Compassionate Use Study of Methylnaltrexone
Study Details
Study Description
Brief Summary
This is an open label compassionate use study of subcutaneously administered methylnaltrexone (MNTX) in participants with advance medical illness and opioid-induced constipation.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| N/A |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Methylnaltrexone Participants will receive single dose of MNTX 0.15 milligrams per kilogram (mg/kg) subcutaneously (SC). Subsequent dosing could be adjusted upward (to a maximum of 0.3 mg/kg) to achieve a desired clinical response or decreased to improve tolerability. |
Drug: Methylnaltrexone
Methylnaltrexone will be administered as per the dose and schedule specified in the arm.
|
Outcome Measures
Primary Outcome Measures
- Number of Participants With Opioid Induced Side Effects [From start of treatment until end of study (up to maximum 3.4 years)]
Opioid induced side effects included nausea, myoclonus, mental clouding (confusional state), vomiting, sedation, pruritus, sweating (hyperhidrosis), constipation, and urinary retention. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Advanced medical illness (that is; terminal illness, such as incurable cancer or end stage aqcuired immunodeficiency syndrome [AIDS]) with a life expectancy of one to six months.
-
On an opioid regimen for the control of pain/discomfort for at least seven days.
-
Has opioid-induced constipation.
-
Stable vital signs and systolic blood pressure greater than or equal to (>=) 85 millimeters of mercury (mmHg), and diastolic blood pressure >=45 mmHg (Supine or sitting).
-
Females of childbearing potential must have a negative pregnancy test (serum or urine).
-
On a laxative regimen (for example, stool softener and SENNA or equivalent) for at least 3 days prior to treatment. Participants who have discontinued laxatives due to intolerability or lack of efficacy are also eligible.
Exclusion Criteria:
-
Participants with known hypersensitivity to methylnaltrexone, naltrexone or naloxone.
-
Participants who received any investigational new drug (experimental) except for methylnaltrexone in the previous 30 days.
-
Participants who are constipated with a disease process suggestive of gastrointestinal obstruction, impaction or diagnosed with a current abdominal pathologic process which may represent a non-opioid cause of bowel dysfunction.
-
Participants who are constipated and have active, clinically significant diverticulitis.
-
Participants with a surgically acute abdomen.
-
Participants being treated with opioids for diarrhea, dyspnea, cough, pulmonary edema, or congestive heart failure.
-
Individuals with a known drug addiction.
-
Females who are pregnant or nursing.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Progenics Pharmaceuticals, Inc. | Tarrytown | New York | United States | 10591 |
Sponsors and Collaborators
- Bausch Health Americas, Inc.
Investigators
- Study Director: Lindsey Mathew, Bausch Health Companies
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- MNTX 901
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Methylnaltrexone |
|---|---|
| Arm/Group Description | Participants received single dose of methylnaltrexone (MNTX) 0.15 milligrams per kilogram (mg/kg) subcutaneously (SC) initially. Subsequent dosing was adjusted upward (to a maximum of 0.3 mg/kg) to achieve a desired clinical response or decreased to improve tolerability. Median treatment duration was 25 days. |
| Period Title: Overall Study | |
| STARTED | 26 |
| Received at Least 1 Dose of Study Drug | 25 |
| COMPLETED | 7 |
| NOT COMPLETED | 19 |
Baseline Characteristics
