nyuuzyou/wikis huggingface dataset:
Experimental design

The purpose of this page is to explain details relevant to experimental trial design, as well as to explain our notations for such designs. It will be expanded over time.
=Experimental arm design=
By definition, the experimental arm of a trial does something different from the comparator/baseline arm. In hematology/oncology trials, what that difference is falls into four general categories:
*Escalations: arms where one or more of the four following types of escalation are undertaken:
**New drug: eg control arm is A and experimental arm is A + B
**More cycles: eg control arm is A x 3 and experimental arm is A x 6
**Higher dose: eg control arm is A 300 mg and experimental arm is A 600 mg
**Higher intensity: eg control arm is A every 3 weeks and experimental arm is A every 2 weeks
*De-escalations: the converse of escalations
*In-class switches: different drugs that are within the same general class (e.g., chemotherapy, immunotherapy). Two common flavors of this:
**A versus B
**A + B versus A + C, where A is the "backbone"
*Out-of-class switches: different drugs that are within different classes (eg, control is chemotherapy, experimental is immunotherapy)
These classes are not always mutually exclusive. For example, the substitution of standard chemotherapy for high-dose chemotherapy with autologous hematopoietic stem cell transplant in the treatment of breast cancer (e.g., Schmid et al. 2005) is an example of an in-class escalation design.
==Labeling==
Experimental arms are labeled using the following schema:
*Escalation arms: (E-esc)
*De-escalation arms: (E-de-esc)
*In-class switch arms: (E-switch-ic)
*Out-of-class switch arms (E-switch-ooc)
==Registration trials==
These represent a special subset of clinical trials that were used to make an approval or labeling decision by the FDA or similar agencies. We have labeled all trials that are explicitly mentioned in regulatory labels with (E-RT) if the experimental arm of an RCT was used to justify approval or a labeling change, and as (RT) if a non-randomized trial was used for such justification.
==Putting it together==
For trials that are registration trials and RCTs, the label is combined. For example an escalation arm would be labeled as (E-RT-esc).
==Placebos==
==Complex multipart regimens==
Some trial designs are much more complicated than the examples described above. In these cases, it can be more difficult to determine the type of difference between the comparator and experimental arms. See the dedicated page for more details.
=Endpoints=
Of equal importance to the trial design is the choice of endpoint(s). Traditionally, the sample size of a trial is calculated such that there is adequate power to detect one or more primary endpoints (also called primary objective, main endpoint, major endpoint). All other measured endpoints are considered secondary. See the response to treatment page for more details about endpoints.
==Superiority designs==
==Non-inferiority and equivalence designs==
Category:Evidence-based oncology
External beam radiotherapy

Also known as external beam radiation therapy, EBT, or EBRT. This is the most common type of radiotherapy and consists of ionizing radiation delivered from outside the body.
==Diseases for which it is used==
*Anal cancer
*Anaplastic glioma
*Bladder cancer
*B-cell acute lymphoblastic leukemia
*Cervical cancer
*Chronic lymphocytic leukemia
*CNS leukemia
*CNS lymphoma
*Diffuse large B-cell lymphoma
*Esophageal cancer
*Extranodal NK- and T-cell lymphoma, nasal type
*Ewing sarcoma
*Gastric cancer
*Glioblastoma
*Head and neck cancer
*HIV-associated lymphoma
*Classical Hodgkin lymphoma
*Low-grade glioma
*Mantle cell lymphoma
*Multiple myeloma
*Nasopharyngeal carcinoma
*Non-small cell lung cancer
*Pancreatic cancer
*Peripheral T-cell lymphoma
*Primary mediastinal B-cell lymphoma
*Prostate cancer
*Rectal cancer
*Small cell lung cancer
*T-cell acute lymphoblastic leukemia
*Testicular cancer
*Thymoma
*Endometrial cancer
Category:Radiation
Category:Anal cancer medications
Category:Anaplastic glioma medications
Category:Bladder cancer medications
Category:B-cell acute lymphoblastic leukemia medications
Category:Cervical cancer medications
Category:Chronic lymphocytic leukemia medications
Category:CNS leukemia medications
Category:CNS lymphoma medications
Category:Diffuse large B-cell lymphoma medications
Category:Esophageal cancer medications
Category:Extranodal NK- and T-cell lymphoma, nasal type medications
Category:Ewing sarcoma medications
Category:Gastric cancer medications
Category:Glioblastoma medications
Category:Head and neck cancer medications
Category:HIV-associated lymphoma medications
Category:Classical Hodgkin lymphoma medications
Category:Low-grade glioma medications
Category:Mantle cell lymphoma medications
Category:Multiple myeloma medications
Category:Nasopharyngeal carcinoma medications
Category:Non-small cell lung cancer medications
Category:Pancreatic cancer medications
Category:Peripheral T-cell lymphoma medications
Category:Primary mediastinal B-cell lymphoma medications
Category:Prostate cancer medications
Category:Rectal cancer medications
Category:Small cell lung cancer medications
Category:T-cell acute lymphoblastic leukemia medications
Category:Testicular cancer medications
Category:Thymoma medications
Category:Endometrial cancer medications
FT-596

