Trial Outcomes & Findings for A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients (NCT NCT05744063)

Last Updated: 2026-03-13

Results Overview

Number of participants permanently discontinuation of study drug due to emapalumab-related adverse event as judged by Investigator, until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

13 participants

Primary outcome timeframe

Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose

Results posted on

2026-03-13

Participant Flow

Participant milestones

Participant milestones
Measure	Emapalumab Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Overall Study STARTED	13
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Only applicable to female participants

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Emapalumab n=13 Participants Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Age, Continuous	7 years n=13 Participants
Sex: Female, Male Female	5 Participants n=13 Participants
Sex: Female, Male Male	8 Participants n=13 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=13 Participants
Race (NIH/OMB) Asian	13 Participants n=13 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=13 Participants
Race (NIH/OMB) Black or African American	0 Participants n=13 Participants
Race (NIH/OMB) White	0 Participants n=13 Participants
Race (NIH/OMB) More than one race	0 Participants n=13 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=13 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=13 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=13 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=13 Participants
Child-bearing potential Yes	1 Participants n=5 Participants • Only applicable to female participants
Child-bearing potential No	4 Participants n=5 Participants • Only applicable to female participants

PRIMARY outcome

Timeframe: Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose

Outcome measures

Outcome measures
Measure	Emapalumab n=13 Participants Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event	0 Participants

SECONDARY outcome

Timeframe: End of treatment or week 8 (whichever occurs earlier)

Number of participants with an overall response i.e., achievement of either Complete Response or Partial Response or HLH Improvement, at end of treatment or week 8 (whichever occurs earlier).

Outcome measures

Outcome measures
Measure	Emapalumab n=13 Participants Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Overall Response Overall response	10 Participants
Overall Response No Response	2 Participants
Overall Response Missing	1 Participants

SECONDARY outcome

Timeframe: End of treatment, likely within 6 months from first dose

Population: Two participants with no response are censored at the date of EOT visit or last assessment. The analysis is conducted in all participants treated.

Time to first overall response from first dose of study drug to the first achievement of response (Complete Response, Partial Response, or HLH Improvement)

Outcome measures

Outcome measures
Measure	Emapalumab n=11 Participants Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Time to First Overall Response	4 Days Interval 3.0 to 5.0

SECONDARY outcome

Timeframe: End of treatment, likely within 6 months from first dose

Population: The analysis is conducted in all participants treated excluding those (2 participants) who do not achieve response at least once between the date of first dose and EOT.

Cumulative duration of response is defined as total time in response from the first achievement of an Overall Response until EOT. For patients who achieve a response, lost that response, and then achieve it subsequently, the total time in response is calculated by adding together these separate periods in response.

Outcome measures

Outcome measures
Measure	Emapalumab n=11 Participants Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Cumulative Duration of Response	55.0 Days Standard Deviation 33.36

SECONDARY outcome

Timeframe: End of treatment, likely within 6 months from first dose

Number of participants able to reduce glucocorticoids by 50% or more of the baseline dose at any time point of the treatment period

Outcome measures

Outcome measures
Measure	Emapalumab n=13 Participants Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Ability to Reduce Glucocorticoids by 50% or More	6 Participants

SECONDARY outcome

Timeframe: End of treatment

Population: Two participants were not assessed at EOT or last assessment

Investigator's assessment of how patient responds to treatment and rated as complete response, partial response, or no response

Outcome measures

Outcome measures
Measure	Emapalumab n=11 Participants Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Investigator Assessed Response Complete Response	2 Participants
Investigator Assessed Response Partial Response	6 Participants
Investigator Assessed Response No response	3 Participants

SECONDARY outcome

Timeframe: End of study (1 year)

Population: All participants are included in the Survival to start of HSCT conditioning. For survival after HSCT only participants that underwent HSCT are included in the measure, i.e. 9 participants

Number of participants surviving to start of HSCT conditioning and Number of participants that underwent HSCT surviving after HSCT to end of study

Outcome measures

Outcome measures
Measure	Emapalumab n=13 Participants Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Survival Survival to start of HSCT conditioning	11 Participants
Survival Survival after HSCT (1 year post HSCT)	9 Participants

Adverse Events

Emapalumab

Serious events: 7 serious events

Other events: 13 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Emapalumab n=13 participants at risk Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Infections and infestations Pneumonia respiratory syncytial viral	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Cytomegalovirus viraemia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Encephalitis viral	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Pneumonia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Sepsis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
General disorders Condition aggravated	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Immune system disorders Graft versus host disease	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Immune system disorders Haemophagocytic lymphohistiocytosis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Blood and lymphatic system disorders Thrombocytopenia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Stomatitis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Renal and urinary disorders Cystitis haemorrhagic	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).

