anthonyyazdaniml/CT-ADE-SOC huggingface dataset:
Intervention: Odevixibat
Trial phase: Phase 3
ATC code: A05AX05
SMILES: CCCCC1(CCCC)CN(c2ccccc2)c2cc(SC)c(OCC(=O)N[C@@H](C(=O)N[C@@H](CC)C(=O)O)c3ccc(O)cc3)cc2S(=O)(=O)N1
Sex: All
Age groups: Child | Adult
Healthy volunteers: No
Participants at risk: 23
Group description: Capsules for oral administration (40 ug/kg) once daily for 24 weeks

A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
Eligibility criteria:
Key Inclusion Criteria:

A male or female participant with a clinical diagnosis of PFIC Type 1 or 2 and with a body weight above 5 kg
Participant must have clinical genetic confirmation of PFIC-1 or PFIC-2
Participant must have elevated serum bile acid (s-BA) concentration
Participant must have history of significant pruritus and a caregiver reported observed scratching in the eDiary
Participant and/or legal guardian must sign informed consent (and assent) as appropriate.
Participants will be expected to have a consistent caregiver(s) for the duration of the study
Caregivers and age-appropriate participants (≥8 years of age) must be willing and able to use an eDiary device as required by the study

Key Exclusion Criteria:

Participant with pathologic variations of the ABCB11 gene that predict complete absence of the bile salt export pump (BSEP) protein

Participant with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:

Biliary atresia of any kind
Benign recurrent intrahepatic cholestasis, indicated by any history of normal s BAs
Suspected or proven liver cancer or metastasis to the liver on imaging studies
Histopathology on liver biopsy that is suggestive of alternate non-PFIC related etiology of cholestasis
Participant with past medical history or ongoing chronic diarrhea
Any participant with suspected or confirmed cancers except for basal cell carcinoma
Participant with a past medical history of chronic kidney disease with an impaired renal function and a glomerular filtration rate <70 mL/min/1.73 m^2
Participant with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to start of Screening Period
Participant has had a liver transplant or a liver transplant is planned within 6 months of randomization
Decompensated liver disease
Participant suffers from uncontrolled, recalcitrant pruritic condition other than PFIC
Participant who has been previously treated with an IBAT inhibitor whose pruritus has not responded to treatment
Intervention: Odevixibat
Trial phase: Phase 3
ATC code: A05AX05
SMILES: CCCCC1(CCCC)CN(c2ccccc2)c2cc(SC)c(OCC(=O)N[C@@H](C(=O)N[C@@H](CC)C(=O)O)c3ccc(O)cc3)cc2S(=O)(=O)N1
Sex: All
Age groups: Child | Adult
Healthy volunteers: No
Participants at risk: 19
Group description: Capsules for oral administration (120 ug/kg) once daily for 24 weeks

A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
Eligibility criteria:
Key Inclusion Criteria:

A male or female participant with a clinical diagnosis of PFIC Type 1 or 2 and with a body weight above 5 kg
Participant must have clinical genetic confirmation of PFIC-1 or PFIC-2
Participant must have elevated serum bile acid (s-BA) concentration
Participant must have history of significant pruritus and a caregiver reported observed scratching in the eDiary
Participant and/or legal guardian must sign informed consent (and assent) as appropriate.
Participants will be expected to have a consistent caregiver(s) for the duration of the study
Caregivers and age-appropriate participants (≥8 years of age) must be willing and able to use an eDiary device as required by the study

Key Exclusion Criteria:

Participant with pathologic variations of the ABCB11 gene that predict complete absence of the bile salt export pump (BSEP) protein

Participant with past medical history or ongoing presence of other types of liver disease including, but not limited to, the following:

Biliary atresia of any kind
Benign recurrent intrahepatic cholestasis, indicated by any history of normal s BAs
Suspected or proven liver cancer or metastasis to the liver on imaging studies
Histopathology on liver biopsy that is suggestive of alternate non-PFIC related etiology of cholestasis
Participant with past medical history or ongoing chronic diarrhea
Any participant with suspected or confirmed cancers except for basal cell carcinoma
Participant with a past medical history of chronic kidney disease with an impaired renal function and a glomerular filtration rate <70 mL/min/1.73 m^2
Participant with surgical history of disruption of the enterohepatic circulation (biliary diversion surgery) within 6 months prior to start of Screening Period
Participant has had a liver transplant or a liver transplant is planned within 6 months of randomization
Decompensated liver disease
Participant suffers from uncontrolled, recalcitrant pruritic condition other than PFIC
Participant who has been previously treated with an IBAT inhibitor whose pruritus has not responded to treatment
Intervention: Azithromycin
Trial phase: Phase 3
ATC code: J01FA10 | J01RA07 | J01RA16 | S01AA26
SMILES: CCC1OC(=O)C(C)C(OC2CC(C)(OC)C(O)C(C)O2)C(C)C(OC2OC(C)CC(N(C)C)C2O)C(C)(O)CC(C)CN(C)C(C)C(O)C1(C)O
Sex: All
Age groups: Child
Healthy volunteers: No
Participants at risk: 161
Group description: children in this arm will receive one dose of azithromycin

Azithromycin: Children enrolled in the trial and randomized to azithromycin will receive a single directly observed dose of oral azithromycin at 20 mg/kg up to the maximum adult dose of 1 g. Oral azithromycin will be administered at the time of enrollment. Enrolled children will also receive RUTF.
Eligibility criteria:
Inclusion Criteria:

Age 6-59 months
WHZ <-3 SD or MUAC <115 mm
No nutritional edema
Able and willing to participate in full 8-week study
Has not been admitted to a nutritional program for the treatment of SAM in the 3 preceeding months
No antibiotic use in past 7 days
No clinical complications requiring inpatient treatment (except for routine admission for children <6 months)
No congenital abnormality or chronic debilitating illness that would lead to predictable growth faltering or reduce likelihood of SAM treatment benefit (such as cerebral palsy, Down syndrome, congenital heart disease, cleft lip/palate, etc)
No allergy to macrolides/azalides
Sufficient appetite according to a feeding test with ready-to-use therapeutic food (RUTF)
Appropriate consent from at least one parent or guardian

Exclusion Criteria:

Age <6 months or >59 months
WHZ ≥-3 SD or MUAC ≥115 mm
Nutritional edema
Not able or willing to participate in full 8-week study
Admission to a nutritional program for the treatment of SAM in the 3 preceeding months
Antibiotic use in past 7 days
Clinical complications requiring inpatient treatment
Congenital abnormality or chronic debilitating illness that would lead to predictable growth faltering or reduce likelihood of SAM treatment benefit (such as cerebral palsy, Down syndrome, congenital heart disease, cleft lip/palate, etc)
Allergy to macrolides/azalides
Insufficient appetite according to a feeding test with ready-to-use therapeutic food (RUTF)
Parent or guardian refuses to provide consent
Intervention: Amoxicillin
Trial phase: Phase 3
ATC code: A02BD01 | A02BD03 | A02BD04 | A02BD05 | A02BD06 | A02BD07 | A02BD10 | A02BD11 | A02BD12 | A02BD13 | A02BD14 | A02BD15 | A02BD16 | J01CA04 | J01CR50
SMILES: [H][C@]12SC(C)(C)[C@H](C(=O)O)N1C(=O)[C@H]2NC(=O)[C@H](N)c1ccc(O)cc1
Sex: All
Age groups: Child
Healthy volunteers: No
Participants at risk: 140
Group description: Children in this arm will receive a 7 day course of amoxicillin (standard of care)

Amoxicillin: Children enrolled in the trial and randomized to azithromycin will receive a 7-day course of oral amoxcillin given in twice-daily doses of 50-100 mg/kg, per the Burkina Faso guidelines for the management of severe acute malnutrition. The first dose will be administered at the time of enrollment and the remaining dose will be administered by the caregiver at home. Enrolled children will also receive RUTF.
Eligibility criteria:
Inclusion Criteria:

Age 6-59 months
WHZ <-3 SD or MUAC <115 mm
No nutritional edema
Able and willing to participate in full 8-week study
Has not been admitted to a nutritional program for the treatment of SAM in the 3 preceeding months
No antibiotic use in past 7 days
No clinical complications requiring inpatient treatment (except for routine admission for children <6 months)
No congenital abnormality or chronic debilitating illness that would lead to predictable growth faltering or reduce likelihood of SAM treatment benefit (such as cerebral palsy, Down syndrome, congenital heart disease, cleft lip/palate, etc)
No allergy to macrolides/azalides
Sufficient appetite according to a feeding test with ready-to-use therapeutic food (RUTF)
Appropriate consent from at least one parent or guardian

Exclusion Criteria:

Age <6 months or >59 months
WHZ ≥-3 SD or MUAC ≥115 mm
Nutritional edema
Not able or willing to participate in full 8-week study
Admission to a nutritional program for the treatment of SAM in the 3 preceeding months
Antibiotic use in past 7 days
Clinical complications requiring inpatient treatment
Congenital abnormality or chronic debilitating illness that would lead to predictable growth faltering or reduce likelihood of SAM treatment benefit (such as cerebral palsy, Down syndrome, congenital heart disease, cleft lip/palate, etc)
Allergy to macrolides/azalides
Insufficient appetite according to a feeding test with ready-to-use therapeutic food (RUTF)
Parent or guardian refuses to provide consent
Intervention: Vilaprisan
Trial phase: Phase 2
SMILES: C[C@]12C[C@H](c3ccc(S(C)(=O)=O)cc3)C3=C4CCC(=O)C=C4CC[C@H]3[C@@H]1CC[C@@]2(O)C(F)(F)C(F)(F)F
Sex: Female
Age groups: Adult | Older Adult
Healthy volunteers: No
Participants at risk: 2
Group description: Premenopausal women 18 years and older with endometriosis received the treatment of Vilaprisan 2 mg.
Eligibility criteria:
Inclusion Criteria:

Signed and dated informed consent
Pre-menopausal women 18 years (inclusive) and above at the time of Visit 1
Women with endometriosis confirmed by laparoscopy or laparotomy OR diagnosed based on imaging
Moderate to severe endometriosis-associated pelvic pain (EAPP)
Adherence to screening period diary entries
Willingness to use only standardized pain medication if needed
Good general health (except for findings related to endometriosis)
Normal or clinically insignificant cervical cytology not requiring further follow-up
An endometrial biopsy performed at the screening phase without significant histological disorder
Use of an acceptable non-hormonal method of contraception
Willingness / ability to comply with electronic diary entry for the duration of study participation

Exclusion Criteria:

Pregnancy or lactation (less than 3 months since delivery, abortion, or lactation before Visit 1)
Hypersensitivity to any ingredient of the study treatments
Laboratory values outside the inclusion range before randomization, and considered clinically relevant
Any diseases or conditions that can compromise the function of the body systems and could result in altered absorption, excessive accumulation, impaired metabolism, or altered excretion of the study drug including elevated liver enzymes
Any diseases or conditions that might interfere with the conduct of the study or the interpretation of the results
Undiagnosed abnormal genital bleeding
Abuse of alcohol, drugs, or medicines (e.g. laxatives) as evaluated by the investigator
Use of other treatments that might interfere with the conduct of the study or the interpretation of the results
Endometriosis-specific treatments for symptom relief except rescue pain medication according to protocol
Simultaneous participation in another clinical trial with investigational medicinal product(s). Participation in another trial prior to study entry that might have an impact on the study objectives, at the discretion of the investigator
Inability to cooperate with the study procedures for any reason
Previous assignment to treatment (e.g. randomization) during this study (allowing previously randomized subjects to be re-included into the study may lead to bias)
Hypersensitivity to any ingredient of standardized pain medication
Wish for pregnancy during the study
Regular use of pain medication due to other underlying diseases
Non-responsiveness of EAPP to GnRH-a (Gonadotropin-releasing hormone agonists)
Intervention: Vilaprisan
Trial phase: Phase 2
SMILES: C[C@]12C[C@H](c3ccc(S(C)(=O)=O)cc3)C3=C4CCC(=O)C=C4CC[C@H]3[C@@H]1CC[C@@]2(O)C(F)(F)C(F)(F)F
Sex: Female
Age groups: Adult | Older Adult
Healthy volunteers: No
Participants at risk: 4
Group description: Premenopausal women 18 years and older with endometriosis received the treatment of Vilaprisan 4 mg
Eligibility criteria:
Inclusion Criteria:

Signed and dated informed consent
Pre-menopausal women 18 years (inclusive) and above at the time of Visit 1
Women with endometriosis confirmed by laparoscopy or laparotomy OR diagnosed based on imaging
Moderate to severe endometriosis-associated pelvic pain (EAPP)
Adherence to screening period diary entries
Willingness to use only standardized pain medication if needed
Good general health (except for findings related to endometriosis)
Normal or clinically insignificant cervical cytology not requiring further follow-up
An endometrial biopsy performed at the screening phase without significant histological disorder
Use of an acceptable non-hormonal method of contraception
Willingness / ability to comply with electronic diary entry for the duration of study participation

Exclusion Criteria:

Pregnancy or lactation (less than 3 months since delivery, abortion, or lactation before Visit 1)
Hypersensitivity to any ingredient of the study treatments
Laboratory values outside the inclusion range before randomization, and considered clinically relevant
Any diseases or conditions that can compromise the function of the body systems and could result in altered absorption, excessive accumulation, impaired metabolism, or altered excretion of the study drug including elevated liver enzymes
Any diseases or conditions that might interfere with the conduct of the study or the interpretation of the results
Undiagnosed abnormal genital bleeding
Abuse of alcohol, drugs, or medicines (e.g. laxatives) as evaluated by the investigator
Use of other treatments that might interfere with the conduct of the study or the interpretation of the results
Endometriosis-specific treatments for symptom relief except rescue pain medication according to protocol
Simultaneous participation in another clinical trial with investigational medicinal product(s). Participation in another trial prior to study entry that might have an impact on the study objectives, at the discretion of the investigator
Inability to cooperate with the study procedures for any reason
Previous assignment to treatment (e.g. randomization) during this study (allowing previously randomized subjects to be re-included into the study may lead to bias)
Hypersensitivity to any ingredient of standardized pain medication
Wish for pregnancy during the study
Regular use of pain medication due to other underlying diseases
Non-responsiveness of EAPP to GnRH-a (Gonadotropin-releasing hormone agonists)
Intervention: Daridorexant
Trial phase: Phase 3
ATC code: N05CJ03
SMILES: COc1ccc(-n2nccn2)c(C(=O)N2CCC[C@@]2(C)c2nc3c(C)c(Cl)ccc3[nH]2)c1
Sex: All
Age groups: Adult | Older Adult
Healthy volunteers: No
Participants at risk: 306
Group description: Daridorexant was administered as tablets, orally, once daily in the evening.
Eligibility criteria:
Inclusion Criteria:

Signed informed consent prior to any study-mandated procedure;
Male or female aged ≥ 18 years;
Insomnia disorder according to DSM-5 criteria;
Insomnia Severity Index score ≥ 15;
Insufficient sleep quantity as collected subjectively in the sleep diary and validated objectively by polysomnography;
Women of childbearing potential must have a negative and urine pregnancy test and use the contraception scheme up to at least 30 days after last study treatment intake.

Exclusion Criteria:

Body mass index below 18.5 or above 40.0 kg/m2;
Any lifetime history of of related breathing disorder, periodic limb movement disorder, restless legs syndrome, circadian rhythm disorder, rapid eye movement (REM) behavior disorder, narcolepsy, or apnea/hypopnea;
Cognitive behavioral therapy (CBT) only allowed if, the treatment started at least 1 month prior to Visit 3 and the subject agrees to continue this CBT throughout the study;
Self-reported usual daytime napping ≥ 1 hour per day and ≥ 3 days per week;
Acute or unstable psychiatric conditions diagnosed by the Mini International Neuropsychiatric Interview;
Mini Mental State Examination (MMSE) score < 25 in subjects ≥ 50 years;
For female subjects: pregnant, lactating or planning to become pregnant during projected duration of the study;
History or clinical evidence of any disease or medical condition or treatment, which may put the subject at risk of participation in the study or may interfere with the study assessments.
Any circumstances or conditions, which, in the opinion of the investigator, may affect the subject's full participation in the study or compliance with the protocol.
Intervention: Daridorexant
Trial phase: Phase 3
ATC code: N05CJ03
SMILES: COc1ccc(-n2nccn2)c(C(=O)N2CCC[C@@]2(C)c2nc3c(C)c(Cl)ccc3[nH]2)c1
Sex: All
Age groups: Adult | Older Adult
Healthy volunteers: No
Participants at risk: 308
Group description: Daridorexant was administered as tablets, orally, once daily in the evening.
Eligibility criteria:
Inclusion Criteria:

Signed informed consent prior to any study-mandated procedure;
Male or female aged ≥ 18 years;
Insomnia disorder according to DSM-5 criteria;
Insomnia Severity Index score ≥ 15;
Insufficient sleep quantity as collected subjectively in the sleep diary and validated objectively by polysomnography;
Women of childbearing potential must have a negative and urine pregnancy test and use the contraception scheme up to at least 30 days after last study treatment intake.

Exclusion Criteria:

Body mass index below 18.5 or above 40.0 kg/m2;
Any lifetime history of of related breathing disorder, periodic limb movement disorder, restless legs syndrome, circadian rhythm disorder, rapid eye movement (REM) behavior disorder, narcolepsy, or apnea/hypopnea;
Cognitive behavioral therapy (CBT) only allowed if, the treatment started at least 1 month prior to Visit 3 and the subject agrees to continue this CBT throughout the study;
Self-reported usual daytime napping ≥ 1 hour per day and ≥ 3 days per week;
Acute or unstable psychiatric conditions diagnosed by the Mini International Neuropsychiatric Interview;
Mini Mental State Examination (MMSE) score < 25 in subjects ≥ 50 years;
For female subjects: pregnant, lactating or planning to become pregnant during projected duration of the study;
History or clinical evidence of any disease or medical condition or treatment, which may put the subject at risk of participation in the study or may interfere with the study assessments.
Any circumstances or conditions, which, in the opinion of the investigator, may affect the subject's full participation in the study or compliance with the protocol.
Intervention: Colchicine
Trial phase: Phase 2 | Phase 3
ATC code: M04AC01
SMILES: COc1cc2c(c(OC)c1OC)-c1ccc(OC)c(=O)cc1[C@@H](NC(C)=O)CC2
Sex: All
Age groups: Child
Healthy volunteers: No
Participants at risk: 9
Group description: Colchicine will be given at 0.6 mg once daily for the duration of chest tube output plus 24 hours after chest tube removal with a maximum of 4 weeks duration.

Colchicine: Colchicine is an alkaloid approved in 1961 for the use in Familial Mediterranean Fever in adults and children 4 years of age or older. It has been widely used for decades in other indications, such as Gout, recurrent pericarditis, pericardial effusions and other inflammatory diseases. This drug is commercially available and is approved in children 4 years and older.
Eligibility criteria:
Inclusion Criteria:

Ages 20 months to 5 years and 364 days are eligible
Diagnosed with single ventricle heart disease requiring Fontan palliation
Undergoing Fontan palliation at the University of Michigan Congenital Heart Center
Ability to take oral or enteral medication

Exclusion Criteria:

Treatment with another investigational drug within 3 months
Pre-existing myelosuppression or decreased bone marrow activity.
Current or recent treatment with certain drugs
Renal or hepatic impairment deemed by the study team
Conditions and/or post-operative complications that would increase risk to the patient such as mechanical support (ECMO) or issues in the intensive care unit (ICU)
Intervention: ABROCITINIB
Trial phase: Phase 3
ATC code: D11AH08
SMILES: CCCS(=O)(=O)N[C@H]1C[C@@H](N(C)c2ncnc3[nH]ccc23)C1
Sex: All
Age groups: Child | Adult | Older Adult
Healthy volunteers: No
Participants at risk: 158
Group description: Participants were randomized to receive a tablet of PF-04965842 (abrocitinib) 100 milligrams (mg) and a tablet of matching placebo orally once daily for 12 weeks. Participants who discontinued early from treatment or who were not eligible for LTE study, were followed up to 4 weeks after last dose of study drug.
Eligibility criteria:
Inclusion Criteria:

12 years of age or older with a minimum body weight of 40 kg
Diagnosis of atopic dermatitis (AD) for at least 1 year and current status of moderate to severe disease (>= the following scores: BSA 10%, IGA 3, EASI 16, Pruritus NRS severity 4)
Recent history of inadequate response or inability to tolerate topical AD treatments or require systemic treatments for AD control

Exclusion Criteria:

Unwilling to discontinue current AD medications prior to the study or require treatment with prohibited medications during the study
Prior treatment with JAK inhibitors
Other active nonAD inflammatory skin diseases or conditions affecting skin
Medical history including thrombocytopenia, coagulopathy or platelet dysfunction, Q wave interval abnormalities, current or history of certain infections, cancer, lymphoproliferative disorders and other medical conditions at the discretion of the investigator
Pregnant or breastfeeding women, or women of childbearing potential who are unwilling to use contraception
Intervention: ABROCITINIB
Trial phase: Phase 3
ATC code: D11AH08
SMILES: CCCS(=O)(=O)N[C@H]1C[C@@H](N(C)c2ncnc3[nH]ccc23)C1
Sex: All
Age groups: Child | Adult | Older Adult
Healthy volunteers: No
Participants at risk: 155
Group description: Participants were randomized to receive PF-04965842 200 mg (2 tablets of 100 mg each) orally once daily for 12 weeks. Participants who discontinued early from treatment or who were not eligible for LTE study, were followed up to 4 weeks after last dose of study drug.
Eligibility criteria:
Inclusion Criteria:

12 years of age or older with a minimum body weight of 40 kg
Diagnosis of atopic dermatitis (AD) for at least 1 year and current status of moderate to severe disease (>= the following scores: BSA 10%, IGA 3, EASI 16, Pruritus NRS severity 4)
Recent history of inadequate response or inability to tolerate topical AD treatments or require systemic treatments for AD control

Exclusion Criteria:

Unwilling to discontinue current AD medications prior to the study or require treatment with prohibited medications during the study
Prior treatment with JAK inhibitors
Other active nonAD inflammatory skin diseases or conditions affecting skin
Medical history including thrombocytopenia, coagulopathy or platelet dysfunction, Q wave interval abnormalities, current or history of certain infections, cancer, lymphoproliferative disorders and other medical conditions at the discretion of the investigator
Pregnant or breastfeeding women, or women of childbearing potential who are unwilling to use contraception