==Mechanism of action==
From the NCI Drug Dictionary: An allogeneic, off-the-shelf, chimeric antigen receptor (CAR)-natural killer (NK) cell product derived from a clonal master induced pluripotent stem cell (iPSC) line, and engineered to express a NK cell-specific anti-CD19 CAR, a high-affinity, non-cleavable CD16 (hnCD16) Fc receptor, and a recombinant fusion of IL-15 and IL-15 receptor alpha (IL-15RF), with potential immunostimulatory and antineoplastic activities. Upon administration, iPSC-derived CD16/IL-15RF-expressing Anti-CD19 CAR-NK Cells FT596 recognize, bind to and induce selective cytotoxicity in CD19-expressing tumor cells. IL-15RF enhances the cytotoxic effect of the NK cells and the activated anti-tumor T-cells. When used in combination with monoclonal antibodies, the hnCD16 Fc receptor of FT596 binds to the Fc portion of tumor cell-bound monoclonal antibodies, leading to NK cell activation, cytokine secretion and enhanced antibody-dependent cellular cytotoxicity (ADCC).
==Preliminary data==
Note: This is not meant to be an exhaustive list of preliminary results. References included in this section include non-randomized studies published in high-profile journals (e.g., NEJM) and experimental arms of phase 3 RCTs. Inclusion here is not an indication of impending regulatory approval, although many of these drugs do end up receiving approval by a regulatory authority.
===B-cell lymphoma===
#Ghobadi A, Bachanova V, Patel K, Park JH, Flinn I, Riedell PA, Bachier C, Diefenbach CS, Wong C, Bickers C, Wong L, Patel D, Goodridge J, Denholt M, Valamehr B, Elstrom RL, Strati P. Induced pluripotent stem-cell-derived CD19-directed chimeric antigen receptor natural killer cells in B-cell lymphoma: a phase 1, first-in-human trial. Lancet. 2025 Jan 11;405(10473):127-136. link to original article EuroPMC NCT04245722
==Also known as==
*Code name: FT-596
Category:Drugs
Category:Intravenous medications
Category:B-cell lymphoma medications (investigational)
Category:Stub
Factor IX, human

==General information==
Class/mechanism: Factor IX; replaces the missing clotting factor IX that is required for hemostasis in patients with hemophilia B.Please choose from the products below to find specific drug/manufacturer information: 
*Alphanine SD Factor IX, human (Alphanine SD) package insertFactor IX, human (Alphanine SD) package insert (locally hosted backup)
*Mononine Mononine manufacturer's websiteFactor IX, human (Mononine) package insertFactor IX, human (Mononine) package insert (locally hosted backup)
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias or the prescribing information.
==Patient drug information==
*Patient drug information (UpToDate)Patient drug information (UpToDate)
==Also known as==
*Generic names: Christmas Factor, coagulation factor 9, FIX
*Brand names: Alphanine SD, Mononine
==References==
Category:Intravenous medications
Category:Fractionated plasma products
Category:Inherited coagulopathy medications
Category:Hemostasis medications
Category:Coagulation factors
Category:Hematology medications
Factor IX, recombinant