Other adverse events

Other adverse events
Measure	Emapalumab n=13 participants at risk Emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Metabolism and nutrition disorders Hypercholesterolaemia	15.4% 2/13 • Number of events 6 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hyperkalaemia	15.4% 2/13 • Number of events 4 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hyponatraemia	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hypomagnesaemia	7.7% 1/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Alkalosis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hyperlactacidaemia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Blood fibrinogen decreased	7.7% 1/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Blood fibrinogen increased	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Epstein-Barr virus test positive	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Glucose urine present	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Neutrophil count increased	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Serum ferritin decreased	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Urinary occult blood positive	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Urine ketone body	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations White blood cell count increased	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hypokalaemia	53.8% 7/13 • Number of events 10 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hypoalbuminaemia	38.5% 5/13 • Number of events 5 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Electrolyte imbalance	30.8% 4/13 • Number of events 5 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hypoproteinaemia	23.1% 3/13 • Number of events 7 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hypertriglyceridaemia	23.1% 3/13 • Number of events 5 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Blood and lymphatic system disorders Myelosuppression	76.9% 10/13 • Number of events 10 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Blood and lymphatic system disorders Anaemia	23.1% 3/13 • Number of events 3 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Blood and lymphatic system disorders Hypofibrinogenaemia	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Blood and lymphatic system disorders Coagulopathy	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Blood and lymphatic system disorders Iron deficiency anaemia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Blood and lymphatic system disorders Lymphopenia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Blood and lymphatic system disorders Splenomegaly	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Hepatobiliary disorders Hepatic function abnormal	76.9% 10/13 • Number of events 11 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Hepatobiliary disorders Hyperbilirubinaemia	15.4% 2/13 • Number of events 3 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Hepatobiliary disorders Liver injury	7.7% 1/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Serum ferritin increased	53.8% 7/13 • Number of events 7 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Bile acids increased	38.5% 5/13 • Number of events 5 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations C-reactive protein increased	38.5% 5/13 • Number of events 5 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Blood glucose decreased	30.8% 4/13 • Number of events 9 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Fibrin D dimer increased	23.1% 3/13 • Number of events 3 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations International normalised ratio increased	15.4% 2/13 • Number of events 4 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Prothrombin time prolonged	15.4% 2/13 • Number of events 4 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Blood magnesium decreased	15.4% 2/13 • Number of events 3 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Activated partial thromboplastin time prolonged	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Blood urea increased	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Platelet count decreased	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations Protein urine present	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Investigations T-lymphocyte count increased	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hypernatraemia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Metabolism and nutrition disorders Hypoglycaemia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Upper respiratory tract infection	38.5% 5/13 • Number of events 10 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Bronchitis	23.1% 3/13 • Number of events 3 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Pneumonia	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Cytomegalovirus infection	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Bacteraemia	7.7% 1/13 • Number of events 6 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Adenovirus infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Fungal infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Fungal sepsis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Gastroenteritis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations JC virus infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Localised infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Norovirus infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Respiratory syncytial virus infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Respiratory tract infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Infections and infestations Urinary tract infection	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Constipation	23.1% 3/13 • Number of events 4 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Diarrhoea	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Stomatitis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Anal fissure	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Ascites	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Gastritis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Gastrointestinal haemorrhage	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Oral disorder	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Gastrointestinal disorders Vomiting	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Vascular disorders Hypertension	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Vascular disorders Shock	15.4% 2/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Vascular disorders Jugular vein thrombosis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Vascular disorders Peripheral vein thrombosis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
General disorders Pyrexia	23.1% 3/13 • Number of events 7 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
General disorders Generalised oedema	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
General disorders Multiple organ dysfunction syndrome	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
General disorders Swelling	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Cardiac disorders Myocardial injury	23.1% 3/13 • Number of events 4 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Cardiac disorders Cardiac failure acute	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Cardiac disorders Sinus tachycardia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Cardiac disorders Supraventricular tachycardia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Epistaxis	15.4% 2/13 • Number of events 10 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Dyspnoea	7.7% 1/13 • Number of events 2 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Acute respiratory failure	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Asthma	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Cough	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Pleural effusion	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Pulmonary bulla	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Respiratory, thoracic and mediastinal disorders Sputum increased	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Immune system disorders Acute graft versus host disease	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Immune system disorders Engraftment syndrome	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Immune system disorders Immunosuppression	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Injury, poisoning and procedural complications Allergic transfusion reaction	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Injury, poisoning and procedural complications Clavicle fracture	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Injury, poisoning and procedural complications Postimplantation syndrome	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Skin and subcutaneous tissue disorders Dermatitis contact	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Skin and subcutaneous tissue disorders Pruritus	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Skin and subcutaneous tissue disorders Rash morbilliform	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Skin and subcutaneous tissue disorders Urticaria	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Eye disorders Astigmatism	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Eye disorders Ocular hypertension	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Musculoskeletal and connective tissue disorders Bone pain	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Musculoskeletal and connective tissue disorders Synovitis	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Nervous system disorders Headache	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Nervous system disorders Hypoaesthesia	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Renal and urinary disorders Renal impairment	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).
Congenital, familial and genetic disorders Rathke's cleft cyst	7.7% 1/13 • Number of events 1 • AEs were collected from the time of the first dose of investigational medication until end of study (1 year).

Additional Information

Medical Info

Swedish Orphan Biovitrum (Sobi)

Phone: +1 774 548 5650

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60