ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)
ISSN 2383-9457 (ONLINE)
| Active ingredient | NCT number | FDA-approved indication | Measured PRO concept as primary endpoint | PROM |
|---|---|---|---|---|
| Diseases of the Nervous System (n=21) | ||||
| Amifampridine |
NCT01377922 NCT02970162 |
To treat Lambert-Eaton myasthenic syndrome in adults | All aspects of patients’ health and an amount of improvement or decline in clinical status | SGI |
| Atogepant |
NCT03777059 NCT02848326 |
To prevent episodic migraines | Migraine days | (eDiary) Migraine days |
| Cannabidiol |
NCT02224690 NCT02224560 NCT02091375 |
To treat rare, severe forms of epilepsy | Seizure frequency | (Diary) Frequency of drop seizures |
| Cenobamate |
NCT01866111 NCT01397968 |
To treat partial onset seizures | Seizure frequency | (Diary) seizure frequency |
| Efgartigimod alfa-fcab |
NCT03669588 |
To treat generalized myasthenia gravis | Severity of Myasthenia Gravis symptom | MG-ADL |
| Eptinezumab-jjmr |
NCT02559895 NCT02974153 |
For the preventive treatment of migraine in adults | Migraine days | (eDiary) Frequency of migraine days/Headache days |
| Erenumab-aooe |
NCT02456740 NCT02483585 NCT02066415 |
For the preventive treatment for migraine | Migraine days | (eDiary) Migraine days |
| Fremanezumab-vfrm |
NCT02629861 NCT02621931 |
For the preventive treatment of migraine in adults | Migraine days | (eDiary) Occurrence of headache |
| Galcanezumab-gnlm |
NCT02614183 NCT02614196 NCT02614261 |
For the preventive treatment of migraine in adults | Migraine days | (Diary) Migraine headache days |
| Ganaxolone |
NCT03572933 |
To treat seizures in cyclin-dependent kinaselike 5 deficiency disorder | Seizure frequency | (eDiary) Daily seizure calendar noting seizure type and frequency |
| Istradefylline |
NCT00456586 NCT00199407 NCT00455507 NCT00955526 |
To treat adult patients with Parkinson’s disease experiencing “off” episodes | Functional status in patients with Parkinson’s disease with motor fluctuations and dyskinesia | (Diary) 24-hour ON/OFF Patient Diary |
| Lasmiditan |
NCT02605174 NCT02439320 |
For the acute treatment of migraine with or without aura, in adults | Headache pain | (eDiary) Migraine pain freedom |
| Lemborexant |
NCT02952820 NCT02783729 |
To treat insomnia | Time of subjective sleep onset latency | (eDiary) sTST |
| Oliceridine |
NCT02815709 NCT02820324 |
To manage acute pain in certain adults Pain | (Diary) 48-hr NRS | |
| Opicapone |
NCT01568073 NCT01227655 |
To treat patients with Parkinson’s disease experiencing “off” episodes | Functional status in patients with Parkinson’s disease with motor fluctuations and dyskinesia | Patient self-rating diary (Houser diary) |
| Pitolisant |
NCT01067222 NCT01638403 |
To treat excessive daytime sleepiness in adult patients with narcolepsy | Subject’s usual level of daytime sleepiness or average sleep propensity | ESS |
| Rimegepant |
NCT03461757 |
To treat patients with Parkinson’s disease experiencing “off” episodes Pain freedom, | Severity of most bothersome symptoms | (eDiary) Pain and most bothersome symptoms |
| Solriamfetol |
NCT02348593 NCT02348606 NCT02348619 NCT02348632 |
To treat excessive sleepiness in adult patients with narcolepsy or obstructive sleep apnea | Subject’s usual level of daytime sleepiness or average sleep propensity | ESS |
| Stiripentol Not identified (STICLO FRANCE study/STICLO ITALY study) | To treat seizures associated with Dravet syndrome in patients | 2 years of age and older taking clobazam | Seizure frequency | (Diary) Seizure diary |
| Ubrogepant |
NCT02828020 NCT02867709 |
to treat acute treatment of migraine with or without aura in adults Pain freedom, | Severity of most bothersome symptoms | (eDiary) Headache severity, migraine associated symptoms |
| Zavegepant |
NCT04571060 NCT03872453 NCT04571060 NCT03872453 |
To treat migraine Pain freedom, | Severity of most bothersome symptoms | (eDiary) FDS, Onset of migraine, headache severity ratings, MBS |
| Diseases of the Genitourinary System (n=3) | ||||
| Elagolix sodium |
NCT01620528 NCT01620528 NCT01931670 NCT01931670 |
For the management of moderate to severe pain associated with endometriosis | Intensity of endometriosis | (eDiary) CPSSS |
| Plazomicin |
NCT02486627 NCT02486627 |
To treat complicated urinary tract infections who have limited or no alternative treatment options | Pain | Patients were asked about improvement/worsening in their urinary symptoms |
| Vibegron |
NCT03492281 NCT03492281 |
To treat overactive bladder | Number of micturitions and UUI episodes | (Diary) Patient Voiding Diarymicturitions, |
| Diseases of the Musculoskeletal System and Connective Tissue (n=2) | ||||
| Baricitinib |
NCT01721057 NCT01721044 NCT01721057 NCT01721044 |
To treat moderately to severely active rheumatoid arthritis | Pain intentsity, Disease activity, Physical function | ACR20 |
| Upadacitinib |