| Arm/Group Title | Methylnaltrexone |
|---|---|
| Arm/Group Description | Participants received single dose of MNTX 0.15 mg/kg SC initially. Subsequent dosing was adjusted upward (to a maximum of 0.3 mg/kg) to achieve a desired clinical response or decreased to improve tolerability. Median treatment duration was 25 days. |
| Overall Participants | 26 |
| Age (years) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [years] |
59.4
(13.63)
|
| Sex: Female, Male (Count of Participants) | |
| Female |
14
53.8%
|
| Male |
12
46.2%
|
| Race/Ethnicity, Customized (Count of Participants) | |
| Caucasian |
24
92.3%
|
| Hispanic |
2
7.7%
|
Outcome Measures
| Title | Number of Participants With Opioid Induced Side Effects |
|---|---|
| Description | Opioid induced side effects included nausea, myoclonus, mental clouding (confusional state), vomiting, sedation, pruritus, sweating (hyperhidrosis), constipation, and urinary retention. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module. |
| Time Frame | From start of treatment until end of study (up to maximum 3.4 years) |
Outcome Measure Data
| Analysis Population Description |
|---|
| All enrolled participants who provided a signed and dated written ICF. |
| Arm/Group Title | Methylnaltrexone |
|---|---|
| Arm/Group Description | Participants received single dose of MNTX 0.15 mg/kg SC initially. Subsequent dosing was adjusted upward (to a maximum of 0.3 mg/kg) to achieve a desired clinical response or decreased to improve tolerability. Median treatment duration was 25 days. |
| Measure Participants | 26 |
| Nausea |
3
11.5%
|
| Myoclonus |
1
3.8%
|
| Vomiting |
3
11.5%
|
| Sedation |
1
3.8%
|
| Constipation |
1
3.8%
|
| Urinary retention |
3
11.5%
|
| Mental clouding |
4
15.4%
|
| Sweating |
2
7.7%
|
| Pruritus |
0
0%
|
Adverse Events
| Time Frame | From start of treatment until end of study (up to maximum 3.4 years) | |
|---|---|---|
| Adverse Event Reporting Description | Safety evaluable participants included all enrolled participants who received at least one dose of study drug. | |
| Arm/Group Title | Methylnaltrexone | |
| Arm/Group Description | Participants received single dose of MNTX 0.15 mg/kg SC initially. Subsequent dosing was adjusted upward (to a maximum of 0.3 mg/kg) to achieve a desired clinical response or decreased to improve tolerability. Median treatment duration was 25 days. | |
All Cause Mortality |
||
| Methylnaltrexone | ||
| Affected / at Risk (%) | # Events | |
| Total | / (NaN) | |
Serious Adverse Events |
||
| Methylnaltrexone | ||
| Affected / at Risk (%) | # Events | |
| Total | 11/25 (44%) | |
| Cardiac disorders | ||
| Myocardial infarction | 1/25 (4%) | |
| Gastrointestinal disorders | ||
| Small intestinal obstruction | 1/25 (4%) | |
| Ascites | 1/25 (4%) | |
| General disorders | ||
| Asthenia | 1/25 (4%) | |
| Pain | 1/25 (4%) | |
| Hepatobiliary disorders | ||
| Hepatic failure | 1/25 (4%) | |
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||
| Malignant neoplasm progression | 8/25 (32%) | |
| Nervous system disorders | ||
| Convulsion | 1/25 (4%) | |
| Respiratory, thoracic and mediastinal disorders | ||
| Acute respiratory failure | 1/25 (4%) | |
| Dyspnoea | 1/25 (4%) | |
Other (Not Including Serious) Adverse Events |
||
| Methylnaltrexone | ||
| Affected / at Risk (%) | # Events | |
| Total | 15/25 (60%) | |
| Blood and lymphatic system disorders | ||
| Anaemia | 1/25 (4%) | |
| Cardiac disorders | ||
| Cyanosis | 1/25 (4%) | |
| Bradycardia | 1/25 (4%) | |
| Atrial fibrillation | 1/25 (4%) | |
| Angina pectoris | 1/25 (4%) | |
| Endocrine disorders | ||
| Hyperthyroidism | 1/25 (4%) | |
| Eye disorders | ||
| Lacrimation increased | 1/25 (4%) | |
