Topical rapamycin combined with pulsed dye laser in the treatment of capillary vascular malformations in Sturge-Weber syndrome: Phase II, randomized, double-blind, intraindividual placebo-controlled clinical trial

Background: Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive fibrotic lung disease characterized by worsening dyspnea and lung function and has a median survival of 2–3 years. Forced vital capacity (FVC) is the primary endpoint used most commonly in IPF clinical trials as it is the best surrogate for mortality. This study assessed quantitative scores from high-resolution computed tomography (HRCT) developed by machine learning as a secondary efficacy endpoint in a 26-week phase II study of BMS-986020 – an LPA1 receptor antagonist – in patients with IPF. Methods: HRCT scans from 96% (137/142) of randomized subjects were utilized. Quantitative lung fibrosis (QLF) scores were calculated from the HRCT images. QLF improvement was defined as ⩾2% reduction in QLF score from baseline to week 26. Results: In the placebo arm, 5% of patients demonstrated an improvement in QLF score at week 26 compared with 15% and 27% of patients in the BMS-986020 600 mg once daily (QD) and twice daily (BID) arms, respectively [ versus placebo: p = 0.08 (600 mg QD); p = 0.0098 (600 mg BID)]. Significant correlations were found between changes in QLF and changes in percent predicted FVC, diffusing capacity for carbon monoxide (DLCO), and shortness of breath at week 26 ( ρ = −0.41, ρ = −0.22, and ρ = 0.27, respectively; all p < 0.01). Conclusions: This study demonstrated the utility of quantitative HRCT as an efficacy endpoint for IPF in a double-blind, placebo-controlled clinical trial setting. The reviews of this paper are available via the supplemental material section.

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Pulsed dye laser for Sturge-Weber syndrome

Archives of Disease in Childhood ◽

10.1136/adc.87.5.434 ◽

2002 ◽

Vol 87 (5) ◽

pp. 434-435 ◽

Cited By ~ 18

Author(s):

C Leaute-Labreze

Keyword(s):

Dye Laser ◽

Pulsed Dye Laser ◽

Weber Syndrome ◽

Sturge Weber Syndrome

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157 The effect of cox-2 inhibition on molecular responses of rectal cancer to preoperative chemoradiation in a phase ii randomized, double blind, placebo-controlled clinical trial

Radiotherapy and Oncology ◽

10.1016/s0167-8140(06)80636-5 ◽

2006 ◽

Vol 78 ◽

pp. S53

Author(s):

A. Debucquoy ◽

L. Goethals ◽

W.H. McBride ◽

K. Haustermans

Keyword(s):

Clinical Trial ◽

Rectal Cancer ◽

Phase Ii ◽

Preoperative Chemoradiation ◽

Controlled Clinical Trial ◽

Double Blind ◽

Molecular Responses ◽

Double Blind Placebo ◽

Cox 2

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The Increase of the Magnitude of Spontaneous Viral Blips in Some Participants of Phase II Clinical Trial of Therapeutic Optimized HIV DNA Vaccine Candidate

Vaccines ◽

10.3390/vaccines7030092 ◽

2019 ◽

Vol 7 (3) ◽

pp. 92 ◽

Cited By ~ 2

Author(s):

Ekaterina Akulova ◽

Boris Murashev ◽

Sergey Verevochkin ◽

Alexey Masharsky ◽

Ruslan Al-Shekhadat ◽

...

Keyword(s):

Clinical Trial ◽

Placebo Group ◽

Dna Vaccine ◽

Phase Ii ◽

Phase Ii Clinical Trial ◽

Controlled Clinical Trial ◽

Viral Reservoirs ◽

Double Blind ◽

Infected People ◽

Hiv 1

We developed a candidate DNA vaccine called “DNA-4”consisting of 4 plasmid DNAs encoding Nef, Gag, Pol(rt), and gp140 HIV-1 proteins. The vaccine was found to be safe and immunogenic in a phase I clinical trial. Here we present the results of a phase II clinical trial of “DNA-4”. This was a multicenter, double-blind, placebo-controlled clinical trial of safety, and dose selection of “DNA-4” in HIV-1 infected people receiving antiretroviral therapy (ART). Fifty-four patients were randomized into 3 groups (17 patients—group DNA-4 0.25 mg, 17 patients—group DNA-4 0.5 mg, 20 patients—the placebo group). All patients were immunized 4 times on days 0, 7, 11, and 15 followed by a 24-week follow-up period. “DNA-4” was found to be safe and well-tolerated at doses of 0.25 mg and 0.5 mg. We found that the amplitudes of the spontaneous viral load increases in three patients immunized with the candidate DNA vaccine were much higher than that in placebo group—2800, 180,000 and 709 copies/mL, suggesting a possible influence of therapeutic DNA vaccination on viral reservoirs in some patients on ART. We hypothesize that this influence was associated with the reactivation of proviral genomes.

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