and efficacious for preventing rCDI with clinical durability to 24 months after treatment, independent of age or sex, and RBX2660 durability associated with durable microbiome shifts from pre-treatment to a healthier composition. Disclosures Robert Orenstein, DO, Rebiotix Inc. (Advisor or Review Panel member), Sarah Mische, PhD, Rebiotix Inc. (Employee), Ken Blount, PhD, Rebiotix Inc. (Employee), Lindy Bancke, PharmD, Rebiotix Inc. (Employee), Xin Su, MD, MSci, Rebiotix Inc. (Employee), Dana Walsh, PhD, Rebiotix Inc. (Employee), Adam Harvey, PhD, Rebiotix Inc. (Employee), Carlos Gonzalez, MS, Rebiotix Inc. (Consultant), Dale N. Gerding, MD, Rebiotix Inc. (Board Member).

ELARA: A phase II, single-arm, multicenter, open-label trial investigating the efficacy and safety of tisagenlecleucel in adult patients with refractory/relapsed follicular lymphoma (r/r FL).

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. TPS7573-TPS7573 ◽  
Author(s):  
Michael Dickinson ◽  
Leslie Popplewell ◽  
Arne Kolstad ◽  
Joy Ho ◽  
Takanori Teshima ◽  
...  
Keyword(s):  
B Cell ◽  
Systemic Therapy ◽  
Response Rate ◽  
Adult Patients ◽  
Phase 2 ◽  
Efficacy And Safety ◽  
Open Label ◽  
Cd20 Antibody ◽  
Open Label Trial ◽  
Anti Cd20

TPS7573 Background: Tisagenlecleucel is an anti-CD19 chimeric antigen receptor-T cell (CAR-T) therapy that was approved for the treatment of pediatric and young adult patients up to 25 years of age with r/r B-cell acute lymphoblastic leukemia in 2017 (Maude et al. NEJM. 2018), as well as for the treatment of adult patients with r/r diffuse large B-cell lymphoma in 2018 (Schuster et al. NEJM. 2018). FL is the second most common non-Hodgkin lymphoma in the Western hemisphere, with limited treatment options in patients refractory to or relapsing after standard therapies. In a phase 2a study of patients with r/r CD19+ lymphomas, 10 of 14 (71%) patients with r/r FL treated with tisagenlecleucel achieved a durable complete remission at a median follow-up of 28.6 months (Schuster et al. NEJM. 2017). Here we introduce ELARA, a phase 2 study evaluating the efficacy and safety of tisagenlecleucel in patients with r/r FL. Methods: ELARA is a phase 2, single-arm, multicenter, open label trial. Eligible patients must be ≥18 years of age, have radiographically measurable grade 1, 2, or 3A r/r FL that is refractory to a second or later line of systemic therapy (including an anti-CD20 antibody and an alkylator), or relapsed within 6 months after completion of a second or later line of systemic therapy, or relapsed during anti-CD20 antibody maintenance (following ≥2 lines of therapy as above) or within 6 months after maintenance completion, or relapsed after autologous hematopoietic stem cell transplant (HSCT). Patients with central nervous system involvement, or those who received prior anti-CD19 therapy, gene therapy, adoptive T-cell therapy, or allogeneic HSCT are not eligible. The primary endpoint of this study is complete response rate based on Lugano classification response criteria. Secondary outcomes include overall response rate, duration of response, overall survival, cellular kinetics, immunogenicity, safety, and patient-reported outcomes. Estimated enrollment for this study is 113 patients. The study is currently open to patient enrollment. Clinical trial information: NCT03568461.

Sign in / Sign up

Export Citation Format

Share Document