==General information==
Class/mechanism: Recombinant factor IX; replaces the missing clotting factor IX that is required for hemostasis in patients with hemophilia B.Please choose from the products below to find specific drug/manufacturer information: 
*Benefix BeneFix manufacturer's websiteFactor IX, recombinant (BeneFix) package insertFactor IX, recombinant (BeneFix) package insert (locally hosted backup)
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias or the prescribing information.
==Patient drug information==
*Factor IX, recombinant (BeneFix) package insert PDF pages 22-30Factor IX, recombinant (BeneFix) package insert PDF pages 22-30
*Factor IX, human and recombinant (coagulation factor 9) patient drug information (UpToDate)Factor IX, human and recombinant (coagulation factor 9) patient drug information (UpToDate)
==Also known as==
*Generic names: recombinant Christmas Factor, recombinant coagulation factor 9, recombinant FIX
*Brand name: BeneFix
==References==
Category:Drugs
Category:Intravenous medications
Category:Recombinant medications
Category:Inherited coagulopathy medications
Category:Coagulation factors
Category:Hematology medications
Factor IX, recombinant, Fc fusion protein (Alprolix)

To be completed
==Also known as==
*Brand name: Alprolix
==References==
# Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, Josephson NC, Perry D, Manco-Johnson MJ, Apte S, Baker RI, Chan GC, Novitzky N, Wong RS, Krassova S, Allen G, Jiang H, Innes A, Li S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Nugent K, Vigliani G, Brennan A, Luk A, Pierce GF; B-LONG Investigators. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013 Dec 12;369(24):2313-23. Epub 2013 Dec 4. link to original article EuroPMC
Category:Drugs
Category:Intravenous medications
Category:Inherited coagulopathy medications
Category:Recombinant medications
Category:Hematology medications
Category:Stub
Factor VIII, human

==General information==
Class/mechanism: Factor VIII; replaces the missing clotting factor VIII that is required for hemostasis in patients with hemophilia A.Please choose from the products below to find specific drug/manufacturer information:
*Hemofil-MHemofil-M manufacturer's websiteFactor VIII, human (Hemofil-M) package insert Factor VIII, human (Hemofil-M) package insert (locally hosted backup)
*Koate-DVIKoate-DVI manufacturer's websiteFactor VIII, human (Koate-DVI) package insert Factor VIII, human (Koate-DVI) package insert (locally hosted backup)
*Monarc-MFactor VIII, human (Monarc-M) package insert Factor VIII, human (Monarc-M) package insert (locally hosted backup)
*Monoclate-PMonoclate-P manufacturer's websiteFactor VIII, human (Monoclate-P) package insert Factor VIII, human (Monoclate-P) package insert (locally hosted backup)
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias or the prescribing information.
==Diseases for which it is used==
*Hemophilia A
==Also known as==
*Generic name: coagulation factor 8
*Brand names: Hemofil-M, Koate-DVI, Koate-HP, Monarc-M, Monoclate-P
==References==
Category:Fractionated plasma products
Category:Intravenous medications
Category:Inherited coagulopathy medications
Category:Hematology medications
Category:Hemostasis medications
Category:Coagulation factors
Factor VIII, recombinant