| Gastrointestinal disorders | ||
| Diarrhoea | 5/25 (20%) | |
| Nausea | 3/25 (12%) | |
| Dyspepsia | 1/25 (4%) | |
| Dizziness | 1/25 (4%) | |
| Dysphagia | 2/25 (8%) | |
| Abdominal pain | 8/25 (32%) | |
| Faecaloma | 1/25 (4%) | |
| Flatulence | 5/25 (20%) | |
| Constipation | 1/25 (4%) | |
| Stomach discomfort | 1/25 (4%) | |
| Vomiting | 3/25 (12%) | |
| General disorders | ||
| Catheter related complication | 1/25 (4%) | |
| Chest discomfort | 1/25 (4%) | |
| Secretion discharge | 2/25 (8%) | |
| Oedema peripheral | 1/25 (4%) | |
| Pyrexia | 2/25 (8%) | |
| Feeling abnormal | 1/25 (4%) | |
| Fatigue | 2/25 (8%) | |
| Asthenia | 1/25 (4%) | |
| Hepatobiliary disorders | ||
| Jaundice | 1/25 (4%) | |
| Infections and infestations | ||
| Upper respiratory tract infection | 2/25 (8%) | |
| Bronchitis bacterial | 1/25 (4%) | |
| Abdominal distension | 1/25 (4%) | |
| Escherichia bacteraemia | 1/25 (4%) | |
| Lower respiratory tract infection | 1/25 (4%) | |
| Urinary tract infection | 2/25 (8%) | |
| Sedation | 1/25 (4%) | |
| Injury, poisoning and procedural complications | ||
| Fall | 1/25 (4%) | |
| Skin laceration | 2/25 (8%) | |
| Endotracheal intubation complication | 1/25 (4%) | |
| Hand fracture | 1/25 (4%) | |
| Investigations | ||
| Creatinine renal clearance decreased | 1/25 (4%) | |
| Blood potassium increased | 1/25 (4%) | |
| Blood magnesium decreased | 1/25 (4%) | |
| Metabolism and nutrition disorders | ||
| Hypoglycaemia | 1/25 (4%) | |
| Hypokalaemia | 1/25 (4%) | |
| Anorexia | 1/25 (4%) | |
| Arthralgia | 1/25 (4%) | |
| Dehydration | 1/25 (4%) | |
| Oral intake reduced | 1/25 (4%) | |
| Musculoskeletal and connective tissue disorders | ||
| Muscular weakness | 1/25 (4%) | |
| Haemarthrosis | 1/25 (4%) | |
| Bursa disorder | 1/25 (4%) | |
| Back pain | 3/25 (12%) | |
| Pain in extremity | 2/25 (8%) | |
| Nervous system disorders | ||
| Headache | 2/25 (8%) | |
| Tremor | 2/25 (8%) | |
| Dizziness | 1/25 (4%) | |
| Loss of consciousness | 1/25 (4%) | |
| Lethargy | 2/25 (8%) | |
| Dysarthria | 1/25 (4%) | |
| Myoclonus | 1/25 (4%) | |
| Psychiatric disorders | ||
| Agitation | 1/25 (4%) | |
| Anxiety | 2/25 (8%) | |
| Restlessness | 2/25 (8%) | |
| Confusional state | 4/25 (16%) | |
| Depression | 1/25 (4%) | |
| Oedema peripheral | 1/25 (4%) | |
| Delirium | 1/25 (4%) | |
| Hallucination, visual | 1/25 (4%) | |
| Renal and urinary disorders | ||
| Haematuria | 1/25 (4%) | |
| Bladder spasm | 1/25 (4%) | |
| Urinary hesitation | 1/25 (4%) | |
| Nocturia | 1/25 (4%) | |
| Urinary retention | 3/25 (12%) | |
| Dysuria | 1/25 (4%) | |
| Respiratory, thoracic and mediastinal disorders | ||
| Rhinorrhoea | 4/25 (16%) | |
| Yawning | 2/25 (8%) | |
| Cough | 3/25 (12%) | |
| Increased bronchial secretion | 1/25 (4%) | |
| Respiratory tract congestion | 1/25 (4%) | |
| Nasal congestion | 1/25 (4%) | |
| Pharyngolaryngeal pain | 1/25 (4%) | |
| Productive cough | 1/25 (4%) | |
| Dyspnoea exertional | 1/25 (4%) | |
| Upper respiratory tract congestion | 2/25 (8%) | |
| Wheezing | 1/25 (4%) | |
| Pulmonary congestion | 1/25 (4%) | |
| Sputum retention | 1/25 (4%) | |
| Pulmonary oedema | 1/25 (4%) | |
| Skin and subcutaneous tissue disorders | ||
| Rash | 1/25 (4%) | |
| Decubitus ulcer | 2/25 (8%) | |
| Hyperhidrosis | 2/25 (8%) | |
| Erythema | 2/25 (8%) | |
| Vascular disorders | ||
| Pallor | 1/25 (4%) | |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
Please contact Sponsor directly for additional information.
Results Point of Contact
| Name/Title | Director of Clinical Operations |
|---|---|
| Organization | Bausch Health Companies |
| Phone | |
| Lindsey.Mathew@bauschhealth.com |
- MNTX 901