ds4dh/ct-dosing-errors huggingface dataset:
Clinical trial NCT01733316
Conditions: Cystinosis
Keywords: Nephropathic Cystinosis Cysteamine Delayed-Release Cysteamine Orphan Disease CTNS Protein, Human
Sponsor: Amgen
Start date: 2013-01-31; Completion date: 2017-07-10
Enrollment: 41 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
The purpose of this study is to gather information about the effectiveness (how well it works to treat cystinosis) and safety of a new form of cysteamine bitartrate called RP103, compared to the already-approved drug cystinosis patients are taking called Cystagon®.

In cystinosis, the body builds up cystine. When taken regularly, the active ingredient of Cystagon® (cysteamine bitartrate) reduces cystine in the body. RP103 has the same active ingredient as Cystagon® and is designed to reduce cystine in a similar way that Cystagon® does. To decide if RP103 is better than Cystagon®, the study will look at two types of blood tests. One test is pharmacodynamics (PD), which measures the amount of white blood cell (WBC) cystine after taking study drug. WBC cystine is a laboratory test used to find out if cysteamine bitartrate is reducing cystine levels in the body. The second test is pharmacokinetics (PK), which measures the amount of cysteamine in the blood after taking the drug.

RP103 is different from Cystagon®: Instead of the cysteamine bitartrate being absorbed from the stomach, RP103 is designed to be absorbed from the small intestine. This may make the effects of the drug last longer, so that it can be taken twice a day instead of four times a day like Cystagon®.

Some cystinosis patients have bad breath (halitosis) when they take Cystagon®. Study participants who experience bad breath with Cystagon® will be asked if they would like to participate in an optional "halitosis substudy" to investigate this issue by collecting some extra PK blood samples.

Detailed description:
Study with completed results acquired from Horizon in 2024.

Arms: arm 1: Cystagon® Phase: From Screening and during Months 1, 2, 3 participants receive their usual dose of Cystagon® every 6 hours (Q6H).

RP103 Phase: During Months 3.5, 4, 5, 6, 7 participants receive RP103 every 12 hours (Q12H).

Long Term Phase: On or after Month 7, for the remainder of study participants receive RP103 Q12H.
Interventions: intervention 1: RP103 intervention 2: Cystagon®
Intervention descriptions: intervention 1: None intervention 2: None

Protocol documents:
Clinical trial NCT02038647
Conditions: Small Cell Lung Cancer
Keywords: Drug therapy
Sponsor: Millennium Pharmaceuticals, Inc.
Start date: 2014-05-12; Completion date: 2017-07-10
Enrollment: 178 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
This is a two-arm, randomized, double-blind, placebo-controlled, multicenter, phase 2 study designed to is to determine if the combination treatment can improve progression free survival (defined as the time from the date of randomization to the date of first documentation of disease progression or death, whichever occurs first) when compared with placebo + paclitaxel.

Detailed description:
The drug tested in this study is called alisertib. Alisertib is being tested to treat people who have Small Cell Lung Cancer (SCLC). This study determined the safety and efficacy for alisertib when given twice a day along with paclitaxel.

This open label study enrolled 178 patients. Participants were randomly assigned (by chance, like flipping a coin) to one of the two treatment groups-which remained undisclosed to the patient and study doctor during the study (unless there is an urgent medical need) and participants were stratified at baseline as to whether brain mets were present or not; whether they were sensitive to prior therapy or were relapsed/refractory to prior therapy; and by world region:

* Alisertib 40 mg + Paclitaxel 60 mg/m\^2
* Paclitaxel 80 mg/m\^2 + Placebo (dummy inactive pill) - this is a tablet that looks like the study drug but has no active ingredient

All participants received treatment until their disease progressed or they experienced unacceptable alisertib-related toxicity.

This multi-center trial was conducted world-wide. The overall time to participate in this study was approximately up to 22 months. Participants made multiple visits to the clinic, and were contacted by telephone every month for 6 months after the end of treatment (EOT) for follow-up assessment of progression free survival and for overall survival every 2 months until death, study closure, or 14 months after randomization of the last participant.