NCT02706873 NCT02706951 NCT02675426 NCT02629159 NCT02706847 |
To treat adults with moderately to severely active rheumatoid arthritis | Pain, Disease activity (Global assessment), Physical function | ACR20/50 |
| Diseases of the Digestive System (n=4) | ||||
| Lactitol |
NCT02481947 NCT02819297 |
To treat chronic idiopathic constipation in adults | Frequency of complete spontaneous bowel movement | (Diary) Bowel movement diary |
| Prucalopride |
NCT01116206 NCT01147926 NCT00488137 NCT00483886 NCT00485940 |
To treat chronic idiopathic constipation | Frequency of spontaneous complete bowel movements | (eDiary) Bowel movement |
| Rifamycin |
NCT01142089 NCT01208922 |
To treat Travelers’ Diarrhea in adults | Stool consistency | (Diary) 120 hours postinitial dose of study medication that recorded data, time of each stool, its consistency as either formed or unformed |
| Tenapanor |
NCT02686138 NCT02621892 |
To treat irritable bowel syndrome with constipation in adults. | Frequency of complete spontaneous bowel movement and abdominal pain | (Diary) stool frequency, abdominal pain intensity |
| Diseases of the Skin and Subcutaneous Tissue (n=3) | ||||
| Daxibotulinumtoixn A-lanm |
NCT03014622 NCT03014635 |
To treat moderate-to-severe glabellar lines associated with corrugator and/or procerus muscle activity | Severity of Facial skin folds | PFWS |
| Difelikefalin |
NCT03422653 NCT03636269 |
To treat moderate-to-severe pruritus associated with chronic kidney disease in certain populations | Intensity of itching | WI-NRS |
| PrabotulinumtoxinA-xvfs |
NCT02334423 NCT02334436 |
For the temporary improvement in the appearance of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adult patients | Facial skin folds | GLS |
| Endocrine, Nutritional, and Metabolic Diseases (n=3) | ||||
| Afamelanotide |
NCT00979745 NCT01605136 |
To increase pain-free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria | Skin pain | (Diary) Time spent in shade |
| Inotersen |
NCT01737398 |
To treat polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults | Patients’ perceptions of the effects of neuropathy | Norfolk QoL-DN |
| Teprotumumab-trbw |
NCT01868997 NCT03298867 |
To treat Thyroid eye disease | Orbital pain | CAS (Spontaneous Orbital pain, gazeevoked Orbital pain) |
| Mental, Behavioral, and Neurodevelopmental Disorders (n=2) | ||||
| Bremelanotide |
NCT02333071 NCT02338960 |
To treat hypoactive sexual desire disorder in premenopausal women. | Female sexual arousal, Sexually-related personal distress | FSFI (Questions 1 and 2) FSDS-DAO |
| Lofexidine hydrochloride |
NCT01863186 NCT00235729 |
For the non-opioid treatment for management of opioid withdrawal symptoms in adults | Opioid withdrawal symptom severity | SOWS-Gossop |
| Symptoms, Signs and Abnormal Clinical and Laboratory Findings, Not Elsewhere Classified (n=1) | ||||
| Fosnetupitant and palonosetron |
NCT02557035 |
To prevent acute and delayed nausea and vomiting associated with initial and repeat courses of highly emetogenic cancer chemotherapy | Emetic episode, Rescue medication during drug administration | (Diary) Episodes of retching and vomiting, rescue medication intake, daily nausea assessment, dexamethasone intake at home |
| Certain Infectious and Parasitic Diseases (n=1) | ||||
| Ibrexafungerp |
NCT03734991 NCT03987620 |
To treat vulvovaginal candidiasis | Presense of vulvovaginal symptoms | (Diary) Vulvovaginal symptoms (itching, burning, or irritation) |
| Disease of the Blood and Blood-Forming Organs, Certain Immune Disorders (n=1) | ||||
| Emicizumab |
NCT02622321 |
To prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A who have developed antibodies called Factor VIII inhibitors. | Bleeding frequency | BMQ |
| Diseases of the Respiratory System (n=1) | ||||
| Baloxavir marboxil |
NCT02954354 Not identified (1518T0821) |
To treat acute uncomplicated influenza in patients who have been symptomatic for no more than 48 hours. | Alleviation of influenza symptoms | (eDiary) Influenza symptoms |
BMQ, Bleeds and Medication Questionnaire; CPSSS, Composite Pelvic Signs and Symptoms Score; DYS, Dysmenorrhea; DYSP, Dyspareunia; ESS, Epworth Sleepiness Scale; FDS, Functional Disability Scale; FSDS-DAOQ13, Female Sexual Distress Scale-Desire/Arousal/Orgasm Question 13; FSFI, Female Sexual Function Index; GLS, Glabellar Line Scale; HAQ-DI, Health Assessment Questionnaire-Disability Index; MBS, Most Bothersome Symptom; MG-ADL, Myasthenia Gravis-Specific Activities of Daily Living; NMPP, Non-Menstrual Pelvic Pain; NRS, Numeric Pain Rating Scale; PFWS, Patient Frown Wrinkle Severity; QoL-DN, Norfolk Quality of Life-Diabetic Neuropathy; SGI, Subject Global Impression; SOWS-Gossop, Short Opiate Withdrawal Scale of Gossop; sTST, subjective Total Sleep Time; UUI, Urge Urinary Incontinence; VAS, Visual Analogue Scale; WI-NRS, Worst Itching Intensity Numerical Rating Scale