Molybdenum Cofactor Deficiency, Type A
Conditions
Keywords
Molybdenum cofactor deficiency (MoCD), molybdenum cofactor (MoCo) biosynthesis, sulfite oxidase (SO), xanthine dehydrogenase, aldehyde oxidase, S sulfocysteine (SSC)
Brief summary
This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.
Detailed description
Patients will receive daily IV infusions of ORGN001 (formerly ALXN1101) starting on Day 1. After a prescribed period, dosing will increase monthly based on defined patient safety measures. After Month 6, patients will continue daily dosing at their last tolerated dose.
Interventions
IV infusion
Sponsors
Origin Biosciences
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation) * Currently treated with rcPMP infusions
Exclusion criteria
\- Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety of ORGN001 (Formerly ALXN1101) | Baseline to Month 24 for all patients plus additional follow-up up to Month 90 | Treatment Emergent Serious Adverse Events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | First 6 months at each dose level, where available | ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints |
| S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Baseline to Month 24 for all patients plus additional follow-up to Month 90 | Analyses were performed on urine SSC, a biomarker of the MoCD pathway. Levels of SSC measured in urine were normalized to urine creatinine levels. The observed value, change, and percent change in urine and blood SSC levels from baseline were summarized by visit over time. |
| Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Baseline to Month 24 for all patients plus additional follow-up until Month 30 | Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam. |
| Long-term Safety of ORGN001 (Formerly ALXN1101) | Baseline to Month 24 for all patients plus additional follow up until Month 72 | Change from baseline in Seizure frequency |
Countries
Australia, Netherlands, Tunisia, United Kingdom, United States
Participant flow
Pre-assignment details
Patients who were currently receiving rcPMP infusions were enrolled and transitioned to ORGN001.
Participants by arm
| Arm | Count |
|---|---|
| Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 Patients currently receiving rcPMP infusions at baseline are transitioned to ORGN001 (formerly ALXN1101), starting at their current rcPMP dose and escalating to a target dose of cPMP 0.9 mg/kg per protocol | 8 |
| Total | 8 |
Baseline characteristics
| Characteristic | Patients Currently Receiving rcPMP Infusions at Baseline and Transitioned to ORGN001 |
|---|---|
| Age, Categorical <=18 years | 8 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 45.2 Months STANDARD_DEVIATION 22.96 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 3 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 5 Participants |
| Region of Enrollment Australia | 1 participants |
| Region of Enrollment Netherlands | 2 participants |
| Region of Enrollment Tunisia | 1 participants |
| Region of Enrollment United Kingdom | 3 participants |
| Region of Enrollment United States | 1 participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 8 |
| other Total, other adverse events | 8 / 8 |
| serious Total, serious adverse events | 8 / 8 |
Outcome results
Primary
Safety of ORGN001 (Formerly ALXN1101)
Treatment Emergent Serious Adverse Events
Time frame: Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Population: Full Analysis Set
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Vascular disorders | 2 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Gastrointestinal disorders | 1 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | General disorders and administration site conditions | 5 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Injury, poisoning and procedural complications | 2 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Metabolism and nutrition disorders | 2 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Musculoskeletal and connective tissue disorders | 1 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Nervous system disorders | 2 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Product issues | 1 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Respiratory, thoracic and mediastinal disorders | 2 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Skin and subcutaneous tissue disorders | 1 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Surgical and medical procedures | 1 events |
| Patient Currently Receiving rcPMP Infusions at Baseline | Safety of ORGN001 (Formerly ALXN1101) | Infections and infestations | 6 events |
Secondary
Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination
Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.
Time frame: Baseline to Month 24 for all patients plus additional follow-up until Month 30
Population: All patients entering the study had complete examinations throughout the study to identify Normal vs Abnormal Neurologic Function on the parameters presented. Date shown here are from Baseline and final examination where all 8 patients have data.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Quality of Spontaneous Movement at Baseline | Normal | 3 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Quality of Spontaneous Movement at Baseline | Abnormal | 5 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Quality of Spontaneous Movement at Month 30 | Normal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Quality of Spontaneous Movement at Month 30 | Abnormal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Dystonic at Baseline | Normal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Dystonic at Baseline | Abnormal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Dystonic at Month 30 | Normal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Dystonic at Month 30 | Abnormal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Opistonic at Baseline | Normal | 7 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Opistonic at Baseline | Abnormal | 1 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Opistonic at Month 30 | Normal | 6 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Opistonic at Month 30 | Abnormal | 2 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Truncal Tone at Baseline | Normal | 2 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Truncal Tone at Baseline | Abnormal | 5 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Truncal Tone at Month 30 | Normal | 2 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Truncal Tone at Month 30 | Abnormal | 6 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Appendicular Tone at Baseline | Normal | 2 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Appendicular Tone at Baseline | Abnormal | 6 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Appendicular Tone at Month 30 | Normal | 1 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Appendicular Tone at Month 30 | Abnormal | 7 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Deep Tendon reflexes at Baseline | Normal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Deep Tendon reflexes at Baseline | Abnormal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Deep Tendon reflexes at Month 30 | Normal | 3 