Arms: arm 1: Alisertib 40 mg, tablets, orally, twice a day, 3 days on/4 days off for 3 weeks on Days 1-3, 8-10, and 15-17 in a 28-day cycle along with paclitaxel 60 mg/m\^2 intravenously (IV) once a week for 3 weeks on Days 1, 8, and 15 in a 28-day until disease progression (Up to 17 Cycles). arm 2: Alisertib placebo-matching tablets, orally, twice a day, 3 days on/4 days off for 3 weeks on Days 1-3, 8-10, and 15-17 in a 28-day cycle along with paclitaxel 80 mg/m\^2 IV once a week for 3 weeks on Days 1, 8, and 15 in a 28-day cycle until disease progression (Up to 22 Cycles).
Interventions: intervention 1: Alisertib intervention 2: Placebo intervention 3: Paclitaxel
Intervention descriptions: intervention 1: Alisertib tablets intervention 2: Placebo matching tablets intervention 3: Paclitaxel intravenous injection

Protocol documents:
Clinical trial NCT02196558
Conditions: Arthritis, Rheumatoid
Keywords: Rheumatoid Arthritis Japanese subjects
Sponsor: Eisai Co., Ltd.
Start date: 2014-05-26; Completion date: 2017-07-13
Enrollment: 53 participants; Allocation: NON_RANDOMIZED; Intervention model: PARALLEL

Brief summary:
This study consist of treatment phase (12 weeks) and extension phase (40 weeks). This study is a multicenter, open-label, uncontrolled, multiple ascending dose (MAD) study to evaluate mainly the safety and tolerability of 12-week repeated subcutaneous administration of E6011 in Japanese subjects with Rheumatoid Arthritis. First 12 subjects will enroll as 100 mg group, next 12 subjects will enroll as 200 mg group, and last 9 subjects will enroll as 400 mg group. A total of 33 subjects will receive repeated subcutaneous administration of E6011. Subjects who roll over onto the Extension phase will have continued monitoring until 52 weeks after the initial administration.

Detailed description:

Arms: arm 1: E6011 will be administered repeatedly, subcutaneously at Week 0, 1, 2, followed by every 2 weeks upto 10 weeks (treatment phase).100 mg group will receive E6011 subcutaneously, 1 ml. If a subject intends to continue administrations; the subject will receive a total of 20 subsequent biweekly administrations (40 weeks) at stable dose (Extension phase). arm 2: E6011 will be administered repeatedly, subcutaneously at Week 0, 1, 2, followed by every 2 weeks upto 10 weeks (treatment phase). 200 mg group will receive E6011 subcutaneously, 1 ml each at two sites. If a subject intends to continue administrations; the subject will receive a total of 20 subsequent biweekly administrations (40 weeks) at stable dose (Extension phase). arm 3: E6011 will be administered repeatedly, subcutaneously at Week 0, 1, 2, followed by every 2 weeks up to 10 weeks (treatment phase). 400 mg group will receive E6011 subcutaneously, 1 ml each at four sites or 2 ml each at two sites. If a subject intends to continue administrations, the subject will receive a total of 20 subsequent biweekly administrations (40 weeks) at stable dose (Extension Phase).
Interventions: intervention 1: E6011
Intervention descriptions: intervention 1: None

Protocol documents:
Clinical trial NCT02047253
Conditions: Metastatic Castration-resistant Prostate Cancer
Keywords: carfilzomib prostate cancer PSA (prostate-specific antigen) CRPC (castration-resistant prostate cancer)
Sponsor: University of Alabama at Birmingham
Start date: 2014-04-01; Completion date: 2017-07-14
Enrollment: 28 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
This study will test how effective the drug, Carfilzomib, reduces progression of prostate cancer in patients who have previously received chemotherapy and androgen inhibitors. Carfilzomib is approved for multiple myeloma but is not approved for prostate cancer. Therefore, it is considered investigational. Other approved methods of treatment for metastatic prostate cancer have demonstrated only modest benefits. Novel and tolerable agents are necessary to make further gains and extend overall survival.

Detailed description:
First-line chemotherapy for metastatic castration-resistant prostate cancer (CRPC) combined with androgen inhibitors modestly extends overall survival. Carfilzomib is anticipated to enhance progression-free survival (PFS) as well as reduce pain and toxicities.

Proteasome inhibitors are promising agents used in the therapy of prostate cancer. Carfilzomib is a more potent and irreversible proteasome inhibitor than the frequently used proteasome inhibitor, Bortezomib. In Phase I trials Carfilzomib demonstrated substantial antitumor activity while exhibiting tolerable side effects.

Carfilzomib has been approved by the FDA for patients with multiple myeloma. The drug, however, is not approved for the use with CRPC patients. This trial will evaluate the tolerance and effectiveness of Carfilzomib in men with metastatic progressive CRPC following chemotherapy and androgen inhibitors.