==General information==
Class/mechanism: Recombinant Factor VIII; replaces the missing clotting factor VIII that is required for hemostasis in patients with hemophilia A.Please choose from the products below to find specific drug/manufacturer information:
*Advate Advate manufacturer's websiteFactor VIII, recombinant (Advate) package insert Factor VIII, recombinant (Advate) package insert (locally hosted backup)
*Helixate FS Helixate FS manufacturer's websiteFactor VIII, recombinant (Helixate FS) package insert Factor VIII, recombinant (Helixate FS) package insert (locally hosted backup)
*Kogenate FS Kogenate FS manufacturer's websiteFactor VIII, recombinant (Kogenate FS) package insert Factor VIII, recombinant (Kogenate FS) package insert (locally hosted backup)
*Recombinate Recombinate manufacturer's websiteFactor VIII, recombinant (Recombinate) package insert Factor VIII, recombinant (Recombinate) package insert (locally hosted backup)
*ReFacto Factor VIII, recombinant (ReFacto) package insert Factor VIII, recombinant (ReFacto) package insert (locally hosted backup)
*Xyntha Xyntha manufacturer's websiteFactor VIII, recombinant (Xyntha) package insert Factor VIII, recombinant (Xyntha) package insert (locally hosted backup)
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias or the prescribing information.
==Diseases for which it is used==
*Hemophilia A
==Patient drug information==
*Factor VIII, recombinant (Advate) package insert
*Factor VIII, recombinant (Helixate FS) package insert
*Factor VIII, recombinant (Kogenate FS) package insert
*Factor VIII, recombinant (Recombinate) package insert
*Factor VIII, recombinant (ReFacto) package insert
*Factor VIII, recombinant (Xyntha) package insert
*Factor VIII, recombinant (antihemophilic factor 8) patient drug information (UpToDate)Factor VIII, recombinant (antihemophilic factor 8) patient drug information (UpToDate)
==Also known as==
*Generic name: recombinant coagulation factor 8
*Brand names: Advate rAHF-PFM, Helixate, Helixate FS, Kogenate, Kogenate FS, Recombinate, Refacto, Xyntha
==References==
Category:Drugs
Category:Intravenous medications
Category:Inherited coagulopathy medications
Category:Recombinant medications
Category:Hematology medications
Category:Hemostasis medications
Category:Coagulation factors
Factor VIIa, recombinant (NovoSeven RT)

==General information==
Class/mechanism: Recombinant factor VIIa; complexes with tissue factor and can activate coagulation factors X to Xa and IX to IXa, which results in conversion of prothrombin to thrombin, and fibrinogen to fibrin to form a fibrin clot. There is still uncertainty about the precise mechanism, and activation of factor X on platelet surfaces may also play an important role. Clinical applications include use for patients with inhibitors to factors VIII and IX, as can be the case in hemophilia A and hemophilia B. The NovoSeven RT formulation contains sucrose and L-Methionine which "allows the product to be stored at room temperature (up to 77 degrees Fahrenheit) for up to two years."Factor VIIa, recombinant (NovoSeven RT) package insert Factor VIIa, recombinant (NovoSeven RT) package insert (locally hosted backup)NovoSeven RT manufacturer's website5/9/2008 FDA approval announcement for NovoSeven RT (fda.gov)
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias or the prescribing information.
==Patient drug information==
*Brief patient counseling information can be found in the Factor VIIa, recombinant (NovoSeven RT) package insert
*Factor VIIa, recombinant (NovoSeven RT) patient drug information (UpToDate)Factor VIIa, recombinant (NovoSeven RT) patient drug information (UpToDate)
==History of changes in FDA indication==
*1999-03-25: Initial approval for use in the treatment of bleeding episodes in hemophilia A or B patients with inhibitors to Factor VIII or Factor IX.
*2008-05-09: Approval of NovoSeven RT formulation for treatment of bleeding and the prevention of surgical bleeding in patients with hemophilia A or B, who have antibodies that neutralize the action of clotting Factors VIII or IX; the treatment of bleeding and the prevention of surgical bleeding in patients with congenital Factor VII deficiency; and the prevention of surgical bleeding in patients with acquired hemophilia.
==Also known as==
*Generic names: recombinant coagulation factor VIIa, eptacog alfa (activated), recombinant factor 7a
*Brand names: AryoSeven, NiaStase, NovoSeven, NovoSeven RT, Novo-Seven, NovoSeven room temperature stable
==References==
Category:Drugs
Category:Intravenous medications
Category:Recombinant medications
Category:Hematology medications
Category:Hemostasis medications
Category:Coagulation factors
Category:FDA approved in 1999
Factor X, human (Coagadex)

==General information==
Class/mechanism: Factor X; replaces the missing clotting factor X that is required for hemostasis in patients with inherited or acquired factor X deficiency.
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias or the prescribing information.
==Also known as==
*Brand name: Coagadex
==References==
Category:Intravenous medications
Category:Fractionated plasma products
Category:Inherited coagulopathy medications
Category:Hemostasis medications
Category:Coagulation factors
Category:Hematology medications
Factor XIII concentrate, human (Corifact)