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Deep Tendon reflexes at Month 30 | Abnormal | 5 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Primitive reflexes at Baseline | Normal | 5 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Primitive reflexes at Baseline | Abnormal | 1 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Primitive reflexes at Month 30 | Normal | 6 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Primitive reflexes at Month 30 | Abnormal | 1 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Clonus presence at Baseline | Normal | 7 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Clonus presence at Baseline | Abnormal | 1 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Clonus presence at Month 30 | Normal | 6 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Ambulation at Baseline | Normal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Ambulation at Baseline | Abnormal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Ambulation at Month 30 | Normal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Ambulation at Month 30 | Abnormal | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination | Clonus presence at Month 30 | Abnormal | 2 Participants |
Secondary
Long-term Safety of ORGN001 (Formerly ALXN1101)
Change from baseline in Seizure frequency
Time frame: Baseline to Month 24 for all patients plus additional follow up until Month 72
Population: Number of patients with Seizures in observation period
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Patient Currently Receiving rcPMP Infusions at Baseline | Long-term Safety of ORGN001 (Formerly ALXN1101) | Screening | 3 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Long-term Safety of ORGN001 (Formerly ALXN1101) | Baseline to Month 6 | 4 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Long-term Safety of ORGN001 (Formerly ALXN1101) | Month 6 to Month 12 | 3 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Long-term Safety of ORGN001 (Formerly ALXN1101) | Month 12 to Month 24 | 3 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Long-term Safety of ORGN001 (Formerly ALXN1101) | Month 24 to Month 36 | 3 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Long-term Safety of ORGN001 (Formerly ALXN1101) | Month 36 to Month 48 | 3 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Long-term Safety of ORGN001 (Formerly ALXN1101) | Month 48 to Month 60 | 3 Participants |
| Patient Currently Receiving rcPMP Infusions at Baseline | Long-term Safety of ORGN001 (Formerly ALXN1101) | Month 60 to Month 72 | 3 Participants |
Secondary
Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)
ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints
Time frame: First 6 months at each dose level, where available
Population: Measurement is actual plasma concentration of ORGN001 at measured timepoints, starting at their current rcPMP dose. 6 patients started at a dose of 240 mcg/kg, 1 patient at 248 mcg/kg, and 1 patient at 280 mcg/kg. (EOI = End of Infusion). 1 patient is missing a pre-infusion dose measurement.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 1 Pre-infusion (240 mcg/kg) | 3.95 ng/mL | Standard Deviation 9.68 |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 1 Pre-infusion (248 mcg/kg) | 0 ng/mL | — |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 1 EOI (240 mcg/kg) | 669.00 ng/mL | Standard Deviation 236.06 |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 1 EOI (280 mcg/kg) | 770.00 ng/mL | — |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 7 EOI (240 mcg/kg) | 699.50 ng/mL | Standard Deviation 246.72 |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 7 EOI (248 mcg/kg) | 694.00 ng/mL | — |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 60 EOI (480 mcg/kg) | 880.84 ng/mL | Standard Deviation 558.56 |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 90 EOI (240 mcg/kg) | 1230.00 ng/mL | — |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 90 EOI (480 mcg/kg) | 762.00 ng/mL | — |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 90 EOI (720 mcg/kg) | 1888.33 ng/mL | Standard Deviation 271.1 |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 120 EOI (480 mcg/kg) | 2810.00 ng/mL | — |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 120 EOI (720 mcg/kg) | 671.00 ng/mL | — |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 120 EOI (960 mcg/kg) | 4213.33 ng/mL | Standard Deviation 4520.31 |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 150 EOI (720 mcg/kg) | 3810.00 ng/mL | — |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 150 EOI (960 mcg/kg) | 2045.67 ng/mL | Standard Deviation 1009.84 |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 150 EOI (1200 mcg/kg) | 3173.33 ng/mL | Standard Deviation 656.53 |
| Patient Currently Receiving rcPMP Infusions at Baseline | Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101) | Day 1 EOI (248 mcg/kg) | 36.00 ng/mL | — |
Secondary
S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time
Analyses were performed on urine SSC, a biomarker of the MoCD pathway. Levels of SSC measured in urine were normalized to urine creatinine levels. The observed value, change, and percent change in urine and blood SSC levels from baseline were summarized by visit over time.
Time frame: Baseline to Month 24 for all patients plus additional follow-up to Month 90
Population: Not all patients had samples taken at each expected timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Baseline | 21.1 umol/mmol | Standard Deviation 12.89 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Day 4 Change from Baseline | -1.7 umol/mmol | Standard Deviation 12.7 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Day 7 Change from Baseline | -7.8 umol/mmol | Standard Deviation 9.7 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Day 14 Change from Baseline | -2.2 umol/mmol | Standard Deviation 14.51 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Day 28 Change of Baseline | 2.8 umol/mmol | Standard Deviation 14.17 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Day 57 Change from Baseline | -3.0 umol/mmol | Standard Deviation 10.29 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Day 127 Change from Baseline | -12.2 umol/mmol | Standard Deviation 14.77 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Month 6 Change from Baseline | -13.6 umol/mmol | Standard Deviation 11.77 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Month 12 Change from Baseline | -8.6 umol/mmol | Standard Deviation 20.15 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Month 24 Change from Baseline | -14.3 umol/mmol | Standard Deviation 14.73 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Month 36 Change from Baseline | -13.2 umol/mmol | Standard Deviation 18.4 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Month 48 Change from Baseline | -6.7 umol/mmol | Standard Deviation 6.65 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Month 60 Change from Baseline | -17.9 umol/mmol | Standard Deviation 14.59 |
| Patient Currently Receiving rcPMP Infusions at Baseline | S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time | Month 90 Change from Baseline | 1.7 umol/mmol | Standard Deviation 10.25 |