Arms: arm 1: Carfilzomib is administered twice-weekly on consecutive days (days 1, 2, 8, 9, 15, 16) as a 30-minute intravenous infusion for 3 weeks every 4 weeks (1 cycle) with dexamethasone given as pre-medication. The dose of carfilzomib is 20 mg/m2 on days 1 and 2 of cycle 1 and is then escalated in succeeding weeks and cycles to 56 mg/m2 if no dose limiting toxicities occur. Acyclovir is given orally at 400 mg twice daily during therapy but may be discontinued at some point. Therapy will continue until side effects become unacceptable, the disease progresses, or the doctor withdraws the patient.
Interventions: intervention 1: Carfilzomib intervention 2: Dexamethasone intervention 3: Acyclovir
Intervention descriptions: intervention 1: Carfilzomib will be administered on days 1, 2, 8, 9, 15, 16 within each 4 week cycle. intervention 2: Administered prior to administration of Study drug intervention 3: Administered orally twice daily

Protocol documents:
Prot_SAP_000.pdf:
Clinical trial NCT01198145
Conditions: Diarrhea Gastrointestinal Complications Unspecified Adult Solid Tumor, Protocol Specific
Keywords: diarrhea gastrointestinal complications unspecified adult solid tumor, protocol specific
Sponsor: Alliance for Clinical Trials in Oncology
Start date: 2011-04-01; Completion date: 2017-07-15
Enrollment: 87 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
RATIONALE: Sulfasalazine may relieve diarrhea in patients with cancer who are undergoing pelvic radiation therapy.

PURPOSE: This randomized phase III trial is studying sulfasalazine to see how well it works in preventing acute diarrhea in patients with cancer who are undergoing pelvic radiation therapy.

Detailed description:
OBJECTIVES:

Primary

* To determine whether sulfasalazine is effective in reducing the acute treatment-related diarrhea in patients receiving pelvic radiotherapy as measured by NCI CTC v4.0 in patients receiving pelvic external-beam radiotherapy as adjuvant or primary treatment for malignancy.

Secondary

* To determine whether sulfasalazine can reduce chronic treatment-related bowel dysfunction following completion of therapy.
* To determine whether sulfasalazine causes any toxicity in this situation.

Tertiary

* To bank blood products for future studies, as part of ongoing research for NCCTG studies (Mayo Clinic Rochester only). (Translational)

OUTLINE: This is a multicenter study. Patients are stratified according to history of anterior resection of the rectum (yes vs no); total planned cumulative dosing, including boost fields of external-beam radiotherapy (4500-5350 cGy vs \> 5350 cGy); and concurrent radiosensitizing fluorouracil, capecitabine, or oxaliplatin (yes vs no). Patients are randomized to 1 of 2 treatment arms.

* Arm I: Patients receive oral sulfasalazine twice daily during radiotherapy\* and for 4 weeks after completion of radiotherapy.
* Arm II: Patients receive oral placebo twice daily during radiotherapy\* and for 4 weeks after completion of radiotherapy.

NOTE: \*Patients must start study treatment by the third radiotherapy fraction.

Patients may undergo blood sample collection at baseline and then weekly during radiotherapy. All patients complete quality of life and bowel function questionnaires at baseline, weekly during radiotherapy, and at 6 weeks after completion of radiotherapy.

After completion of radiotherapy, patients are followed up at 6 weeks and at 12 and 24 months.

Arms: arm 1: Patients receive oral sulfasalazine twice daily during radiotherapy and for 4 weeks after completion of radiotherapy. arm 2: Patients receive oral placebo twice daily during radiotherapy and for 4 weeks after completion of radiotherapy.
Interventions: intervention 1: sulfasalazine intervention 2: placebo
Intervention descriptions: intervention 1: Given orally intervention 2: Given orally

Protocol documents:
Clinical trial NCT02246218
Conditions: Urea Cycle Disorder
Keywords: Urea Cycle Disorder UCD Buphenyl Glycerol Phenylbutyrate Ravicti Hyperammonemia Sodium Phenylbutyrate GPB HPN-100
Sponsor: Amgen
Start date: 2014-12-31; Completion date: 2017-07-17
Enrollment: 27 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
This is an open-label study consisting of a transition period to RAVICTI, followed by a safety extension period for at least 6 months and up to 24 months of treatment with RAVICTI, depending on age at enrollment. It is designed to capture information important for evaluating safety, pharmacokinetics and efficacy in young children.

Subjects who are followed by or referred to the Investigator for management of their UCD. Subjects eligible for this study will include patients ranging from newborn to \< 2 years of age with either a diagnosed or clinically suspected UCD.