==General information==
Class/mechanism: Human factor XIII; in the coagulation cascade, factor XIII is activated by thrombin (factor IIa) to factor XIIIa, which promotes cross-linking of fibrin. Factor XIIIa promotes stabilization of the fibrin clot by cross-linking alpha-2 plasmin inhibitor to the alpha chain of fibrin. Clinically used in patients with congenital factor XIII deficiency.Factor XIII concentrate, human (Corifact) package insertFactor XIII concentrate, human (Corifact) package insert (locally hosted backup)Corifact manufacturer's website
Route: IV
Extravasation: no information
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias or the prescribing information.
==Patient drug information==
*Factor XIII concentrate, human (Corifact) package insert PDF page 5Factor XIII concentrate, human (Corifact) package insert PDF page 5
*Factor XIII concentrate, human (Corifact) patient drug information (UpToDate)Factor XIII concentrate, human (Corifact) patient drug information (UpToDate)
==Also known as==
*Generic names: coagulation factor 13, FXIII
*Brand name: Corifact
==References==
Category:Fractionated plasma products
Category:Intravenous medications
Category:Inherited coagulopathy medications
Category:Hemostasis medications
Category:Coagulation factors
Category:Hematology medications
Fadrozole (Afema)

==Mechanism of action==
From the NCI Data Dictionary: The hydrochloride salt of the nonsteroidal aromatase inhibitor fadrozole with potential antineoplastic activity. Fadrozole specifically inhibits aromatase, blocking the aromatization of androstenedione and testosterone into estrone and estradiol, respectively, the final step in estrogen biosynthesis; the reduction in estrogen levels may inhibit growth in estrogen-dependent cancers.
==Diseases for which it is used==
*Breast cancer
==History of changes in PMDA indication==
*1995: Approved for the management of breast cancer.
==Also known as==
*Code name: CGS-16949A, FAD-286
*Brand name: Afema
Category:Drugs
Category:Oral medications
Category:Aromatase inhibitors, second generation
Category:Breast cancer medications
Category:PMDA approved in 1995
Farletuzumab (MORAb-003)

==Mechanism of action==
From the NCI Drug Dictionary: A humanized, immunoglobulin G1 monoclonal antibody with potential antitumor activity. Farletuzumab specifically targets at glycoprotein 3 (GP-3), a cell surface antigen that is overexpressed on many epithelial-derived cancer cells. Upon binding to the GP-3 antigen, farletuzumab triggers a host immune response against GP-3 expressing cells resulting in cell lysis.
==Also known as==
*Code name: MORAb-003
Category:Drugs
Category:Intravenous medications
Category:Anti-GP-3 antibodies
Category:Ovarian cancer medications (investigational)
Category:Investigational drugs
Category:Stub
Fedratinib (Inrebic)

==General information==
Class/mechanism from the NCI Drug Dictionary: An orally bioavailable, small-molecule, ATP-competitive inhibitor of Janus-associated kinase 2 (JAK2) with potential antineoplastic activity. Fedratinib competes with JAK2 as well as the mutated form AK2V617F for ATP binding, which may result in inhibition of JAK2 activation, inhibition of the JAK-STAT signaling pathway, and the induction of tumor cell apoptosis.
Route: PO
Extravasation: n/a
For conciseness and simplicity, HemOnc.org currently will focus on treatment regimens and not list information such as: renal/hepatic dose adjustments, metabolism (including CYP450), excretion, monitoring parameters (although this will be considered for checklists), or manufacturer. Instead, for the most current information, please refer to your preferred pharmacopeias or the prescribing information.
==Diseases for which it is used==
*Myelofibrosis
==History of changes in FDA indication==
* 2019-08-16: Initial approval for adults with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF). (Based on JAKARTA)
==History of changes in EMA indication==
*2021-02-08: Initial authorization as Inrebic
*Inrebic is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis who are Janus Associated Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.
== Patient Drug Information==
*Fedratinib (Inrebic) Package InsertFedratinib (Inrebic) Package Insert
==Also known as==
*Code names: SAR-302503, TG-101348
*Brand names: Inrebic
==References==
Category:Drugs
Category:Oral medications
Category:JAK2 inhibitors
Category:Myelofibrosis medications
Category:FDA approved in 2019
Category:EMA approved in 2021