scholarly journals A Phase 3 Multicenter, Prospective, Open-Label Efficacy and Safety Study of Immune Globulin (Human) 10% Caprylate/Chromatography Purified in Patients with Myasthenia Gravis Exacerbations

2019 ◽  
Vol 81 (5-6) ◽  
pp. 223-230 ◽  
Author(s):  
Guntis Karelis ◽  
Rodica Balasa ◽  
Jan L. De Bleecker ◽  
Tima Stuchevskaya ◽  
Andres Villa ◽  
...  
Keyword(s):  
Myasthenia Gravis ◽  
Immune Globulin ◽  
Autoimmune Disorder ◽  
Efficacy And Safety ◽  
Open Label ◽  
Safety Study ◽  
Serious Adverse Events ◽  
Good Tolerability ◽  
Secondary Endpoints ◽  
Patients Experience

Background: Myasthenia gravis (MG) is an autoimmune disorder affecting neuromuscular transmission. Exacerbations may involve increasing bulbar weakness and/or sudden respiratory failure, both of which can be critically disabling. Management of MG exacerbations includes plasma exchange and intravenous immunoglobulin (IVIG); they are equally effective, but patients experience fewer side effects with IVIG. The objective of this study was to assess the efficacy and safety of immune globulin caprylate/chromatography purified (IGIV-C) in subjects with MG exacerbations. Methods: This prospective, open-label, non-controlled 28-day clinical trial was conducted in adults with MG Foundation of America class IVb or V status. Subjects received IGIV-C 2 g/kg over 2 consecutive days (1 g/kg/day) and were assessed for efficacy/safety on Days 7, 14, 21, and 28. The primary efficacy endpoint was the change from Baseline in quantitative MG (QMG) score to Day 14. Secondary endpoints of clinical response, Baseline to Day 14, included at least a 3-point decrease in QMG and MG Composite and a 2-point decrease in MG-activities of daily living (MG-ADL). Results: Forty-nine subjects enrolled. The change in QMG score at Day 14 was significant (p < 0.001) in the Evaluable (–6.4, n = 43) and Safety (–6.7, n = 49) populations. Among evaluable subjects, Day 14 response rates were 77, 86, and 88% for QMG, MG Composite, and MG-ADL, respectively. IGIV-C showed good tolerability with no serious adverse events. Conclusions: The results of this study show that IGIV-C was effective, safe, and well tolerated in the treatment of MG exacerbations.

Subcutaneous immunoglobulin in myasthenia gravis exacerbation

Neurology ◽  
2017 ◽  
Vol 89 (11) ◽  
pp. 1135-1141 ◽  
Author(s):  
Grayson Beecher ◽  
Dustin Anderson ◽  
Zaeem A. Siddiqi
Keyword(s):  
Adverse Events ◽  
Myasthenia Gravis ◽  
Manual Muscle Testing ◽  
Subcutaneous Immunoglobulin ◽  
Open Label ◽  
Serious Adverse Events ◽  
Muscle Testing ◽  
Open Label Phase ◽  
Secondary Endpoints ◽  
Comparative Safety

Objective:To investigate the efficacy, tolerability, and safety of subcutaneous immunoglobulin (SCIg) in patients with mild to moderate myasthenia gravis (MG) exacerbation.Methods:We performed a prospective, open-label, phase 3 trial in patients with MG aged 18 years or older and mild to moderate worsening (transition from Myasthenia Gravis Foundation of America class I to II/III or class II to III), treated with SCIg (2 g/kg), self-administered over 4 weeks. The primary endpoint was change in quantitative MG (QMG) score from baseline to study end at 6 weeks. Secondary endpoints included change in manual muscle testing (MMT), MG activities of daily living (MG-ADL), and MG composite (MGC) scores, as well as occurrence of adverse events, and tolerability as assessed via Treatment Satisfaction Questionnaire for Medication (TSQM).Results:Twenty-two of 23 patients completed the study. QMG score decreased from 14.9 ± 4.1 to 9.8 ± 5.6 (p < 0.0001), MMT score decreased from 16.8 ± 9.5 to 5.2 ± 4.5 (p < 0.0001), MG-ADL score decreased from 9.5 ± 3.0 to 4.6 ± 3.0 (p < 0.0001), and MGC score decreased from 17.4 ± 5.0 to 5.6 ± 4.5 (p < 0.0001). Satisfaction by TSQM was high (79.6 ± 15.6%). Common adverse events included headache and injection site reactions. No serious adverse events occurred.Conclusions:SCIg is well-tolerated, safe, and effective in mild to moderate MG exacerbation. Comparative safety and efficacy must be established with randomized controlled trials.Classification of evidence:This study provides Class IV evidence that in patients with mild to moderate MG exacerbation, SCIg is safe and effective in reducing MG disability measures.

485 Efficacy and Safety of Once- and Twice-Nightly Dosing of Lower-Sodium Oxybate in Adults With Idiopathic Hypersomnia

SLEEP ◽  
2021 ◽  
Vol 44 (Supplement_2) ◽  
pp. A191-A192
Author(s):  
Isabelle Arnulf ◽  
Anne Marie Morse ◽  
Patricia Chandler ◽  
Rupa Parvataneni ◽  
Dan Chen ◽  
...  
Keyword(s):  
Sodium Oxybate ◽  
Mean Difference ◽  
Controlled Study ◽  
Efficacy And Safety ◽  
Open Label ◽  
Double Blind ◽  
Patient Global Impression ◽  
Idiopathic Hypersomnia ◽  
Secondary Endpoints ◽  
Global Impression Of Change

Abstract Introduction Idiopathic hypersomnia (IH) is a rare central hypersomnolence disorder. In a randomized, controlled study of lower-sodium oxybate (LXB; Xywav™) in adults with IH (NCT03533114), significant differences for LXB compared with placebo were observed in Epworth Sleepiness Scale (ESS; primary efficacy endpoint), self-reported Patient Global Impression of Change (PGIc), and IH Severity Scale (IHSS; key secondary endpoints). In this clinical study, investigators were permitted to initiate LXB dosing on a once-nightly or twice-nightly regimen. Methods Eligible participants aged 18–75 years began LXB treatment, administered once or twice nightly during an open-label treatment/titration and optimization period (OLTTOP; 10–14 weeks); dose amount/regimen could be adjusted during this period. Participants next entered a 2-week, open-label, stable-dose period (SDP), then were randomized to placebo or to continue LXB treatment during a 2-week, double-blind, randomized withdrawal period (DBRWP). P values are nominal for this exploratory analysis. Results Of 154 enrolled participants, 40 (26%) initiated LXB treatment on a once-nightly regimen. In the efficacy population (n=115), 27 participants were on a once-nightly regimen during SDP (48.1% of whom initiated treatment once nightly during OLTTOP) and 88 participants were on a twice-nightly regimen during SDP (86.4% of whom initiated treatment twice nightly during OLTTOP). During SDP, median (min, max) LXB total dose was 4.5 (2.5, 6) g/night (once-nightly group) and 7.5 (4.5, 9) g/night (twice-nightly group). ESS scores worsened in participants randomized to placebo vs those continuing LXB in the once-nightly group (n=11 and n=15, respectively; LS mean difference [95% CI]: −4.93 [−7.41, −2.46]; P=0.0004) and twice-nightly group (n=47 and n=41, respectively; LS mean difference [95% CI]: −7.44 [−9.15, −5.72]; P&lt;0.0001). Worsening was also observed in PGIc (once-nightly: 81.8% [placebo] vs 26.7% [LXB]; P=0.0077; twice-nightly: 89.4% [placebo] vs 19.5% [LXB]; P&lt;0.0001) and IHSS score (estimated median difference [95% CI], once-nightly: −9.00 [−16.0, −3.0]; P=0.0028; twice-nightly: −12.00 [−15.0, −8.0]; P&lt;0.0001). Common adverse events included nausea (21.4%), headache (16.2%), anxiety (14.9%), dizziness (11.7%), insomnia (11.7%), and vomiting (10.4%). Conclusion The efficacy and safety of LXB in IH were demonstrated for both once-nightly and twice-nightly regimens. The majority of participants initiated and remained on a twice-nightly regimen. Support (if any) Jazz Pharmaceuticals

First long-term results on efficacy and safety of long-acting pasireotide in combination with everolimus in patients with advanced carcinoids (NET) of the lung/thymus: Phase II LUNA trial.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 8574-8574
Author(s):  
Eric Baudin ◽  
Alfredo Berruti ◽  
Mario Giuliano ◽  
Wasat Mansoor ◽  
Catalin Bobirca ◽  
...  
Keyword(s):  
Phase Ii ◽  
Dose Intensity ◽  
Extension Phase ◽  
Long Term Results ◽  
Tolerability Profile ◽  
Efficacy And Safety ◽  
Open Label ◽  
Secondary Endpoints ◽  
Core Phase ◽  
Long Acting

8574 Background: Everolimus (EVE) improves progression-free survival (PFS) in patients (pts) with progressive non-functioning thoracic and digestive advanced neuroendocrine tumors (NET). The LUNA trial aimed to assess the efficacy and safety of long-acting pasireotide (PAS) and EVE alone or in combination in pts with progressive bronchial or thymic carcinoids. Core phase results for primary endpoint (PFS) and secondary endpoints at 9 and 12 months (mo) were previously published. Cumulative data results at the end of the extension phase are presented here. Methods: LUNA was a prospective, multicenter, randomized, open-label, 3-arm, phase II trial. Adult pts with carcinoids of lung/thymus were randomized (1:1:1) to receive either PAS (60 mg/mo i.m.) or EVE (10 mg/day orally) or PAS + EVE. The key secondary endpoints assessed in this extension phase, including all the patients who were still not progressing at 12 months, were PFS, duration of biochemical response (DBR), and biochemical PFS (BPFS). Results: Of the total 124 pts included in the core phase, 41 pts with a median age of 61 years entered the extension phase including PAS (12), EVE (14) and PAS + EVE (15). Lung was the primary site of cancer in 95.1% and 82.9% had non-functioning tumors. Surgery/local or regional therapy was the preferred prior treatment in 63.4% pts. Disease progression was the primary reason for discontinuation among 3 arms with 65.9% in overall extension phase; no pts in PAS arm discontinued due to adverse events (AEs). Mean relative dose intensity (RDI) was higher for PAS (95.6% alone and 90.4% in combination) when compared to EVE (76.6% alone and 72.4% in combination); 38.1% pts in the EVE arm and 43.9% pts in the combination arm with EVE had RDI <70%. PAS +EVE combination showed clinical benefit in terms of PFS and BPFS compared to PAS and EVE alone as shown in Table. At least one dose reduction of PAS or EVE was reported in >50% pts. Most common AEs of any grade regardless of the study drug in PAS +EVE arm were hyperglycemia (87.8%), diarrhea (80.5%), and weight loss (58.5%), while stomatitis was reported in 34.1%. Twelve deaths were reported during the study and up to 56 days from last study treatment dose. Duration of exposure and efficacy. Conclusions: Mature median PFS and BPFS data suggest a benefit of PAS+EVE combination. The safety and tolerability profile of PAS and EVE alone or in combination were consistent with prior experience of these treatments in the oncology setting, with no new safety signals being reported during the study. Post-hoc prognostic studies are ongoing. Clinical trial information: NCT01563354. [Table: see text]

scholarly journals Efficacy and safety of artemether-lumefantrine and dihydroartemisinin-piperaquine for the treatment of uncomplicated Plasmodium falciparum malaria and prevalence of molecular markers associated with artemisinin and partner drug resistance in Uganda

2021 ◽  
Vol 20 (1) ◽  
Author(s):  
Chris Ebong ◽  
Asadu Sserwanga ◽  
Jane Frances Namuganga ◽  
James Kapisi ◽  
Arthur Mpimbaza ◽  
...  
Keyword(s):  
Molecular Markers ◽  
Falciparum Malaria ◽  
Uncomplicated Malaria ◽  
Plasmodium Falciparum Malaria ◽  
Uncomplicated Falciparum Malaria ◽  
Pharmacokinetic Studies ◽  
Efficacy And Safety ◽  
Open Label ◽  
Serious Adverse Events ◽  
Line Therapy

Abstract Background In Uganda, artemether-lumefantrine (AL) is first-line therapy and dihydroartemisinin-piperaquine (DP) second-line therapy for the treatment of uncomplicated malaria. This study evaluated the efficacy and safety of AL and DP in the management of uncomplicated falciparum malaria and measured the prevalence of molecular markers of resistance in three sentinel sites in Uganda from 2018 to 2019. Methods This was a randomized, open-label, phase IV clinical trial. Children aged 6 months to 10 years with uncomplicated falciparum malaria were randomly assigned to treatment with AL or DP and followed for 28 and 42 days, respectively. Genotyping was used to distinguish recrudescence from new infection, and a Bayesian algorithm was used to assign each treatment failure a posterior probability of recrudescence. For monitoring resistance, Pfk13 and Pfmdr1 genes were Sanger sequenced and plasmepsin-2 copy number was assessed by qPCR. Results There were no early treatment failures. The uncorrected 28-day cumulative efficacy of AL ranged from 41.2 to 71.2% and the PCR-corrected cumulative 28-day efficacy of AL ranged from 87.2 to 94.4%. The uncorrected 28-day cumulative efficacy of DP ranged from 95.8 to 97.9% and the PCR-corrected cumulative 28-day efficacy of DP ranged from 98.9 to 100%. The uncorrected 42-day efficacy of DP ranged from 73.5 to 87.4% and the PCR-corrected 42-day efficacy of DP ranged from 92.1 to 97.5%. There were no reported serious adverse events associated with any of the regimens. No resistance-associated mutations in the Pfk13 gene were found in the successfully sequenced samples. In the AL arm, the NFD haplotype (N86Y, Y184F, D1246Y) was the predominant Pfmdr1 haplotype, present in 78 of 127 (61%) and 76 of 110 (69%) of the day 0 and day of failure samples, respectively. All the day 0 samples in the DP arm had one copy of the plasmepsin-2 gene. Conclusions DP remains highly effective and safe for the treatment of uncomplicated malaria in Uganda. Recurrent infections with AL were common. In Busia and Arua, the 95% confidence interval for PCR-corrected AL efficacy fell below 90%. Further efficacy monitoring for AL, including pharmacokinetic studies, is recommended. Trial registration The trail was also registered with the ISRCTN registry with study Trial No. PACTR201811640750761

Peri-Operative Eltrombopag or Immune Globulin for Patients with Immune Thrombocytopaenia (The Bridging ITP Trial): Methods and Rationale

2019 ◽  
Vol 119 (03) ◽  
pp. 500-507 ◽  
Author(s):  
Donald Arnold ◽  
Erin Jamula ◽  
Nancy Heddle ◽  
Richard Cook ◽  
Cyrus Hsia ◽  
...  
Keyword(s):  
Platelet Count ◽  
Randomized Trial ◽  
Immune Globulin ◽  
Elective Surgery ◽  
Dose Titration ◽  
Major Surgery ◽  
Open Label ◽  
Rescue Treatment ◽  
Minor Surgery ◽  
Secondary Endpoints

Background The Bridging ITP Trial is an open-label randomized trial designed to compare the oral thrombopoietin receptor agonist eltrombopag and intravenous immune globulin (IVIG) for patients with immune thrombocytopaenia (ITP) who require an increase in platelet count before elective surgery. Here, we report the study methods and rationale. Methods We designed a multi-centre, non-inferiority randomized trial comparing daily oral eltrombopag starting 3 weeks pre-operatively, and IVIG administered 1 week pre-operatively for patients with ITP requiring a platelet count increase prior to surgery. Starting dose of eltrombopag is 50 mg daily with a weekly pre-operative dose titration schedule, and treatment is continued for 1 week after surgical haemostasis is achieved. IVIG is administered at a dose of 1 to 2 g/kg 1 week pre-operatively with the allowance for a second dose within 1 week after surgical haemostasis. The objective of the study is to demonstrate non-inferiority of eltrombopag for the primary endpoint of achieving the pre-operative platelet count threshold (50 × 109/L for minor surgery; or 100 × 109/L for major surgery) and sustaining platelet count levels above the threshold for 1 week after surgical haemostasis is achieved, without the use of rescue treatment. Secondary endpoints include thrombosis, bleeding and patient satisfaction. Conclusion The Bridging ITP Trial will evaluate the efficacy and safety of eltrombopag as an alternative to IVIG in the peri-operative setting for patients with ITP. The protocol was designed to provide a management strategy that can be applied in clinical practice. ClinicalTrials.gov Identifier NCT01621204.

scholarly journals Cardiac Conduction Safety during Coadministration of Artemether-Lumefantrine and Lopinavir/Ritonavir in HIV-Infected Ugandan Adults

2011 ◽  
Vol 2011 ◽  
pp. 1-4 ◽  
Author(s):  
Pauline Byakika-Kibwika ◽  
Mohammed Lamorde ◽  
Peter Lwabi ◽  
Wilson B. Nyakoojo ◽  
Violet Okaba-Kayom ◽  
...  
Keyword(s):  
Single Dose ◽  
Cardiac Conduction ◽  
Hiv Positive ◽  
Cyp3a4 Inhibitor ◽  
Open Label ◽  
Safety Study ◽  
Serious Adverse Events ◽  
Normal Limits ◽  
Hiv Positive Adults ◽  
Cyp3a4 Substrate

Background. We aimed to assess cardiac conduction safety of coadministration of the CYP3A4 inhibitor lopinavir/ritonavir (LPV/r) and the CYP3A4 substrate artemether-lumefantrine (AL) in HIV-positive Ugandans. Methods. Open-label safety study of HIV-positive adults administered single-dose AL (80/400 mg) alone or with LPV/r (400/100 mg). Cardiac function was monitored using continuous electrocardiograph (ECG). Results. Thirty-two patients were enrolled; 16 taking LPV/r -based ART and 16 ART naïve. All took single dose AL. No serious adverse events were observed. ECG parameters in milliseconds remained within normal limits. QTc measurements did not change significantly over 72 hours although were higher in LPV/r arm at 24 (424 versus 406; P=.02) and 72 hours (424 versus 408; P=.004) after AL intake. Conclusion. Coadministration of single dose of AL with LPV/r was safe; however, safety of six-dose AL regimen with LPV/r should be investigated.

Every-Three-Week (Q3W) Maintenance Dosing of Epoetin Alfa in Anemic Cancer Patients Receiving Chemotherapy: 60,000 U SC QW to Target Hb 12 g/dl, Followed by 80,000 U SC Q3W.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 4227-4227 ◽  
Author(s):  
Vernon Montoya ◽  
Denise Williams
Keyword(s):  
Health Care Providers ◽  
Response Rate ◽  
Study Drug ◽  
Maintenance Phase ◽  
Care Providers ◽  
Epoetin Alfa ◽  
Open Label ◽  
Serious Adverse Events ◽  
Initiation Phase ◽  
Secondary Endpoints

Abstract The efficacy of epoetin alfa (EPO) 40,000 U SC once weekly (QW) has been established in clinical studies in anemic patients (pts) with cancer receiving chemotherapy (CT). Higher starting doses of EPO may increase initial hemoglobin (Hb) response and subsequent less frequent maintenance dosing may improve dosing flexibility for pts and health care providers. This ongoing, open-label, multicenter, 24-week (wk) study was designed to evaluate the efficacy and safety of EPO at a starting dose of 60,000 U SC QW to a target Hb of 12 g/dL in the Initiation Phase (IP; maximum of 12 wks), followed by 80,000 U SC every 3 wks (Q3W) in the Maintenance Phase (MP) to maintain Hb in the range 11.5–12.5 g/dL. Eligible pts were ≥18 years with nonmyeloid malignancy, had baseline (BL) Hb <11 g/dL, and were scheduled to receive CT Q3W for ≥15 wks. Pts who achieved Hb ≥12 g/dL during the IP subsequently entered the MP; however, pts did not receive the first Q3W maintenance dose until start of the next Q3W CT cycle. Pts were withdrawn if Hb decreased to <11 g/dL during the MP. The primary endpoint was proportion of pts in the IP who achieved an Hb ≥12 g/dL or an Hb increase ≥2 g/dL from BL. Secondary endpoints included Hb increase ≥1 g/dL but <2.0 g/dL from BL in IP, maintenance of mean Hb 11.5–12.5 g/dL in the MP, maintenance of mean Hb >11.0 g/dL but <11.5 g/dL in MP, Hb over time, and transfusions. All Hb and response rates were independent of RBC transfusion within the previous 28 days. Study enrollment was terminated at 115 pts. In this preliminary analysis, the first 69 pts (mean age, 62 years; 65% women; 87% ECOG 0-1) enrolled and who received ≥1 dose of study drug were evaluable for safety and efficacy. Mean BL Hb was 10.2 ± 0.8 g/dL. The proportion of pts in the IP who achieved an Hb ≥12 g/dL or an Hb increase ≥2 g/dL from BL was 74% (49/69). The proportion of pts in the IP with an Hb increase ≥1 g/dL but <2 g/dL from BL was 9% (6/69). Forty-one (59%) pts entered the MP after a median of 5.0 wks in the IP and spent an average of 7.1 wks in the MP. Of these pts, 33 (80%) maintained an average Hb of 11.5–12.5 g/dL and 3 (7%) maintained an average Hb >11.0 g/dL but <11.5 g/dL over the course of the MP. During the MP, mean Hb was 12.1 ± 0.6 g/dL at entry, 11.8 ± 1.0 g/dL (n=15) after 8 wks of Q3W dosing, and 11.6 ± 1.1 g/dL (n=38) at final value. Eleven (27%) pts were withdrawn during the MP because Hb decreased to <11 g/dL. EPO dose was reduced in 25% (17/69) of pts in the IP and in 39% (16/41) of pts in the MP due to Hb >13 g/dL or Hb increase >1.3-g/dL in 2 wks. Thirty-five percent (24/69) of pts had a dose of EPO held at any time during the study for Hb >13 g/dL or another reason. Five (7%) pts were transfused after study day 28. Twenty-six (38%) pts experienced serious adverse events (AEs). Eight (12%) pts experienced a clinically relevant thrombovascular event. Two (3%) pts discontinued due to AEs. Nine (13%) pts died during the study or within the 90-day follow-up period. These preliminary data suggest that initial dosing of EPO 60,000 U QW is associated with a hematopoietic response rate (Hb ≥12 g/dL or Hb increase ≥2 g/dL from BL) of ~75% in pts with cancer and anemia receiving CT, similar to the response rate reported historically for EPO 40,000 U SC QW with dose escalation to 60,000 U SC QW. In addition, among pts who achieved a target Hb ≥12 g/dL with QW dosing and then received Q3W maintenance dosing, EPO 80,000 U SC Q3W maintained mean Hb above 11 g/dL in >85% of pts.

A phase II, open-label, single-arm, efficacy, and safety study of MDV3100 in patients with hormone-naïve prostate cancer.

2011 ◽  
Vol 29 (7_suppl) ◽  
pp. 177-177
Author(s):  
E. S. Baskin-Bey ◽  
G. M. Holtkamp ◽  
M. R. Smith ◽  
T. Ouatas ◽  
D. Phung ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Phase Ii ◽  
Response Rate ◽  
Locally Advanced ◽  
Dropout Rate ◽  
Efficacy And Safety ◽  
Mineral Density ◽  
Open Label ◽  
Safety Study ◽  
Psa Response

177 Background: MDV3100 is a novel androgen receptor (AR) antagonist in clinical development for the treatment of prostate cancer (PCa). Compared to bicalutamide, MDV3100 has higher in vitro AR binding affinity and no evidence of partial agonism. Preliminary phase I-II data show antitumor activity with MDV3100 in men with advanced PCa who were concurrently using androgen deprivation therapy (ADT; Scher HI, et al. Lancet. 2010;375:1437-46). Phase II and III studies in men with progressive and earlier-stage PCa are ongoing. This abstract describes the design of a phase II study of MDV3100 in men with hormone-naïve PCa (HNPCa) who are candidates for ADT. Methods: This 25-week, open-label, single-arm, efficacy and safety study of MDV3100 (160 mg/d orally) will be initiated at ∼20 investigational sites in late 2010 (planned countries: Austria, Belgium, Czech Republic, Denmark, Germany). Inclusion criteria include histologically confirmed, locally advanced PCa (all stages), noncastrate testosterone (T; ≥230 ng/dL), prostate-specific antigen (PSA) ≥2 ng/mL (screening), Eastern Cooperative Oncology Group score of 0, and life expectancy ≥1 year. Exclusion criteria include, among others, previous/current hormonal therapy or chemotherapy for PCa. The primary endpoint is PSA response, defined as an ≥80% decrease from baseline to wk 25. A binary PSA response per patient (wk 25) will be determined, allowing for generation of a PSA response rate (95% CI) from all patients for the study. Secondary endpoints include: PSA dynamics; pharmacokinetics; change in gonadotropin, T, dihydrotestosterone, estradiol, sex-hormone binding globulin, and prolactin levels; and safety/tolerability. Exploratory endpoints include changes in bone mineral density, bone turnover markers, metabolic parameters, and quality of life. Results: The planned enrollment is 60 patients. With a 20% dropout rate, the study will have 80% power to reject the unwanted PSA response rate of ≤50% (5% significance). Efficacy and safety data will be published later. Conclusions: Previous data were in men with castrate levels of T (≤50 ng/dL). This trial will be the first to investigate the use of MDV3100 monotherapy in HNPCa. [Table: see text]

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