Detailed description:
Study acquired from Horizon in 2024.

Arms: arm 1: RAVICTI Oral Liquid should be administered just prior to breastfeeding or intake of formula or food. The recommended dosing regimen is 3-6 times per day depending on feeding schedule and at the discretion of the Investigator.
Interventions: intervention 1: RAVICTI
Intervention descriptions: intervention 1: None

Protocol documents:
Clinical trial NCT00494442
Conditions: Ovarian Neoplasm
Keywords: Advanced ovarian cancer Poly(ADP ribose) polymerases KU-0059436 AZD2281 BRCA1 protein BRCA2 protein
Sponsor: AstraZeneca
Start date: 2007-06-11; Completion date: 2017-07-20
Enrollment: 58 participants; Allocation: NON_RANDOMIZED; Intervention model: PARALLEL

Brief summary:
The purpose of the study is to see if the drug KU 0059436 is effective and well tolerated in treating patients with measurable BRCA1- or BRCA2-positive advanced ovarian cancer and for whom no curative therapeutic option exists.

Detailed description:

Arms: arm 1: None arm 2: None
Interventions: intervention 1: KU-0059436 (AZD2281)(PARP inhibitor) intervention 2: KU-0059436 (AZD2281)(PARP inhibitor)
Intervention descriptions: intervention 1: oral intervention 2: oral

Protocol documents:
Clinical trial NCT00593554
Conditions: Acute Myeloid Leukemia Acute Lymphoblastic Leukemia Myelodysplasia Chronic Myeloid Leukemia
Sponsor: Sherif S. Farag
Start date: 2007-08-07; Completion date: 2017-07-28
Enrollment: 9 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
The purpose of this study is to determine if haplotype-mismatched HSCT is associated with an improvement in treatment-related mortality (TRM) rate at 6 months.

Detailed description:

Arms: arm 1: Total body Irradiation; Thiotepa; Fludarabine; Rabbit ATG; arm 2: Palifermin; Total Body Irradiation; Thiotepa; Fludarabine; Rabbit ATG
Interventions: intervention 1: Total Body Irradiation intervention 2: Thiotepa intervention 3: Fludarabine intervention 4: Rabbit ATG intervention 5: Palifermin
Intervention descriptions: intervention 1: 8 Gy on Day -9 intervention 2: 5 mg/kg/d on Day -8 to -7 intervention 3: 40 mg/m2/d on Day -6 to -3 intervention 4: 2.5 mg/kg/d on Day -5 to -2 intervention 5: 60 ug/kg (actual body weight) on Day -9 to -7 and Day 0 to +2

Protocol documents:
Clinical trial NCT01303965
Conditions: Multiple Myeloma
Sponsor: Sherif S. Farag
Start date: 2011-02-07; Completion date: 2017-07-28
Enrollment: 14 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
One of the complications that can occur after a stem cell transplant is called graft versus host disease (GVHD). Another complication is that multiple myeloma may come back (relapse). In this study, a drug called lenalidomide will be started 1-2 months after a transplant, or possibly later depending on recovery of your side effects. Lenalidomide and sirolimus have been shown to work together against multiple myeloma. Therefore, lenalidomide will be combined with sirolimus with the hope that this will help prolong the amount of time the disease is in remission. Researchers hope these steps will help prolong the amount of time the multiple myeloma is in remission and will decrease the chance of GvHD.

Detailed description:

Arms: arm 1: Use sirolimus and tacrolimus as GvHD prophylaxis with sirolimus and lenalidomide as post-transplant maintenance
Interventions: intervention 1: Sirolimus intervention 2: Tacrolimus intervention 3: Lenalidomide
Intervention descriptions: intervention 1: Start on Day -3 and continue for 1 year intervention 2: Start on Day -3 and begin tapering on Day +100 until Day +180. intervention 3: Start between Day +30 and +120 and continue for 1 year.

Protocol documents:
Clinical trial NCT01604824
Conditions: Hypercholesterolemia
Sponsor: Regeneron Pharmaceuticals
Start date: 2012-02-22; Completion date: 2017-07-28
Enrollment: 23 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
The primary objective of the study is to assess the pharmacodynamic (PD) effect of alirocumab on serum low density lipoprotein cholesterol (LDL-C) during 14 weeks of subcutaneous (SC) administered alirocumab in patients with autosomal dominant hypercholesterolemia (ADH) and gain-of-function mutation (GOFm) in 1 or both alleles of the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or with loss-of-function mutation (LOFm) in 1 or more alleles of the apolipoprotein (ApoB) gene.

