Clinical trial NCT03147443: Evaluating the Effects of Traditional Chinese Medicine by N-of-1 Trials
Official title: The Study on the Effects of Traditional Chinese Medicine Through a Series of N-of-1 Trials Based on the Mathematical Model of "Carryover Effect"
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Bronchiectasis
Quality of Life
Traditional Chinese Medicine
Sputum
Symptoms
Individuality
Keywords: traditional Chinese medicine
Drugs, Chinese Herbal
N-of-1 trials
sputum
symptoms
individualized treatment
stable bronchiectasis
Lead sponsor: Shanghai University of Traditional Chinese Medicine
Sponsor class: OTHER
First posted: 2017-05-10
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: randomized, double-blind, crossover comparisons of individualized herbal decoction with control decoction within individual patients. Each n-of-1 trial has 3 pairs of treatment periods. The duration of each treatment period will be 4 weeks.
Primary purpose: TREATMENT

Brief summary: Treatment based on syndrome differentiation is the essence of traditional Chinese Medicine(TCM) and forms the individualized treatment. N-of-1 trials may be a good clinical effect evaluation method for the individualized treatment of TCM. This study aims to compare: (1) the efficacy of syndrome differentiation(individualized decoction) with placebo, (2)the efficacy of syndrome differentiation(individualized decoction) with the same prescription minus heat-clearing Chinese herbs, for patients with stable bronchiectasis through N-of-1 trials.

Detailed description: Traditional Chinese medicine is one of the important part of complementary and alternative medicine in the world, and it plays increasingly important role in the international medical practice. Treatment based on syndrome differentiation is the essence of traditional Chinese Medicine (TCM) and forms the individualized treatment. However, this individualized TCM intervention often makes it difficult for population-based RCTs to carry out a standard form. N-of-1 trials may be a good clinical effect evaluation method for the individualized treatment of TCM.

The key hypothesis of this study is that: (1) the efficacy of syndrome differentiation(individualized decoction) will be better than placebo in patients with stable bronchiectasis through N-of-1 trials. (2) the efficacy of syndrome differentiation(individualized decoction) will be better than the same prescription minus heat-clearing Chinese herbs in patients with stable bronchiectasis through N-of-1 trials. The difference of efficacy may be various according to the individual severity of phlegm-heat.

In this study, We will conduct a single center N-of-1 trials in 36 patients with stable bronchiectasis without hemoptysis and respiratory failure. These N-of-1 trials will be randomized, double-blind, crossover comparisons of individualized herbal decoction with control decoction within individual patients. Each n-of-1 trial has 3 pairs of treatment periods. The duration of each treatment period will be 4 weeks. We will compare :(1) the efficacy of syndrome differentiation(individualized decoction) with placebo, (2) the efficacy of syndrome differentiation(individualized decoction) with the same prescription minus heat-clearing Chinese herbs, in patients with stable bronchiectasis through N-of-1 trials. The primary outcome is patient self-reported symptoms(such as cough, expectoration, shortness of breath, chest pain, and fatigue) scores on a 7 point visual analogue scale. Secondary outcomes are 24-hour sputum volume and CAT（The COPD Assessment Test） scores. We use paired t test for single case. Mixed effects model taking account of "Carryover Effects" and meta-analysis will be used for a series of N-of-1 trials as a group.

Eligibility criteria: Inclusion Criteria:

* the diagnostic criteria based on the consensus of Chinese experts and the guidelines for noncystic fibrosis bronchiectasis issued by the British Thoracic Society in 2010;
* male or female, aged 18-70 years;
* being in the stable stage, and no acute exacerbation of bronchiectasis within the past three weeks;
* frequency of acute exacerbation of bronchiectasis ≤ 3 times every year;
* signed informed consent for participation.

Exclusion Criteria:

* having developed respiratory failure with estimated survival time less than one year;
* having hemoptysis as a comorbidity;
* having complications by active tuberculosis;
* being pregnant or with severe heart, liver,and kidney dysfunctions;
* participating in other pharmacological clinical trials within the past 3 months.

Primary outcomes:
measure: Patient Self-Rated Symptom Score
timeFrame: 4 weeks
Secondary outcomes:
measure: 24 h Sputum Volume
timeFrame: 4 weeks
Other outcomes:
measure: COPD Assessment Test
timeFrame: 4 weeks
Locations:
city: Shanghai
country: China
facility: Yueyang Hospital of Integrated Traditional Chinese and Western Medicine,Shanghai University of Traditional Chinese Medicine
state: Shanghai
Study officials:
References:
citation: Huang H, Yang P, Xue J, Tang J, Ding L, Ma Y, Wang J, Guyatt GH, Vanniyasingam T, Zhang Y. Evaluating the Individualized Treatment of Traditional Chinese Medicine: A Pilot Study of N-of-1 Trials. Evid Based Complement Alternat Med. 2014;2014:148730. doi: 10.1155/2014/148730. Epub 2014 Nov 11.
citation: Lee BY, Lee S, Lee JS, Song JW, Lee SD, Jang SH, Jung KS, Hwang YI, Oh YM. Validity and Reliability of CAT and Dyspnea-12 in Bronchiectasis and Tuberculous Destroyed Lung. Tuberc Respir Dis (Seoul). 2012 Jun;72(6):467-74. doi: 10.4046/trd.2012.72.6.467. Epub 2012 Jun 29.
citation: Zi S, Huang H, Yang P, Xu M, Wu Y, Wang Z, Ge F, Chen X. Evaluating the Effects of Heat-Clearing Traditional Chinese Medicine in Stable Bronchiectasis by a Series of N-of-1 Trials. Evid Based Complement Alternat Med. 2022 Jan 17;2022:6690638. doi: 10.1155/2022/6690638. eCollection 2022.