Detailed description:

Arms: arm 1: Participants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene (Cohort 1): Alirocumab 150 mg subcutaneous (SC) injection at Week 0 (Day 1), Week 2 (Day 15), Week 4, 6 and 10 (matching placebo at Week 8, 12 and 14) during the double-blind period (Group A). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years. arm 2: Participants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene (Cohort 1): Alirocumab 150 mg subcutaneous (SC) injection at Week 2 (Day 15), Week 4, 6, 8 and 12 (\[matching placebo at Week 0 (Day 1), 10 and 14\]) during the double-blind period (Group B). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years. arm 3: Participants with gain-of-function mutation (GOFm) in the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or loss-of-function mutation (LOFm) in the apolipoprotein (Apo) B gene (Cohort 2): Alirocumab 150 mg subcutaneous (SC) injection at Week 0 (Day 1), Week 2 (Day 15), Week 4, 6 and 10 (matching placebo at Week 8, 12 and 14) during the double-blind period (Group C). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years. arm 4: Participants with gain-of-function mutation (GOFm) in proprotein convertase subtilisin/kexin type 9 (PCSK9) gene or loss-of-function mutation (LOFm) in apolipoprotein (Apo) B gene (Cohort 2): Alirocumab 150 mg subcutaneous (SC) injection at Week 2 (Day 15), Week 4, 6, 8 and 12 (\[matching placebo at Week 0 (Day 1), 10 and 14\]) during the double-blind period (Group D). Afterwards, participants have the possibility to continue in an open-label extension period with 150 mg alirocumab SC twice per week (Q2W) for an additional 3 years.
Interventions: intervention 1: Alirocumab intervention 2: Placebo
Intervention descriptions: intervention 1: SC injection in the abdomen intervention 2: SC injection in the abdomen

Protocol documents:
Clinical trial NCT00780234
Conditions: Lung Cancer Endobronchial Dysplasia
Keywords: chemoprevention lung cancer pioglitazone
Sponsor: VA Office of Research and Development
Start date: 2009-08-01; Completion date: 2017-08-01
Enrollment: 92 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
This is a chemoprevention trial evaluating the diabetic agent pioglitazone. Non-diabetic subjects at risk for lung cancer (based on smoking history, lung function testing, and atypical cells in a sputum sample) receive either placebo or pioglitazone and have chest computerized tomography (CAT) scans and examinations of their airways with a bronchoscope at the start of the trial and after 6 months on treatment. Compensation will be provided to the subject after completing the trial.

Detailed description:
This trial evaluates the oral peroxisome proliferator-activated receptor gamma (PPARgamma) agonist pioglitazone in a double-blind placebo controlled trial. The high risk current and former smokers qualify based on tobacco exposure, airflow limitation on lung function testing, and sputum cytologic atypia. Subjects have a quantitative high resolution thoracic CT scan and a fluorescent bronchoscopy at study entry and after 6 months on drug or placebo. Biologic samples are collected at both time points. The primary outcome is endobronchial histology and determining if pioglitazone can retard progression. Secondary endpoints related to the PPAR gamma signaling pathway will also be analyzed.

Arms: arm 1: Current or former smokers receive 6 months of treatment with pioglitazone arm 2: Current or former smokers receive 6 months of treatment with placebo
Interventions: intervention 1: fluorescence bronchoscopy intervention 2: quantitative high resolution CT scan intervention 3: PIOGLITAZONE VS. PLACEBO 30 mg
Intervention descriptions: intervention 1: examination of the central airways with a bronchoscope. Both white light and fluorescent light will be used. intervention 2: High resolution CT scan of the chest intervention 3: Patients will be randomized to receive either pioglitazone or placebo. Pioglitazone hydrochloride, a thiazolidinedione antidiabetic agent and a potent peroxisome proliferator-

activated receptor-gamma agonist. It is FDA approved for the treatment of Type II diabetes. It has been previously administered to non-diabetic subjects. The most common side effect of pioglitazone is fluid retention and modest weight gain. There is a potential risk that pioglitazone may cause an elevation in liver enzymes and more serious hepatotoxicity (rare). There is risk of edema and weight gain associated with pioglitazone therapy. 5% experienced peripheral edema in clinical trials. fluid retention may result in new onset heart failure or exacerbation of existing heart failure. Small risk of hypoglycemia, anemia, myalgia, bone fracture, headache, and macular retinal edema exists. There is insufficient information to confirm its safety in Pregnancy/Breastfeeding. Bladder cancer is more serious but rare.

Protocol documents: